“In January of this year, we reported positive topline results from our PULSAR Phase 2 placebo-controlled trial of sotatercept in patients with PAH,” said Habib Dable, President and Chief Executive Officer of Acceleron. “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. Thus, we’re thrilled that the FDA has granted this Breakthrough Therapy designation—a first for an Acceleron-discovered medicine and for a therapeutic candidate in PAH—as it supports and aligns with our mission to deliver novel therapeutic options to patients in need as quickly as possible.”
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.
Source: Company Press Release