RxOutlook® 1st Quarter 2019

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

2019 Possible launch date Ophthalmological DS-300 DS-300 Eton undisclosed SC Filed NDA 2019 unknown N disease anti-sclerostin Evenity romosozumab Amgen Osteoporosis SC Filed NDA 2/2019 Y N monoclonal antibody tetrahydrofolate iclaprim iclaprim Motif Bio Bacterial IV Filed NDA 2/13/2019 Y Y dehydrogenase inhibitor tazarotene/ IDP-118 Valeant / Psoriasis TOP Filed NDA 2/15/2019 N N halobetasol deaminase Mavenclad Merck/ Teva resistant Multiple sclerosis PO Filed NDA 2/15/2019 Y N deoxyadenosine analog Lotemax Gel loteprednol Valeant corticosteroid Ocular inflammation OP Filed NDA 2/25/2019 N N Nex Gen etabonate turoctocog alfa glyco-PEGylated factor NN-7088 Novo Nordisk Hemophilia IV/SC Filed BLA 2/27/2019 Y N pegol VIII derivative selective sphingosine-1 BAF-312 siponimod Novartis phosphate Multiple sclerosis PO Filed NDA 3/1/2019 Y N agonist midazolam UCB Seizures Intranasal Filed NDA 3/1/2019 N Y (USL-261)

XeriSol glucagon Xeris glucagon analog Diabetes mellitus SC Filed NDA 3/1/2019 N N Glucagon

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RxOutlook® 1st Quarter 2019

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

receptor JZP-507 Jazz Narcolepsy PO Filed NDA 3/1/2019 Y N agonist NMDA receptor Major depressive esketamine Janssen Intranasal Filed NDA 3/8/2019 N N antagonist disorder (MDD) prostaglandin agonist/ latanoprost/ rho kinase- Glaucoma/ Ocular Roclatan Aerie OP Filed NDA 3/14/2019 N N netarsudil mesylate transport hypertension protein inhibitor HER2/neu receptor Herceptin trastuzumab Halozyme Breast cancer SC Filed BLA 3/15/2019 Y N antagonist gamma aminobutyric allopregnanolone acid-A (GABA-A) Postpartum SAGE-547 SAGE/ IV Filed NDA 3/19/2019 Y Y (brexanolone) receptor allosteric depression/ Seizures modulator Jazz Narcolepsy/ Sleep ADX-N05 Pharmaceuticals/ derivative PO Filed NDA 3/20/2019 N Y apnea Aerial BioPharma corticosteroid/ GSP-301 furoate/ Glenmark Allergic rhinitis NA Filed NDA 3/21/2019 N N antihistamine olopatadine HCl sodium-dependent glucose transporter 1 Zynquista sotagliflozin Sanofi/ Lexicon Diabetes mellitus PO Filed NDA 3/22/2019 N N (SGLT-1) and SGLT-2 inhibitor fosphenytoin fosphenytoin sodium/ sodium/ sulfobutylether sulfobutylether Sedor anticonvulsant Seizures IM/IV Filed NDA 3/22/2019 Y N beta- beta-cyclodextrin cyclodextrin sodium sodium anti- Recro Pharma/ N-1539 meloxicam inflammatory drug Pain IV Filed NDA 3/24/2019 Y N Alkermes (NSAID)

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RxOutlook® 1st Quarter 2019

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

NRL-1 Neurelis benzodiazepine Seizures Intranasal Filed NDA 3/25/2019 N Y long-acting muscarinic receptor Chronic obstructive aclidinium/ AstraZeneca/ (mAChR) Duaklir Pressair pulmonary disease INH Filed NDA 3/31/2019 N N formoterol Actavis/ Circassia antagonist/long-acting (COPD) beta 2 agonist (LAMA/LABA) AMG-504-1 glucagon Eli Lilly/ Locemia glucagon analog Diabetes mellitus Intranasal Filed NDA 2Q2019 N N Rizaport IntelGenx / Red Hill rizatriptan triptans Acute migraines PO Filed NDA 4/1/2019 N N (VersaFilm) Biopharma EVK-001 metoclopramide Evoke Pharma antidopiminergics Diabetic gastroparesis Intranasal Filed NDA 4/1/2019 N Y

Primary immunodeficiency/ RI-002 RI-002 ADMA Biologics immunoglobulin Respiratory syncytial IV Filed BLA 4/2/2019 Y N virus (RSV)/ Bacterial infections

Multiple myeloma/ Sarcoma/ Diffuse large Karyopharm selective inhibitor of B-cell lymphoma KPT-330 selinexor PO Filed NDA 4/6/2019 Y Y Therapeutics nuclear export (SINE) (DLBCL)/ Acute myeloid leukemia (AML) calcitonin gene-related Aimovig (140 Filed erenumab-aooe Amgen peptide (CGRP) Migraine headaches SC 4/15/2019 N N mg dose) sNDA Human dolutegravir/ GlaxoSmithKline/ Tivicay/ Epivir integrase inhibitor/ NRTI immunodeficiency virus PO Filed NDA 4/18/2019 Y N lamivudine Shionogi (HIV) Psoriasis/ Crohn's BI-655066 Boehringer interleukin-23 (IL-23) risankizumab disease/ Ankylosing IV/SC Filed BLA 4/25/2019 Y Y (ABBV-066) Ingelheim/ AbbVie inhibitor spondylitis/ Asthma

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RxOutlook® 1st Quarter 2019

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

Nabriva Contepo fosfomycin cell wall inhibitor Bacterial infections IV Filed NDA 4/30/2019 Y N Therapeutics anesthetic/ Nonsteroidal bupivacaine/ HTX-011 Heron Therapeutics Anti-inflammatory Drug Pain Instillation Filed NDA 4/30/2019 N N meloxicam (NSAID) Dengue fever Dengvaxia Sanofi Pasteur vaccine Dengue fever SC Filed BLA 5/1/2019 N N vaccine

onasemnogene Spinal muscular Zolgensma Novartis/ Regenxbio gene therapy IV Filed BLA 5/1/2019 Y Y abeparvovec atrophy (SMA)

APD-421 amisulpride Acacia Nausea/ Vomiting IV Filed NDA 5/5/2019 N N antagonist FLT-3 receptor Acute myeloid quizartinib quizartinib Daiichi Sankyo PO Filed NDA 5/25/2019 Y Y kinase inhibitor leukemia (AML) Slinda Exeltis Pregnancy prevention PO Filed NDA 5/27/2019 N N

NKTR-181 NKTR-181 Nektar opioid agonist Pain PO Filed NDA 5/28/2019 N N Imvamune; Imvamune; MVA- Bavarian Nordic vaccine Smallpox SC Filed BLA 6/1/2019 Y N MVA-BN BN osmotic gradient Bronchitol mannitol Pharmaxis enhancer; mucus Asthma/ Cystic fibrosis INH Filed NDA 6/20/2019 N Y clearance enhancer melanocortin type 4 Female sexual disorder PT-141 bremelanotide Palatin/ AMAG SC Filed NDA 6/23/2019 Y N receptor agonist (FSD) alpha-2/beta-1 vascular Ehlers-Danlos Edsivo celiprolol HCl Acer Therapeutics PO Filed NDA 6/25/2019 Y Y adrenergic agent Syndrome (vEDS) Thiola tiopronin Retrophin reducing agent Cystinuria PO Filed NDA 6/30/2019 N N human Ryplazim ProMetic/ Hematech plasminogen Plasminogen deficiency IV Filed BLA Mid-2019 Y Y plasminogen tadalafil phosphodiesterase-5 tadalafil VersaFilm IntelGenx Erectile dysfunction PO Filed NDA Mid-2019 Y N VersaFilm (PDE-5) inhibitor

