FDA Updates for Week of Nov. 15, 2021


In COVID-19 news, FDA expands EUA for Moderna/Pfizer boosters and Pfizer seeks EUA for oral antiviral. Keytruda receives another indication, first therapy is approved for dwarfism, FDA extends review of mavacamten, and Gilead submits application for chronic hepatitis therapy.

FDA expands EUA for Moderna and Pfizer COVID-19 boosters.

The FDA has extended both the Moderna and Pfizer-BioNTech COVID-19 vaccines authorizing use of a single booster dose for all people 18 years of age and older.

Previously, a single booster dose of both vaccines was authorized for people 65 years of age and older and those at risk of severe COVID-19 or those with occupational exposure.

The boosters are now authorized for those after at least six months after completion of the primary series for the Moderna or BioNTech vaccines or at least two months after completion with the Janssen vaccine.

Pfizer seeks EUA for COVID-19 oral antiviral.

Pfizer has submitted an application for emergency use authorization for Paxlovid (ritonavir), an oral antiviral for the treatment of mild-to-moderate COVID-19 in patients at increased risk of hospitalizations or death.

If authorized, this would be the first oral antiviral of its kind, a 3CL protease inhibitor specifically designed to combat the virus that causes COVID-19.

This submission includes clinical data from the phase 2/3 EPIC-HR, which enrolled non-hospitalized adults aged 18 and older with confirmed COVID-19 who are at increased risk of progressing to severe illness.

FDA approves first drug for a form of dwarfism.

The FDA has granted accelerated approval to BioMarin Pharmaceutical’s Voxzogo (vosoritide) injection to improve growth in children five years of age and older with achondroplasia, the most common form of dwarfism. People with achondroplasia have a genetic mutation that causes fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth.

Voxzogo works by binding to a specific receptor called natriuretic peptide receptor-B that reduces the growth regulation gene’s activity and stimulates bone growth.

Continued approval for this indication is contingent upon confirmatory studies. BioMarin is conducting ongoing open-label extension studies.

Gilead submits application for chronic hepatitis therapy.

Gilead Sciences has submitted a biologics license application (BLA) to the FDA for bulevirtide for injection, a potential first-in-class antiviral medicine for the treatment of chronic hepatitis delta virus (HDV) infection in adults. Bulevirtide has been granted breakthrough therapy and orphan drug designations by the FDA.

The submission is supported by data from completed and ongoing phase 2 studies and the ongoing phase 3 MYR301 study, which supports the safety and efficacy of bulevirtide once daily after 24 weeks of therapy.

Interim results from the phase 3 MYR301 study indicate that after 24 weeks of therapy, the proportion of people with HDV infection achieving the combined virological and biochemical response was 36.7% with bulevirtide 2 mg, 28% in participants receiving bulevirtide 10 mg and 0% in participants currently under observation who have not received antiviral treatment at this stage of the study.

In Europe, Hepcludex (bulevirtide) has been granted conditional marketing authorization by the European Commission and PRIority MEdicines (PRIME) scheme eligibility by the European Medicines Agency, as the first approved treatment in Europe for adults with chronic HDV infection with compensated liver disease.

FDA extends review of mavacamten.

The FDA has extended the review of Bristol Myers Squibb’s new drug application for mavacamten, which is being reviewed as a treatment for patients with symptomatic obstructive hypertrophic cardiomyopathy, to April 28, 2022.

The agency has extended the PDUFA date to assess information pertaining to updates to the proposed risk evaluation mitigation strategy (REMS). A REMS program was included in the initial application for mavacamten. No additional data or studies have been requested.

The Institute for Clinical and Economic Review (ICER), in a final evidence report, has found concerns about long-term safety indicate there may not be a benefit for mavacamten when added to first-line therapy. ICER estimates that for mavacamten to be cost-effective, it would need to be priced between $12,000 and $15,000 annually.

FDA grants approval to Keytruda for early stage RCC.

The FDA has approved Merck’s Keytruda (pembrolizumab) for the treatment of patients with renal cell carcinoma (RCC) after surgery. This approval is the fourth indication in earlier stages of cancer. Other early cancer indications include: early stage, triple negative breast cancer; as a first-line treatment of head and neck cancer; and as a first-line treatment in nonsmall cell lung cancer.

The RCC indication is based on a phase 3 trial in which Keytruda demonstrated an improvement in disease-free survival. The median duration of response was 11.1 months. Severe reactions occurred in 20% of patients and included: acute renal injury, adrenal insufficiency, pneumonia, colitis and diabetic ketoacidosis. The trial is ongoing to assess overall survival as a secondary outcome measure.

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