Acceleron ‘Breakthrough’ for Pulmonary Hypertension Drug

FDA has granted Acceleron Pharma a breakthrough therapy designation for sotatercept and its use in treating patients with pulmonary arterial hypertension (PAH). Last year, the agency granted an orphan drug designation for the therapy. Sotatercept is being developed under a licensing agreement with Bristol Myers Squibb.

Acceleron says sotatercept was designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR-II signaling, which is a key molecular driver of PAH. In preclinical studies, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure, it says. “Recent topline analysis of the PULSAR Phase 2 trial of sotatercept in patients with PAH revealed 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,” it adds.