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Specialty Pipeline: March 2021

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Specialty Pipeline: March 2021 Specialty Pipeline MONTHLY UPDATE Critical updates in an ever changing environment March 2021 NEW DRUG INFORMATION ™ ● Amondys 45 (casimersen): The United States Food and Drug Administration (FDA) has granted accelerated approved Sarepta Therapeutics’ Amondys 45 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping. Approximately 8% of patients have exon 45 skipping DMD. Sarepta already markets Vyondys 53® (golodirsen) for patients with mutations amenable to exon 53 skipping, and Exondys 51® (eteplirsen) for those with mutations amenable to exon 51 skipping. Amondys 45 is administered via a once-weekly intravenous infusion. Amondys 45’s approval is based on preliminary data from Phase 3 clinical trial, ESSENCE, that demonstrated a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45.1 The Institute for Clinical and Economic Review (ICER) states that, “The clinical efficacy of exon-skipping therapies (including Amondys 45) is still unclear. There is limited or no evidence demonstrating improvements in functions, as comparison with historical controls with conditions such as DMD.”2 Amondys 45 full approval will be determined upon completion of the ESSENSE trial, which is expected to conclude in 2024. Amondys 45 uses weight- based dosing and has launched with an annual wholesale acquisition cost (WAC) similar to other Sarepta exon-skipping therapies. ™ ● Nulibry (fosdenopterin): Origin Biomed’s Nulibry has been approved as the first therapy to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. MoCD Type A is a rare, inborn error of metabolism caused by mutations in the MOCS1 gene that results in toxic sulfite accumulation in the brain. Nulibry was approved by the FDA based on three clinical trials that demonstrated Nulibry reduced the risk of death by 82% when compared to no treatment at all.3 The survival rate at 3 years was reported to be 84% in the Nulibry arm compared to 55% for untreated patients in the historical group. Nulibry is a once daily weight-based IV formulation that is intended for health care administration; however if appropriate Nulibry can be administered at home by a caregiver or patient after detailed instructions. Nulibry has launched with a WAC of $1,370 per vial. While the information in this newsletter is from sources we believe to be reliable, we do not warrant that the information in this document is free from error. Use it only as a guide. Statements regarding drugs or manufacturers are not intended as promotion; those statements should not be used to make assumptions about formulary status. Each trademarked drug name is the property of its respective owner. Specialty Pipeline Monthly Update: March 2021 Page 2 ™ ● Pepaxto (melphalan flufenamide): The FDA has granted accelerated approval to Oncopeptides’ Pepaxto in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38- directed monoclonal antibody. Pepaxto’s approval was based on the HORIZON study that demonstrated Pepaxto in combination with dexamethasone had an overall response rate (ORR) of 23.7% and a median duration of response of 4.2 months.4 Continued approval for this indication may be contingent upon verification of confirmatory trial. Pepaxto has launched with a WAC of $9,500/vial or $19,000 per 28 days. ™ ● Fotivda (tivozanib): The FDA has approved Aveo Oncology’s Fotivda for the treatment of adults with relapsed or refractory advanced renal cell carcinoma (RCC) who have received two or more prior systemic therapies. Fotivda is an oral, next-generation vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI). Fotivda’s approval was based on a Phase 3 Study, Tivo-3 that delayed cancer progression by a median of 5.6 months for Fotivda compared with 3.9 months for the Bayer’s Nexavar™ (sorafenib) patients.5 Fotivda did not demonstrate it could help patients live longer because Fotivda had an overall survival of 16.4 months compared to 19.2 months with Nexavar. Aveo plans to make Fotivda available by March 31, 2021 with a WAC of $24,150 per month. NEW INDICATIONS ® ● Libtayo (cemiplimab-rwlc): The FDA granted approval of Regeneron’s Libtayo to include the treatment of locally advanced or metastatic basal cell carcinoma (BCC). ® ● Actemra (tocilizumab): Genentech’s Actemra has been granted a new indication by the FDA to slow the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease. Specialty Pipeline Monthly Update: March 2021 