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RxOutlook® 4th Quarter 2017

OptumRx brand pipeline forecast

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

2017 Possible launch date sodium zirconium ZS-9 ZS Pharma potassium-binding agent Hyperkalemia PO CRL 4Q2017 N N cyclosilicate recombinant human LR-769 eptacog beta LFB SA Hemophilia IV Filed BLA 4Q2017 Y N Factor VIIa FMX-102 (FXFM-244) Foamix tetracyclines Impetigo TOP InTrial 4Q2017 N N

MK-8228 letermovir Merck quinazoline Cytomegalovirus IV/PO Filed NDA 11/8/2017 N Y Heron neurokinin-1 (NK1) Cinvanti Nausea and vomiting IV Filed NDA 11/12/2017 N N Therapeutics antagonist Mucopolysaccharidosis UX-003 (rhGUS) beta-glucuronidase replacement IV Filed BLA 11/16/2017 Y Y VII Otsuka/ Proteus Schizophrenia/ Bipolar/ Abilify atypical PO Filed NDA 11/23/2017 N N Digital Health Major Depression receptor RBP-6000 depot Indivior/ QLT Drug addiction SC Filed NDA 11/30/2017 Y N (partial) non- reverse Human GlaxoSmithKline/ transcriptase inhibitor/ rilpivirine/ dolutegravir rilpivirine/ dolutegravir immunodeficiency virus PO Filed NDA 12/1/2017 Y N J&J/ ViiV Integrase strand transfer (HIV) inhibitor ertugliflozin ertugliflozin Merck/ SGLT2 inhibitor diabetes mellitus PO Filed NDA 12/3/2017 N N

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RxOutlook® 4th Quarter 2017

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

SGLT-2 inhibitor/ ertugliflozin/ sitagliptin ertugliflozin/ sitagliptin Merck/ Pfizer dipeptidyl peptidase-4 Diabetes mellitus PO Filed NDA 12/3/2017 N N (DPP-4) inhibitor

ertugliflozin/ ertugliflozin/ metformin Merck/ Pfizer SGLT2 inhibitor/ biguanide Diabetes mellitus PO Filed NDA 12/3/2017 N N metformin glucagon-like -1 semaglutide semaglutide Novo Nordisk Diabetes mellitus SC Filed NDA 12/5/2017 Y N (GLP-1) receptor agonist long-acting muscarinic SUN-101 (EP-101) glycopyrrolate Sunovion COPD INH Filed NDA 12/15/2017 N N mammalian target of Opsiria sirolimus Santen rapamycin (mTOR) Uveitis Intravitreal Filed NDA 12/24/2017 Y Y inhibitor ethinyl / Agile hormonal combination Twirla Pregnancy prevention TOP Filed NDA 12/26/2017 N N Therapeutics contraceptive Clarus Jatenzo Hypogonadism PO Filed NDA 12/27/2017 N N Therapeutics Luminesse brimonidine tartrate Bausch and Lomb alpha-2 agonist Ocular rednes OP Filed NDA 12/27/2017 N N Macrilen acetate Aeterna Zentaris agonist PO Filed NDA 12/30/2017 Y Y deficiency lubiprostone Takeda/ Sucampo chloride channel activator Constipation PO InTrial Late 2017 N N Tarsa/ Unigene Tbria salmon calcitonin osteoclast inhibitor Osteoporosis PO Filed NDA Late 2017 N N Laboratories Non-small cell lung FastTrk/ MVA-MUC1-IL2 TG-4010 Transgene vaccine SC Late 2017 N N cancer (NSCLC) Breakthru arbaclofen extended- Ontinua ER Osmotica muscle relaxant Spasticity PO Filed NDA Late 2017 Y N release Aclaris A-101 (V-101) hydrogen peroxide peroxide Benign pigmented lesions TOP Filed NDA Late 2017 Y N Therapeutics

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RxOutlook® 4th Quarter 2017

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

interleukin-5 receptor benralizumab benralizumab AstraZeneca / COPD SC Filed BLA Late 2017 Y N alpha inhibitor Dengvaxia Dengue fever vaccine Sanofi Pasteur vaccine Dengue fever SC Filed BLA Late 2017 N N / Aft Maxigesic acetaminophen/ NSAID Pain PO Filed NDA Late 2017 N N Pharmaceuticals 2018 Possible launch date Non-small cell lung PF-00299804 dacomitinib Pfizer pan-HER inhibitor PO InTrial 2018 Y N cancer (NSCLC) Ovarian cancer/ Non- Hodgkin's lymphoma (NHL)/ Small cell lung MLN-8237 alisertib Millennium aurora kinase A inhibitor PO InTrial 2018 Y Y cancer (SCLC)/ Breast cancer/ Peripheral T-cell lymphoma selective -A Xinlay AbbVie Diabetic nephropathy PO InTrial 2018 Y N receptor antagonist Transition scyllo- beta-amyloid (Abeta) Alzheimer's disease/ scyllo-cyclohexanehexol Therapeutics/ PO InTrial 2018 N N cyclohexanehexol oligomerization inhibitor Down's syndrome Niemann-Pick Disease/ FastTrk/ arimoclomol arimoclomol CytRx cytoprotectives Amyotrophic lateral PO 2018 Y Y Breakthru sclerosis phosphatidylserine Non-small cell lung FastTrk/ Tarvacin bavituximab Peregrine IV 2018 Y N receptor antagonist cancer (NSCLC) Breakthru bone marrow-derived Bone PREOB mesenchymal stem cell stem cell therapy Fracture/ Bone necrosis Intraosseus InTrial 2018 Y N Therapeutics therapy DSC-127 aclerastide Derma Sciences angiotensin II analog Diabetic foot ulcers TOP InTrial 2018 N N

KAE-609 cipargamin protein synthesis inhibitor Malaria PO InTrial 2018 N N

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RxOutlook® 4th Quarter 2017

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

aldosterone synthase LCI-699 Novartis Cushing's syndrome PO InTrial 2018 N Y inhibitor Primary IVIG IVIG Prometic immune globulin IV InTrial 2018 Y N immunodeficiency ALKS-8700 (RDC-1559) ALKS-8700 (RDC-1559) prodrug Multiple sclerosis PO InTrial 2018 Y N Major depressive FastTrk/ esketamine Janssen NMDA receptor antagonist Intranasal 2018 N N disorder (MDD) Breakthru FastTrk/ SHP-621 Shire Eosinophilic esophagitis PO 2018 - 2019 Y Y Breakthru United Pulmonary arterial RemoSynch analog IV/SC Filed NDA 1H2018 Y Y Therapeutics hypertension (PAH) Immune Remune HIV-1 vaccine Response vaccine HIV IM Filed BLA 1H2018 Y Y BioPharma Xeglyze abametapir Hatchtech metalloprotease inhibitor Lice TOP CRL 1H2018 N N

TX-004HR, TX-12-004HR estradiol TherapeuticsMD receptor agonist Vaginal atrophy VG CRL 1H2018 N N

PSD-502 lidocaine/ prilocaine Plethora/Sciele sodium Premature ejaculation TOP InTrial 1Q2018 N N

HEPLISAV-B HBV vaccine Merck/ Dynavax vaccine Hepatitis B IM Filed BLA 1Q2018 N N anti-sclerostin monoclonal Evenity romosozumab Osteoporosis SC CRL 1Q2018 Y N antibody Breast cancer/ Pancreatic cancer/ Pancreatic cancer/ Small cell lung RS7-SN-38 antibody-drug cancer (SCLC)/ Non-small FastTrk/ IMMU-132 sacituzumab govitecan Seattle Genetics IV 1Q2018 Y Y conjugate cell lung cancer (NSCLC)/ Breakthru Colorectal cancer/ Esophageal cancer/ Urinary bladder cancer

