FDA grants Orphan Drug Designation to sotatercept

Orphan Drug Designation has been given to sotatercept for the treatment of patients with pulmonary arterial hypertension.

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH).

“We’re pleased that the FDA has granted this designation for sotatercept,” said Janethe de Oliveira Pena, MD, PhD, Vice President, Pulmonary Medical Research at Acceleron, which manufactures the treatment. “In pre-clinical studies, sotatercept has demonstrated an ability to target the underlying mechanisms of PAH, which is a rare disease of high unmet medical need.

We believe that if similar effects are seen in a clinical setting, sotatercept has the potential to become an important addition to the standard of care in PAH.”

Sotatercept is being evaluated in two Phase II trials in patients with PAH: the PULSAR trial, which completed its target enrollment in June 2019 and the SPECTRA exploratory trial, which is currently enrolling. The company expects to report top-line results from the PULSAR trial during the first quarter of 2020.

ODD is granted by the FDA to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the US.

Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits and seven years of market exclusivity in the US following marketing approval by the FDA. 

Sotatercept is an investigational therapy that is not approved for any use in any country.