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RxOutlook® 1st Quarter 2019

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 (S1PR1) Multiple sclerosis/ RPC-1063 ozanimod Celgene PO CRL 3Q2019 Y N and 5 (S1PR5) receptor Ulcerative colitis (UC) modulator

TRV-130 oliceridine Trevena opioid receptor agonist Pain IV CRL 3Q2019 N N astodrimer sodium VivaGel Starpharma viral attachment inhibitor Bacterial infections VG CRL 2H2019 N N (SPL-7013) Zalviso sufentanil, ARX-01 AcelRx opioid analgesic Pain SL CRL 2H2019 Y N Breast cancer/ Pancreatic cancer/ Pancreatic cancer/ Small cell lung cancer sacituzumab Immunomedics/ RS7-SN-38 antibody- IMMU-132 (SCLC)/ Non-small cell IV CRL 2H2019 Y Y govitecan Royalty Pharma drug conjugate lung cancer (NSCLC)/ Colorectal cancer/ Esophageal cancer/ Urinary bladder cancer

Intracerebral/ FastTrk/ VTS-270 kleptose Sucampo/ INDPA sugar molecule Niemann-Pick disease 2H2019 Y Y Intrathecal Breakthru

phosphoinositide 3- Breast cancer/ Head BYL-719 Novartis kinase CA (PIK3CA) PO InTrial 2H2019 Y N and neck cancer inhibitor MNK-812 oxycodone Mallinckrodt opioid agonist Pain PO CRL 2H2019 N N

Clostridium difficile Filed Vancocin vancomycin HCl ANI antibacterial (CDI)/ PO 7/1/2019 N N sNDA Enterocolitis

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RxOutlook® 1st Quarter 2019

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

Amyloid cardiomyopathy/ amyloid accumulation Vyndaqel meglumine Pfizer Familial amyloid PO Filed NDA 7/1/2019 Y Y inhibitor polyneuropathy (FAP)/ Cardiomyopathy Erythropoietic protoporphyria (EPP)/ melanocortin receptor 1 Scenesse afamelanotide Clinuvel Polymorphous light SC Filed NDA 7/8/2019 Y Y (MC-1) agonist eruption (PLE/PMLE)/ Vitiligo Amyotrophic lateral Portage/ Biohaven/ glutamate release sclerosis (ALS)/ SL/ BHV-0223 Filed NDA 7/15/2019 N Y Catalent inhibitor Migraines/ Social Transmucosal anxiety disorder beta-lactamase inhibitor/ relebactam/ carbapenem/ MK-7655 imipenem/ Merck Bacterial infections IV Filed NDA 7/16/2019 Y N dehydropeptidase-1 cilastatin inhibitor Shield Therapeutics/ Anemia/ Irritable bowel Feraccru ferric trimaltol AOP Orphan iron replacement PO Filed NDA 7/23/2019 N N syndrome (IBS) Pharma Non-small cell lung cancer (NSCLC)/ FastTrk/ entrectinib entrectinib Ignyta tyrosine kinase inhibitor Colorectal cancer/ PO 8/1/2019 N Y Breakthru Solid tumors/ Neuroblastoma Tenosynovial giant cell selective macrophage PLX108-01 tumor (TGCT)/ pexidartinib Daiichi Sankyo colony stimulating factor PO Filed NDA 8/3/2019 N Y (PLX-3397) Pigmented Villonodular 1 receptor inhibitor Synovitis (PVNS) Dravet syndrome/ serotonin receptor Fintepla Zogenix Lennox-Gastaut PO Filed NDA 8/6/2019 Y Y agonist syndrome (LGS)

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RxOutlook® 1st Quarter 2019

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

loteprednol KPI-121 0.25% Kala corticosteroid Dry eyes OP Filed NDA 8/15/2019 N N etabonate

Nabriva BC-3781 lefamulin pleuromutilins Bacterial infections IV/PO Filed NDA 8/20/2019 Y N Therapeutics

morpholino antisense Duchenne muscular SRP-4053 golodirsen Sarepta IV Filed NDA 8/20/2019 Y N oligonucleotide dystrophy (DMD)

Aimmune/ AR-101 AR-101 peanut protein capsule Peanut allergy PO Filed BLA 8/21/2019 N N Regeneron/ Sanofi sodium-hydrogen Irritable bowel RDX-5791 tenapanor Ardelyx exchanger-3 (NHE-3) syndrome (IBS)/ PO Filed NDA 9/13/2019 N N (AZD-1722) inhibitor Hyperphosphatemia pan-fibroblast growth JNJ-42756493 Johnson & Johnson/ erdafitinib factor receptor (FGFR) Urothelial cancer PO Filed NDA 9/18/2019 Y N (JNJ-493) Otsuka tyrosine kinase inhibitor Intra-Cellular Schizophrenia/ Bipolar ITI-007 (ITI-722) lumateperone Therapies/ Bristol- antipsychotic disorder/ Alzheimer's PO Filed NDA 9/27/2019 N N Myers Squibb disease (AD) Brinavess potassium channel vernakalant Correvio Arrhythmia IV InTrial 4Q2019 Y N (Kynapid) blocker Chronic lymphocytic ublituximab leukemia (CLL)/ Small (LFB-R603, cell lymphocytic CD-20 monoclonal TG20, TGTX- ublituximab TG Therapeutics lymphoma (SLL)/ IV InTrial 4Q2019 Y Y antibody 1101, TG-1101, Utuxin) (MCL)/ Multiple sclerosis Head and neck cancer/ cytotoxic T lymphocyte- Solid tumors/ tremelimumab tremelimumab AstraZeneca associated antigen 4 Mesothelioma/ Non- IV InTrial 4Q2019 Y Y (CTLA4) inhibitor small cell lung cancer (NSCLC)

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RxOutlook® 1st Quarter 2019

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

Novo Nordisk/ glucagon-like peptide-1 NN-9924 semaglutide Emisphere (GLP-1) receptor Diabetes mellitus PO InTrial 4Q2019 Y N (OG-217SC) Technologies agonist