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RxOutlook® 4th Quarter 2017

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

Advaxis/ Biocon/ FusionVax/ Anal cancer/ Cervical FastTrk/ ADXS-HPV axalimogene filolisbac Sorrento vaccine cancer/ Head and neck IV 1Q2018 Y Y Breakthru Therapeutics/ cancer Taiwan Biotech furoate/ corticosteroid/ GSP-301 Glenmark Allergic rhinitis NA InTrial 1Q2018 N N HCl -donating Bausch and Glaucoma/ Ocular Vesneo (Vyzulta) latanoprostene bunod F2-alpha TOP Filed NDA 1Q2018 N N Lomb/ NicOx hypertension analog Ocular Pain/ Allergic Dextenza corticosteroid OP CRL 1Q2018 N N sustained-release Therapeutix conjunctivitis Blastic plasmacytoid dendritic dell neoplasm/ Acute mylegenous leukemia/ Myelodysplastic Stemline interleukin-3 receptor syndrome/ Multiple SL-401 SL-401 IV InTrial 1Q2018 Y Y Therapeutics antagonist myeloma/ Hypereosinophilic syndrome/ Myelofibrosis/ Chronic myelomonocytic leukemia / Charleston anti-emetic/ opioid/ CL-108 / Nausea/ Vomiting/ Pain PO Filed NDA 1Q2018 N N Laboratories analgesic acetaminophen Evosyal A Alphaeon botulinum toxins Glabellar lines/ Spasticity IM Filed BLA 1Q2018 Y N Theratechnologie Human TNX-355 ibalizumab s/ / Roche/ viral entry inhibitor immunodeficiency virus IM/IV/SC Filed sBLA 1/3/2018 Y Y TaiMed (HIV)

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RxOutlook® 4th Quarter 2017

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

RESOLVE (Sinuva) mometasone furoate Intersect ENT corticosteroid Chronic sinusitis Implant Filed NDA 1/7/2018 Y N Spark Luxturna voretigene neparvovec gene therapy Retinal disease Intraocular Filed BLA 1/12/2018 Y Y Therapeutics Camurus/ agonist CAM-2038 buprenorphine Opioid dependence/ Pain SC Filed NDA 1/19/2018 Y N Braeburn (partial) Cystic fibrosis/ Non-cystic Linhaliq Aradigm/ Grifols fluoroquinolone INH Filed NDA 1/26/2018 Y Y fibrosis bronchiectasis [177]Lutetium- DOTA[0]-Tyr[3]- receptor Gastroenteropancreatic Lutathera Novartis INJ Filed NDA 1/26/2018 Y Y Octreotate (177-Lu- agonist neuroendocrine tumors Dotatate) Portola/ Pfizer/ recombinant Factor Xa AndexXa andexanet alfa Bristol-Myers Reversal agent IV Filed NDA 2/2/2018 Y N inhibitor antidote Squibb Tlando testosterone Lipocine androgen Hypogonadism PO Filed NDA 2/8/2018 N N dexamethasone Dexycu Icon Bioscience corticosteroid Ocular inflammation Intraocular Filed NDA 2/12/2018 N/A N controlled-release HIV integrase inhibitors/ bictegravir/ Human nucleoside reverse GS-9883/F/TAF emtricitabine/ Gilead immunodeficiency virus PO Filed NDA 2/12/2018 Y N transcriptase inhibitors tenofovir alafenamide (HIV) (NRTI)/ NRTI hydroxyprogesterone Makena Lumara Health progestin Preterm labor SC Filed sNDA 2/14/2018 Y Y caproate RayVa (Vitaros) alprostadil / Apricus Erectile dysfunction TOP Filed NDA 2/17/2018 N N agonist benzhydrocodone/ Apadaz KemPharm opioid receptor agonist Pain PO Filed NDA 2/23/2018 N N acetaminophen factor IXa/ factor X bi- ACE-910 emicizumab Roche/ Chugai Hemophilia SC Filed BLA 2/23/2018 Y Y specific antibody

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RxOutlook® 4th Quarter 2017

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

angiotensin II La Jolla renin-angiotensin peptide LJPC-501 Hypotension IV Filed NDA 2/28/2018 Y N (synthetic human) Pharmaceutical agonist rho kinase- Glaucoma/ Ocular Rhopressa netarsudil mesylate Aerie TOP Filed NDA 2/28/2018 N N transport protein inhibitor hypertension corrector ivacaftor/ tezacaftor ivacaftor/ tezacaftor Vertex Cystic fibrosis PO Filed NDA 2/28/2018 Y Y modulator ZTlido lidocaine Scilex sodium channel blocker Postherpetic neuralgia TOP Filed NDA 2/28/2018 N N

Yonsa abiraterone acetate Churchill/ Iroko kinase inhibitor Prostate cancer PO Filed NDA 3/19/2018 Y N IntelGenx / Red VersaFilm Headache PO CRL 2Q2018 N N Hill Biopharma Zoptrex zoptarelin doxorubicin Aeterna Zentaris anthracycline derivative Endometrial cancer IV InTrial 2Q2018 Y N Acute myeloid leukemia/ anti-CD45 monoclonal- anti-CD45 monoclonal Iomab-B Actinium Myelodysplastic IV InTrial 2Q2018 Y Y antibody BC8-I-131 antibody syndrome Amyotrophic lateral FastTrk/ CK-2017357 (CK-357) tirasemtiv Cytokinetics troponin activator PO 2Q2018 Y Y sclerosis Breakthru Vernalis/ Tris CCP-07 CCP-07 undisclosed Cough and cold PO CRL 2Q2018 N N Pharma Vernalis/ Tris CCP-08 CCP-08 undisclosed Viral rhinitis PO CRL 2Q2018 Y N Pharma Radiation poisoning/ FastTrk/ Protectan CBLB-502 entolimod BioLabs TLR5 SC 2Q2018 Y Y Colorectal cancer Breakthru AbbVie/ gonadotropin-releasing Endometriosis/ NBI-56418 Neurocrine hormone (GnRH) receptor PO Filed NDA 2Q2018 Y N Menstrual bleeding Biosciences antagonist selective NK1 receptor / Helsinn Group/ Akynzeo antagonist/ 5-HT3 Nausea and vomiting IV Filed sNDA 2Q2018 N N Eisai receptor antagonist

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RxOutlook® 4th Quarter 2017

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

selective Ella Actavis () Uterine bleeding PO Filed NDA 2Q2018 N N receptor modulator ProMetic/ Ryplazim human plasminogen plasminogen Plasminogen deficiency IV Filed BLA 4/14/2018 Y Y Hematech Immune Tavalisse fostamatinib Rigel oral SYK inhibitor PO Filed NDA 4/17/2018 Y Y thrombocytopenia Ultragenyx/ KRN-23 burosumab Kyowa Hakko fibroblast growth factor Hypophosphatemia IV/SC Filed BLA 4/17/2018 Y Y Kirin polyethylene glycol-based Plenvu NER-1006 Norgine Bowel preparation PO Filed NDA 4/28/2018 N N preparation / / EB-178 Astellas beta-3 receptor Overactive bladder PO Filed sNDA 4/28/2018 N N mirabegron agonist Inbrija levodopa Acorda antiparkinson Parkinson's disease INH CRL 5/3/2018 N N calcitonin gene-related Aimovig Amgen Headache SC Filed BLA 5/17/2018 Y N peptide (CGRP) antagonist tacrolimus (extended- Astagraf XL Astellas immunosuppressant Organ transplantation PO Filed sNDA 5/18/2018 Y N release) LuMeBlue (CB-17-01) Cosmo imaging dye Colonoscopy PO Filed NDA 5/21/2018 N N tildrakizumab tildrakizumab interleukin-23 (IL-23) Merck Psoriasis SC Filed BLA 5/23/2018 Y N (MK-3222) (MK-3222) inhibitor nonsteroidal anti- Recro Pharma/ N-1539 inflammatory drug Pain IV Filed NDA 5/26/2018 Y N Alkermes (NSAID) Peg-Pal pegvaliase BioMarin enzyme replacement Phenylketonuria SC Filed BLA 5/28/2018 Y Y NSAID/ calcium channel Hypertension/ KIT-302 / amlodipine Kitov PO Filed NDA 5/31/2018 N N blocker Osteoarthritis

