Acceleron Receives EMA PRIME Designation for Sotatercept in Pulmonary Arterial Hypertension

May 05, 2020 News /BioonBIOON/ -- Acceleron Pharma, a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapies targeting the TGF-β superfamily for the treatment of severe and rare diseases, recently announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to sotatercept (ACE-011, ActRIIA-Fc) for the treatment of patients with pulmonary arterial hypertension (PAH). Notably, this is the first drug to receive PRIME designation for PAH since the EMA established the program in 2016.

In April this year, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH) (WHO Group 1). In September 2019,FDASotatercept was also granted Orphan Drug Designation (ODD).

PRIME is a fast-track approval program launched by the EMA in March 2016, similar to the United States’FDAsimilar to the Breakthrough Therapy Designation (BTD) program, aims to accelerate the review process for key medicines in areas of unmet medical need, enabling patients to benefit as early as possible. Experimental drugs selected for the PRIME scheme willClinical Trialand received strong support from the EMA in drug development to accelerate the development and approval of truly innovative medicines, thereby addressing the medical need for promising new therapies. To qualify for PRIME designation, a medicine must have preliminary clinical and non-clinical evidence demonstrating that it offers a substantial improvement over existing treatments.

Breakthrough Therapy Designation (BTD) is a new drug review pathway established by the FDA in 2012, aimed at accelerating the development and review of new drugs intended to treat serious or life-threatening diseases, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies. Drugs granted BTD can receive, during development, includingFDAMore intensive guidance, including from senior officials, with eligibility for rolling review and priority review during the new drug marketing application assessment, to ensure that new treatment options are made available to patients in the shortest possible time.

Habib Dable, President and Chief Executive Officer of Acceleron Pharma, stated: “InFDAJust weeks after sotatercept was granted Breakthrough Therapy designation, it received Priority Medicines (PRIME) designation from the EMA, further strengthening our belief that sotatercept has the potential to significantly transform the treatment landscape for pulmonary arterial hypertension (PAH). We are pleased that, in ourClinical Trial“During the preliminary evaluation of the data, regulatory authorities in the United States and Europe proposed a pathway that could potentially help us accelerate the provision of sotatercept to patients in need of new treatment options.”

Sotatercept is an investigational drug,Designed as a selective ligand trap for members of the TGF-β superfamily to rebalance BMPR-II signaling, a key driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vascular muscularization and improved indicators of right heart failure.

In late January this year, Acceleron announced the top-line results from the Phase II PULSAR trial, demonstrating that the study met its primary endpoint, key secondary endpoints, and other secondary endpoints. The adverse events were consistent with those previously reported for sotatercept in other indications. As part of the licensing agreement with Celgene (acquired by Bristol-Myers Squibb), sotatercept is also being evaluated in the Phase II SPECTRA trial.

It is worth noting that in early April this year, Reblozyl (luspatercept), another TGF-β ligand trap drug developed by Acceleron in collaboration with Celgene, was approved by the U.S. FDA for a new indication: the treatment of adult patients with lower-risk myelodysplastic syndromes (MDS).Anemia。Reblozyl is the first and onlyFDAApproved erythroid maturation agents, representing a novel class of therapies, help patients reduce the burden of red blood cell transfusions by modulating the late stages of erythroid maturation.In the United States, Reblozyl was first approved in November 2019 for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.

It is worth mentioning that,Reblozyl is the first FDA-approved drug for the treatment of anemia associated with beta-thalassemia, and also the first in over a decade to be approvedFDAApproval of a New Treatment Regimen for MDS Patients Requiring Red Blood Cell (RBC) Transfusions Who Have Failed Treatment with One Erythropoiesis-Stimulating AgentIt should be noted that Reblozyl is not indicated as a substitute for red blood cell transfusion in patients requiring immediate correction of anemia.

Recently, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion, recommending the approval of Reblozyl for the treatment of β-thalassemia and MDS-related anemia. The European Commission is expected to approve the drug in June this year.

The active pharmaceutical ingredient of Reblozyl is luspatercept, a first-in-class erythroid maturation agent (EMA) that modulates late-stage erythrocyte maturation. This drug is a soluble fusion protein composed of the Fc domain of human IgG1 fused to the extracellular domain of the activin receptor type IIB (ActRIIB). Acting as a ligand trap, it selectively binds specific ligands of the transforming growth factor (TGF)-β superfamily that regulate late-stage red blood cell (RBC) maturation, thereby reducing activation of the Smad2/3 signaling pathway, ameliorating ineffective erythropoiesis, promoting late-stage erythrocyte maturation, and increasing hemoglobin levels. (Bioon.com)

Original Source: Acceleron Receives Priority Medicines (PRIME) Designation from European Medicines Agency (EMA) for Sotatercept in Pulmonary Arterial Hypertension