FastTrk/ Pedmark (STS) sodium thiosulfate Fennec reducing agent Hearing loss IV 4Q2019 Y Y Breakthru Rexista XR oxycodone ER Intellipharmaceutics opioid agonist Pain PO CRL 4Q2019 N N CD-20 monoclonal Chronic lymphocytic antibody/ leukemia (CLL)/ Diffuse ublituximab/ TGR- TG-1303 TG Therapeutics phosphoinositide-3 large B-cell lymphoma IV/PO InTrial 4Q2019 Y Y 1202 kinase (PI3K) delta (DLBCL)/ Non-Hodgkin inhibitor lymphoma (NHL) FastTrk/ MitoGel UroGen alkylating agent Bladder cancer Intravesical 4Q2019 N Y Breakthru PPP-002 PPP-002 Tetra Bio-Pharma botanical drug Pain Undisclosed InTrial 4Q2019 N N Tentative Xyrosa doxycycline Sun Pharma tetracyclines Rosacea PO 4Q2019 N N Approval PF-708 teriparatide Pfenex/ Alvogen parathyroid hormone Osteoporosis SC Filed NDA 10/1/2019 Y N monomethyl ALKS-8700 fumarate (diroximel Biogen/ Alkermes prodrug Multiple sclerosis (MS) PO Filed NDA 10/17/2019 Y N (RDC-1559) fumarate) synthetic ACTH adrenocorticotropic adrenocortical cosyntropin Assertio INJ Filed NDA 10/18/2019 Y N depot hormone (ACTH) insufficiency triamcinolone intraocular/ CLS-1001 Clearside corticosteroid Macular edema Filed NDA 10/19/2019 Y N acetonide subretinal FMX-101 minocycline Foamix tetracyclines Acne vulgaris/ Rosacea TOP Filed NDA 10/21/2019 N N (ARK-E021) phosphodiesterase-5 APC-8000 tadalafil Adamis Erectile dysfunction PO Filed NDA 10/28/2019 Y N (PDE-5) inhibitor

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RxOutlook® 1st Quarter 2019

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

Naloxone naloxone Adamis opioid antagonist Opioid dependence IM Filed NDA 10/31/2019 N N Symject Amyloid cardiomyopathy/ tafamidis free acid amyloid accumulation Vyndaqel Pfizer Familial amyloid PO Filed NDA 11/1/2019 Y Y form inhibitor polyneuropathy (FAP)/ Cardiomyopathy dihydrofolate reductase RediTrex Cumberland Psoriasis; arthritis SC Filed NDA 11/1/2019 Y N (DHFR) inhibitor neurally Acting Anti- LY-573144 CoLucid/ Ill dong/ Eli lasmiditan Migraine Agent Acute migraines PO Filed NDA 11/14/2019 Y N (COL-144) Lilly (NAAMA)

Aquestive glutamate release Amyotrophic lateral SL/ AQST-117 riluzole Filed NDA 11/15/2019 N Y Therapeutics inhibitor sclerosis (ALS) Transmucosal

SK YKP-3089 cenobamate undisclosed Seizure PO Filed NDA 11/21/2019 Y N Biopharmaceuticals Rheumatoid arthritis janus associated kinase (RA)/ Psoriatic arthritis/ ABT-494 upadacitinib AbbVie PO Filed NDA 12/20/2019 N N (JAK) inhibitor Crohn's disease (CD)/ Atopic dermatitis (AD) granulocyte colony- SPI-2012 eflapegrastim Spectrum/ Hanmi stimulating factor Neutropenia SC Filed BLA 12/27/2019 Y N (GCSF) orexin receptor Insomnia/ Sleep-wake E-2006 lemborexant Eisai/ Purdue PO Filed NDA 12/27/2019 N N antagonist rhythm disorder ethinyl / hormonal combination Twirla Agile Therapeutics Pregnancy prevention TOP CRL Late 2019 N N levonorgestrel contraceptive Kyowa Hakko A2A Nouriast istradefylline Parkinson's disease PO CRL Late 2019 N N Kogyo/ Biovail antagonist 5-HT-1A receptor Major depressive Travivo gepirone ER GSK/Fabre-Kramer PO CRL Late 2019 N N agonist disorder (MDD)

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RxOutlook® 1st Quarter 2019

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

protein absorption FastTrk/ CM-AT CM-AT Curemark Autism PO Late 2019 Y N enhancer Breakthru aldoxorubicin aldoxorubicin NantWorks/ CytRx derivative Soft tissue sarcoma IV InTrial Late 2019 Y Y Anti-VEGF Age-related macular abicipar pegol Allergan VEGF-A inhibitor Intravitreal InTrial Late 2019 Y N DARPin degeneration (AMD) Hemolytic uremic anti-MASP-2 FastTrk/ OMS-721 OMS-721 Omeros syndrome (HUS)/ IV/SC Late 2019 Y Y monoclonal antibody Breakthru Renal diseases RNA polymerase rifabutin/ inhibitor/ penicillin/ FastTrk/ Talicia amoxicillin/ RedHill Biopharma Bacterial infections PO Late 2019 N N proton pump inhibitor Breakthru pantoprazole (PPI) Human C-C chemokine receptor immunodeficiency virus FastTrk/ PRO-140 leronlimab CytoDyn IV/SC Late 2019 Y Y 5 (CCR5) antagonist (HIV)/ Graft vs. host Breakthru disease (GvHD) oxycodone/ ELI-200 Elite opioid agonist Pain PO CRL Late 2019 N N naltrexone Sumitomo non-ergoline dopamine APL-130277 Dainippon/ MonoSol Parkinson's disease SL CRL Late 2019 N N agonist Rx/ Sunovion alpha galactosidase FastTrk/ PRX-102 Protalix replacement Fabry disease IV Late 2019 Y N (pegunigalsidase Breakthru alfa) intercellular adhesion Atlantic Healthcare/ AP-1007 alicaforsen molecule-1 (ICAM-1) Ulcerative colitis (UC) IV/Rectal Filed NDA Late 2019 Y Y Ionis inhibitor adrenoleucodystro Adrenomyeloneuropath FastTrk/ Lenti-D Bluebird Bio gene therapy Undisclosed Late 2019 Y Y phy gene therapy y Breakthru

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RxOutlook® 1st Quarter 2019

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

modified type II activin ACE-536 FastTrk/ luspatercept Acceleron/ Celgene receptor recombinant Anemia SC Late 2019 Y Y (RAP-536) Breakthru fusion protein Macular degeneration/ Intraocular/ RTH-258 Diabetic macular Subretinal/ (ESBA-1008, brolucizumab Novartis/ Delenex anti-VEGF antibody InTrial Late 2019 Y N edema (DME)/ Retinal Subconjunctiv DLX-1008) vein occlusion (RVO) al

tamsulosin Female Health/ alpha-adrenergic Benign prostatic tamsulosin DRS PO InTrial Late 2019 N N delayed-release Aspen antagonist hyperplasia (BPH)

Stallergenes/ Dust mite allergic Oralair Mites dust mite peptide vaccine SL InTrial Late 2019 Y N Shionogi rhinitis SA-237 interleukin-6 (IL-6) Neuromyelitis optica FastTrk/ satralizumab Roche/ Chugai SC Late 2019 Y Y (RG-6168) monoclonal antibody (NMO) Breakthru Vernalis/ Tris CCP-08 CCP-08 undisclosed Viral rhinitis PO CRL Late 2019 Y N Pharma

gonadotropin-releasing FP-001 (LMIS) leuprolide mesylate Foresee Prostate cancer SC InTrial Late 2019 Y N hormone (GnRH) analog

