FDA grants breakthrough designation to sotatercept for PAH

The FDA has granted breakthrough therapy designation to sotatercept for treatment of pulmonary arterial hypertension, according to a press release from Acceleron Pharma.

Sotatercept is an investigational agent designed to be a selective ligand trap for members of the transforming growth factor-beta superfamily to rebalance bone morphogenetic protein receptor type II (BMPR-II). It has been shown in preclinical studies to reverse pulmonary vessel muscularization and improve indicators of right heart failure. In 2019, the drug received orphan drug designation for PAH from the FDA, and earlier this year, sotatercept met the primary and key secondary endpoints of the PULSAR phase 2 trial.

“In January of this year, we reported positive top-line results from our PULSAR phase 2 placebo-controlled trial of sotatercept in patients with PAH,” Habib Dable, president and CEO of Acceleron Pharma, said in the release. “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 ... as it supports and aligns with our mission to deliver novel therapeutic options to patients in need as quickly as possible.”

The FDA has granted breakthrough therapy designation to sotatercept for treatment of pulmonary arterial hypertension, according to a press release from Acceleron Pharma.

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Sotatercept is also being evaluated in the SPECTRA phase 2 trial in patients with PAH, according to the release.