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RxOutlook® 4th Quarter 2017

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

ITCA-650 (sustained sustained- Intarcia/ glucagon-like peptide-1 Diabetes mellitus SC implant CRL 2H2018 Y N release exenatide) release Quintiles/ Servier (GLP-1) receptor agonist

MAP Semprana Pharmaceuticals/ dihydroergotamine Headache INH InTrial 2H2018 N N mesylate Allergan Resolor Shire 5-HT-4 receptor agonist Chronic constipation PO InTrial 2H2018 N N telapristone acetate Repros/ Gedeon progesterone receptor Uterine fibroids/ Proellex PO InTrial 2H2018 N N (CBD4124) Richter antagonist Endometriosis Clostridium difficile FastTrk/ cadazolid cadazolid quinolonyl-oxazolidinone PO 2H2018 N Y infection Breakthru Rheumatoid arthritis/ janus associated kinase Olumiant baricitinib Eli Lilly/ Incyte Psoriasis/ Diabetic PO CRL 2H2018 Y N 1/2 (JAK1/2) inhibitor nephropathy Alzheimer's disease/ LMTX methylthioninum Roche apoptosis inhibitor PO InTrial 2H2018 N Y Blepharitis/ Ocular Dexasite dexamethasone InSite Vision corticosteroid TOP InTrial 2H2018 N N inflammation Neurotrophic keratitis/ RGN-259 thymosin beta 4 RegeneRx actin regulating peptide TOP InTrial 2H2018 N Y Dry eyes Non-small cell lung HyperAcute Lung tergenpantucel-L NewLink Genetics immunotherapy IV InTrial 2H2018 Y N cancer (NSCLC) Benign prostatic NX-1207 fexapotide triflutate Nymox pro-apoptotic hyperplasia/ Prostate Intratumoral InTrial 2H2018 Y N cancer FastTrk/ Zeftera ceftobiprole Basilea cephalosporin antibiotic Bacterial infections IV 2H2018 Y N Breakthru calcium sensitizing Septic shock/ Heart Tenax FastTrk/ Simdax levosimendan phosphodiesterase failure/ Low cardiac IV 2H2018 Y N Therapeutics Breakthru inhibitor output syndrome (LCOS)

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RxOutlook® 4th 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/ cytotoxic T lymphocyte- Solid tumors/ tremelimumab tremelimumab AstraZeneca associated antigen 4 Mesothelioma/ Non- IV InTrial 2H2018 Y Y inhibitor small cell lung cancer (NSCLC) Cosmo/ Hana/ Paion/ CNS-7056 (ONO-2745) remimazolam Pharmascience/ benzodiazepine Procedural sedation IV InTrial 2H2018 Y N R-Pharm/ Yichang Humanwell / angiotensin aspirin/ lisinopril/ converting enzyme CardiaPill CardioPharma Cardiovascular disease PO InTrial 2H2018 N N simvastatin inhibitor/ 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 Photocure/ Cervical cancer/ Bladder Cevira hexaminolevulinate heme precursor Intravesical InTrial 2H2018 Y N Ipsen/ Juno cancer (imaging) human normal Primary IVIG-SN Green Cross immune globulin IV CRL 2H2018 Y N immunoglobulin G immunodeficiency hydrocodone opioid receptor agonist/ TV-46763 bitartrate/ Teva Pain PO InTrial 2H2018 N N analgesic acetaminophen glycopyrronium DRM-04B Dermira Hyperhidrosis TOP InTrial 2H2018 N N tosylate P-005672 sarecycline Allergan/ Paratek tetracyclines Acne vulgaris PO InTrial 2H2018 N N

ZP-triptan Zosano Headache TOP InTrial 2H2018 N N naloxone Amphastar Opioid dependence Intranasal CRL 2H2018 N N

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RxOutlook® 4th 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/ ALXN-1210 ALXN-1210 Alexion C-5 monoclonal antibody IV InTrial 2H2018 Y Y Hemolytic uremic syndrome gonadotropin-releasing FP-001 (LMIS) leuprolide mesylate Foresee Prostate cancer SC InTrial 2H2018 Y N hormone (GnRH) analog Insys opioid receptor agonist SL/ buprenorphine buprenorphine Pain Filed NDA 2H2018 N N Therapeutics (partial) Transmucosal EUSA/ Aperion/ APN-311 dinutuximab beta Endo/ Gen Ilac/ disialoganglioside Neuroblastoma SC InTrial 2H2018 Y Y Medison Parkinson's disease (PD) Nuplazid Acadia 5-HT-2A receptor agonist PO InTrial 2H2018 Y N psychosis sodium-dependent glucose transporter 1 LX-4211 sotagliflozin Sanofi/ Lexicon Diabetes mellitus PO InTrial 2H2018 N N (SGLT-1) and SGLT-2 inhibitor Head and Neck cancer/ Multikine Leukocyte Interleukin CEL-SCI immunomodulator SC InTrial Mid-2018 Y Y Squamous cell carcinoma Bilirubin Stanate stannsoporfin InfaCare heme oxygenase inhibitor encephalopathy/ IM Filed NDA Mid-2018 Y N Hyperbilirubemia receptor APD-421 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

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RxOutlook® 4th Quarter 2017

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

Pulmonary arterial Trevyent trevyent SteadyMed prostacyclin analog SC CRL Mid-2018 Y Y hypertension Sun Pharma Advanced Glaucoma/ Ocular Xelpros prostaglandin agonist OP CRL Mid-2018 N N Research hypertension Company Primatene HFA epinephrine Amphastar catecholamine Asthma INH CRL Mid-2018 N N

IDP-122 halobetasol propionate Valeant corticosteroid Psoriasis TOP InTrial Mid-2018 N N next-generation plazomicin plazomicin Achaogen/ Ionis Bacterial infections IV Filed NDA Mid-2018 Y N aminoglycoside Melanoma/ Fallopian Array BioPharma/ tube cancer/ Ovarian MEK-162 binimetinib MEK inhibitor PO Filed NDA 6/30/2018 Y N Novartis cancer/ Peritoneal cancer/ Colorectal cancer GW Epidiolex product Seizures PO Filed NDA 6/30/2018 Y Y Pharmaceuticals Array BioPharma/ rapidly accelerated LGX-818 encorafenib Pierre Fabre Melanoma PO Filed NDA 6/30/2018 Y Y fibrosarcoma inhibitor Medicament tazarotene/ IDP-118 Valeant / corticosteroid Psoriasis TOP Filed NDA 7/5/2018 N N halobetasol Indivior/ Reckitt RBP-7000 Schizophrenia SC Filed NDA 3Q2018 Y N Benckiser Jazz ADX-N05 (ARL-N05, JZP- ADX-N05 (ARL-N05, Pharmaceuticals/ derivative Narcolepsy PO InTrial 3Q2018 N Y 110, SKL-N05) JZP-110, SKL-N05) Aerial BioPharma VS-EBOV (rVSV-EBOV; Merck/ NewLink ZEBOV vaccine Ebola IM InTrial 3Q2018 Y N rVSV-ZEBOV-GP) Genetics BAX-817 BAX-817 Baxter factor VIIa stimulant Hemophilia IV InTrial 3Q2018 Y N

