FDA Updates for Week of November 1, 2021

Merck receives first approval for oral antiviral to treat COVID-19.

The United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has authorized molnupiravir, the first oral antiviral medicine authorized for the treatment of mild-to-moderate COVID-19. Developed by Merck and Ridgeback Biotherapeutics, the therapy will have the brand name Lagevrio in the UK.

The authorization is based on positive results from a planned interim analysis from the phase 3 MOVe-OUT clinical trial, which evaluated molnupiravir 800 mg twice-daily in non-hospitalized, unvaccinated adult patients with mild-to-moderate COVID-19. Molnupiravir reduced the risk of hospitalization or death by about 50%. Through Day 29, no deaths were reported in patients who received molnupiravir, as compared with eight deaths in patients who received placebo. The incidence of any adverse event was comparable in the molnupiravir and placebo groups.

Last month Merck submitted its application to the FDA, and an FDA advisory committee will meet on Nov. 30, 2021, to discuss an emergency use authorization for molnupiravir. Merck has also submitted an application for molnupiravir to the European Medicines Agency.

FDA rejects EUA for COVID-19 respiratory failure therapy.

The FDA has declined to issue an emergency use authorization (EUA) for NRx Pharmaceuticals’ Zyesami (aviptadil) to treat patients with COVID-19-related respiratory failure. Regulatory officials said there is not enough data on the risks and benefits of the therapy; officials have reviewed safety data for only 131 randomized patients treated with Zyesami.

NRx officials said in a statement that 150 or more additional patients have already been treated with Zyesami in the National Institutes of Health ACTIV-3b trial. Last week, the study’s data safety and monitoring board found no new safety issues. The NIH study, which has enrolled more than 300 patients, is a confirmatory study that randomized patients with COVID-19 respiratory failure to Zyesami or Veklury (remdesivir) and placebo.

A second nationwide trial to determine if similar benefits may be achieved in critically ill patients with an inhaled version is being conducted on the I-SPY platform, maintained by the Quantum Leap Healthcare Collaborative. NRx also continues to study the effect of inhaled Zyesami in patients with severe but not critical COVID-19 in a placebo-controlled trial that aims to demonstrate the ability of the therapy. to keep patients from requiring intensive care.

FDA approves novel therapy for chronic myeloid leukemia.

The FDA has approved Novartis’ Scemblix (asciminib) to treat patients with chronic myeloid leukemia (CML). The FDA issued accelerated approval for adults with Philadelphia chromosome-positive CML in the chronic phase and full approval for adults with Philadelphia chromosome-positive CML with the T315I mutation.

Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available tyrosine kinase inhibitor (TKI) therapies. This is a first-in-class therapy of a STAMP (specifically targeting ABL myristoyl pocket) inhibitor. The BCR-ABL1 gene is associated with the overproduction of leukemia cells.

FDA approves therapy for age-related blurry vision.

The FDA has approvedVuity (pilocarpine HCl ophthalmic solution) 1.25% for the treatment of adults with presbyopia, age-related blurry near vision. This is the only FDA-approved eye drop to treat this progressive eye condition that affects 128 million Americans, nearly half of the U.S. adult population. Vuity was developed by Allergan, an AbbVie company.

Vuity is a daily, prescription eye drop that works in as little as 15 minutes and lasts up to 6 hours to improve near and intermediate vision without impacting distance vision. Specifically designed for presbyopia, Vuity uses a proprietary technology allows the therapy to rapidly adjust to the physiologic pH of the tear film. It uses the eye’s own ability to reduce pupil size, improving near vision without affecting distance vision.

FDA extends review of Janssen’s CAR T therapy for multiple myeloma.

The FDA has extended the review of Janssen’s biologics license application for ciltacabtagene autoleucel (cilta-cel) to Feb. 28, 2022. The extension was implemented to give the agency time to review information provided by the company on the analytical method. This information was requested by the FDA. No additional clinical data have been requested.

Cilta-cel is a B-cell maturation antigen (BCMA) chimeric antigen receptor T cell (CAR-T) therapy, is being investigated for the treatment of adults with multiple myeloma. Janssen had initiated its rolling submission in December 2020.

Studies are ongoing.

Cilta-cel was developed by Legend Biotech, and in December 2017, Janssen Biotech entered into a collaboration agreement with Legend Biotech to develop and commercialize cilta-cel.