177 lu-dota- tetulomab (177 lu- tetraxetan- anti-CD37 antibody Non-Hodgkin FastTrk/ Betalutin tetulomab, 177 lu- Nordic Nanovector IV Late 2019 Y Y radionuclide lymphoma (NHL) Breakthru lilotomab satetraxetan, 177 lu-DOTA-HH1) RVT-802 RVT-802 Enzyvant tissue-based therapy DiGeorge syndrome Undisclosed Filed NDA Late 2019 Y Y Myovant Sciences/ gonadotropin-releasing Uterine fibroids/ TAK-385 relugolix Roivant Sciences/ hormone (GnRH) Endometriosis/ PO InTrial Late 2019 Y N Takeda receptor antagonist Prostate cancer

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RxOutlook® 1st Quarter 2019

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

Waldenström's selective inhibitor of Macroglobulinemia FastTrk/ BGB-3111 zanubrutinib BeiGene Bruton tyrosine kinase (WM)/ Chronic PO Late 2019 Y Y Breakthru (BTK) lymphocytic leukemia (CLL)

human monoclonal anti- Acute coronary ACZ-885 canakinumab Novartis SC CRL Late 2019 Y N IL-1 beta antibody syndrome (ACS) integrin receptor Ulcerative colitis (UC)/ Entyvio vedolizumab Takeda SC InTrial Late 2019 Y N antagonist Crohn's disease (CD) Harmony Narcolepsy/ Excessive tiprolisant Biosciences/ inverse histamine H-3 Wakix daytime sleepiness/ PO InTrial Late 2019 N N () BioProject/ Grupo receptor antagonist Schizophrenia Ferrer ASG-22M6E Astellas/ Seattle FastTrk/ (ASG-22CE, enfortumab vedotin nectin-4 antagonist Bladder cancer IV Late 2019 Y N Genetics Breakthru ASG-22ME) phosphopantothenate FastTrk/ RE-024 fosmetpantotenate Retrophin Neurodegeneration IV Late 2019 Y Y replacement therapy Breakthru TG-101348 Impact janus kinase 2 (JAK-2) (SAR-503, SAR- fedratinib Myelofibrosis (MF) PO Filed NDA Late 2019 Y N Biomedicines inhibitor 302503) Melanoma/ Solid tumors/ PD-1 checkpoint PDR-001 PDR-001 Novartis Neuroendocrine tumors IV InTrial Late 2019 Y Y inhibitor (NET)/ Non-Hodgkin lymphoma (NHL) cortexolone Winlevi/ 17alpha-propionate Intrepid androgen antagonist Acne vulgaris/ alopecia TOP InTrial Late 2019 N N Breezula (CB-03-01) Pfizer/ JDP second generation JDP-205 cetirizine Urticaria IV InTrial Late 2019 N N Therapeutics antihistamine

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RxOutlook® 1st Quarter 2019

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

selective KIT and Gastrointestinal stromal FastTrk/ BLU-285 avapritinib Blueprint Medicines PO Late 2019 Y Y PDGFRa inhibitor tumors (GIST) Breakthru FastTrk/ ODM-201 darolutamide Bayer/ Endo/ Orion Prostate cancer PO End 2019 Y N agonist Breakthru Aquestive SL/ Libervant diazepam benzodiazepine Seizures InTrial End 2019 N Y Therapeutics Transmucosal 2020 Possible launch date GW Multiple sclerosis (MS)/ FastTrk/ Sativex nabiximols Pharmaceuticals/ cannabinoid product SL/ SPR 2020 N N Pain Breakthru Otsuka Blepharitis/ Ocular Dexasite dexamethasone InSite Vision corticosteroid TOP InTrial 2020 N N inflammation Leukocyte Head and Neck cancer/ Multikine Interleukin (CS- CEL-SCI immunomodulator Squamous cell SC InTrial 2020 Y Y 001P3) carcinoma IdeS Kidney transplant/ (immunoglobulin Thrombotic G-degrading thrombocytopenic FastTrk/ imlifidase Hansa Medical bacterial enzyme IV Early 2020 Y Y enzyme of purpura Breakthru Streptococcus (TTP)/Goodpasture's pyogenes) disease DCVax-L, glioblastoma Northwest vaccine Glioblastoma SC InTrial Early 2020 Y Y DCVax-Brain multiforme vaccine Biotherapeutics neurotrophic tyrosine kinase receptor type 1 FastTrk/ tanezumab tanezumab Pfizer/ Eli Lilly Osteoarthritis/ Pain IV/SC 2020 Y N (TrkA) antagonist Breakthru (monoclonal antibody) Viaskin Peanut Viaskin Peanut DBV Technologies immunotherapy Peanut allergy TOP CRL 2020 N N

mycolic acid Deltyba delamanid Otsuka Tuberculosis PO InTrial 2020 N N biosynthesis inhibitor

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RxOutlook® 1st Quarter 2019

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

dopamine precursor/ levodopa/ Parkinson's disease ND-0612L NeuroDerm dopa-decarboxylase SC InTrial 2020 Y N (PD) inhibitor dopamine precursor/ levodopa/ Parkinson's disease ND-0612H NeuroDerm dopa-decarboxylase SC InTrial 2020 Y N carbidopa (PD) inhibitor corticosteroid/ long- acting muscarinic budesonide/ Chronic obstructive BGF-MDI receptor antagonist glycopyrronium/ AstraZeneca pulmonary diseaser INH InTrial 2020 N N (PT-010) (LAMA)/ long-acting formoterol (COPD)/ Asthma beta 2 adrenergic receptor agonist (LABA) Aveo/ Astellas/ Tivopath tivozanib VEGF inhibitor Renal cell cancer PO InTrial 2020 Y N Kyowa Hakko Kirin ALS-8176 (ALS- RSV polymerase Respiratory syncytial 008176, JAL- lumicitabine Johnson & Johnson PO InTrial 2020 N N inhibitor virus (RSV) 8176) synthase LCI-699 Novartis Cushing's syndrome PO InTrial 2020 N Y inhibitor

Vicinium oportuzumab Eleven anti-ECAM exotoxin A FastTrk/ Bladder cancer Intravesical 2020 Y N (VB-4-845) monatox Biotherapeutics fusion protein Breakthru

neo-recombinant GZ-402666 human acid alpha Sanofi enzyme therapy Pompe disease IV InTrial 2020 Y N (NeoGAA) glucosidase ursodeoxycholic ursodeoxycholic Retrophin/ Primary biliary bile acid derivative PO InTrial 2020 Y N acid acid Asklepion cirrhosis/cholangitits autologous Histogenics/ autologous chondrocyte NeoCart chondrocyte tissue Joint repair Undisclosed InTrial 2020 Y N Purpose tissue implant implant

