FDA decision on sotatercept for PAH expected in March

In clinical trial, therapy increased walk time for those given the drug

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking approval of sotatercept as a treatment for adults with pulmonary arterial hypertension (PAH).

The FDA has given the application priority review, shortening the review time from the usual 10 months to six months. A decision is expected by March 26.

“The FDA’s acceptance of this application is an exciting milestone in our journey to bring this novel activin signaling inhibitor to patients,” Joerg Koglin, MD, PhD, senior vice president of global clinical development at Merck Research Laboratories, said in a company press release. Merck acquired exclusive rights to sotatercept in pulmonary hypertension through the acquisition of Acceleron Pharma.

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PAH is a disorder marked by increased pressure in the vessels that carry blood from the heart through the lungs. Structural alterations in these vessels make them narrow, increasing blood pressure and putting strain on the heart.

“Despite advances in the treatment of PAH over the last two decades, there is still a significant need to improve outcomes for patients,” Koglin said.

Sotatercept is designed to reduce the abnormal growth of blood vessel cells in PAH by rebalancing the activity of signaling molecules that control cell growth.

STELLAR clinical trial data

Merck’s application seeking approval of the therapy is based on data from a Phase 3 clinical trial called STELLAR (NCT04576988), which evaluated 323 adults with PAH. Participants were given either sotatercept or a placebo, on top of standard treatments, for about six months. The study drug was administered via subcutaneous (under-the-skin) injection every three weeks, with sotatercept started at a dose of 0.3 mg/kg and then increased up to 0.7 mg/kg.

The main goal of STELLAR was to assess the impact of sotatercept on the distance that patients could walk in six minutes, which is a common measure of exercise capacity in people who are able to walk. Results showed that, after six months, patients given sotatercept could walk on average about 40 meters (131 feet) more than those given placebo.

Data from STELLAR also indicated that sotatercept improved measures of heart health and of blood flow in the lungs.

“Based on the profound improvements across primary and secondary outcome measures in the Phase 3 STELLAR trial, we believe sotatercept has the potential to transform the treatment of patients with PAH,” Koglin said. “We look forward to working closely with the FDA to bring sotatercept to patients in need.”

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