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RxOutlook® 4th Quarter 2017

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

ALNCD Alkermes antipsychotic Schizophrenia IV Filed NDA 3Q2018 Y N NanoCrystal Dispersion loteprednol etabonate Dry eyes/ Ocular Inveltys Kala corticosteroid TOP Filed NDA 3Q2018 N N MPP inflammation Familial chylomicronemia ISIS 304801 volanesorsen Ionis antisense drug SC Filed NDA 3Q2018 Y Y syndrome/ Lipodystrophy Sumitomo Attention deficit SEP-225289 dasotraline Dainippon/ triple inhibitor hyperactivity disorder PO Filed NDA 3Q2018 N N Sunovion (ADHD)/ Eating disorders Sumitomo Dainippon/ non- dopamine FastTrk/ APL-130277 Parkinson's disease PO/SL 3Q2018 N N MonoSol Rx/ agonist Breakthru Sunovion intraocular/ CLS-1001 Clearside corticosteroid Uveitis InTrial 3Q2018 Y N acetonide subretinal Thrombocytopenia/ thrombopoietin receptor Immune AKR-501 avatrombopag Astellas/ Dova PO Filed NDA 3Q2018 Y N agonist thrombocytopenic purpura (ITP) darunavir/ Human emtricitabine/ protease inhibitor/ NRTI/ Symtuza Janssen immunodeficiency virus PO Filed NDA 3Q2018 Y N cobicistat/ tenofovir boosting agent/ NRTI (HIV) alafenamide Dr. Reddy's Tentative Zenavod doxycycline tetracyclines Rosacea PO 8/7/2018 N N Laboratories Approval BAY94-9027 damoctocog alfa pegol Bayer anithemophilic factor Hemophilia IV Filed BLA 8/31/2018 Y N estrogen receptor estradiol/ TX-001HR TherapeuticsMD agonist/progesterone Menopause PO InTrial 4Q2018 N N progesterone receptor agonist ArQule/ Daiichi c-Met receptor tyrosine ARQ-197 tivantinib Hepatic cancer PO InTrial 4Q2018 Y N Sankyo kinase inhibitor

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RxOutlook® 4th Quarter 2017

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

alpha-galactosidase A FastTrk/ Galafold migalastat Amicus/ GSK Fabry disease PO 4Q2018 Y Y enhancer Breakthru 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 mu-opioid receptor Remoxy Pain Therapeutics Pain PO CRL 4Q2018 N N agonist Zalviso sufentanil, ARX-01 AcelRx opioid analgesic Pain SL CRL 4Q2018 Y N 1-phosphate 1 Multiple sclerosis/ RPC-1063 ozanimod Celgene (S1PR1) and 5 (S1PR5) PO InTrial 4Q2018 Y N Ulcerative colitis receptor modulator RNA polymerase inhibitor/ rifabutin/ amoxicillin/ RedHill FastTrk/ Talicia / proton pump Bacterial infections PO 4Q2018 N N pantoprazole Biopharma Breakthru inhibitor Non-cystic fibrosis bronchiectasis/ Cystic Cipro Inhaled ciprofloxacin DPI Nektar/ Bayer fluoroquinolone INH InTrial 4Q2018 N Y fibrosis bacterial infections Asthma/ GST/ Mastocytosis/ Multiple myeloma/ Prostate cancer/ Crohn's disease/ protein tyrosine kinase masitinib masitinib AB Science Multiple sclerosis/ PO InTrial 4Q2018 N Y inhibitor Rheumatoid arthritis/ Pancreatic cancer/ Amyotrophic lateral sclerosis ARX-04 sufentanil AcelRx opioid analgesic Pain SL CRL 4Q2018 Y N

Durasert fluocinolone acetonide Alimera/pSivida corticosteroid Posterior uveitis INJ InTrial 4Q2018 Y N

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RxOutlook® 4th Quarter 2017

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

non-nucleoside reverse doravirine/ transcriptase inhibitor Human MK-1439A lamivudine/ tenofovir Merck (NNRTI)/ Nucleoside immunodeficiency virus PO InTrial 4Q2018 Y N disoproxil fumarate reverse transcriptase (HIV) inhibitor (NRTI)/ NRTI dopamine receptor / Schizophrenia/ Bipolar ALKS-3831 Alkermes antagonist/ opioid PO InTrial 4Q2018 N N samidorphan disorder receptor antagonist Alnylam/ Ionis/ FastTrk/ ALN-TTR02 patisiran RNAi therapeutic Polyneuropathy IV 4Q2018 Y Y Sanofi/ Tekmira Breakthru dexamethasone EGP-437 phosphate 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 Short stature/ Growth VRS-317 somavaratan Versartis/ Amunix growth hormone analog SC InTrial 4Q2018 Y Y hormone deficiency Trevi PW-4142 ER Therapeutics/ opioid agonist/ antagonist Prurigo nodularis PO InTrial 4Q2018 N N Endo Theravance TD-4208 revefenacin Biopharma/ muscarinic antagonist COPD INH InTrial 4Q2018 N N FastTrk/ TRV-130 oliceridine Trevena opioid receptor agonist Pain IV 4Q2018 N N Breakthru intercellular adhesion Atlantic AP-1007 alicaforsen molecule-1 (ICAM-1) Ulcerative colitis IV/Rectal Filed NDA 4Q2018 Y Y Healthcare/ Ionis inhibitor Acute lymphocytic EZN-2285 calaspargase pegol Baxalta enzyme therapy IM/IV InTrial 4Q2018 Y N leukemia FMX-101 (ARK-E021) minocycline Foamix tetracyclines Acne vulgaris/ Rosacea TOP InTrial 4Q2018 N N

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RxOutlook® 4th Quarter 2017

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

non-nucleoside reverse Human MK-1439 doravirine Merck transcriptase inhibitor immunodeficiency virus PO InTrial 4Q2018 Y N (NNRTI) (HIV) Neurocrine catechol-O- Ongentys opicapone Biosciences/ Bial/ methyltransferase (COMT) Parkinson disease PO InTrial 4Q2018 N N Ono inhibitor human monoclonal anti-IL- ACZ-885 canakinumab Novartis Acute coronary syndrome SC InTrial 4Q2018 Y N 1 beta antibody phosphodiesterase-5 VersaFilm tadalafil VersaFilm IntelGenx Erectile dysfunction PO InTrial 4Q2018 Y N (PDE-5) inhibitor JNJ-56021927 Janssen Prostate cancer PO Filed NDA 4Q2018 N N antagonist calcitonin gene-related TEV-48125 fremanezumab Teva/ Otsuka Headache SC Filed BLA 4Q2018 Y N peptide (CGRP) antagonist liprotamase Exocrine pancreatic pancreatic enzyme Sollpura (amylase/lipase/ Eli Lilly/ Anthera insufficiency/ Low PO CRL 4Q2018 N Y replacement therapy protease) digestive Sarcoma/ Diffuse large B- Karyopharm selective inhibitor of cell lymphoma/ Acute KPT-330 selinexor PO InTrial 4Q2018 Y Y Therapeutics nuclear export myeloid leukemia/ Multiple myeloma FastTrk/ DX-2930 lanadelumab Dyax kallikrein inhibitor Hereditary angioedema SC Late 2018 Y Y Breakthru Erythropoietic melanocortin receptor 1 protoporphyria/ Scenesse afamelanotide Clinuvel SC implant Filed NDA Late 2018 Y Y (MC-1) agonist Polymorphous light eruption/ Vitiligo aspartyl-alanyl Ampio Ampion diketopiperazine (DA- immunomodulator Osteoarthritis Intra-articular InTrial Late 2018 Y N Pharmaceuticals DKP)