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RxOutlook® 1st Quarter 2019

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

EUSA/ Aperion/ APN-311 dinutuximab beta Endo/ Gen Ilac/ disialoganglioside Neuroblastoma SC InTrial 2020 Y Y Medison Multiple myeloma/ Acute lymphoblastic isatuximab isatuximab Sanofi/ ImmunoGen CD38 antagonist leukemia (ALL) or IV InTrial 2020 Y Y lymphoblastic lymphoma (LBL) chimeric antigen Celgene/ Bluebird Multiple myeloma/ bb-2121 bb-2121 receptor (CAR) T cell IV InTrial 2020 Y Y Bio Brain cancer therapy ornithine decarboxylase CPP-1X/ Familial adenomatous eflornithine/ Cancer Prevention inhibitor/ non-steroidal FastTrk/ sulindac polyposis (FAP)/ PO 2020 Y Y sulindac Pharma/ Zeria anti-inflammatory drug Breakthru (DFMO) Colorectal cancer (NSAID) indacaterol maleate/ long-acting beta 2 QMF-149 Novartis/ Merck Asthma INH InTrial 2020 N N mometasone agonist/ corticosteroid furoate Ophthalmological DS-200 DS-200 Eton undisclosed SC InTrial 2020 unknown N disease Ophthalmological DS-100 DS-100 Eton undisclosed SC InTrial 2020 unknown N disease NNC-0195-0092 recombinant human Short stature/ Growth somapacitan Novo Nordisk SC InTrial 2020 Y N (NN-8640) growth hormone (rhGH) hormone deficiency Non-small cell lung INCB-028060 capmatinib Novartis/ Incyte cMET inhibitor PO InTrial 2020 Y N cancer (NSCLC) JNJ-872 (VX- Johnson & Johnson/ FastTrk/ JNJ-872 (VX-787) viral protein inhibitor PO 2020 N N 787) Vertex Breakthru ITCA-650 glucagon-like peptide-1 (sustained exenatide Intarcia/ Quintiles/ (GLP-1) receptor Diabetes mellitus SC implant CRL 1Q2020 Y N release sustained-release Servier agonist exenatide)

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RxOutlook® 1st Quarter 2019

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

ATP citrate (pro-S)- lyase and stimulating ETC-1002/ bempedoic acid/ Esperion AMP-activated protein Hypercholesterolemia PO InTrial 1Q2020 N N ezetimibe ezetimibe Therapeutics kinase (AMPK)/ cholesterol absorption inhibitor Cosmo/ Hana/ Paion/ CNS-7056 Pharmascience/ R- benzodiazepine Procedural sedation IV InTrial 1Q2020 Y N (ONO-2745) Pharm/ Yichang Humanwell Pulmonary arterial Trevyent trevyent SteadyMed prostacyclin analog SC CRL 1Q2020 Y Y hypertension (PAH) calcitonin gene-related ALD-403 eptinezumab Alder peptide (CGRP) Migraine prevention IV/SC InTrial 1Q2020 N N receptor antagonist donepezil Corplex Corium International Alzheimer's disease TOP InTrial 1Q2020 N N transdermal system D-threo- Attention deficit KP-415 KemPharm CNS hyperactivity disorder PO InTrial 1Q2020 N N controlled-release (ADHD) calcitonin gene-related MK-1602 (AGN- ubrogepant Allergan/ Merck peptide (CGRP) Acute migraines PO InTrial 1Q2020 Y N 241689) receptor antagonist cystic fibrosis transmembrane VX-659 VX-659 Vertex Cystic fibrosis (CF) PO InTrial 1Q2020 Y N conductance regulator (CFTR) corrector cystic fibrosis transmembrane FastTrk/ VX-445 VX-445 Vertex Cystic fibrosis (CF) PO 1Q2020 Y N conductance regulator Breakthru (CFTR) corrector

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RxOutlook® 1st Quarter 2019

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

Graft vs. Host disease (GvHD)/ Crohn's Mesoblast/ JCR/ disease/ Prochymal remestemcel-L Mallinckrodt/ Osiris mesenchymal stem cells Gastrointestinal injury IV InTrial 1Q2020 Y Y Therapeutics post radiation exposure/ Heart failure (HF)

ATP citrate (pro-S)- Esperion lyase and stimulating ETC-1002 bempedoic acid Hypercholesterolemia PO InTrial 1Q2020 N N Therapeutics AMP-activated protein kinase (AMPK) naloxone nasal FastTrk/ naloxone Insys Therapeutics opioid antagonist Opioid dependence Intranasal 1Q2020 N N spray Breakthru Neurocrine catechol-O- Ongentys Biosciences/ Bial/ methyltransferase Parkinson disease PO InTrial 1Q2020 N N Ono (COMT) inhibitor Acute respiratory FastTrk/ Traumakine -beta -1a Faron/ Maruishi interferon distress syndrome IV 1Q2020 Y N Breakthru (ARDS) NX-1207 (NYM- Benign prostatic 4805, REC fexapotide triflutate Nymox pro-apoptotic hyperplasia (BPH)/ Intratumoral InTrial 1H2020 Y N 0482) Prostate cancer VS-EBOV (rVSV- Merck/ NewLink ZEBOV EBOV; rVSV- vaccine Ebola IM Filed BLA 1H2020 Y N Genetics ZEBOV-GP) Strongbridge COR-003 levoketoconazole azole antifungal Cushing's syndrome PO InTrial 1H2020 N Y Biopharma Shionogi/ S-649266 cefiderocol cephalosporin antibiotic Bacterial infections IV InTrial 1H2020 Y N GlaxoSmithKiline Pancreatic cancer/ pegvorhyaluronidas FastTrk/ PEGPH-20 Halozyme/ Nektar hyaluronic acid Non-small cell lung IV 1H2020 Y Y e alfa Breakthru cancer (NSCLC)

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RxOutlook® 1st Quarter 2019

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

S-265744 LAP Human (S/GSK- cabotegravir (long- GlaxoSmithKline/ HIV integrase inhibitor immunodeficiency virus SC InTrial 1H2020 Y N 1265744 LAP; acting) Pfizer/ Shionogi (HIV) GSK-744 LA) sodium oxybate dopamine receptor FT-218 Avadel Narcolepsy PO InTrial 1H2020 Y N extended-release agonist Polycythemia vera ropeginterferon ropeginterferon PharmaEssentia/ (PV)/ Myelofibrosis interferon SC InTrial 1H2020 Y Y alfa-2b alfa-2b AOP Orphan (MF)/ Essential thrombocythemia (ET) lentiviral beta- Sickle cell disease/ FastTrk/ Lentiglobin globin gene Bluebird Bio gene therapy IV 1H2020 Y Y Beta thalassemia Breakthru transfer Cosmetic/ Cervical daxibotulinumtoxin Revance RT-002 botulinum toxins dystonia/ Plantar IM InTrial 1H2020 Y Y A Therapeutics fasciitis FastTrk/ SEG-101 crizanlizumab Novartis P-selectin antagonist Sickle cell disease IV 1H2020 Y Y Breakthru

MC2-01 calcipotriene/ vitamin D analog/ MC2 Therapeutics Psoriasis TOP InTrial 1H2020 N N (MC-201) betamethasone corticosteroid

FastTrk/ ALN-AS1 givosiran Alnylam RNAi therapeutic agent Porphyria SC 1H2020 Y Y Breakthru Pregnancy prevention/ Amphora Amphora Neothetics spermicidal agent VG CRL 1H2020 N N Bacterial infections interferon receptor Systemic lupus FastTrk/ MEDI-546 anifrolumab AstraZeneca/ BMS IV 1H2020 Y N antagonist erythematosus (SLE) Breakthru Apealea Oasmia Ovarian cancer IV InTrial 1H2020 Y Y (Paclical)

