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RxOutlook® 3rd Quarter 2017

OptumRx brand pipeline forecast

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

2017 Possible launch date

Travivo gepirone ER GSK/ Fabre-Kramer 5-HT-1A receptor agonist Major depressive disorder PO CRL n/a N N salmon calcitonin (TAR01- Tarsa/ Unigene Tbria osteoclast inhibitor Osteoporosis PO Filed NDA 2H2017 N N 201) Laboratories Ontinua ER arbaclofen extended-release Osmotica muscle relaxant Spasticity PO Filed NDA 2H2017 Y N

GF-00100100 ozenoxacin Medimetriks/ Cipher quinolone (non-fluorinated) Bacterial infections TOP Filed NDA 3Q2017 N N methylphenidate modified- Attention deficit hyperactivity Benjorna Highland Therapeutics CNS stimulant PO Filed NDA 3Q2017 N N release disorder (ADHD) urate transporter1 and organic anion transporter 4 Duzallo lesinurad/ allopurinol AstraZeneca Gout PO Filed NDA 8/2017 N N inhibitor/ hypoxanthine analog anti-CD22 antibody-drug Acute myeloid leukemia Besponsa / UCB IV Filed NDA 8/20/2017 Y Y conjugate (AML) carbapenem/ beta- Carbavance meropenem/ vaborbactam Medicines Company Bacterial infections IV Filed NDA 8/20/2017 N N lactamase inhibitor Dyskinesia in Parkinson's Nurelin amantadine ER Adamas antiviral PO Filed NDA 8/24/2017 N Y disease/ Multiple sclerosis anti-rabies immunoglobulin KamRAB Kamada/ Kedrion immune globulin Rabies IM Filed BLA 8/29/2017 Y N (human) -6 (IL-6) Plivensia Janssen / GSK SC Filed BLA 9/2017 Y N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

angiotensin receptor blocker EZR-104 valsartan controlled-release Ezra Innovations Heart failure/ Hypertension PO Filed NDA 9/10/2017 N N (ARB) amphetamine XR- amphetamine extended- Attention deficit hyperactivity Neos Therapeutics stimulant PO Filed NDA 9/17/2017 N N liquid release disorder (ADHD) Solosec secnidazole Symbiomix 5-nitroimidazole antibiotic Urinary tract infection PO Filed NDA 9/17/2017 N N phosphodiesterase-5 (PDE- tadalafil pharmafilm tadalafil pharmafilm MonoSol Rx Erectile dysfunction PO Filed NDA 9/18/2017 Y N 5) inhibitor ITCA-650 (sustained Intarcia/ Quintiles/ glucagon-like peptide-1 exenatide sustained-release Diabetes mellitus SC implant Filed NDA 9/21/2017 Y N release exenatide) Servier (GLP-1) receptor agonist Rexista XR oxycodone ER Intellipharmaceutics opioid agonist Pain PO Filed NDA 9/25/2017 N N antibody-drug conjugate (CD-33 Ab linked to Acute myeloid leukemia Mylotarg Pfizer IV Filed BLA 9/30/2017 Y Y calicheamicin, a cytotoxic (AML) agent) insulin aspart (ultra fast FIAsp Novo Nordisk fast-acting insulin Diabetes mellitus SC CRL 9/30/2017 N N acting) pregabalin controlled- Postherpetic neuralgia Lyrica CR Pfizer GABA structural analogue PO Filed sNDA 10/2/2017 N N release (PHN)/ Fibromyalgia Acute lymphoblastic leukemia (ALL)/ Diffuse chimeric receptor CTL-019 tisagenlecleucel large B-cell lymphoma IV Filed BLA 10/3/2017 Y Y (CAR) T cell therapy (DLBCL)/ Chronic lymphocytic leukemia (CLL) Zilretta acetonide Flexion corticosteroid Osteoarthritis Intra-articular Filed NDA 10/6/2017 Y N

ARX-04 sufentanil AcelRx opioid analgesic Pain SL Filed NDA 10/12/2017 Y N Vibex QST (QuickShot testosterone Antares androgen Hypogonadism SC Filed NDA 10/20/2017 N N Testosterone) recombinant human Factor LR-769 eptacog beta LFB SA Hemophilia IV Filed BLA 10/23/2017 Y N VIIa

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

Duchenne muscular dystrophy/ Translarna ataluren PTC Therapeutics gene transcription modulator PO Filed NDA 10/24/2017 Y Y Mucopolysaccharidosis (MPS) neurokinin-1 (NK-1) receptor Varubi rolapitant Tesaro/Opko Health Nausea and vomiting (N/V) IV Filed NDA 10/25/2017 Y N antagonist folate analog metabolic Non-small cell lung cancer EP-5101 pemetrexed Eagle Pharmaceuticals IV Filed NDA 10/30/2017 Y N inhibitor (NSCLC)/ Mesothelioma neurokinin-1 (NK1) receptor Cinvanti aprepitant Heron Therapeutics Nausea and vomiting (N/V) IV Filed NDA 11/12/2017 N N antagonist beta-glucuronidase Mucopolysaccharidosis VII UX-003 (rhGUS) replacement IV Filed BLA 11/16/2017 Y Y (recombinant) (MPS-VII) Non-Hodgkin's lymphoma phosphatidylinositol-3 kinase BAY-80-6946 copanlisib (NHL)/ Diffuse large B-cell IV/PO Filed NDA 11/17/2017 Y Y (PI3K) inhibitor lymphoma inhaled corticosteroid (ICS)/ fluticasone furoate/ fluticasone furoate/ long-acting muscarinic agent Chronic obstructive umeclidinium/ GSK/ Innoviva INH Filed NDA 11/21/2017 N N umeclidinium/ (LAMA)/ long-acting beta pulmonary disease (COPD) vilanterol agonist (LABA) Otsuka/ Proteus Digital Schizophrenia/ Bipolar/ Abilify aripiprazole atypical antipsychotic PO Filed NDA 11/23/2017 N N Health Major Depression AstraZeneca/ / glucagon-like peptide-1 Bydureon exenatide Bristol-Myers Squibb/ Eli Diabetes mellitus SC Filed sNDA 11/27/2017 N N (GLP-1) receptor agonist Lilly Non-Hodgkin lymphoma/ Mantle cell lymphoma/ Diffuse Large B-Cell chimeric antigen receptor CAR-T ciloleucel axicabtagene ciloleucel Kite Lymphoma (DLBCL)/ IV Filed NDA 11/29/2017 Y Y (CAR) T cell therapy Chronic lymphocytic leukemia (CLL)/ Small cell lymphocytic lymphoma (SLL) opioid receptor agonist RBP-6000 buprenorphine depot / QLT Drug addiction SC Filed NDA 11/30/2017 Y N (partial)

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

sodium zirconium ZS-9 ZS Pharma potassium-binding agent Hyperkalemia PO CRL 4Q2017 N N cyclosilicate cyclosporine A (Nova- Dry eye/ Vernal Cyclokat Santan SAS immunosuppressant TOP InTrial 4Q2017 Y Y 22007) keratoconjunctivitis Apealea (Paclical) paclitaxel Oasmia taxane IV InTrial 4Q2017 Y Y FastTrk/ Prostvac rilimogene glafolivec BN ImmunoTherapeutics Prostate cancer SC 4Q2017 Y N Breakthru Diffuse large B-cell lymphoma (DLBCL)/ Pixuvri pixantrone Cell Therapeutics anthracycline derivative IV CRL 4Q2017 Y N Follicular lymphoma/ Non- Hodgkin's lymphoma (NHL) Pancreatic islet reparixin reparixin Dompe interleukin-8 (IL-8) inhibitor IV InTrial 4Q2017 Y Y transplantation FastTrk/ Civacir immunoglobulin Biotest immunoglobulin Hepatitis C (HCV) IV 4Q2017 Y Y Breakthru DCVax-L, DCVax- glioblastoma multiforme Northwest vaccine Glioblastoma SC InTrial 4Q2017 Y Y Brain vaccine Biotherapeutics Non-small cell lung cancer tertomotide tertomotide KAEL-GemVax vaccine Intradermal InTrial 4Q2017 Y N (NSCLC)/ RNA polymerase inhibitor/ rifabutin/ amoxicillin/ RHB-105 RedHill Biopharma penicillin/ proton pump Bacterial infections PO InTrial 4Q2017 N N pantoprazole inhibitor (PPI) Primary immunodeficiency/ RI-002 RI-002 ADMA Biologics immunoglobulin Respiratory syncytial virus/ IV CRL 4Q2017 Y N Bacterial infections ABX-203 ABX-203 Abivax vaccine Hepatitis B (HBV) SC/ intranasal InTrial 4Q2017 N N amantadine extended- Osmolex ER (OS-320) Osmotica antiviral Dyskinesia PO InTrial 4Q2017 N Y release Medicines Development MDL moxidectin chloride channel agonist Parasitic infections PO InTrial 4Q2017 N N for Global Health Aeroquin levofloxacin HCl Raptor fluoroquinolone Cystic fibrosis (CF) INH InTrial 4Q2017 Y Y

