Update for June 30, 2020

Remdesivir – Update on Pricing and availability
 
Additional information became available on Monday, 29 June, 2020, so we have provided an update regarding pricing and availability.
 
The FDA approved an emergency use authorization (EUA) for remdesivir to treat hospitalized patients with severe COVID-19. HHS signed an Agreement with Gilead for 500,000 treatment courses of remdesivir on 6/29/2020. This represents 100% of July production, 90% of August production and 90% of September production. The U.S. The Department of Health and Human Services will control distribution through AmerisourceBergen and hospitals will pay no more than WAC for the drug. The government will tell AmerisourceBergen how many vials can be shipped to a state. The department of health in each state will direct the wholesaler to deliver doses to the hospitals with the highest number of inpatient COVID-19 cases. The initial supply from the Federal Stockpile of 5 million doses were exhausted at the end of June 2020. HHS will stop managing distribution of remdesivir at the end of September, when supplies are expected to stabilize.
 
Gilead has set AWP for remdesivir (Veklury) at $520 per vial or $3,120 for five days of treatment. This is the price for private insurance patients in the U.S. The U.S. government price for Medicaid and military hospitals is $390 per vial or $2,340 for a five-day course of treatment. This is also the price for developed countries. Pricing for remdesivir is more aligned with estimates from ICER that take into account the potential effect of dexamethasone. Gilead has entered into agreements with generic manufacturers to make the drug available at a substantially lower cost to developing countries, where healthcare resources, infrastructure and economics are lower. 
 
Regulatory Update
 
The FDA rejected the BLA for abicipar pegol in June 2020 due to an unfavorable benefit-risk ratio in the treatment of neovascular (wet) age-related macular degeneration.
 
CSL Behring licensed worldwide marketing rights for hemophilia B gene therapy etranacogene dezaparvovec from uniQure.
 
Sarepta submitted an NDA for casimersen for the treatment of Duchenne muscular dystrophy with genetic mutations that are amenable to skipping exon 45 of the Duchenne gene.
 
Ascendis submitted a BLA for lonapegsomatropin for the treatment for pediatric growth hormone deficiency.

The FDA granted seviprotimut-L a Fast Track designation as an adjuvantive treatment of stage IIB/IIC melanoma patients, post-resection to improve recurrence-free survival.
 
Announced Research Updates
 
Orphazyme announced that in the 6-month, 37 patient, Phase II, dose-ranging, ORARIGAU-01 trial, treatment with arimoclomol 100mg, 200mg, or 400mg did not reduce serum chitotriosidase activity compared to placebo in patients with Gaucher disease.
 
Acceleron announced that in the 24-week, 106 patient, Phase II, PULSAR trial, treatment with sotatercept reduced pulmonary vascular resistance by 20.5% with 0.3 mg/kg (n = 32) and 33.9% with 0.7 mg/kg (n=42) compared to a 2.1% reduction with placebo in patients with pulmonary arterial hypertension.
 
Rhythm Pharmaceuticals announced 12-week interim results from 75 patients enrolled in a Phase II trial, where a weekly setmelanotide formulation achieved similar weight loss to a daily formulation in healthy obese patients.
 
Published Research Updates
 
In the 87 patient, Phase II MAJA trial, maintenance therapy with vinflunine plus best supportive care resulted in a mortality rate of 59.1% compared to 74.4% mortality with only best supportive care in parents with advanced urothelial carcinoma.
 
In the 527 patient, Phase III FALUCA trial, treatment with fruquintinib did not improve overall survival compared to placebo (8.9 months vs 10.4 months) in patients with locally advanced/metastatic nonsquamous non-small-cell lung cancer.