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RxOutlook® 1st Quarter 2019

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

daratumumab (with recombinant Johnson & Johnson humanized anti-CD38 Multiple myeloma/ Darzalex SC InTrial 1H2020 Y Y human / Genmab monoclonal antibody Amyloidosis hyaluronidase) hypoxia-inducible factor FG-4592 FibroGen/ Astellas/ roxadustat prolyl hydroxylase (HIF- Anemia PO InTrial 1H2020 Y N (ASP-1517) AstraZeneca PHI) cabotegravir HIV integrase inhibitor/ cabotegravir (long- (long-acting)/ non- reverse acting)/ rilpivirine ViiV Healthcare HIV IM/SC InTrial 1H2020 Y N rilpivirine (long- transcriptase inhibitor (long-acting) acting) (NNRTI) dexamethasone EGP-437 phosphate EyeGate corticosteroid Uveitis OP InTrial 2Q2020 Y N (iontophoretic)

pneumococcal FastTrk/ V-114 Merck vaccine Bacterial infection IM 2Q2020 Y N conjugate vaccine Breakthru

valoctocogene FastTrk/ BMN-270 BioMarin gene therapy Hemophilia IV 2Q2020 Y Y roxaparvovec Breakthru ZP-4207 dasiglucagon Zealand Pharma glucagon analog Diabetes mellitus SC InTrial 2Q2020 N Y (ZP-GA-1) apoptosis signal- Non-alcoholic GS-4997 selonsertib Gilead regulating kinase-1 steatohepatitis (NASH)/ PO InTrial 2Q2020 Y N (ASK-1) inhibitor Alcoholic hepatitis dopamine receptor / Schizophrenia/ Bipolar ALKS-3831 Alkermes antagonist/ opioid PO InTrial Mid-2020 N N samidorphan disorder receptor antagonist lidocaine/ PSD-502 Plethora/ Recordati sodium channel blocker Premature ejaculation TOP InTrial Mid-2020 N N prilocaine Duchenne muscular idebenone idebenone Santhera co-enzyme Q-10 analog PO CRL Mid-2020 Y Y dystrophy

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RxOutlook® 1st Quarter 2019

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

Hepatitis D (HDV); Hutchinson-Gilford EBP-994 (rEBP- Eiger Progeria Syndrome FastTrk/ lonafarnib prenylation inhibitor PO Mid-2020 Y Y 994) Biopharmaceuticals (HGPS or progeria) Breakthru and progeroid laminopathies

Diffuse large B-cell lymphoma (DLBCL)/ chimeric antigen lisocabtagene Acute lymphocytic FastTrk/ JCAR-017 Juno/ Celgene receptor (CAR) T cell IV Mid-2020 Y Y maraleucel leukemia (ALL)/ Breakthru therapy / Mantle cell lymphoma Rheumatoid arthritis/ Crohn's disease/ Galapagos NV/ janus associated kinase- Ulcerative colitis (UC)/ GLPG-0634 filgotinib PO InTrial Mid-2020 Y N Gilead 1 (JAK) inhibitor Sjogren's syndrome/ Ankylosing spondylitis/ Psoriatic arthritis

insulin-like growth factor RV-001 (Roche- Horizon/ Chugai/ FastTrk/ teprotumumab 1 (IGF-1) receptor Thyroid eye disease IV Mid-2020 Y Y 1, R-1507) Roche/ Genmab Breakthru antagonist

long-acting beta 2 indacaterol/ adrenergic receptor glycopyrronium agonist (LABA)/ long- QVM-149 bromide/ Novartis/ Sosei Asthma INH InTrial Mid-2020 N N acting muscarinic mometasone receptor antagonist furoate (LAMA)/ corticosteroid selective FGFR1/2/3 INCB-54828 pemigatinib Incyte Biliary tract cancer PO InTrial Mid-2020 Y Y inhibitor

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RxOutlook® 1st Quarter 2019

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

GBT-440 Global Blood FastTrk/ voxelotor hemoglobin modulator Sickle cell anemia PO Mid-2020 Y Y (GTx-011) Therapeutics Breakthru

Diffuse large B-cell phosphoinositide-3 TG Therapeutics/ lymphoma (DLBCL)/ FastTrk/ TGR-1202 umbralisib kinase (PI3K) delta PO Mid-2020 Y Y Rhizen Chronic lymphocytic Breakthru inhibitor leukemia (CLL) Portage Biotech/ calcitonin gene-related BMS-927711 rimegepant sulfate Biohaven/ Bristol- peptide (CGRP) Acute migraines PO InTrial Mid-2020 Y N (BHV-3000) Myers Squibb receptor antagonist monomethyl Banner Life Tentative Bafiertam prodrug Multiple sclerosis PO 6/20/2020 Y N fumarate Sciences Approval C-C chemokine receptor TBR-652 Tobira Therapeutics/ HIV/ Non-alcoholic FastTrk/ cenicriviroc 5 (CCR5) and receptor 2 PO 3Q2020 Y N (TAK-652, CVC) Takeda steatohepatitis (NASH) Breakthru antagonist Sun Pharma SDP-037, Ocular SDP-037, SDN-037 Advanced Research Corticosteroid OP InTrial 3Q2020 N N SDN-037 inflammation/pain Company (SPARC) delta-9- PPP-001 tetrahydrocannabin Tetra-Bio-Pharma cannabinoid product Pain INH InTrial 3Q2020 Y Y ol/ cannabidiol non-nucleoside reverse Tentative efavirenz efavirenz Micro Labs transcriptase inhibitor HIV PO 3Q2020 Y N Approval (NNRTI) TransCon Growth Short stature/ Growth ACP-001 Ascendis growth hormone prodrug SC InTrial 3Q2020 Y N Hormone hormone deficiency AstraZeneca/ Array selective MEK kinase Uveal melanoma/ selumetinib selumetinib BioPharma/ Cancer PO InTrial 2H2020 Y Y inhibitor Thyroid cancer Research UK RGN-259 (GBT- Neurotrophic keratitis thymosin beta 4 RegeneRx actin regulating peptide OP InTrial 2H2020 N Y 201; RGN-352) (NK)/ Dry eyes FastTrk/ Zeftera ceftobiprole Basilea cephalosporin antibiotic Bacterial infections IV 2H2020 Y N Breakthru

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RxOutlook® 1st Quarter 2019

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

Niemann-Pick Disease (NPD)/ Sporadic FastTrk/ arimoclomol arimoclomol Orphazyme cytoprotectives Inclusion Body Myositis PO 2H2020 Y Y Breakthru (IBM)/ Amyotrophic lateral sclerosis (ALS) MediWound/ BL&H/ peptide hydrolase NexoBrid bromelain CrystalGenomics/ Burns/ Skin injury TOP InTrial 2H2020 N Y replacement agent Kaken gamma-aminobutyric acid (GABA)-ergic baclofen/ Charcot-Marie Tooth FastTrk/ PXT-3003 Pharnext agonist/ opioid receptor PO 2H2020 N Y naltrexone/ sorbitol disease Breakthru antagonist/ sorbitol combination bimatoprost bimatoprost sustained Allergan prostaglandin agonist Glaucoma Implant InTrial 2H2020 N/A N sustained release release End stage renal diease (ESRD)/Peripheral Proteon human elastase FastTrk/ PRT-201 vonapanitase artery disease (PAD)/ TOP 2H2020 Y Y Therapeutics (recombinant) Breakthru Vascular access in hemodialysis Toll-like receptor 9 MGN-1703 lefitolimod Mologen Colorectal cancer SC InTrial 2H2020 Y N (TLR9) agonist