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

vascular endothelial growth Ovarian cancer/ Biliary tract Recentin cediranib AstraZeneca factor receptor (VEGF) PO InTrial 4Q2017 Y Y cancer antagonists FMX-102 (FXFM-244) minocycline Foamix tetracyclines Impetigo TOP InTrial 4Q2017 N N Shingrix (GSK- herpes zoster vaccine GSK vaccine Herpes zoster IM Filed BLA 4Q2017 Y N 1437173A) aspartyl-alanyl Ampion Ampio Pharmaceuticals immunomodulator Osteoarthritis Intra-articular InTrial Late 2017 Y N diketopiperazine (DA-DKP) human plasminogen human plasminogen Kedrion plasminogen Plasminogen deficiency TOP InTrial Late 2017 Y Y

ELI-202 opioid (undisclosed) Elite opioid agonist Pain PO InTrial Late 2017 N N Healthpoint allogeneic neonatal HP802-247 HP802-247 Venous leg ulcer TOP InTrial Late 2017 Y N Biotherapeutics keratinocytes and fibroblasts growth hormone Anorexia/ Cachexia (cancer- FastTrk/ anamorelin anamorelin Helsinn / Novo Nordisk secretagogue receptor PO Late 2017 Y N related) Breakthru (GHSR) agonist lubiprostone Takeda/ Sucampo chloride channel activator Constipation PO InTrial Late 2017 N N janus associated kinase Myeloproliferative disorders/ CYT-387 momelotinib Gilead PO InTrial Late 2017 Y Y (JAK) inhibitor Pancreatic cancer CEM-101 solithromycin Cempra macrolide Bacterial infections IV/PO CRL Late 2017 N N FastTrk/ midazolam (USL-261) midazolam (USL-261) Upsher-Smith benzodiazepine Seizures Intranasal Late 2017 N Y Breakthru FLT-3 receptor tyrosine Acute myeloid leukemia quizartinib quizartinib Ambit Biosciences PO InTrial Late 2017 Y N kinase inhibitor (AML) pegylated arginine Hepacid ADI-PEG-20 Polaris Hepatic cancer IM InTrial Late 2017 Y Y deiminase Portola/ Pfizer/ Bristol- recombinant Factor Xa AndexXa andexanet alfa Reversal agent IV CRL Late 2017 Y N Myers Squibb inhibitor antidote Growth hormone deficiency/ somatropin somatropin Opko/ Pfizer enzyme replacement Small for gestational age SC InTrial Late 2017 Y Y (SGA)

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

transmembrane activator EMD Serono / Bristol- Systemic lupus atacicept (TACI-Ig) atacicept (TACI-Ig) and CAML interactor (TACI) SC InTrial Late 2017 Y N Myers Squibb erythematosus (SLE) receptor-immunoglobulin antiplatelet derived growth Fovista (E-10030; Ophthotech/ Archemix/ Age-related macular FastTrk/ pegpleranib sodium factor subunit B (PDGF-B) Intravitreal Late 2017 Y N OAP-030) Astellas/ Novartis degeneration (AMD) Breakthru aptamer Non-small cell lung cancer FastTrk/ MVA-MUC1-IL2 TG-4010 Transgene vaccine SC Late 2017 N N (NSCLC) Breakthru mycolic acid biosynthesis Deltyba delamanid Otsuka Tuberculosis PO InTrial Late 2017 N N inhibitor Primatene HFA epinephrine Amphastar catecholamine INH CRL Late 2017 N N Seciera (OTX-101) cyclosporine Auven Therapeutics immunosuppressant Dry eyes TOP InTrial Late 2017 Y N Acute lymphocytic leukemia EZN-2285 calaspargase pegol Baxalta enzyme therapy IM/IV InTrial Late 2017 Y N (ALL) Ryplazim human plasminogen ProMetic/ Hematech plasminogen Plasminogen deficiency IV Filed BLA Late 2017 Y Y Primary biliary ursodeoxycholic acid ursodeoxycholic acid Retrophin/ Asklepion bile acid derivative PO InTrial Late 2017 Y N cirrhosis/cholangitits A-101 (V-101) hydrogen peroxide Aclaris Therapeutics peroxide Benign pigmented lesions TOP Filed NDA Late 2017 Y N Benign prostatic hyperplasia tamsulosin DRS tamsulosin DRS Female Health/ Aspen alpha-adrenergic antagonist PO InTrial Late 2017 N N (BPH) naloxone naloxone Amphastar opioid antagonist Opioid dependence Intranasal CRL Late 2017 N N interleukin-5 receptor (IL-5R) Asthma/ Chronic obstructive benralizumab AstraZeneca SC Filed BLA Late 2017 Y N alpha inhibitor pulmonary disease (COPD) Dengvaxia Dengue fever vaccine Sanofi Pasteur vaccine Dengue fever SC Filed BLA Late 2017 N N acetaminophen/ Non- Maxigesic / Aft Pharmaceuticals steroidal anti-inflammatory Pain PO Filed NDA Late 2017 N N drug (NSAID) ertugliflozin ertugliflozin Merck/ Pfizer SGLT2 inhibitor diabetes mellitus PO Filed NDA 12/3/2017 N N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

sodium glucose transporter type 2 (SGLT-2) inhibitor/ ertugliflozin/ sitagliptin ertugliflozin/ sitagliptin Merck/ Pfizer Diabetes mellitus PO Filed NDA 12/3/2017 N N dipeptidyl peptidase-4 (DPP- 4) inhibitor ertugliflozin/ metformin ertugliflozin/ metformin Merck/ Pfizer SGLT2 inhibitor/ biguanide Diabetes mellitus PO Filed NDA 12/3/2017 N N glucagon-like peptide-1 semaglutide semaglutide Novo Nordisk Diabetes mellitus SC/ PO Filed NDA 12/5/2017 Y N (GLP-1) receptor agonist long-acting muscarinic Chronic obstructive SUN-101 (EP-101) glycopyrrolate Sunovion INH Filed NDA 12/15/2017 N N pulmonary diseaser (COPD) mammalian target of Opsiria sirolimus Santen Uveitis Intravitreal Filed NDA 12/24/2017 Y Y rapamycin (mTOR) inhibitor ethinyl estradiol/ hormonal combination Twirla Agile Therapeutics Pregnancy prevention TOP Filed NDA 12/26/2017 N N levonorgestrel contraceptive ACE-910 (RG-6013, factor IXa/ factor X bi- hBS-910, RO- emicizumab Roche/ Chugai Hemophilia SC Filed BLA 12/26/2017 Y Y specific antibody 5534262) Jatenzo testosterone (CLR-610) Clarus Therapeutics androgen Hypogonadism PO Filed NDA 12/27/2017 N N

Luminesse brimonidine tartrate Bausch and Lomb alpha-2 agonist Ocular rednes OP Filed NDA 12/27/2017 N N

Macrilen macimorelin acetate Aeterna Zentaris ghrelin agonist Growth hormone deficiency PO Filed NDA 12/30/2017 Y Y

2018 Possible launch date Non-small cell lung cancer PF-00299804 dacomitinib Pfizer pan-HER inhibitor PO InTrial 2018 Y N (NSCLC)

Ovarian cancer/ Non- Hodgkin's lymphoma (NHL)/ MLN-8237 alisertib Millennium aurora kinase A inhibitor Small cell lung cancer PO InTrial 2018 Y Y (SCLC)/ Breast cancer/ Peripheral T-cell lymphoma