Page 3 MARCH NEWS ● “The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of roxadustat (FibroGen) for treatment of anemia in chronic kidney disease (CKD). The California Technology Assessment Forum (CTAF), one of ICER’s three independent evidence appraisal committees, deliberated over these findings and took votes on the strength of evidence on comparative clinical effectiveness at a February 2021 public meeting. Two weeks after ICER’s public meeting, FibroGen announced that the FDA now plans to convene a public advisory committee meeting to discuss roxadustat’s new drug application.”6 ● “FibroGen will likely have to delay its US rollout for roxadustat once again. In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.”7 ● “Argenx SE, provided a statement in response to news from Immunovant regarding a voluntary pause of dosing in its ongoing trials for efgatigimod, an FcRn antagonist. The announcement by Immunovant was issued following an observed signal of elevated total cholesterol and LDL levels in one of its ongoing trials.”8 ● “As drugmakers respond to the COVID-19 pandemic by developing vaccines and therapeutics, many of them are losing patent protection on older—and once lucrative—medicines. Each year, many of the pharmaceutical industry’s stalwart products lose their exclusive hold on the market, offering an opportunity for generics makers to seize share with cheaper copycats. This year’s expected losses of U.S. exclusivity include the Roche macular degeneration blockbuster Lucentis, two medicines from AbbVie and a Pfizer cancer drug.”9 Specialty Pipeline Monthly Update: March 2021 Page 4 SPECIALTY NEW PRODUCT APPROVALS IN THE PAST TWELVE MONTHS ROUTE OF MONTH GENERIC NAME BRAND NAME MANUFACTURER INDICATION(S) ADMINISTRATION APPROVED fosdenopterin Nulibry® Origin Biosciences Molybdenum cofactor IV February 2021 (BridgeBio) deficiency (MoCD) Type A casimersen Amondys 45® Sarepta Duchenne muscular dystrophy IV February 2021 Therapeutics (DMD) evinacumab Evkeeza™ Regeneron Homozygous familial IV February 2021 hypercholesterolemia (HoFH) voclosporin Lupkyis™ Aurinia Lupus nephritis Oral January 2021 ansuvimab-zykl Ebanga™ Ridgeback Treatment of Zaire ebolavirus IV December 2020 Biotherapeutics (Ebolavirus) infection berotralstat Orladeyo™ BioCryst Prevention of hereditary Oral December 2020 angioedema (HAE) attacks setmelanotide Imcivree™ Rhythm Certain types of genetic obesity SC November 2020 Pharmaceuticals lumasiran Oxlumo™ Alnylam Primary hyperoxaluria type 1 SC December 2020 (PH1) lonafarnib Zokinvy™ Eiger Progeria and progeroid Oral November 2020 BioPharmaceuticals laminopathies mannitol for inhalation Bronchitol™ Chiesi Group and Cystic fibrosis Inhaled November 2020 Pharmaxis remdesivir Veklury™ Gilead COVID-19 IV October 2020 atoltivimab, maftivimab and Inmazeb™ Regeneron Ebola IV October 2020 odesivimab-ebgn somapacitan-beco Sogroya™ Novo Nordisk Adult growth hormone SC August 2020 deficiency ofatumumab Kesimpta™ Novartis and Relapsing forms of multiple SC August 2020 Genmab sclerosis (RMS) cysteamine ophthalmic solution Cystadrops™ Recordati Rare Corneal cystine crystal deposits Eye drop August 2020 0.37% Disease satralizumab-mwge Enspryng™ Roche Neuromyelitis optica spectrum SC August 2020 disorder (NMOSD) risdiplam Evrysdi™ Genentech Spinal muscular atrophy Oral August 2020 viltolarsen Viltepso™ Nippon Shinyaku Duchenne muscular dystrophy IV August 2020 triheptanoin Dojolvi™ Ultragenyx Long-chain fatty acid oxidation Oral July 2020 disorders calcium, magnesium, Xywav™ Jazz Excessive daytime sleepiness Oral July 2020 potassium, and sodium Pharmaceuticals oxybates fenfluramine Fintepla™ Zogenix Seizures associated with Dravet Oral June 2020 syndrome triheptanoin Dojolvi™ Ultragenyx Long-chain fatty acid oxidation Oral June 2020 disorders octreotide Mycapssa™ Chiasma Acromegaly Oral June 2020 inebilizumab-cdon Uplizna™ Viela Bio Neuromyelitis optica spectrum IV June 2020 disorder (NMOSD) apomorphine HCI sublingual Kynmobi™ Sunovion Parkinson’s disease OFF SL film May 2020 film episodes artesunate Artesunate™ Amivas Malaria IV May 2020 continued Specialty Pipeline Monthly Update: March 2021 Page 5 SPECIALTY NEW PRODUCT APPROVALS IN THE PAST TWELVE MONTHS (continued) ROUTE OF MONTH GENERIC NAME BRAND NAME MANUFACTURER INDICATION(S) ADMINISTRATION APPROVED deferiprone Ferriprox™ ApoPharma Thalassemia syndromes Oral May 2020 leuprolide acetate Fensolvi™ Tolmar Central precocious puberty SC May 2020 Pharmaceuticals
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