Sotatercept Receives Breakthrough Therapy Status for Pulmonary Arterial Hypertension

FDA Breakthrough Therapy Status logo
The FDA has granted Breakthrough Therapy designation to sotatercept for the treatment of PAH.

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to sotatercept (Acceleron Pharma) for the treatment of pulmonary arterial hypertension (PAH) (World Health Organization Group 1).

Sotatercept is a ligand trap that has high selectivity for members of the transforming growth factor (TGF)-beta superfamily. The investigational agent is believed to block the activin signaling pathway, which may rebalance bone morphogenetic protein receptor type 2 (BMPR2) signaling and restore vascular homeostasis.

The Breakthrough Therapy designation is supported by positive topline data from the phase 2 PULSAR trial that compared the efficacy and safety of sotatercept to placebo in 106 adult patients with PAH for 24 weeks. Patients were randomized 4:3:3 to receive sotatercept 0.7mg/kg or 0.3mg/kg subcutaneously, or placebo every 21 days in combination with standard of care. 

Results demonstrated that sotatercept met the primary end point achieving a statistically significant reduction in pulmonary vascular resistance at week 24 compared with placebo. Additionally, sotatercept met the key secondary end point, with statistically significant improvements in 6-minute walk distance (6MWD).

With regard to safety, sotatercept was well tolerated, and adverse events were found to be consistent with those reported in previously published data. 

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“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. 

Full detailed results will be presented at a medical conference later this year. In September 2019, the FDA granted Orphan Drug designation to sotatercept for this indication. The Company is also currently investigating sotatercept in the phase 2 SPECTRA trial for PAH. 

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This article originally appeared on MPR