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

selective endothelin-A Xinlay atrasentan AbbVie Diabetic nephropathy PO InTrial 2018 Y N receptor antagonist scyllo- cyclohexanehexol scyllo-cyclohexanehexol Transition Therapeutics/ beta-amyloid (Abeta) Alzheimer's disease/ Down's (ELND-005, D-5, D- (ELND-005, D-5, D-005, PO InTrial 2018 N N oligomerization inhibitor syndrome 005, AZD-103, AZD-103, scylloinositol) scylloinositol) Niemann-Pick Disease FastTrk/ arimoclomol arimoclomol CytRx cytoprotectives (NPD)/ Amyotrophic lateral PO 2018 Y Y Breakthru sclerosis (ALS) phosphatidylserine receptor Non-small cell lung cancer FastTrk/ Tarvacin bavituximab Peregrine IV 2018 Y N antagonist (NSCLC) Breakthru bone marrow-derived PREOB mesenchymal stem cell Bone Therapeutics stem cell therapy Fracture/ Bone necrosis Intraosseus InTrial 2018 Y N therapy JNJ-56021927 (ARN- androgen receptor apalutamide Janssen Prostate cancer PO InTrial 2018 N N 509, JNJ-927) antagonist DSC-127 (USB-001, aclerastide Derma Sciences angiotensin II analog Diabetic foot ulcers TOP InTrial 2018 N N NorLeu-3-A(1-7)) KAE-609 cipargamin Novartis protein synthesis inhibitor Malaria PO InTrial 2018 N N aldosterone synthase LCI-699 osilodrostat Novartis Cushing's syndrome PO InTrial 2018 N Y inhibitor ALKS-8700 (RDC- ALKS-8700 (RDC-1559) Alkermes prodrug Multiple sclerosis (MS) PO InTrial 2018 Y N 1559) FastTrk/ esketamine esketamine Janssen NMDA receptor antagonist Major depressive disorder Intranasal 2018 N N Breakthru IVIG IVIG Prometic immune globulin Primary immunodeficiency IV InTrial 2018 Y N

PSD-502 lidocaine/ prilocaine Plethora/Sciele sodium channel blocker Premature ejaculation TOP InTrial 1Q2018 N N [177]Lutetium-DOTA[0]- Advanced Accelerator somatostatin receptor Gastroenteropancreatic Lutathera Tyr[3]-Octreotate (177-Lu- INJ Filed NDA 1Q2018 Y Y Applications agonist neuroendocrine tumors Dotatate)

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

HEPLISAV-B HBV vaccine Merck/ Dynavax vaccine Hepatitis B (HBV) IM Filed BLA 1Q2018 N N anti-sclerostin monoclonal Evenity romosozumab Osteoporosis SC CRL 1Q2018 Y N antibody Familial chylomicronemia ISIS 304801 (ISIS- volanesorsen Ionis antisense drug syndrome (FCS)/ SC InTrial 1Q2018 Y Y APOCIIIRx) Lipodystrophy Breast cancer/ Pancreatic cancer/ Pancreatic cancer/ Small cell lung cancer RS7-SN-38 antibody-drug FastTrk/ IMMU-132 Seattle Genetics (SCLC)/ Non-small cell lung IV 1Q2018 Y Y conjugate Breakthru cancer (NSCLC)/ Colorectal cancer/ Esophageal cancer/ Urinary bladder cancer Advaxis/ Biocon/ ADXS-HPV (ADXS- Anal cancer/ Cervical FusionVax/ Sorrento FastTrk/ 11-001, Lm-LLO-E7, axalimogene filolisbac vaccine cancer/ Head and neck IV 1Q2018 Y Y Therapeutics/ Taiwan Breakthru lovaxin C) cancer Biotech Azedra (Ultratrace Neuroendrocine tumors iobenguane (I-131) Progenics radiopharmaceuticals IV Filed NDA 1Q2018 Y Y MIBG) (NET) Postherpetic neuralgia ZTlido lidocaine Scilex sodium channel blocker TOP CRL 1Q2018 N N (PHN) anti-DLL3 monoclonal Small cell lung cancer Rova-T AbbVie IV InTrial 1Q2018 Y Y antibody (SCLC) furoate/ GSP-301 Glenmark corticosteroid/ antihistamine Allergic rhinitis NA InTrial 1Q2018 N N olopatadine HCl Mantle cell lymphoma (MCL)/ Chronic lymphocytic ACP-196 acalabrutinib Acerta/ Merck tyrosine kinase inhibitor PO Filed NDA 1Q2018 Y Y leukemia (CLL)/ Small cell lymphocytic lymphoma (SLL) promethazine/ hydrocodone/ anti-emetic/ opioid/ CL-108 Charleston Laboratories Nausea/ Vomiting/ Pain PO CRL 1Q2018 N N acetaminophen analgesic Vesneo (Vyzulta) latanoprostene bunod Bausch and Lomb/NicOx nitric oxide-donating Glaucoma/ Ocular TOP CRL 1Q2018 N N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

F2-alpha hypertension analog dexamethasone sustained- Dextenza Ocular Therapeutix corticosteroid Pain/ Allergic conjunctivitis OP CRL 1Q2018 N N release IDP-118 tazarotene/ halobetasol Valeant / corticosteroid Psoriasis TOP InTrial 1Q2018 N N

Blastic plasmacytoid dendritic dell neoplasm (BPDCN)/ Acute mylegenous leukemia (AML)/ Myelodysplastic interleukin-3 (IL-3) receptor SL-401 SL-401 Stemline Therapeutics syndrome (MDS)/ Multiple IV InTrial 1Q2018 Y Y antagonist myeloma (MM)/ Hypereosinophilic syndrome/ Myelofibrosis/ Chronic myelomonocytic leukemia (CMML) Immune Response Remune HIV-1 vaccine vaccine HIV IM Filed BLA 1Q2018 Y Y BioPharma TNX-355 (TMB-355, Theratechnologies/ viral entry inhibitor HIV IM/IV/SC Filed sBLA 1/3/2018 Y Y Hu-5A8) / Roche/ TaiMed OptiNose (OPN-375; OptiNose AS corticosteroid Nasal polyps Intranasal Filed NDA 1H2018 N N Opt-FP) Pulmonary arterial RemoSynch United Therapeutics analog IV/SC Filed NDA 1H2018 Y Y hypertension (PAH) Polycythemia vera (PV)/ ropeginterferon alfa- PharmaEssentia/ AOP ropeginterferon alfa-2b Myelofibrosis (MF)/ Essential SC InTrial 1H2018 Y Y 2b Orphan thrombocythemia (ET) Xeglyze abametapir Hatchtech metalloprotease inhibitor Lice TOP CRL 1H2018 N N TX-004HR, TX-12- estradiol TherapeuticsMD estrogen receptor agonist Vaginal atrophy VG CRL 1H2018 N N 004HR RESOLVE (Sinuva) mometasone furoate Intersect ENT corticosteroid Chronic sinusitis Implant Filed NDA 1/7/2018 Y N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

Luxturna voretigene neparvovec Spark Therapeutics gene therapy Retinal disease Intraocular Filed BLA 1/12/2018 Y Y opioid receptor agonist CAM-2038 buprenorphine Camurus/ Braeburn Opioid dependence/ Pain SC Filed NDA 1/20/2018 Y N (partial) Breast cancer/ Non-small LY-2835219 bemaciclib (abemaciclib) Eli Lilly cyclin-dependent kinases cell lung cancer (NSCLC)/ PO Filed NDA 1/26/2018 Y N Mantle cell lymphoma non-nucleoside reverse GlaxoSmithKline/ J&J/ transcriptase inhibitor rilpivirine/ dolutegravir rilpivirine/ dolutegravir HIV PO Filed NDA 2/1/2018 Y N ViiV (NNRTI)/ Integrase strand transfer inhibitor (ISTI) dexamethasone controlled- Dexycu Icon Bioscience corticosteroid Ocular inflammation Intraocular Filed NDA 2/12/2018 N/A N release hydroxyprogesterone Makena Lumara Health progestin Preterm labor SC Filed sNDA 2/14/2018 Y Y caproate rho kinase-norepinephrine Glaucoma/ Ocular Rhopressa netarsudil mesylate Aerie TOP Filed NDA 2/28/2018 N N transport protein inhibitor hypertension Yonsa abiraterone acetate Churchill/ Iroko tyrosine kinase inhibitor Prostate cancer PO Filed NDA 3/19/2018 Y N corrector ion channel ivacaftor/ tezacaftor ivacaftor/ tezacaftor Vertex Cystic fibrosis PO Filed NDA 3/26/2018 Y Y modulator IntelGenx / Red Hill VersaFilm rizatriptan triptans Headache PO CRL 2Q2018 N N Biopharma opioid receptor agonist FastTrk/ ALKS-5461 buprenorphine/ samidorphan Alkermes (partial)/ opioid receptor Major depressive disorder PO 2Q2018 N N Breakthru antagonist FastTrk/ MK-8228 letermovir Merck quinazoline Cytomegalovirus (CMV) IV/PO 2Q2018 N Y Breakthru Zoptrex zoptarelin Aeterna Zentaris anthracycline derivative IV InTrial 2Q2018 Y N

