FDA Accepts Merck's (MRK) Filing for Sotatercept in PAH Disease
Merck MRK announced that the FDA had accepted the biologics license application (“BLA”) seeking approval for sotatercept, its novel investigational activin signaling inhibitor, as a potential treatment for adult patients with pulmonary arterial hypertension (WHO Group 1) [“PAH”].
The agency has granted priority review to this filing. A final decision is expected by Mar 26, 2024.
If approved, Merck’s sotatercept will provide a new treatment option for patients and bring a novel approach to PAH treatment.
The BLA filing is based on data from the phase III STELLAR study, which evaluated the safety and efficacy of sotatercept against placebo as an add-on to the current standard of care for adults with PAH. The study achieved its primary endpoint of sotatercept exhibiting significant improvement in exercise capacity, as measured by 6-minute walk distance (6MWD), and eight of nine key secondary outcome measures.
PAH is a rare, progressive blood vessel disorder that affects the arteries in the lungs and the right side of the heart, resulting in elevated blood pressure in the pulmonary circulation. It exerts significant strain on the heart, causing limited physical activity, heart failure and reduced life expectancy. The five-year mortality rate for patients with this disease is around 43%. Per Merck, around 40,000 people in the United States suffer from PAH.
Sotatercept is designed to target cellular signaling associated with vascular hyperproliferation and pathological remodeling to treat PAH. The FDA has granted the drug breakthrough therapy and orphan drug designations for PAH treatment.
Merck added sotatercept following its acquisition of Acceleron Pharma in 2021. The drug is the subject of a licensing agreement with Bristol Myers Squibb BMY. Following the acquisition of Acceleron, Merck also added the anemia drug Reblozyl to its portfolio of marketed drugs. Reblozyl is being developed and commercialized through a global collaboration with Bristol Myers Squibb.
Merck receives milestone and royalty payments on Reblozyl product sales from Bristol Myers as part of the collaboration. Bristol Myers generated $440 million from Reblozyl product sales in first-half 2023, up 34% year over year, driven by higher demand.
A significant player in the target market is United Therapeutics UTHR which markets four drugs to treat PAH in the United States – Remodulin, Orenitram, Tyvaso and Adcirca.
UTHR markets Tyvaso, which is an inhaled formulation of treprostinil while Remodulin is an injectable formulation. United Therapeutics also markets Orenitram, the oral version of treprostinil and Adcirca (tadalafil) tablets.
Last year in May, the FDA approved United Therapeutics’ new drug application (“NDA”) for the dry powder inhalation (“DPI”) formulation for Tyvaso. The NDA also included a device for the administration of the new formulation. As a result of this approval, UTHR has generated $557.3 million from Tyvaso sales in first-half 2023, up 49% year over year.
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