Acceleron Pharma has announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH).
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.
Preclinical studies showed that sotatercept reduced pulmonary arterial pressures and reversed pulmonary vessel muscularization, cellular proliferation, and right ventricular hypertrophy. The Company is currently conducting two phase 2 trials, PULSAR and SPECTRA, to evaluate the efficacy and safety of sotatercept in patients with PAH.
“We’re pleased that the FDA has granted this designation for sotatercept,” said Janethe de Oliveira Pena, MD, PhD, VP, Pulmonary Medical Research at Acceleron. “In preclinical 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.”
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