Acute myeloid leukemia anti-CD45 monoclonal- anti-CD45 monoclonal Iomab-B Actinium (AML)/ Myelodysplastic IV InTrial 2Q2018 Y Y antibody BC8-I-131 antibody syndrome (MDS)

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

darunavir/ emtricitabine/ protease inhibitor/ NRTI/ Symtuza cobicistat/ tenofovir Johnson & Johnson HIV PO InTrial 2Q2018 Y N boosting agent/ NRTI alafenamide Amyotrophic lateral sclerosis FastTrk/ CK-2017357 (CK-357) tirasemtiv Cytokinetics troponin activator PO 2Q2018 Y Y (ALS) Breakthru CCP-07 CCP-07 Vernalis/ Tris Pharma undisclosed Cough and cold PO CRL 2Q2018 N N

CCP-08 CCP-08 Vernalis/ Tris Pharma undisclosed Viral rhinitis PO CRL 2Q2018 Y N Protectan CBLB-502 Radiation poisoning/ FastTrk/ entolimod Cleveland BioLabs TLR5 SC 2Q2018 Y Y (SA-702) Colorectal cancer Breakthru Non-small cell lung cancer FastTrk/ PF-06463922 lorlatinib Pfizer tyrosine kinase inhibitor PO 2Q2018 Y N (NSCLC) Breakthru estrogen receptor TX-001HR (TX-12- estradiol/ progesterone TherapeuticsMD agonist/progesterone Menopause PO InTrial 2Q2018 N N 001HR) receptor agonist nonsteroidal anti- inflammatory drug (NSAID)/ KIT-302 / amlodipine Kitov Hypertension/ Osteoarthritis PO Filed NDA 2Q2018 N N calcium channel blocker (CCB) nonsteroidal anti- N-1539 Recro Pharma/ Alkermes Post-surgical pain IV Filed NDA 2Q2018 Y N inflammatory drug (NSAID) Tavalisse fostamatinib Rigel oral SYK inhibitor Immune thrombocytopenia PO Filed NDA 4/17/2018 Y Y polyethylene glycol-based Plenvu NER-1006 Norgine Bowel preparation PO Filed NDA 4/28/2018 N N preparation / beta- EB-178 solifenacin/ mirabegron Astellas 3 adrenergic receptor Overactive bladder PO Filed sNDA 4/30/2018 N N agonist Inbrija levodopa Acorda antiparkinson Parkinson's disease INH Filed NDA 5/3/2018 N N calcitonin gene-related Aimovig erenumab Amgen Headache SC Filed BLA 5/17/2018 Y N peptide (CGRP) antagonist

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug (MK- interleukin-23 (IL-23) tildrakizumab (MK-3222) Merck Psoriasis SC Filed BLA 5/23/2018 Y N 3222) inhibitor Cystic fibrosis/ Non-cystic Linhaliq ciprofloxacin Aradigm/ Grifols fluoroquinolone INH Filed NDA 5/27/2018 Y Y fibrosis bronchiectasis prostaglandin (PG) receptor RayVa (Vitaros) alprostadil / Apricus Erectile dysfunction TOP CRL Mid-2018 N N agonist brincidofovir DNA-directed DNA Adenovirus/ FastTrk/ CMX001 Chimerix PO Mid-2018 N N hexadecyloxypropyl ester polymerase inhibitor Cytomegalovirus (CMV) Breakthru FastTrk/ BAY-41-6551 amikacin Nektar/ Bayer aminoglycoside Bacterial infections INH Mid-2018 Y N Breakthru Leukocyte Interleukin (CS- Head and Neck cancer/ Multikine CEL-SCI immunomodulator SC InTrial Mid-2018 Y Y 001P3) Squamous cell carcinoma Bilirubin encephalopathy/ Stanate stannsoporfin InfaCare heme oxygenase inhibitor IM Filed NDA Mid-2018 Y N Hyperbilirubemia FastTrk/ Epidiolex GW Pharmaceuticals product Seizures PO Mid-2018 Y Y Breakthru dopamine receptor APD-421 amisulpride Acacia Nausea/ Vomiting IV InTrial Mid-2018 N N antagonist synaptic vesicle protein AGB-101 levetiracetam AgeneBio Alzheimer's disease PO InTrial Mid-2018 N N modulator Shionogi/ S-649266 cefiderocol cephalosporin antibiotic Bacterial infections IV InTrial Mid-2018 Y N GlaxoSmithKiline XaraColl bupivacaine implant Innocoll sodium channel blocker Pain implant CRL Mid-2018 Y N

ELI-200 oxycodone/ naltrexone Elite opioid agonist Pain PO CRL Mid-2018 N N FastTrk/ TRV-130 oliceridine Trevena opioid receptor agonist Pain IV Mid-2018 N N Breakthru Feraccru (ferric iron, Shield Therapeutics/ Anemia/ Irritable bowel ferric trimaltol iron replacement PO InTrial Mid-2018 N N ST-10, ST10-021) AOP Orphan Pharma syndrome (IBS) IDP-122 halobetasol propionate Valeant corticosteroid Psoriasis TOP InTrial Mid-2018 N N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

C-C chemokine receptor 5 FastTrk/ PRO-140 PRO-140 CytoDyn HIV IV/SC 2H2018 Y N (CCR5) antagonist Breakthru dihydroergotamine mesylate MAP Pharmaceuticals/ Semprana dihydroergotamine Headache INH InTrial 2H2018 N N (DHE) Resolor prucalopride Shire 5-HT-4 receptor agonist Chronic constipation PO InTrial 2H2018 N N telapristone acetate progesterone receptor Uterine fibroids/ Proellex Repros/ Gedeon Richter PO InTrial 2H2018 N N (CBD4124) antagonist Endometriosis Clostridium difficile- FastTrk/ cadazolid cadazolid quinolonyl-oxazolidinone PO 2H2018 N N associated diarrhea Breakthru Alzheimer's disease/ LMTX methylthioninum Roche inhibitor PO InTrial 2H2018 N Y Dementia Blepharitis/ Ocular Dexasite dexamethasone InSite Vision corticosteroid TOP InTrial 2H2018 N N inflammation RGN-259 (GBT-201; Neurotrophic keratitis (NK)/ thymosin beta 4 RegeneRx actin regulating peptide TOP InTrial 2H2018 N Y RGN-352) Dry eyes tergenpantucel-L Non-small cell lung cancer HyperAcute Lung (turgenpumatucel-L, NewLink Genetics IV InTrial 2H2018 Y N (NSCLC) tergenpumatucel-L) Benign prostatic hyperplasia NX-1207 fexapotide triflutate Nymox pro-apoptotic Intratumoral InTrial 2H2018 Y N (BPH)/ Prostate cancer

BAY94-9027 damoctocog alfa pegol Bayer anithemophilic factor Hemophilia IV InTrial 2H2018 Y N next-generation FastTrk/ plazomicin plazomicin Achaogen/ Ionis Bacterial infections IV 2H2018 Y N aminoglycoside Breakthru FastTrk/ Zeftera ceftobiprole Basilea cephalosporin antibiotic Bacterial infections IV 2H2018 Y N Breakthru IONIS-TTRrx (ISIS- Transthyretin (TTR) IONIS-TTRrx (ISIS-TTRRx, FastTrk/ TTRRx, ISIS-420915; GlaxoSmithKline/ Ionis antisense RNA modulator amyloidosis/ Transthyretin SC 2H2018 Y Y ISIS-420915; GSK-2998728) Breakthru GSK-2998728) (TTR) cardiomyopathy Septic shock/ Heart failure/ calcium sensitizing FastTrk/ Simdax levosimendan Tenax Therapeutics Low cardiac output IV 2H2018 Y N phosphodiesterase inhibitor Breakthru syndrome (LCOS)

