The European Commission (EC) has approved sotatercept, in combination with other therapies for pulmonary arterial hypertension (PAH), for the treatment of PAH in adult patients with functional class (FC) II to III of the World Health Organization (WHO) to improve exercise capacity. The announcement was made by MSD – known as Merck & Co. in the United States and Canada – in a note specifying that sotatercept is the first and only activin signal inhibitor therapy for PAH approved in all 27 EU Member States, as well as Iceland, Liechtenstein and Norway. The drug works by improving the balance between pro-proliferative and anti-proliferative signaling to regulate the proliferation of vascular cells underlying PAH. The European Commission’s approval of sotatercept is based on the safety and efficacy results of the Phase 3 Stellar study.
“The EC approval of sotatercept is an important milestone for patients, as it is the first therapy to target the activin signaling pathway,” said Joerg Koglin, M.D., SVP and Head of General Medicine, Global Clinical Development, Merck Research Laboratories. “We are proud to bring this innovative treatment to more patients and remain committed to further exploring the potential of sotatercept in areas of unmet need in the management of PAH.”
PAH is a rare, progressive, high-risk blood vessel disorder characterized by narrowing of the small pulmonary arteries and high blood pressure in the pulmonary circulation. This places significant strain on the heart, resulting in limited physical activity, heart failure, and reduced life expectancy. The 5-year mortality rate is approximately 43%, according to data from the Reveal registry. It is “a disabling disease,” explains Marc Humbert, professor of medicine and director of the Pulmonary Hypertension Reference Center at the Université Paris-Saclay. “Patients suffer from debilitating symptoms that can severely limit their daily activities.” For this reason, “new treatment options continue to be needed. Based on the Phase 3 Stellar study, adding sotatercept to standard PAH therapy improved exercise capacity, reduced the risk of death or clinical worsening events, and improved functional class compared with standard PAH therapy alone. These results – adds Humbert – are significant and reinforce the idea that sotatercept, in combination with other therapies for PAH, should be considered a new standard of care for the treatment of adult patients in functional class II and III”.
In detail, the Stellar study compared sotatercept (n=163) with placebo (n=160) both in combination with standard therapies in adult patients with PAH (Group 1, WHO Cf II or III). The primary efficacy endpoint was the change from baseline to week 24 in six-minute walk distance. Treatment with sotatercept resulted in a statistically and clinically significant improvement in six-minute walk distance of 40.8 meters compared to placebo. Treatment also significantly improved several important secondary outcomes, including a reduction in the risk of death or clinical worsening.
In a post hoc analysis provided to the European Medicines Agency (EMA), time to death or clinical worsening was defined as the time from randomisation to the first occurrence of worsening of PAH, PAH-specific hospitalisation, inclusion on a waiting list for lung and/or heart transplant related to worsening, need for atrial septostomy, or death from any cause. The risk of death or clinical worsening was reduced by 82% with sotatercept added to standard therapy compared to standard therapy alone. Sotatercept is administered once every 3 weeks as a single subcutaneous injection and can be administered by appropriately guided and trained patients or caregivers and with follow-up by a healthcare professional. Healthcare professionals and patients/caregivers should refer to the instructions for use containing information on the correct preparation and administration of sotatercept.
The treatment has already obtained the designation of Priority Medicine (Prime) and orphan drug for the treatment of PAH from the EMA. On March 26, 2024, sotatercept was approved by the American Agency Fda in the United States for adults with PAH, WHO Group 1, to increase exercise capacity, improve WHO functional class (Cf) and reduce the risk of worsening clinical events.
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