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RxOutlook® 4th Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug human plasminogen human plasminogen Kedrion plasminogen Plasminogen deficiency TOP InTrial Late 2018 Y Y poly (ADP-) BMN-673 talazoparib BioMarin polymerase (PARP) Breast cancer PO InTrial Late 2018 Y N inhibitor AstraZeneca/ Array BioPharma/ selective MEK kinase Thyroid cancer/ Uveal selumetinib selumetinib PO InTrial Late 2018 Y Y Cancer Research inhibitor melanoma UK Cyclacel / Daiichi sapacitabine sapacitabine antimetabolite Acute myeloid leukemia PO InTrial Late 2018 Y Y Sankyo growth hormone Helsinn / Novo Anorexia/ Cachexia FastTrk/ Adlumiz secretagogue receptor PO Late 2018 Y N Nordisk (cancer-related) Breakthru (GHSR) agonist brincidofovir DNA-directed DNA Adenovirus/ FastTrk/ CMX-001 hexadecyloxypropyl Chimerix PO Late 2018 N N polymerase inhibitor Cytomegalovirus Breakthru ester VivaGel astodrimer sodium Starpharma viral attachment inhibitor Bacterial infections VG Filed NDA Late 2018 N N

CEM-101 solithromycin Cempra macrolide Bacterial infections IV/PO CRL Late 2018 N N FastTrk/ BAY-41-6551 amikacin Nektar/ Bayer aminoglycoside Bacterial infections INH Late 2018 Y N Breakthru GW FastTrk/ Sativex Pharmaceuticals/ cannabinoid product Multiple sclerosis/ Pain SL/ SPR Late 2018 N N Breakthru Otsuka Multiple sclerosis/ FastTrk/ Nerventra laquinimod Teva immunomodulator PO Late 2018 Y N Huntington's disease Breakthru selective sphingosine-1 BAF-312 Novartis phosphate receptor Multiple sclerosis PO InTrial Late 2018 Y N agonist

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RxOutlook® 4th Quarter 2017

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

opioid receptor agonist buprenorphine/ Major depressive ALKS-5461 Alkermes (partial)/ opioid receptor PO Filed NDA Late 2018 N N samidorphan disorder antagonist FastTrk/ midazolam (USL-261) midazolam (USL-261) Upsher-Smith benzodiazepine Seizures Intranasal Late 2018 N Y Breakthru selective A2a tozadenant tozadenant Roche/ UCB Parkinson's disease PO InTrial Late 2018 Y N receptor antagonist Kyowa Hakko A2A adenosine receptor Nouriast Parkinson's disease PO CRL Late 2018 N N Kogyo/ Biovail antagonist Congenital myasthenic amifampridine BioMarin/ syndrome/ Lambert- Firdapse potassium channel blocker PO CRL Late 2018 Y Y phosphate Catalyst Eaton myasthenic syndrome protein absorption FastTrk/ CM-AT CM-AT Curemark Autism PO Late 2018 Y N enhancer Breakthru osmotic gradient Bronchitol mannitol Pharmaxis enhancer; mucus Asthma/ Cystic fibrosis INH InTrial Late 2018 N Y clearance enhancer long-acting muscarinic receptor AstraZeneca/ Duaklir aclidinium/ antagonist/long-acting COPD INH InTrial Late 2018 N N Actavis/ Circassia beta 2 agonist (LAMA/LABA) FLT-3 receptor tyrosine quizartinib quizartinib Ambit Biosciences Acute myeloid leukemia PO InTrial Late 2018 Y N kinase inhibitor moxetumomab moxetumomab MedImmune CD22 antigen inhibitor Hairy cell leukemia IV InTrial Late 2018 Y Y pasudotox pasudotox combretastatin A4 phosphate Neuroendocrine tumors/ FastTrk/ Zybrestat OXiGENE vascular targeting agent IV Late 2018 Y Y (fosbretabulin Glioma Breakthru tromethamine)

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RxOutlook® 4th Quarter 2017

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

pegylated Hepacid ADI-PEG-20 Polaris Hepatic cancer IM InTrial Late 2018 Y Y deiminase Head and Neck cancer/ Oncolytics Ovarian cancer/ Fallopian IV/ FastTrk/ Reolysin pelareorep human reovirus Late 2018 Y Y Biotech tube cancer/ Peritoneal intratumoral Breakthru cancer/ Breast cancer Apealea (Paclical) Oasmia taxane Ovarian cancer IV InTrial Late 2018 Y Y AnGes / beperminogene Peripheral artery disease/ FastTrk/ Collategene Mitsubishi angiogenesis IM Late 2018 Y N perplasmid Critical limb ischemia Breakthru Tanabe recombinant human Reasanz serelaxin Novartis Heart failure IV InTrial Late 2018 Y N relaxin (rhRlx) agonist Growth hormone somatropin somatropin Opko/ Pfizer enzyme replacement deficiency/ Small for SC InTrial Late 2018 Y Y gestational age

glioblastoma Northwest DCVax-L, DCVax-Brain vaccine Glioblastoma SC InTrial Late 2018 Y Y multiforme vaccine Biotherapeutics

FastTrk/ NeuVax nelipepimut-S Galena vaccine Breast cancer intradermal Late 2018 Y N Breakthru Non-small cell lung tertomotide tertomotide KAEL-GemVax vaccine cancer (NSCLC)/ Intradermal InTrial Late 2018 Y N Melanoma amyloidosis/ FastTrk/ IONIS-TTRrx inotersen Ionis antisense RNA modulator SC Late 2018 Y Y Transthyretin Breakthru cardiomyopathy aldoxorubicin aldoxorubicin CytRx anthracycline derivative Soft tissue sarcoma IV InTrial Late 2018 Y Y amikacin liposome FastTrk/ ALIS Insmed aminoglycoside Bacterial infections INH Late 2018 Y Y inhalation suspension Breakthru

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RxOutlook® 4th Quarter 2017

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

interleukin-13 (IL-13) Asthma/ Idiopathic CAT-354 tralokinumab 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) Ophthotech/ antiplatelet derived Age-related macular FastTrk/ Fovista pegpleranib sodium Archemix/ growth factor subunit B Intravitreal Late 2018 Y N degeneration Breakthru Astellas/ Novartis aptamer Primary immunodeficiency/ RI-002 RI-002 ADMA Biologics immunoglobulin Respiratory syncytial IV CRL Late 2018 Y N virus (RSV)/ Bacterial infections melanocortin type 4 PT-141 Palatin Female sexual disorder SC InTrial Late 2018 Y N (bremalanotide) receptor agonist Diffuse large B-cell 1 phosphatidylinositol 3 lymphoma/ Non-Hodgkin BKM-120 buparlisib Novartis PO InTrial Late 2018 Y N kinase inhibitor lymphoma/ Mantle cell lymphoma/ Breast cancer Non-small cell lung cancer (NSCLC)/ FastTrk/ entrectinib entrectinib Ignyta tyrosine kinase inhibitor PO Late 2018 N Y Colorectal cancer/ Solid Breakthru tumors/ Neuroblastoma Chronic lymphocytic Verastem/ phosphatidylinositol 3 IPI-145 (INK-1197) duvelisib leukemia/ Non-Hodgkin PO InTrial Late 2018 N Y Infinity kinase inhibitor lymphoma Boehringer Psoriasis/ Crohn's interleukin-23 (IL-23) BI-655066 (ABBV-066) risankizumab Ingelheim/ disease/ Ankylosing IV/SC InTrial Late 2018 Y Y inhibitor AbbVie spondylitis/ Asthma Novan nitric oxide-releasing SB-204 (NVN-1000) SB-204 (NVN-1000) Acne vulgaris TOP InTrial Late 2018 N N Therapeutics compound

