FDA Grants Acceleron Breakthrough Therapy Designation for Experimental PAH Therapy

Rare Daily Staff

The U.S. Food and Drug Administration awarded Acceleron Pharma Breakthrough Therapy designation to its experimental drug sotatercept for the treatment of patients with the rare and chronic condition pulmonary arterial hypertension.

Pulmonary arterial hypertension (PAH) is a rapidly progressing disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant strain on the heart, often leading to limited physical activity, heart failure, and reduced life expectancy. The five-year survival rate for patients with PAH is approximately 57 percent. Available therapies generally act by promoting the dilation of pulmonary vessels without addressing the underlying cause of the disease.

Sotatercept is an experimental therapy that targets a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure. Recent topline analysis of a phase 2 trial of sotatercept in patients with PAH showed the trial met the primary as well as key and other secondary endpoints, with adverse events consistent with previously published data on sotatercept in other diseases.

“Based on the results, we believe that sotatercept has the potential to shift the current treatment paradigm and provide significant benefit to patients with PAH on top of currently available therapies,” said Habib Dable, president and CEO of Acceleron.

The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may provide substantial improvement on at least one clinically significant endpoint over available therapy.

A Breakthrough Therapy designation conveys more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. In 2019, the FDA granted Orphan Drug designation for sotatercept in PAH.

Photo: Habib Dable, president and CEO of Acceleron