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

COR-003 levoketoconazole Cortendo azole antifungal Cushing's syndrome PO InTrial 2H2018 N Y Head and neck cancer/ Solid cytotoxic T lymphocyte- tumors/ Mesothelioma/ Non- tremelimumab AstraZeneca associated antigen 4 IV InTrial 2H2018 Y Y small cell lung cancer (CTLA4) inhibitor (NSCLC) selective progesterone Esmya ulipristal acetate Actavis (Allergan) Uterine bleeding PO InTrial 2H2018 N N receptor modulator (SPRM) Cosmo/ Hana/ Paion/ CNS-7056 (ONO- Pharmascience/ R- remimazolam benzodiazepine Procedural sedation IV InTrial 2H2018 Y N 2745) Pharm/ Yichang Humanwell Alnylam/ Ionis/ Sanofi/ FastTrk/ ALN-TTR02 patisiran RNAi therapeutic Polyneuropathy IV 2H2018 Y Y Tekmira Breakthru opioid receptor agonist SL/ buprenorphine buprenorphine Insys Therapeutics Pain InTrial 2H2018 N N (partial) Transmucosal / angiotensin converting / CardiaPill aspirin/ lisinopril/ simvastatin CardioPharma Cardiovascular disease PO InTrial 2H2018 N N HMG Co-A reductase inhibitor gamma-aminobutyric acid Fibromyalgia/ Diabetic DS-5565 mirogabalin Daiichi Sankyo PO InTrial 2H2018 N N (GABA)-ergic analog neuropathic pain (DNP) hexaminolevulinate (hexyl Cervical cancer/ Bladder Cevira Photocure/ Ipsen/ Juno heme precursor Intravesical InTrial 2H2018 Y N aminolevulinate) cancer (imaging) human normal IVIG-SN Green Cross immune globulin (IVIG) Primary immunodeficiency IV CRL 2H2018 Y N immunoglobulin G hydrocodone bitartrate/ opioid receptor agonist/ TV-46763 Teva Pain PO InTrial 2H2018 N N acetaminophen analgesic DRM-04B glycopyrronium tosylate Dermira Hyperhidrosis TOP InTrial 2H2018 N N

P-005672 sarecycline Allergan/ Paratek tetracyclines Acne vulgaris PO InTrial 2H2018 N N

ZP-triptan zolmitriptan Zosano triptans Headache TOP InTrial 2H2018 N N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

Paroxysymal nocturnal hemoglobinuria (PNH)/ ALXN-1210 ALXN-1210 Alexion C-5 monoclonal antibody IV InTrial 2H2018 Y Y Hemolytic uremic syndrome (HUS) -releasing FP-001 (LMIS) leuprolide mesylate Foresee Prostate cancer SC InTrial 2H2018 Y N hormone (GnRH) analog Indivior/ Reckitt RBP-7000 risperidone atypical antipsychotic Schizophrenia SC InTrial 2H2018 Y N Benckiser EUSA/ Aperion/ Endo/ APN-311 beta disialoganglioside Neuroblastoma SC InTrial 2H2018 Y Y Gen Ilac/ Medison Zorblisa allantoin Amicus antioxidant Bacterial infections TOP Filed NDA 2H2018 Y Y sodium-dependent glucose LX-4211 sotagliflozin Sanofi/ Lexicon transporter 1 (SGLT-1) and Diabetes mellitus PO InTrial 2H2018 N N SGLT-2 inhibitor HIV integrase inhibitors/ bictegravir/ emtricitabine/ nucleoside reverse GS-9883/F/TAF Gilead HIV PO Filed NDA 6/12/2018 Y N tenofovir alafenamide transcriptase inhibitors (NRTI)/ NRTI pegylated phenylalanine Peg-Pal BioMarin enzyme replacement Phenylketonuria (PKU) SC Filed BLA 6/29/2018 Y Y ammonia lyase Pulmonary arterial Trevyent trevyent SteadyMed prostacyclin analog SC Filed NDA 7/3/2018 Y Y hypertension (PAH) Melanoma/ Fallopian tube Array BioPharma/ cancer/ Ovarian cancer/ MEK-162 binimetinib MEK inhibitor PO Filed NDA 7/5/2018 Y N Novartis Peritoneal cancer/ Colorectal cancer Array BioPharma/ Pierre rapidly accelerated LGX-818 encorafenib Melanoma PO Filed NDA 7/5/2018 Y Y Fabre Medicament fibrosarcoma (RAF) inhibitor ADX-N05 (ARL-N05, ADX-N05 (ARL-N05, JZP- / phenylalanine derivative Narcolepsy PO InTrial 3Q2018 N Y JZP-110, SKL-N05) 110, SKL-N05) Aerial BioPharma VS-EBOV (rVSV-EBOV; Merck/ NewLink ZEBOV vaccine Ebola IM InTrial 3Q2018 Y N rVSV-ZEBOV-GP) Genetics

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

BAX-817 BAX-817 Baxter factor VIIa stimulant Hemophilia IV InTrial 3Q2018 Y N FastTrk/ NKTR-181 NKTR-181 Nektar opioid agonist Pain PO 3Q2018 N N Breakthru gonadotropin-releasing Abbott/Neurocrine Endometriosis/ Menstrual NBI-56418 hormone (GnRH) receptor PO InTrial 3Q2018 Y N Biosciences bleeding antagonist Chronic lymphocytic phosphatidylinositol 3 kinase IPI-145 (INK-1197) duvelisib AbbVie/ Infinity leukemia (CLL)/ Non- PO InTrial 3Q2018 N Y inhibitor Hodgkin lymphoma (NHL) Sumitomo Dainippon/ non-ergoline dopamine FastTrk/ APL-130277 apomorphine Parkinson's disease PO/SL 3Q2018 N N MonoSol Rx/ Sunovion agonist Breakthru CLS-1001 (CLS-1003, intraocular/ triamcinolone acetonide Clearside corticosteroid Uveitis InTrial 3Q2018 Y N CLS-TA) subretinal thrombopoietin receptor FastTrk/ S-888711 lusutrombopag Shionogi Thrombocytopenia PO 3Q2018 Y N agonist Breakthru Tentative Zenavod doxycycline Dr. Reddy's Laboratories tetracyclines Rosacea PO 8/7/2018 N N Approval c-Met receptor tyrosine ARQ-197 tivantinib ArQule/ Daiichi Sankyo Hepatic cancer PO InTrial 4Q2018 Y N kinase inhibitor cholesterol ester transfer Atherosclerosis/ anacetrapib anacetrapib Merck PO InTrial 4Q2018 N N protein (CETP) inhibitor Hyperlipidemia Galafold (Amigal, alpha-galactosidase A migalastat Amicus/ GSK Fabry disease PO InTrial 4Q2018 Y Y HGT-3310, AT-1001) enhancer FastTrk/ tafenoquine tafenoquine GSK/MMV apoptosis modulator Malaria PO 4Q2018 N Y Breakthru DNA-dependent RNA Zemcolo rifamycin Cosmo/ Dr. Falk Traveler's diarrhea PO InTrial 4Q2018 N N polymerase inhibitor Remoxy oxycodone Pain Therapeutics mu-opioid receptor agonist Pain PO CRL 4Q2018 N N

