FDA grants priority review for Merck’s PAH drug label update

Published 02/07/2025, 11:50
FDA grants priority review for Merck’s PAH drug label update

RAHWAY, N.J. - The U.S. Food and Drug Administration has accepted and granted priority review for Merck’s (NYSE:MRK) supplemental application seeking to update the label for WINREVAIR based on results from the Phase 3 ZENITH trial. The FDA has set a target action date of Oct. 25, 2025. Merck, a prominent player in the pharmaceuticals industry with an impressive "GREAT" financial health score according to InvestingPro, continues to demonstrate strong market presence with a substantial market capitalization of $205 billion.

WINREVAIR, approved in 2024 for adults with pulmonary arterial hypertension (PAH), is currently authorized to increase exercise capacity, improve functional class, and reduce clinical worsening events. The new application is based on data from the ZENITH trial, which was stopped early due to efficacy results. This development adds to Merck’s robust pharmaceutical portfolio, which generated revenues of $63.9 billion in the last twelve months, with an impressive gross profit margin of 77%.

According to a company press release, the ZENITH trial demonstrated a 76% reduction in the risk of a composite endpoint including all-cause death, lung transplantation, and PAH-related hospitalization compared to placebo. The study focused on patients with WHO functional class III or IV PAH at high risk of mortality.

"There remains a significant unmet medical need for patients living with PAH who, despite being on background therapy, remain at higher risk of morbidity and mortality," said Dr. Joerg Koglin, senior vice president of global clinical development at Merck Research Laboratories.

WINREVAIR is the first activin signaling inhibitor therapy approved for PAH treatment. The drug works by improving the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation.

Common side effects reported in clinical trials include headache, nosebleeds, rash, and dizziness. The medication may increase hemoglobin levels and decrease platelet count, requiring monitoring.

PAH is a rare, progressive disorder affecting approximately 40,000 people in the U.S. The disease is characterized by constriction of small pulmonary arteries and elevated blood pressure in pulmonary circulation, with a five-year mortality rate of approximately 43%.

WINREVAIR is currently approved in more than 45 countries based on results from a previous trial called STELLAR.

In other recent news, Merck has received significant developments across its product lines. The company’s Enflonsia vaccine gained a key recommendation from the U.S. Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices as a preventive option against respiratory syncytial virus in infants. This recommendation, which still requires final approval, could enhance insurance coverage and access to the vaccine. Additionally, Merck’s Winrevair showed positive results in the Phase 3 HYPERION study for pulmonary arterial hypertension, meeting its primary endpoint and reducing the risk of clinical worsening events. Morgan Stanley maintained an Equalweight rating on Merck following these results, indicating steady confidence in the company’s performance.

In another development, the U.S. Food and Drug Administration approved Merck’s Keytruda for treating locally advanced head and neck squamous cell carcinoma. The approval was based on the Phase 3 KEYNOTE-689 trial, which demonstrated a significant reduction in the risk of event-free survival events. TD Cowen maintained a hold rating on Merck, reflecting a balanced view despite positive vaccine developments. These recent advancements highlight Merck’s ongoing efforts to expand its portfolio and impact various healthcare segments.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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