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RxOutlook® 4th Quarter 2017

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

gamma aminobutyric acid- Seizures/ Postpartum FastTrk/ SAGE-547 SAGE/ A (GABA-A) receptor IV Late 2018 Y Y (brexanolone) depression Breakthru FastTrk/ Viaskin Peanut Viaskin Peanut DBV Technologies immunotherapy Peanut allergy TOP Late 2018 Y N Breakthru Nabriva FastTrk/ BC-3781 lefamulin pleuromutilins Bacterial infections IV/PO Late 2018 Y N Therapeutics Breakthru mycolic acid biosynthesis Deltyba delamanid Otsuka Tuberculosis PO InTrial Late 2018 N N inhibitor FastTrk/ NKTR-181 NKTR-181 Nektar opioid agonist Pain PO Late 2018 N N Breakthru STS sodium thiosulfate Fennec reducing agent Hearing loss IV InTrial Late 2018 Y Y Intellipharmaceut Rexista XR oxycodone ER opioid agonist Pain PO CRL Late 2018 N N ics Myelofibrosis/ Geron/ Johnson Myelodysplastic FastTrk/ GRN-163L imetelstat telomerase inhibitor IV Late 2018 Y Y & Johnson syndrome/ Acute Breakthru myelogenous leukemia Cutaneous T-cell Kyowa Hakko CCR-4 receptor FastTrk/ AMG-761 mogamulizumab lymphoma/ Adult T-cell IV Late 2018 Y Y Kirin antibody Breakthru leukemia-lymphoma

ELI-200 oxycodone/ Elite opioid agonist Pain PO CRL Late 2018 N N

ELI-202 opioid (undisclosed) Elite opioid agonist Pain PO InTrial Late 2018 N N FastTrk/ PTK-0796 omadacycline Paratek aminomethylcyclines Bacterial infections IV/PO Late 2018 N N Breakthru Pollinex Quattro Grass Allergy birch pollen allergen (MPL-103, Ragweed Therapeutics/ vaccine Allergic rhinitis SC InTrial Late 2018 Y N extract MATA MPL) Endo/ GSK/ Teva

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RxOutlook® 4th Quarter 2017

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

Axovant Sciences/ Alzheimer's disease/ RVT-101 intepirdine 5HT-6 receptor antagonist PO InTrial Late 2018 N N GlaxoSmithKline/ Lewy body dementia Roviant Sciences Azedra (Ultratrace iobenguane (I-131) Progenics radiopharmaceuticals Neuroendrocine tumors IV Filed NDA Late 2018 Y Y MIBG) selective macrophage Pigmented Villonodular FastTrk/ PLX108-01 (PLX-3397) pexidartinib Daiichi Sankyo colony stimulating factor 1 PO Late 2018 N Y Synovitis (PVNS) Breakthru receptor inhibitor Medicines MDL moxidectin Development for chloride channel agonist Parasitic infections PO InTrial Late 2018 N N Global Health Acute myelogenous isocitrate dehydrogenase- FastTrk/ AG-120 (AGI-5198) ivosidenib Agios/ Celgene leukemia (AML)/ Biliary PO Late 2018 Y Y 1 inhibitor Breakthru tract cancer

alpha galactosidase PRX-102 Protalix enzyme replacement Fabry's disease IV InTrial Late 2018 Y N (pegunigalsidase alfa)

Humanigen/ benznidazole benznidazole nitroimidazole Chagas disease PO InTrial Late 2018 N Y Savant Zavante FastTrk/ Zolyd fosfomycin cell wall inhibitor Bacterial infections IV Late 2018 Y N Therapeutics Breakthru Thrombotic anti-von Willebrand factor FastTrk/ ALX-0081 caplacizumab Ablynx NV thrombocytopenic IV/SC Late 2018 Y Y (vWF) nanobody Breakthru purpura (TTP) SIGA FastTrk/ Arestvyr tecovirimat Technologies/ antiviral Smallpox IV/PO Late 2018 Y Y Breakthru PharmAthene

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RxOutlook® 4th Quarter 2017

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

long-acting beta 2 / glycopyrronium agonist (LABA)/ long- QVM-149 Novartis/ Sosei Asthma INH InTrial Late 2018 N N bromide/ mometasone acting muscarinic receptor furoate antagonist (LAMA)/ corticosteroid Intraocular/ RTH-258 (ESBA-1008, brolucizumab Novartis/ Delenex anti-VEGF antibody Macular degeneration Subretinal/ InTrial Late 2018 Y N DLX-1008) Subconjunctival FMX-103 minocycline Foamix tetracyclines Rosacea TOP InTrial Late 2018 N N Retrophin/ Primary biliary ursodeoxycholic acid ursodeoxycholic acid derivative PO InTrial Late 2018 Y N Asklepion cirrhosis/cholangitits Dr. Reddy's DFN-02 (DFP-02) DFN-02 (DFP-02) triptans Headache Intranasal InTrial Late 2018 N N Laboratories Female Health/ alpha-adrenergic Benign prostatic tamsulosin DRS tamsulosin DRS PO InTrial Late 2018 N N Aspen antagonist hyperplasia Xigen/ Auris Hearing loss/ Ocular Intratympanic/ FastTrk/ AM-111 brimapitide JNK stress kinase inhibitor Late 2018 Y Y Medical inflammation Intraocular Breakthru D-threo- Attention deficit KP-415 KemPharm CNS stimulant PO InTrial Late 2018 N N hyperactivity disorder controlled-release Polycythemia vera/ PharmaEssentia/ ropeginterferon alfa-2b ropeginterferon alfa-2b interferon Myelofibrosis/ Essential SC InTrial Late 2018 Y Y AOP Orphan thrombocythemia Diffuse large B-cell lymphoma/ Acute chimeric antigen receptor FastTrk/ JCAR-017 JCAR-017 Juno/ Celgene lymphocytic leukemia/ IV Late 2018 Y Y (CAR) T cell therapy Breakthru Follicular lymphoma/ Mantle cell lymphoma phosphoinositide 3-kinase Breast cancer/ Head and BYL-719 alpelisib Novartis PO InTrial Late 2018 Y N CA (PIK3CA) inhibitor neck cancer

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RxOutlook® 4th 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 rovalpituzumab anti-DLL3 monoclonal Rova-T AbbVie Small cell lung cancer IV InTrial Late 2018 Y Y tesirine antibody Diffuse large B-cell TG Therapeutics/ phosphoinositide-3 kinase TGR-1202 TGR-1202 lymphoma/ Chronic PO InTrial Late 2018 Y Y Rhizen (PI3K) delta inhibitor lymphocytic leukemia FastTrk/ MSI-1256F squalamine lactate Ohr angiogenesis inhibitor Macular degeneration OP Late 2018 Y N Breakthru morpholino antisense Duchenne muscular SRP-4053 golodirsen Sarepta IV InTrial Late 2018 Y N oligonucleotide dystrophy thrombopoietin receptor S-888711 lusutrombopag Shionogi Thrombocytopenia PO Filed NDA Late 2018 Y N agonist Spinocerebellar ataxia/ glutamate release FastTrk/ BHV-4157 trigriluzole Portage Biotech Obsessive-compulsive PO Late 2018 Y Y inhibitor Breakthru disorder FastTrk/ RVT-802 RVT-802 Enzyvant tissue-based therapy DiGeorge syndrome Undisclosed Late 2018 Y Y Breakthru Non-small cell lung FastTrk/ PF-06463922 lorlatinib Pfizer tyrosine kinase inhibitor PO Late 2018 Y N cancer (NSCLC) Breakthru SEG-101 crizanlizumab Novartis P-selectin antagonist Sickle cell disease IV InTrial Late 2018 Y Y recombinant human nerve Neurotrophic keratitis/ Oxervate cengermin Dompe Intraocular Filed BLA Late 2018 Y Y growth factor Dry eyes essential fatty acid SC-411 docosahexaenoic acid Sancilio Sickle cell disease PO InTrial Late 2018 Y Y conjugate scPharmaceutical furosemide diuretic Heart failure SC InTrial Late 2018 Y N s receptor Ulcerative colitis/ Crohn's Entyvio vedolizumab Takeda SC InTrial Late 2018 Y N antagonist disease