Acute myeloid iodine I 131 anti-CD45 monoclonal leukemia (AML)/ Iomab-B monoclonal Actinium IV InTrial 2H2020 Y Y antibody Myelodysplastic antibody BC8 syndrome (MDS)

hypoxia-inducible factor- AKB-6548 vadadustat Akebia Therapeutics prolyl hydroxylase (HIF- Anemia PO InTrial 2H2020 Y N PH) inhibitor

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RxOutlook® 1st Quarter 2019

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

Solid tumors/ Diffuse E-7438 (EPZ- methyltransferase EZH2 large B-cell lymphoma FastTrk/ tazemetostat Epizyme/ Eisai PO 2H2020 Y Y 6438) inhibitor (DLBCL)/ Non-Hodgkin Breakthru lymphoma (NHL)

Neutropenia/ Non-small tumor vascular NPI-2358 plinabulin BeyondSpring cell lung cancer IV InTrial 2H2020 Y N disrupting agent (tVDA) (NSCLC) Sci-B-Vac hepatitis B vaccine VBI Vaccines vaccine Hepatitis B (HBV) IM InTrial 2H2020 N N POD- dihydroergotamine INP-104 Impel/ 3M ergot derivative Acute migraines NA InTrial 2H2020 N N mesylate (POD- DHE) FMX-103 minocycline Foamix tetracyclines Rosacea TOP InTrial 2H2020 N N

Attention deficity selective norepinephrine SPN-812 SPN-812 Supernus hyperactivity disorder PO InTrial 2H2020 N N (ADHD)

Hereditary angioedema FastTrk/ BCX-7353 BCX-7353 BioCryst kallikrein inhibitor PO 2H2020 Y Y (HAE) Breakthru IMGN-853 (M- mirvetuximab folate receptor-1 FastTrk/ 9346A-sulfo- ImmunoGen Ovarian cancer IV 2H2020 Y Y soravtansine antagonist Breakthru SPDB-DM4) Liquidia Pulmonary arterial LIQ-861 treprostinil prostacyclin analog INH InTrial 2H2020 Y N Technologies hypertension (PAH) Molluscum/ Verruca VP-102 VP-102 Verrica antiviral TOP InTrial 2H2020 N N vulgaris HER2 oncoprotein MAGH-22 margetuximab MacroGenics Breast cancer IV InTrial 2H2020 Y N antagonist Qtrypta zolmitriptan Zosano triptans Acute migraines TOP InTrial 4Q2020 N N

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RxOutlook® 1st Quarter 2019

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

Diffuse large B-cell lymphoma (DLBCL)/ MOR-208 MOR-208 (MOR- Acute lymphocytic FastTrk/ (MOR-00208, 00208, XmAB- MorphoSys/ Xencor CD-19 antagonist IV 4Q2020 Y Y leukemia (ALL)/ Breakthru XmAB-5574) 5574) Chronic lymphocytic leukemia (CLL) BMS-663068 Human fostemsavir Bristol-Myers FastTrk/ (BMS-626529 HIV attachment inhibitor immunodeficiency virus PO 4Q2020 Y N (temsavir prodrug) Squibb Breakthru prodrug) (HIV) The Medicines proprotein convertase ALN-PCSsc inclisiran Company/ Alnylam/ subtilisin/kexin 9 (PCSK- Hyperlipidemia SC InTrial 4Q2020 Y Y (PCSK9si) Arbutus Biopharma 9) inhibitor Avenue tramadol tramadol opioid receptor agonist Pain IV InTrial 4Q2020 N N Therapeutics dehydrated human Plantar fasciitis/ amnion/chorion amniotic tissue AmnioFix MiMedx Achilles tendonitis/ INJ InTrial 4Q2020 Y N membrane membrane Osteoarthritis (dHACM) estetrol/ Mithra/ Fuji/ Zhejian PO/SL/ Estelle Pregnancy prevention InTrial 4Q2020 N N drospirenone Xianju agonist Transmucosal Colorectal cancer/ Pancreatic cancer/ Sumitomo BBI-608 napabucasin stem cell inhibitor Non-small cell lung PO InTrial 4Q2020 Y Y Dainippon cancer (NSCLC)/ Gastric cancer opioid receptor agonist Opioid dependence/ Tentative CAM-2038 buprenorphine Camurus/ Braeburn SC 11/1/2020 Y N (partial) Pain Approval Acute myeloid DNA methylation leukemia (AML)/ FastTrk/ azacitidine Celgene PO Late 2020 Y Y inhibitor Myelodysplastic Breakthru syndromes

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RxOutlook® 1st Quarter 2019

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

Duchenne muscular gene transcription dystrophy/ Translarna ataluren PTC Therapeutics PO CRL Late 2020 Y Y modulator Mucopolysaccharidosis (MPS) selective peroxisome Non-alcoholic proliferator-activated FastTrk/ GFT-505 elafibranor Genfit steatohepatitis (NASH)/ PO Late 2020 N N receptor (PPAR) Breakthru Primary biliary cirrhosis modulator Non-small cell lung FastTrk/ MVA-MUC1-IL2 TG-4010 Transgene vaccine SC Late 2020 N N cancer (NSCLC) Breakthru Myelofibrosis (MF)/ Myelodysplastic Geron/ Johnson & FastTrk/ GRN-163L imetelstat telomerase inhibitor syndrome (MDS)/ IV Late 2020 Y Y Johnson Breakthru Acute myelogenous leukemia (AML) Lupus nephritis/ FastTrk/ Luveniq voclosporin ILJIN calcineurin inhibitor Psoriasis/ Transplant PO Late 2020 Y Y Breakthru rejection Mycapssa octreotide Chiasma somatostatin analog Acromegaly PO CRL Late 2020 Y Y (Octreolin) vitamin K epoxide ARYx Therapeutics/ ATI-5923 tecarfarin reductase enzyme Anticoagulation PO InTrial Late 2020 N N Armetheon inhibitor Non-cystic fibrosis Linhaliq ciprofloxacin Aradigm/ Grifols fluoroquinolone bronchiectasis/ Cystic INH CRL Late 2020 Y Y fibrosis Orphan V-1 () Hepato-renal syndrome Lucassin IV CRL Late 2020 Y Y Therapeutics/ Ikaria agonist (HRS)

interleukin-13 (IL-13) MILR-1444A lebrikizumab Chugai Atopic dermatitis (AD) SC InTrial Late 2020 Y Y inhibitor

dexamethasone Otividex Otonomy corticosteroid Meniere's disease Intratympanic InTrial Late 2020 Y N sustained-release

