Sotatercept granted first FDA Breakthrough Therapy designation in pulmonary arterial hypertension since the Agency established the designation in 2012
CAMBRIDGE, MA, USA I April 08, 2020 I Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH) (World Health Organization Group 1).
“In January of this year, we reported positive topline results from our PULSAR Phase 2 placebo-controlled trial of sotatercept in patients with PAH,” said Habib Dable, President and Chief Executive Officer of Acceleron. “Based on the results, we believe that sotatercept has the potential to shift the current treatment paradigm and provide significant benefit to patients with PAH on top of currently available therapies. Thus, we’re thrilled that the FDA has granted this Breakthrough Therapy designation—a first for an Acceleron-discovered medicine and for a therapeutic candidate in PAH—as it supports and aligns with our mission to deliver novel therapeutic options to patients in need as quickly as possible.”
The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may provide substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy designation conveys more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. For more information please visit the FDA website at www.fda.gov.
In 2019, the FDA granted Orphan Drug designation for Sotatercept in PAH.
About Sotatercept
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR-II signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure. Recent topline analysis of the PULSAR Phase 2 trial of sotatercept in patients with PAH revealed the trial met the primary as well as key and other secondary endpoints, with adverse events consistent with previously published data on sotatercept in other diseases. Sotatercept, which is part of a licensing agreement with Bristol Myers Squibb, is also being evaluated in the SPECTRA Phase 2 trial in patients with PAH. For more information, please visit www.clinicaltrials.gov
Sotatercept is an investigational therapy that is not approved for any use in any country.
About PAH
PAH is a rare and chronic, rapidly progressing disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant strain on the heart, often leading to limited physical activity, heart failure, and reduced life expectancy. The 5-year survival rate for patients with PAH is approximately 57%. Available therapies generally act by promoting the dilation of pulmonary vessels without addressing the underlying cause of the disease. As a result, PAH often progresses rapidly for many patients despite standard of care treatment. A growing body of research has implicated imbalances in BMP and TGF-beta signaling as a primary driver of PAH in familial, idiopathic, and acquired forms of the disease.
About Acceleron
Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron’s leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body’s ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting newly approved REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States and are developing luspatercept for the treatment of chronic anemia in myelofibrosis. Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial and actively enrolling patients in the Phase 2 SPECTRA trial.
For more information, please visit www.acceleronpharma.com. Follow Acceleron on social media: @AcceleronPharma and LinkedIn.
SOURCE: Acceleron
Post Views: 279
Sotatercept granted first FDA Breakthrough Therapy designation in pulmonary arterial hypertension since the Agency established the designation in 2012
CAMBRIDGE, MA, USA I April 08, 2020 I Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH) (World Health Organization Group 1).
“In January of this year, we reported positive topline results from our PULSAR Phase 2 placebo-controlled trial of sotatercept in patients with PAH,” said Habib Dable, President and Chief Executive Officer of Acceleron. “Based on the results, we believe that sotatercept has the potential to shift the current treatment paradigm and provide significant benefit to patients with PAH on top of currently available therapies. Thus, we’re thrilled that the FDA has granted this Breakthrough Therapy designation—a first for an Acceleron-discovered medicine and for a therapeutic candidate in PAH—as it supports and aligns with our mission to deliver novel therapeutic options to patients in need as quickly as possible.”
The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may provide substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy designation conveys more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. For more information please visit the FDA website at www.fda.gov.
In 2019, the FDA granted Orphan Drug designation for Sotatercept in PAH.
About Sotatercept
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR-II signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure. Recent topline analysis of the PULSAR Phase 2 trial of sotatercept in patients with PAH revealed the trial met the primary as well as key and other secondary endpoints, with adverse events consistent with previously published data on sotatercept in other diseases. Sotatercept, which is part of a licensing agreement with Bristol Myers Squibb, is also being evaluated in the SPECTRA Phase 2 trial in patients with PAH. For more information, please visit www.clinicaltrials.gov
Sotatercept is an investigational therapy that is not approved for any use in any country.
About PAH
PAH is a rare and chronic, rapidly progressing disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant strain on the heart, often leading to limited physical activity, heart failure, and reduced life expectancy. The 5-year survival rate for patients with PAH is approximately 57%. Available therapies generally act by promoting the dilation of pulmonary vessels without addressing the underlying cause of the disease. As a result, PAH often progresses rapidly for many patients despite standard of care treatment. A growing body of research has implicated imbalances in BMP and TGF-beta signaling as a primary driver of PAH in familial, idiopathic, and acquired forms of the disease.
About Acceleron
Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron’s leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body’s ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting newly approved REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States and are developing luspatercept for the treatment of chronic anemia in myelofibrosis. Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial and actively enrolling patients in the Phase 2 SPECTRA trial.
For more information, please visit www.acceleronpharma.com. Follow Acceleron on social media: @AcceleronPharma and LinkedIn.
SOURCE: Acceleron
Post Views: 279