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RxOutlook® 4th Quarter 2017

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

Non-small cell lung cancer (NSCLC)/ Regeneron/ programmed death ligand- FastTrk/ REGN-2810 cemiplimab Cutaneous squamous cell IV Late 2018 Y N Sanofi 1 (PD-L1) inhibitor Breakthru carcinoma/ Basal cell carcinoma B-244 B-244 AOBiome antibacterial Acne TOP InTrial Late 2018 Y N Harmony Narcolepsy/ Excessive Biosciences/ inverse H-3 Wakix tiprolisant (pitolisant) daytime sleepiness/ PO InTrial Late 2018 N N BioProject/ Grupo receptor antagonist Schizophrenia Ferrer EM-100 EM-100 Eton undisclosed Opthalmological disease SC InTrial Late 2018 N Human HIV-1 protease inhibitor/ Tentative lopinavir/ritonavir lopinavir/ritonavir Cipla immunodeficiency virus PO Late 2018 Y N CYP3A4 inhibitor Approval (HIV) nucleoside reverse transcriptase inhibitor tenofovir disoproxyl tenofovir disoproxyl Human (NRTI)/ NRTI/ non- Tentative fumarate/ lamivudine/ fumarate/ lamivudine/ Hetero Labs immunodeficiency virus PO Late 2018 Y N nucleoside reverse Approval efavirenz (HIV) transcriptase inhibitor (NNRTI) tenofovir disoproxyl tenofovir disoproxyl Human Tentative fumarate/ lamivudine/ fumarate/ lamivudine/ Matrix Labs NRTI/ NRTI/ NNRTI immunodeficiency virus PO Late 2018 Y N Approval efavirenz efavirenz (HIV) tenofovir disoproxyl tenofovir disoproxyl Human Tentative fumarate/ lamivudine/ fumarate/ lamivudine/ Aurobindo NRTI/ NRTI/ NNRTI immunodeficiency virus PO Late 2018 Y N Approval efavirenz efavirenz (HIV) tenofovir disoproxyl tenofovir disoproxyl Human Tentative fumarate/ lamivudine/ fumarate/ lamivudine/ Cipla NRTI/ NRTI/ NNRTI immunodeficiency virus PO Late 2018 Y N Approval efavirenz efavirenz (HIV)

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RxOutlook® 4th Quarter 2017

Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug tenofovir disoproxyl tenofovir disoproxyl Human Tentative fumarate/ lamivudine/ fumarate/ lamivudine/ Macleods NRTI/ NRTI/ NNRTI immunodeficiency virus PO Late 2018 Y N Approval efavirenz efavirenz (HIV) GDC-0032 (RG7604) taselisib Roche/ Chugai PI3 Kinase Inhibitor Breast cancer PO InTrial Late 2018+ Y N

2019 Possible launch date Acute myleoid leukemia/ FastTrk/ azacitidine azacitidine Celgene DNA methylation inhibitor Myelodysplastic PO 2019 Y Y Breakthru syndromes Clostridium difficile Clostridium difficile FastTrk/ ACAM-CDIFF Sanofi Pasteur vaccine IM 2019 Y N toxoid vaccine infection Breakthru ALS-8176 (ALS-008176, ALS-8176 (ALS-008176, Johnson & Respiratory syncytial RSV polymerase inhibitor PO InTrial 2019 N N JAL-8176) JAL-8176) Johnson virus pan-fibroblast growth Johnson & JNJ-42756493 (JNJ-493) JNJ-42756493 (JNJ-493) factor receptor tyrosine Urothelial cancer PO InTrial 2019 Y N Johnson/ Otsuka kinase inhibitor

EVK-001 Evoke Pharma antidopiminergics Diabetic gastroparesis Intranasal InTrial 1Q2019 N Y Post-traumatic stress PO/SL/ FastTrk/ Tonmya Tonix/ Vela muscle relaxant 1Q2019 N N disorder Transmucosal Breakthru prostaglandin agonist/ rho latanoprost/ 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) interleukin-5 (IL-5) Cinqair (SC) reslizumab Teva Asthma SC InTrial 1Q2019 Y N antagonist Tetraphase fully synthetic tetracycline FastTrk/ eravacycline eravacycline Bacterial infections IV/PO 1Q2019 Y N Pharmaceuticals antibiotic Breakthru

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RxOutlook® 4th Quarter 2017

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

tetrahydrofolate FastTrk/ iclaprim iclaprim Motif Bio Bacterial infections IV 1Q2019 Y Y dehydrogenase inhibitor Breakthru

Chronic lymphocytic leukemia/ Small cell CD-20 monoclonal ublituximab ublituximab TG Therapeutics lymphocytic lymphoma/ IV InTrial 1Q2019 Y Y antibody Mantle cell lymphoma/ Multiple sclerosis Shield Therapeutics/ Anemia/ Irritable bowel Feraccru ferric trimaltol iron replacement PO InTrial 1Q2019 N N AOP Orphan syndrome Pharma CD-20 monoclonal Chronic lymphocytic antibody/ TG-1303 ublituximab/ TGR-1202 TG Therapeutics leukemia/ Diffuse large B- IV/PO InTrial 1Q2019 Y Y phosphoinositide-3 kinase cell lymphoma (PI3K) delta inhibitor Human C-C chemokine receptor 5 immunodeficiency virus FastTrk/ PRO-140 PRO-140 CytoDyn IV/SC 2H2019 Y Y (CCR5) antagonist (HIV)/ Graft vs. host Breakthru disease FastTrk/ TTP-488 azeliragon vTv Therapeutics RAGE antagonist Alzheimer's disease PO 2H2019 N N Breakthru factor D monoclonal Age-related macular lampalizumab lampalizumab Roche Intravitreal InTrial 2H2019 Y N antibody degeneration FastTrk/ Brabafen Zogenix receptor agonist Dravet syndrome PO 2H2019 Y Y Breakthru

CoLucid/ Ill dong/ neurally Acting Anti- LY-573144 Headache PO InTrial 2H2019 Y N Eli Lilly Migraine Agent (NAAMA)

Irritable bowel sodium-hydrogen RDX-5791 tenapanor Ardelyx syndrome/ PO InTrial 2H2019 N N exchanger-3 inhibitor Hyperphosphatemia

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RxOutlook® 4th Quarter 2017

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

hypoxia-inducible factor- Akebia AKB-6548 vadadustat prolyl hydroxylase Anemia PO InTrial 2H2019 Y N Therapeutics inhibitor Rheumatoid arthritis/ janus associated kinase ABT-494 upadacitinib AbbVie Psoriatic arthritis/ PO InTrial 2H2019 N N (JAK) inhibitor Crohn's disease transdermal Corium Corplex anticholinergic Alzheimer's disease TOP InTrial 2H2019 N N system International anesthetic/ Nonsteroidal bupivacaine/ Heron FastTrk/ HTX-011 Anti-inflammatory Drug Pain Instillation 2H2019 N N meloxicam Therapeutics Breakthru (NSAID) Human dolutegravir/ GlaxoSmithKline/ Tivicay/ Epivir integrase inhibitor/ NRTI immunodeficiency virus PO InTrial 2H2019 Y N lamivudine Shionogi (HIV)

