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Acceleron's Pulmonary Arterial Hypertension Asset Receives Orphan Drug Designation

Shanthi Rexaline

Cambridge, Massachusetts-based Acceleron Pharma Inc (NASDAQ: XLRN) said Monday that the FDA has accorded Orphan Drug Designation to its sotatercept.

The drug is an investigational asset to treat pulmonary arterial hypertension, or PAH. Sotatercept is a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key driver of PAH.

An ODD is granted by the FDA to facilitate development of effective and safe therapies for rare diseases that affect fewer than 200,000 people in the U.S.

The benefits of an ODD include grants for meeting clinical trial costs, tax advantages, FDA user-fee benefits and seven years of market exclusivity in the U.S. following FDA approval.

Sotatercept, which hasn't been approved in any country, is being evaluated in two Phase 2 trials — dubbed PULSAR and SPECTRA — in patients with PAH.

Acceleron expects to report top-line results from the PULSAR trial in the first quarter of 2020. 

Enrollment is underway for the SPECTRA study, which is an exploratory trial.

Acceleron shares were trading higher by 1.27% at $45.10 at the time of publication Monday. 

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