Zalviso sufentanil, ARX-01 AcelRx opioid analgesic Pain SL CRL 4Q2018 Y N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

sphingosine 1-phosphate 1 Multiple sclerosis/ Ulcerative RPC-1063 ozanimod Celgene (S1PR1) and 5 (S1PR5) PO InTrial 4Q2018 Y N colitis (UC) receptor modulator EVK-001 metoclopramide Evoke Pharma antidopiminergics Diabetic gastroparesis Intranasal InTrial 4Q2018 N Y angiotensin II (synthetic renin-angiotensin peptide LJPC-501 La Jolla Pharmaceutical Hypotension IV InTrial 4Q2018 Y N human) agonist Cystic fibrosis bacterial infections/ Non-cystic Cipro Inhale ciprofloxacin DPI Nektar/ Bayer fluoroquinolone INH InTrial 4Q2018 N Y fibrosis bronchiectasis/ Bacterial infections Asthma/ GST/ Mastocytosis/ Multiple myeloma/ Prostate cancer/ Crohn's disease/ protein tyrosine kinase Multiple sclerosis/ masitinib masitinib AB Science PO InTrial 4Q2018 N Y inhibitor Rheumatoid arthritis/ Pancreatic cancer/ Amyotrophic lateral sclerosis (ALS) Durasert fluocinolone acetonide Alimera/pSivida corticosteroid Posterior uveitis INJ InTrial 4Q2018 Y N non-nucleoside reverse transcriptase inhibitor doravirine/ lamivudine/ MK-1439A Merck (NNRTI)/ Nucleoside reverse HIV PO InTrial 4Q2018 Y N tenofovir disoproxil fumarate transcriptase inhibitor (NRTI)/ NRTI dopamine receptor ALKS-3831 olanzapine/ samidorphan Alkermes antagonist/ opioid receptor Schizophrenia PO InTrial 4Q2018 N N antagonist dexamethasone phosphate EGP-437 EyeGate corticosteroid Uveitis OP InTrial 4Q2018 Y N (iontophoretic) calcitonin gene-related FastTrk/ LY-2951742 galcanezumab Eli Lilly/ Arteaus Headache SC 4Q2018 Y N peptide (CGRP) antagonist Breakthru

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

Short stature/ Growth VRS-317 (hGH-XTEN) somavaratan Versartis/ Amunix growth hormone analog SC InTrial 4Q2018 Y Y hormone deficiency PW-4142 (T-111) nalbuphine ER Trevi Therapeutics/ Endo opioid agonist/ antagonist Prurigo nodularis PO InTrial 4Q2018 N N TD-4208 (GSK- Theravance Biopharma/ Chronic obstructive revefenacin muscarinic antagonist INH InTrial 4Q2018 N N 1160724) pulmonary disease (COPD) Attention deficit hyperactivity SEP-225289 (DSP- Sumitomo Dainippon/ dasotraline triple reuptake inhibitor disorder (ADHD)/ Eating PO InTrial 4Q2018 N N 225289, SEP-289) Sunovion disorders AP-1007 (AP-1431; intercellular adhesion AP-1450; AP-1451; alicaforsen Atlantic Healthcare/ Ionis molecule-1 (ICAM-1) (UC) IV/Rectal Filed NDA 4Q2018 Y Y ISIS-2302) inhibitor FMX-101 (ARK-E021) minocycline Foamix tetracyclines Acne vulgaris/ Rosacea TOP InTrial 4Q2018 N N non-nucleoside reverse MK-1439 doravirine Merck transcriptase inhibitor HIV PO InTrial 4Q2018 Y N (NNRTI) catechol-O- Neurocrine Biosciences/ Ongentys opicapone methyltransferase (COMT) Parkinson disease PO InTrial 4Q2018 N N Bial/ Ono inhibitor human monoclonal anti-IL-1 Acute coronary syndrome ACZ-885 Novartis SC InTrial 4Q2018 Y N beta antibody (ACS) Exocrine pancreatic liprotamase (amylase/lipase/ pancreatic enzyme Sollpura Eli Lilly/ Anthera insufficiency (EPI)/ Low PO CRL 4Q2018 N Y protease) (ALTU-135) replacement therapy (PERT) digestive Sarcoma/ Diffuse large B- Karyopharm selective inhibitor of nuclear cell lymphoma (DLBCL)/ KPT-330 selinexor PO InTrial 4Q2018 Y Y Therapeutics export (SINE) Acute myeloid leukemia (AML)/ Multiple myeloma Hereditary angioedema FastTrk/ DX-2930 Dyax kallikrein inhibitor SC Late 2018 Y Y (HAE) Breakthru Erythropoietic protoporphyria melanocortin receptor 1 Scenesse afamelanotide Clinuvel (EPP)/ Polymorphous light SC implant Filed NDA Late 2018 Y Y (MC-1) agonist eruption (PLE/PMLE)/Vitiligo

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

poly (ADP-ribose) BMN-673 talazoparib BioMarin Breast cancer PO InTrial Late 2018 Y N polymerase (PARP) inhibitor AstraZeneca/ Array selective MEK kinase Thyroid cancer/ Uveal selumetinib selumetinib BioPharma/ Cancer PO InTrial Late 2018 Y Y inhibitor melanoma Research UK / Daiichi Acute myeloid leukemia sapacitabine sapacitabine antimetabolite PO InTrial Late 2018 Y Y Sankyo (AML) astodrimer sodium (SPL- VivaGel Starpharma viral attachment inhibitor Bacterial infections VG Filed NDA Late 2018 N N 7013) GW Pharmaceuticals/ FastTrk/ Sativex cannabinoid product Multiple sclerosis (MS)/ Pain SL/ SPR Late 2018 N N Otsuka Breakthru Multiple sclerosis/ FastTrk/ Nerventra laquinimod Teva immunomodulator PO Late 2018 Y N Huntington's disease Breakthru selective sphingosine-1 BAF-312 siponimod Novartis Multiple sclerosis PO InTrial Late 2018 Y N phosphate receptor agonist selective adenosine A2a tozadenant tozadenant Roche/ UCB Parkinson's disease PO InTrial Late 2018 Y N receptor antagonist Congenital myasthenic Firdapse amifampridine phosphate BioMarin/ Catalyst potassium channel blocker syndrome (CMS)/ Lambert- PO CRL Late 2018 Y Y Eaton myasthenic syndrome osmotic gradient enhancer; Bronchitol mannitol Pharmaxis Asthma/ Cystic fibrosis INH InTrial Late 2018 N Y mucus clearance enhancer long-acting muscarinic acetylcholine receptor AstraZeneca/ Actavis/ Chronic obstructive Duaklir aclidinium/ (mAChR) antagonist/long- INH InTrial Late 2018 N N Circassia pulmonary disease (COPD) acting beta 2 agonist (LAMA/LABA) Tetraphase fully synthetic tetracycline FastTrk/ eravacycline eravacycline Bacterial infections IV/PO Late 2018 Y N Pharmaceuticals antibiotic Breakthru moxetumomab MedImmune CD22 antigen inhibitor Hairy cell leukemia IV InTrial Late 2018 Y Y pasudotox combretastatin A4 vascular targeting agent Ovarian cancer/ FastTrk/ Zybrestat OXiGENE IV Late 2018 Y Y phosphate (fosbretabulin (VTA) Neuroendocrine tumors Breakthru

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

tromethamine) (NET)/ Glioma

Peripheral artery disease AnGes / Mitsubishi FastTrk/ Collategene beperminogene perplasmid angiogenesis (PAD)/ Critical Limb IM Late 2018 Y N Tanabe Breakthru Ischemia (CLI) recombinant human relaxin Reasanz serelaxin Novartis Heart failure IV InTrial Late 2018 Y N (rhRlx) agonist FastTrk/ NeuVax nelipepimut-S Galena vaccine Breast cancer intradermal Late 2018 Y N Breakthru aldoxorubicin aldoxorubicin CytRx anthracycline derivative Soft tissue sarcoma IV InTrial Late 2018 Y Y interleukin-13 (IL-13) Asthma/ Idiopathic CAT-354 AstraZeneca SC InTrial Late 2018 Y Y inhibitor pulmonary fibrosis granulocyte colony- SPI-2012 eflapegrastim Spectrum/ Hanmi Neutropenia SC InTrial Late 2018 Y N stimulating factor (GCSF) bremelanotide melanocortin type 4 receptor PT-141 Palatin Female sexual disorder SC InTrial Late 2018 Y N (bremalanotide) agonist Diffuse large B-cell lymphoma (DLBCL)/ Non- 1 phosphatidylinositol 3 BKM-120 buparlisib Novartis Hodgkin lymphoma (NHL)/ PO InTrial Late 2018 Y N kinase inhibitor Mantle cell lymphoma (MCL)/ Breast cancer Neuroblastoma/ NSCLC/ FastTrk/ entrectinib entrectinib Ignyta tyrosine kinase inhibitor Colorectal cancer/ Solid PO Late 2018 N Y Breakthru tumors FastTrk/ Brabafen fenfluramine Zogenix serotonin receptor agonist Dravet syndrome PO Late 2018 Y Y Breakthru benzhydrocodone/ Apadaz KemPharm opioid receptor agonist Pain PO CRL Late 2018 N N acetaminophen calcitonin gene-related TEV-48125 fremanezumab Teva/ Otsuka Headache SC InTrial Late 2018 Y N peptide (CGRP) antagonist BI-655066 (ABBV- Boehringer Ingelheim/ interleukin-23 (IL-23) Psoriasis/Crohn's disease/ IV/SC InTrial Late 2018 Y Y 066) AbbVie inhibitor Ankylosing