Summit Duchenne muscular FastTrk/ BMN-195 ezutromid utrophin inducer PO 2H2019 Y Y Therapeutics dystrophy Breakthru

FibroGen/ hypoxia-inducible factor FG-4592 roxadustat Astellas/ Anemia PO InTrial Mid-2019 Y N prolyl hydroxylase AstraZeneca Diffuse large B-cell lymphoma/ Follicular Pixuvri pixantrone Cell Therapeutics anthracycline derivative IV CRL Mid-2019 Y N lymphoma/ Non- Hodgkin's lymphoma Intra-Cellular Schizophrenia/ Bipolar Therapies/ ITI-007 lumateperone antipsychotic disorder/ Alzheimer's PO InTrial Mid-2019 N N Bristol-Myers disease Squibb dopamine precursor/ ND-0612L levodopa/ carbidopa NeuroDerm dopa-decarboxylase Parkinson's disease SC InTrial Mid-2019 Y N inhibitor

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RxOutlook® 4th Quarter 2017

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

dopamine precursor/ ND-0612H levodopa/ carbidopa NeuroDerm dopa-decarboxylase Parkinson's disease SC InTrial Mid-2019 Y N inhibitor Auven Seciera cyclosporine immunosuppressant Dry eyes TOP InTrial 3Q2019 Y N Therapeutics tenofovir disoproxyl Human Tentative TLE-400 fumarate/ lamivudine/ Mylan NRTI/ NRTI/ NNRTI immunodeficiency virus PO 3Q2019 Y N Approval efavirenz (HIV) hyaluronic acid/ hyaluronic acid/ Cingal triamcinolone Anika Osteoarthritis Intra-articular InTrial 4Q2019 Y N corticosteroid hexacetonide selective peroxisome Non-alcoholic proliferator-activated FastTrk/ GFT-505 elafibranor Genfit steatohepatitis (NASH)/ PO 4Q2019 N N receptor (PPAR) Breakthru Primary biliary cirrhosis modulator Attention deficit ADAIR dextroamphetamine Alcobra CNS stimulant hyperactivity disorder PO InTrial 4Q2019 N N (ADHD) Tentative Xyrosa doxycycline Sun Pharma tetracyclines Rosacea PO 4Q2019 N N Approval Roche/ AC amyloid beta-protein MABT-5102A crenezumab Alzheimer's disease IV/SC InTrial 12/1/2019 Y N Immune inhibitor Lupus nephritis/ FastTrk/ Luveniq voclosporin ILJIN calcineurin inhibitor Psoriasis/ Transplant PO Late 2019 Y Y Breakthru rejection ARYx vitamin K epoxide ATI-5923 tecarfarin Therapeutics/ reductase enzyme Anticoagulation PO InTrial Late 2019 N N Armetheon inhibitor beta-amyloid (Abeta) gantenerumab gantenerumab Roche Alzheimer's disease SC InTrial Late 2019 Y N inhibitor

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RxOutlook® 4th Quarter 2017

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

gamma-aminobutyric acid (GABA)-ergic agonist/ / naltrexone/ Charcot-Marie Tooth PXT-3003 Pharnext opioid receptor PO InTrial Late 2019 N Y sorbitol disease antagonist/ sorbitol combination End stage renal diease Proteon human elastase (ESRD)/Peripheral artery FastTrk/ PRT-201 vonapanitase TOP Late 2019 Y Y Therapeutics (recombinant) disease (PAD)/ Vascular Breakthru access in hemodialysis Toll-like receptor 9 (TLR9) MGN-1703 lefitolimod Mologen Colorectal cancer SC InTrial Late 2019 Y N agonist ATP citrate (pro-S)-lyase Esperion and stimulating AMP- ETC-1002 bempedoic acid Hypercholesterolemia PO InTrial Late 2019 N N Therapeutics activated protein kinase (AMPK) Amphora Amphora Neothetics spermicidal agent Pregnancy prevention VG CRL Late 2019 N N corticosteroid/ long-acting budesonide/ muscarinic receptor BGF-MDI glycopyrronium/ AstraZeneca antagonist (LAMA)/ long- COPD/ Asthma INH InTrial Late 2019 N N formoterol acting beta 2 adrenergic receptor agonist (LABA) Aimmune/ FastTrk/ AR-101 (AR101) AR-101 (AR101) Regeneron/ peanut protein capsule Peanut allergy PO Late 2019 Y N Breakthru Sanofi calcitonin gene-related ALD-403 eptinezumab Alder Headache IV/SC InTrial Late 2019 Y N peptide (CGRP) antagonist Human cabotegravir (long- GlaxoSmithKline/ S-265744 LAP HIV integrase inhibitor immunodeficiency virus SC InTrial Late 2019 Y N acting) Pfizer/ Shionogi (HIV) OSE Pharma/ Non-small cell lung OSE-2101 tedopi vaccine SC InTrial Late 2019 Y Y Takeda cancer (NSCLC)

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RxOutlook® 4th Quarter 2017

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

chemoattractant receptor- Asthma/ Atopic QAW-039 Novartis homologous molecule PO InTrial Late 2019 Y N dermatitis antagonist Stallergenes/ Oralair Mites dust mite peptide vaccine Dust mite allergic rhinitis SL InTrial Late 2019 Y N Shionogi interleukin-6 (IL-6) SA-237 satralizumab Roche/ Chugai Neuromyelitis optica SC InTrial Late 2019 Y Y monoclonal antibody skQ1 visomitin Mitotech plastoquinone derivative Dry eyes OP InTrial Late 2019 Y N Nordic anti-CD37 antibody Betalutin 177 lu-dota-tetulomab Non-Hodgkin lymphoma IV InTrial Late 2019 Y Y Nanovector radionuclide Roche/ PTC RG-7916 (RO-7034067) RG-7916 (RO-7034067) SMN2 splicing modifier Spinal muscular atrophy PO InTrial Late 2019 Y Y Therapeutics End-stage renal disease FastTrk/ Humacyl human acellular vessel Humacyte cellular therapy (ESRD)/ Peripheral artery Implant Late 2019 Y N Breakthru disease (PAD) Insomnia/ Sleep-wake E-2006 Eisai/ Purdue receptor antagonist PO InTrial Late 2019 N N rhythm disorder digitalis-like factor FastTrk/ DigiFab digoxin immune fab AMAG/ Velo Preeclampsia IV Late 2019 Y Y antagonist Breakthru Portage Biotech/ calcitonin gene-related BMS-927711 rimegepant sulfate Biohaven/ Bristol- Headache PO InTrial Late 2019 Y N peptide (CGRP) Myers Squibb indacaterol maleate/ long-acting beta 2 agonist/ QMF-149 Novartis/ Merck Asthma INH InTrial Late 2019 N N mometasone furoate corticosteroid

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, NSCLC = Non-small cell lung cancer, COPD = Chronic Obstructive Pulmonary Disease

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RxOutlook® 4th Quarter 2017

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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.

OptumRx specializes in the delivery, clinical management and affordability of prescription and consumer health products. We are an Optum® company — a leading provider of integrated health services. Learn more at optum.com.

All Optum® trademarks and logos are owned by Optum, Inc. All other brand or product names are trademarks or registered marks of their respective owners. This document contains information that is considered proprietary to OptumRx and should not be reproduced without the express written consent of OptumRx.

RxOutlook® is published by the OptumRx Clinical Services Department.

© 2017 Optum, Inc. All rights reserved. ORX6204A_160128

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