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RxOutlook® 1st Quarter 2019

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

protein synthesis KAE-609 cipargamin Novartis Malaria PO InTrial Late 2020 N N inhibitor / BAY 94-8862 finerenone Bayer PO InTrial Late 2020 N N receptor antagonist Heart failure respiratory Respiratory syncytial FastTrk/ RSV-F syncytial virus Novavax vaccine IM Late 2020 Y N virus (RSV) infection Breakthru vaccine OSE-2101 OSE Pharma/ Non-small cell lung (IDM-2101, EP- tedopi vaccine SC InTrial Late 2020 Y Y Takeda cancer (NSCLC) 2101) chemoattractant QAW-039 receptor-homologous Asthma/ Atopic (NVP-QAW- fevipiprant Novartis PO InTrial Late 2020 Y N molecule (CRTH2) dermatitis 039) antagonist MD-1003 MD-1003 MedDay biotin Multiple sclerosis PO InTrial Late 2020 Y N granulocyte Pulmonary alveolar Molgradex molgramostim Serendex/ Savara macrophage-colony proteinosis (PAP)/ Host INH InTrial Late 2020 Y Y stimulating factor defense Eosinophilic FastTrk/ SHP-621 budesonide Shire corticosteroid PO Late 2020 Y Y esophagitis Breakthru vascular endothelial Ovarian cancer/ Biliary Recentin cediranib AstraZeneca growth factor receptor PO InTrial Late 2020 Y Y tract cancer (VEGF) antagonists R-1658 (RG- DalCor/ Japan cholesteryl ester transfer Acute coronary 1658, JTT-705, dalcetrapib PO InTrial Late 2020 Y N Tobacco/ Roche protein inhibitor syndrome (ACS) RO-4607381) FastTrk/ cannabidiol cannabidiol Insys Therapeutics cannabinoid product Seizures/ Prader-Willi PO Late 2020 Y N Breakthru skQ1 visomitin Mitotech plastoquinone derivative Dry eyes OP InTrial Late 2020 Y N RG-7916 (RO- Roche/ PTC Spinal muscular Risdiplam SMN2 splicing modifier PO InTrial Late 2020 Y Y 7034067) Therapeutics atrophy

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RxOutlook® 1st Quarter 2019

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

SCY-078 (MK- Scynexis/ R-Pharm glucan synthase FastTrk/ ibrexafungerp Fungal infections IV/PO Late 2020 N Y 3118) JSC/ Merck inhibitors Breakthru ChemoCentryx/ C5a receptor (C5aR) Vasculitis/ CCX-168 avacopan PO InTrial Late 2020 Y Y Galencia antagonist Glomerulopathy Obesity/ Bardet-Biedl BIM-22493 Rhythm/ Camurus/ melanocortin 4 receptor syndrome/ Prader-Willi FastTrk/ setmelanotide SC Late 2020 Y Y (RM-493) Ipsen (MC4R) agonist syndrome/ Diabetes Breakthru mellitus GSK-2696274 GSK-2696274 GlaxoSmithKline gene therapy Leukodystrophy IV InTrial Late 2020 Y Y (OTL-200) (OTL-200) norepinephrine/ Attention deficit NLS-1 (BLK- NLS Pharma dopamine reuptake hyperactivity disorder PO InTrial Late 2020 N N 021) controlled-release inhibitor (NDRI) (ADHD) Roivant Sciences/ selective beta 3 MK-4618 (KRP- vibegron Urovant/ Kissei/ adrenergic receptor Overactive bladder PO InTrial Late 2020 N N 114V, RVT-901) Kyorin/ Merck agonist vosoritide C-type natriuretic BMN-111 BioMarin/ Chugai Achondroplasia SC InTrial Late 2020 Y Y (vasoritide) peptide (CNP) analog Furoscix scPharmaceuticals Heart failure SC CRL Late 2020 Y N AstraZeneca Renal cell cancer AZD-6094 c-Met receptor tyrosine savolitinib (volitinib) (Hutchison (RCC)/ Non-small cell PO InTrial Late 2020 Y N (HMPL-504) kinase inhibitor MediPharma) lung cancer (NSCLC) Irritable bowel disease SHP-647 (PF- SHP-647 (PF- (IBD)/ Crohn's disease Shire MAdCAM-1 antagonist IV/SC InTrial Late 2020 Y Y 00547659) 00547659) (CD)/ Ulcerative colitis (UC) Genmab/ Seattle HuMax-TF ADC tisotumab vedotin tissue factor antibody Solid tumors Undisclosed InTrial Late 2020 Y N Genetics RG-7314 (RO- V1A vasopressin Autism spectrum FastTrk/ balovaptan Roche PO Late 2020 Y N 5285119) receptor antagonist disorder Breakthru allosteric Bcr-Abl Chronic myelogenous ABL-001 asciminib Novartis PO InTrial Late 2020 Y Y inhibitor leukemia (CML)

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RxOutlook® 1st Quarter 2019

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

chemokine (CXCR4) Transplant/ Breast POL-6326 balixafortide Polyphor IV InTrial Late 2020 Y N antagonist cancer MK-0594 (VPD- NK-1 receptor Atopic dermatitis/ FastTrk/ serlopitant Menlo PO Late 2020 Y N 737) antagonist Cough Breakthru BMS-986089 BMS-986089 (RG- Roche/ Bristol- Duchenne muscular anti-myostatin adnectin SC InTrial Late 2020 Y Y (RG-6206) 6206) Myers Squibb dystrophy (DMD) LY-900014 LY-900014 Eli Lilly insulins Diabetes mellitus SC InTrial Late 2020 N N (URLi) Eisai/ Biogen/ BAN-2401 BAN-2401 protofibril antagonist Alzheimer's disease IV InTrial Late 2020 Y N BioArctic therapeutic aryl GSK/ Celestial/ GSK-2894512 hydrocarbon receptor Atopic dermatitis (AD)/ tapinarof Roivant Sciences/ TOP InTrial Late 2020 Y N (WBI-1001) modulating agent Psoriasis Welichem Biotech (TAMA) 2021 Possible launch date brincidofovir Adenovirus/ DNA-directed DNA FastTrk/ CMX-001 hexadecyloxypropy Chimerix Cytomegalovirus PO 2021 N Y polymerase inhibitor Breakthru l ester (CMV)/ Smallpox adrenocorticotropic Rheumatoid arthritis CT-100 corticotrophin Eton INJ InTrial 2021 Y N hormone (ACTH) (RA) TWIN (S6G5T- benzoyl peroxide/ Sol-Gel retinoid Acne vulgaris TOP InTrial 2021 N N 1; S6G5T-3) Technologies toll-like receptor 9 (TLR- SC/ FastTrk/ IMO-2125 tilsotolimod Idera Melanoma 2021 Y Y 9) agonist intratumoral Breakthru Pfizer/ FastTrk/ rivipansel rivipansel synthetic glycomimetic Sickle cell IV 2021 Y Y GlycoMimetics Breakthru Sun Pharma PDP-716 brimonidine Advanced Research alpha-2 agonist Glaucoma OP InTrial 1Q2021 N N Company (SPARC) ALNG-01 (ALN- glycolate oxidase lumasiran Alnylam Hyperoxaluria Intranasal InTrial 1Q2021 Y Y G-01) antagonist

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RxOutlook® 1st Quarter 2019

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

Psoriasis(Ps)/ Psoriatic arthritis (PsA)/ UCB-4940 interleukin-17 (IL-17) bimekizumab UCB Ankylosing spondylitis IV InTrial 1H2021 Y N (CDP-4940) receptor inhibitor (AS)/ Rheumatoid arthritis (RA)

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

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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.

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RxOutlook® is published by the OptumRx Clinical Services Department.

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