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

spondylitis/Asthma

gamma aminobutyric acid-A Seizures/ Postpartum FastTrk/ SAGE-547 allopregnanolone SAGE/ (GABA-A) receptor allosteric IV Late 2018 Y Y depression Breakthru modulator FastTrk/ Viaskin Peanut Viaskin Peanut DBV Technologies immunotherapy Peanut allergy TOP Late 2018 Y N Breakthru FastTrk/ BC-3781 lefamulin Nabriva Therapeutics pleuromutilins Bacterial infections IV/PO Late 2018 Y N Breakthru sodium-hydrogen RDX-5791 (AZD- Irritable bowel syndrome tenapanor Ardelyx exchanger-3 (NHE-3) PO InTrial Late 2018 N N 1722) (IBS)/ Hyperphosphatemia inhibitor STS sodium thiosulfate Fennec reducing agent Hearing loss IV InTrial Late 2018 Y Y Myelofibrosis/ Geron/ Johnson & Myelodysplastic syndrome GRN-163L imetelstat telomerase inhibitor IV InTrial Late 2018 Y Y Johnson (MDS)/ Acute myelogenous leukemia (AML) dexamethasone sustained- FastTrk/ OTO-104 Otonomy corticosteroid Meniere's disease Intratympanic Late 2018 Y N release Breakthru Ultragenyx/ Kyowa FastTrk/ KRN-23 (UX-023) burosumab fibroblast growth factor Hypophosphatemia IV/SC Late 2018 Y Y Hakko Kirin Breakthru FastTrk/ PTK-0796 omadacycline Paratek aminomethylcyclines (AMC) Bacterial infections IV/PO Late 2018 N N Breakthru Allergy Therapeutics/ Pollinex Quattro Grass birch pollen allergen extract vaccine Allergic rhinitis SC InTrial Late 2018 Y N Endo/ GSK/ Teva Axovant Sciences/ Alzheimer's disease/ Lewy RVT-101 intepirdine GlaxoSmithKline/ 5HT-6 receptor antagonist PO InTrial Late 2018 N N body dementia Roviant Sciences selective macrophage Pigmented Villonodular FastTrk/ PLX108-01 pexidartinib Daiichi Sankyo colony stimulating factor 1 PO Late 2018 N Y Synovitis (PVNS) Breakthru receptor inhibitor PRX-102 alpha galactosidase Protalix enzyme replacement Fabry's disease IV InTrial Late 2018 Y N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

(pegunigalsidase alfa) benznidazole benznidazole KaloBios/ Savant DNA synthesis inhibitor Chagas disease PO InTrial Late 2018 Y N FastTrk/ Zolyd fosfomycin Zavante Therapeutics cell wall inhibitor Bacterial infections IV Late 2018 Y N Breakthru SIGA Technologies/ FastTrk/ Arestvyr tecovirimat antiviral Smallpox IV/PO Late 2018 Y Y PharmAthene Breakthru long-acting beta 2 adrenergic receptor agonist / glycopyrronium (LABA)/ long-acting QVM-149 bromide/ mometasone Novartis/ Sosei Asthma INH InTrial Late 2018 N N muscarinic receptor furoate antagonist (LAMA)/ corticosteroid Intraocular/ RTH-258 brolucizumab Novartis/ Delenex anti-VEGF antibody Macular degeneration Subretinal/ InTrial Late 2018 Y N Subconjunctival GDC-0032 taselisib Roche/ Chugai PI3 Kinase Inhibitor Breast cancer PO InTrial Late 2018+ Y N

FMX-103 minocycline Foamix tetracyclines Rosacea TOP InTrial Late 2018 N N

DFN-02 (DFP-02) DFN-02 (DFP-02) Dr. Reddy's Laboratories triptans Headache Intranasal InTrial Late 2018 N N AM-111 (D-JNKI-1, Hearing loss/ Ocular Intratympanic/ FastTrk/ brimapitide Xigen/ Auris Medical JNK stress kinase inhibitor Late 2018 Y Y XG-102) inflammation Intraocular Breakthru D-threo-methylphenidate Attention deficit hyperactivity KP-415 KemPharm CNS stimulant PO InTrial Late 2018 N N controlled-release disorder (ADHD) interleukin-6 (IL-6) SA-237 (RG-6168) SA-237 (RG-6168) Roche/ Chugai Neuromyelitis optica (NMO) SC InTrial Late 2018+ Y Y monoclonal antibody Diffuse large B-cell lymphoma (DLBCL)/ Acute chimeric antigen receptor FastTrk/ JCAR-017 JCAR-017 Juno/ Celgene lymphocytic leukemia (ALL)/ IV Late 2018 Y Y (CAR) T cell therapy Breakthru Follicular lymphoma/ Mantle cell lymphoma

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

FastTrk/ LOXO-101 larotrectinib Loxo Oncology tyrosine kinase inhibitor Solid tumors/ Sarcoma PO Late 2018 Y Y Breakthru Chronic lymphocytic CD-20 monoclonal antibody/ leukemia (CLL)/ Diffuse TG-1303 / TGR-1202 TG Therapeutics phosphoinositide-3 kinase IV/PO InTrial Late 2018 Y Y large B-cell lymphoma (PI3K) delta inhibitor (DLBCL) Diffuse large B-cell phosphoinositide-3 kinase TGR-1202 TGR-1202 TG Therapeutics/ Rhizen lymphoma (DLBCL)/ Chronic PO InTrial Late 2018 Y Y (PI3K) delta inhibitor lymphocytic leukemia (CLL) MSI-1256F (OHR- FastTrk/ squalamine lactate Ohr angiogenesis inhibitor Macular degeneration OP Late 2018 Y N 102) Breakthru FastTrk/ BHV-4157 trigriluzole Portage Biotech glutamate release inhibitor Spinocerebellar ataxia PO Late 2018 Y Y Breakthru FastTrk/ RVT-802 RVT-802 Enzyvant tissue-based therapy DiGeorge syndrome Undisclosed Late 2018 Y Y Breakthru SEG-101 Novartis P-selectin antagonist Sickle cell disease IV InTrial Late 2018 Y Y recombinant human nerve Neurotrophic keratitis (NK)/ Oxervate cengermin Dompe Intraocular Filed BLA Late 2018 Y Y growth factor (rHNGF) Dry eyes HIV-1 protease inhibitor/ Tentative lopinavir/ritonavir lopinavir/ritonavir Cipla HIV PO Late 2018 Y N CYP3A4 inhibitor Approval FastTrk/ SHP-621 Shire corticosteroid Eosinophilic esophagitis PO 2018 - 2019 Y Y Breakthru 2019 Possible launch date Acute myleoid leukemia FastTrk/ azacitidine azacitidine Celgene DNA methylation inhibitor (AML)/ Myelodysplastic PO 2019 Y Y Breakthru syndromes Clostridium difficile toxoid FastTrk/ ACAM-CDIFF Sanofi Pasteur vaccine Clostridium difficile infection IM 2019 Y N vaccine Breakthru ALS-8176 (ALS- ALS-8176 (ALS-008176, Johnson & Johnson RSV polymerase inhibitor Respiratory syncytial virus PO InTrial 2019 N N 008176, JAL-8176) JAL-8176)

