On September 28, MSD announcedThe Biologics License Application (BLA) for Sotatercept for the treatment of pulmonary arterial hypertension (PAH) has been granted Priority Review by the FDA, with a PDUFA date set for March 26, 2024.SotaterceptBLA mainly refers toBased on the positive results of the Phase III STELLAR study.The study enrolled a total of 323 adult PAH patients who were receiving stable background therapy, aiming to evaluate the efficacy and safety of Sotatercept compared to placebo as an add-on therapy.The results showed that at Week 24 of treatment,The 6-minute walking distance (6MWD) of patients in the Sotatercept group increased by 34.4 meters from baseline, compared to 1.0 meter in the placebo group. According toHodges-Lehmann test, the inter-group difference between the two groups is 40.8 meters (95% CI: 27.5 to 54.1; P<0.001）。In addition, the study also successfully reached eight secondary endpoints, including NT-proBNP levels, time to first reported death or clinical worsening event,PAH-SYMPACT Physical Impact Domain Scores, etc.However, one remaining secondary endpoint was not met, namelyPAH-SYMPACTNo significant improvement in the cognitive/emotional impact domain score.
STELLAR StudyEfficacy Data (Source: NEJM)
PAH is a rare, progressive, and life-threatening cardiovascular disease.The clinical manifestations areProliferative remodeling of small pulmonary arteries and gradual luminal narrowing。According toEstimateIt is estimated that there were approximately 40 million PAH patients globally in 2021., with a 5-year mortality rate of approximately 43%.Currently，Existing PAH therapies on the market canBy promoting pulmonary vasodilation to alleviatePatient's condition, but unable to fromFundamentally SolvePulmonary Vascular RemodelingQuestion.Research shows that transformationLong Factorβ（TGF-β) Superfamily member-mediated cell proliferation and anti-Apoptosis SignalPathwayImbalance is one of the key mechanisms driving pulmonary vascular remodeling in PAH patients.ActivinType 2AReceptor(ACVR2A) is one of the members of the TGF-β superfamily. Therefore,TargetedDrugs targeting ACVR2A are a potentially effective approach to reverse pulmonary vascular remodeling.SotaterceptYesAcceleron Pharma has developed a first-in-class ACVR2A-Fc fusion protein that selectively binds to TGF-β superfamily ligands.Restoring the balance between pro-proliferative and anti-proliferative signaling pathways associated with pulmonary artery wall and right ventricular remodeling, which inhibits cell proliferation, reverses vascular remodeling, and promotes vascular patency.Sotatercept is also currently the only drug specifically designed to treat PAH.ACVR2A-targeted drugs.2020April of the year,Sotatercept with Phase IIPositive Results of the PULSAR StudyBlocked by FDA Granted Breakthrough Therapy DesignationGrid.September 2021,Acceleron Pharma wasMSD acquired it for $11.5 billion, and the product also belongs to MSD.Copyright © 2023 PHARMCUBE. All Rights Reserved.
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