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

pan-fibroblast growth factor JNJ-42756493 (JNJ- Johnson & Johnson/ JNJ-42756493 (JNJ-493) receptor (FGFR) tyrosine Urothelial cancer PO InTrial 2019 Y N 493) Otsuka kinase inhibitor Post-traumatic stress PO/SL/ FastTrk/ TNX-102 SL cyclobenzaprine Tonix/ Vela muscle relaxant 1Q2019 N N disorder (PTSD) Transmucosal Breakthru prostaglandin agonist/ rho / netarsudil Glaucoma/ Ocular Roclatan Aerie kinase-norepinephrine TOP InTrial 1Q2019 N N mesylate hypertension transport protein inhibitor alpha-2/beta-1 adrenergic vascular Ehlers-Danlos Edsivo celiprolol HCl Acer Therapeutics PO InTrial 1Q2019 Y Y agent Syndrome (vEDS) tetrahydrofolate FastTrk/ iclaprim iclaprim Motif Bio Bacterial infections IV 1Q2019 Y N dehydrogenase inhibitor Breakthru Chronic lymphocytic ublituximab (LFB- leukemia (CLL)/ Small cell R603, TG20, TGTX- ublituximab TG Therapeutics CD-20 monoclonal antibody lymphocytic lymphoma IV InTrial 1Q2019 Y Y 1101, TG-1101, (SLL)/ Mantle cell lymphoma Utuxin) (MCL)/ Multiple sclerosis FibroGen/ Astellas/ hypoxia-inducible factor FG-4592 (ASP-1517) roxadustat Anemia PO InTrial Mid-2019 Y N AstraZeneca prolyl hydroxylase (HIF-PHI) Schizophrenia/ Bipolar Intra-Cellular Therapies/ ITI-007 (ITI-722) lumateperone antipsychotic disorder/ Alzheimer's PO InTrial Mid-2019 N N Bristol-Myers Squibb disease (AD) dopamine precursor/ dopa- ND-0612L levodopa/ carbidopa NeuroDerm Parkinson's disease SC InTrial Mid-2019 Y N decarboxylase inhibitor dopamine precursor/ dopa- ND-0612H levodopa/ carbidopa NeuroDerm Parkinson's disease SC InTrial Mid-2019 Y N decarboxylase inhibitor BMN-195 (SMT- Duchenne muscular FastTrk/ ezutromid Summit Therapeutics utrophin inducer PO 2H2019 Y Y C1100) dystrophy (DMD) Breakthru FastTrk/ TTP-488 azeliragon vTv Therapeutics RAGE antagonist Alzheimer's disease PO 2H2019 N N Breakthru factor D monoclonal Age-related macular lampalizumab Roche Intravitreal InTrial 2H2019 Y N antibody degeneration

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

neurally Acting Anti-Migraine LY-573144 lasmiditan CoLucid/ Ill dong/ Eli Lilly Headache PO InTrial 2H2019 Y N Agent (NAAMA) hypoxia-inducible factor- AKB-6548 vadadustat Akebia Therapeutics prolyl hydroxylase (HIF-PH) Anemia PO InTrial 2H2019 Y N inhibitor Rheumatoid arthritis (RA)/ janus associated kinase ABT-494 upadacitinib AbbVie Psoriatic arthritis/ Crohn's PO InTrial 2H2019 N N (JAK) inhibitor disease donepezil transdermal Corplex Corium International anticholinergic Alzheimer's disease TOP InTrial 2H2019 N N system anesthetic/ Nonsteroidal HTX-011 bupivacaine/ meloxicam Heron Therapeutics Anti-inflammatory Drug Pain Undisclosed InTrial 2H2019 N N (NSAID) GlaxoSmithKline/ Tivicay/ Epivir dolutegravir/ lamivudine integrase inhibitor/ NRTI HIV PO InTrial 2H2019 Y N Shionogi selective peroxisome Non-alcoholic steatohepatitis FastTrk/ GFT-505 elafibranor Genfit proliferator-activated (NASH)/ Primary biliary PO 4Q2019 N N Breakthru receptor (PPAR) modulator cirrhosis Attention deficit hyperactivity ADAIR dextroamphetamine Alcobra CNS stimulant PO InTrial 4Q2019 N N disorder (ADHD) Tentative Xyrosa doxycycline Sun Pharma tetracyclines Rosacea PO 4Q2019 N N Approval Lupus nephritis/ Psoriasis/ FastTrk/ Luveniq voclosporin ILJIN calcineurin inhibitor PO Late 2019 Y Y Transplant rejection Breakthru ARYx Therapeutics/ vitamin K epoxide reductase ATI-5923 tecarfarin Anticoagulation PO InTrial Late 2019 N N Armetheon enzyme inhibitor Rheumatoid arthritis/ janus associated kinase 1/2 Olumiant baricitinib Eli Lilly/ Incyte Psoriasis/ Diabetic PO CRL Late 2019 Y N (JAK1/2) inhibitor nephropathy beta-amyloid (Abeta) gantenerumab gantenerumab Roche Alzheimer's disease SC InTrial Late 2019 Y N inhibitor gamma-aminobutyric acid Charcot-Marie Tooth PXT-3003 baclofen/ naltrexone/ sorbitol Pharnext PO InTrial Late 2019 N Y (GABA)-ergic agonist/ opioid disease

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

receptor antagonist/ sorbitol combination End stage renal disease human elastase (ESRD)/Peripheral artery FastTrk/ PRT-201 vonapanitase Proteon Therapeutics TOP Late 2019 Y Y (recombinant) disease (PAD)/ Vascular Breakthru access in hemodialysis Toll-like receptor 9 (TLR9) MGN-1703 MGN-1703 Mologen Colorectal cancer SC InTrial Late 2019 Y N agonist ATP citrate (pro-S)-lyase and stimulating AMP- ETC-1002 bempedoic acid Esperion Therapeutics Hypercholesterolemia PO InTrial Late 2019 N N activated protein kinase (AMPK) antisense DNA GED-0301 mongersen Celgene Crohn's disease PO InTrial Late 2019 N Y oligonucleotide corticosteroid/ long-acting muscarinic receptor Chronic obstructive budesonide/ glycopyrronium/ BGF-MDI AstraZeneca antagonist (LAMA)/ long- pulmonary disease (COPD)/ INH InTrial Late 2019 N N formoterol acting beta 2 adrenergic Asthma receptor agonist (LABA) FastTrk/ AR-101 (AR101) AR-101 (AR101) Aimmune peanut protein capsule Peanut allergy PO Late 2019 Y N Breakthru calcitonin gene-related ALD-403 eptinezumab Alder Headache IV/SC InTrial Late 2019 Y N peptide (CGRP) antagonist AL-335/ odalasvir/ AL-335 (JNJ-4178) Johnson & Johnson NS5B polymerase inhibitor Hepatitis C (HCV) PO InTrial Late 2019 Y N simeprevir Non-small cell lung cancer OSE-2101 tedopi OSE Pharma/ Takeda vaccine SC InTrial Late 2019 Y Y (NSCLC) chemoattractant receptor- QAW-039 (NVP- fevipiprant Novartis homologous molecule Asthma/ PO InTrial Late 2019 Y N QAW-039) (CRTH2) antagonist phosphoinositide 3-kinase Breast cancer/ Head and BYL-719 alpelisib Novartis PO InTrial Late 2019 Y N CA (PIK3CA) inhibitor neck cancer skQ1 visomitin Mitotech plastoquinone derivative Dry eyes OP InTrial Late 2019 Y N

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RxOutlook® 3rd Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug

177 lu-dota-tetulomab (177 lu-tetraxetan-tetulomab, 177 anti-CD37 antibody Non-Hodgkin lymphoma Betalutin Nordic Nanovector IV InTrial Late 2019 Y Y lu- satetraxetan, radionuclide (NHL) 177 lu-DOTA-HH1) RG-7916 (RO- Roche/ PTC RG-7916 (RO-7034067) SMN2 splicing modifier Spinal muscular atrophy PO InTrial Late 2019 Y Y 7034067) Therapeutics digitalis-like factor FastTrk/ DigiFab digoxin immune fab AMAG/ Velo Preeclampsia IV Late 2019 Y Y antagonist Breakthru dehydrated human Plantar fasciitis/ Achilles AmnioFix amnion/chorion membrane MiMedx amniotic tissue membrane TOP InTrial Late 2019 Y N tendonitis (dHACM) MABT-5102A crenezumab Roche/ AC Immune amyloid beta-protein inhibitor Alzheimer's disease IV/SC InTrial 12/1/2019 Y N

IM = intramuscular, INH = inhalation, INJ = injection, IUD = intrauterine device, IV = intravenous, OP = ophthalmic, PO = oral, SC = subcutaneous, SL = sublingual, SPR = spray, TOP = topical, VG = vaginal

optum.com/optumrx

The information contained herein is compiled from various sources and is provided for informational purposes only. Due to factors beyond the control of OptumRx, information related to prospective drug launches is subject to change without notice. This information should not be solely relied upon for formulary decision-making purposes.

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RxOutlook® is published by the OptumRx Clinical Services Department.

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