Luspatercept

Summary of opinion: Reblozyl, 22/02/2024 Positive

Retrieved on: 
Sunday, March 10, 2024
Marketing, Anemia, Luspatercept, European Commission, MDS, Civil service commission, Committee, CHMP, Medication package insert, Patient, EEIG

On 22 February 2024, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Reblozyl.

Key Points: 
  • On 22 February 2024, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Reblozyl.
  • The marketing authorisation holder for this medicinal product is Bristol-Myers Squibb Pharma EEIG.
  • For information, the full indications for Reblozyl will be as follows:
    Reblozyl is indicated in adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) (see section 5.1).
  • Reblozyl is indicated in adults for the treatment of anaemia associated with transfusion dependent and non-transfusion dependent beta thalassaemia (see section 5.1).

U.S. FDA Accepts for Priority Review Supplemental Biologics License Application and EMA Validates Application for Reblozyl® (luspatercept-aamt) as First-Line Treatment of Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (MDS)

Retrieved on: 
Monday, May 1, 2023
FDA, Health, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, Epoetin alfa, Bristol Myers Squibb, RBC, Priority review, Failure, ESA, Safety, European Medicines Agency, Transfusion-dependent anemia, Type, BMY, EMA, MDS, HB, Prescription Drug User Fee Act, Acquisition, Patient, Syndrome, Anemia, PDUFA, Merck, Regulation of tobacco by the U.S. Food and Drug Administration, NYSE, Food, Pharmaceutical industry, Luspatercept

In the U.S., the FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of August 28, 2023.

Key Points: 
  • In the U.S., the FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of August 28, 2023.
  • Priority Review designation underscores the high unmet need and value that Reblozyl could bring to this patient population.
  • In Europe, the EMA’s validation of the application confirms the submission is complete and begins the EMA’s centralized review process.
  • Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021.

Versanis Bio Appoints Kenneth Attie, M.D., and Aditya Venugopal, Ph.D., to Key Leadership Roles

Retrieved on: 
Tuesday, October 18, 2022
Cornell University, Kaiser San Francisco Medical Center, Doctor of Philosophy, Bimagrumab, Therapy, Strategic planning, Activin and inhibin, Muscle, Intercept Pharmaceuticals, Medical research, Lists of diseases, Myostatin, Ligand, Biotechnology, Patient, Insming, Business, Genentech, Program, Fatty liver disease, Venugopal, GDF11, Body composition, New York University Grossman School of Medicine, Immunoglobulin G, Multimedia, Technology, Senior, NASH, University, NEW, Acquisition, Atlas, Obesity, Acceleron Pharma, Business development, University of California, Corporate development, Neuromuscular Disorders, Luspatercept, Atlas Venture, Hematology, Metabolism, Navigant Consulting, View, Management, Pharmaceutical industry, Medicine, New York University

NEW YORK, Oct. 18, 2022 /PRNewswire/ -- Versanis Bio ("Versanis"), a clinical stage company advancing novel therapeutics for people living with obesity and cardiometabolic diseases, today announced two key leadership appointments. Versanis welcomed Executive Vice President and Chief Medical Officer, Kenneth Attie, M.D., and Senior Vice President, Corporate Development and Strategic Planning, Aditya Venugopal, Ph.D.

Key Points: 
  • Versanis welcomedExecutive Vice President and Chief Medical Officer, Kenneth Attie, M.D., and Senior Vice President, Corporate Development and Strategic Planning, Aditya Venugopal, Ph.D.
    "Ken and Aditya each have demonstrated success growing biotechnology companies," said Versanis CEO Mark Pruzanski, M.D.
  • He also held leadership roles at Altus Pharmaceuticals and Insmed and spent more than a decade at Genentech.
  • Versanis Bio is a privately held, clinical-stage biopharmaceutical company bringing transformational treatments to people living with cardiometabolic disease.
  • Versanis was founded in 2021 and is a portfolio company of Aditum Bio, Atlas Venture, and Medicxi.

Acceleron Announces Second Quarter 2021 REBLOZYL® Net Sales

Retrieved on: 
Wednesday, July 28, 2021
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Acceleron Pharma, Companies, Life sciences, Health care, Luspatercept, Bristol Myers Squibb

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 net sales of REBLOZYL(luspatercept-aamt) as reported by its global collaborator, Bristol Myers Squibb, were approximately $128 million for the second quarter ended June 30, 2021.

Key Points: 
  • 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 net sales of REBLOZYL(luspatercept-aamt) as reported by its global collaborator, Bristol Myers Squibb, were approximately $128 million for the second quarter ended June 30, 2021.
  • Acceleron expects to report royalty revenue of approximately $25.6 million from net sales of REBLOZYL in the second quarter ended June 30, 2021.
  • This compares with approximately $22.4 million in royalty revenue from approximately $112 million of net sales of REBLOZYL for the first quarter ended March 31, 2021.
  • Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases.

European Hematology Association - Above and BEYOND: Luspatercept is Efficacious and Well-Tolerated in Patients with Non-Transfusion-Dependent β-Thalassemia

Retrieved on: 
Friday, June 11, 2021
Medical specialties, Anemias, Clinical medicine, Blood, Transfusion medicine, Luspatercept, Thalassemia, Beta thalassemia, Transfusion-dependent anemia

Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.

Key Points: 
  • Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.
  • There are currently no approved therapies for the management of anemia in NTD -thalassemia.
  • Luspatercept is a drug that is approved for the treatment of anemia in transfusion-dependent -thalassemia and may also be beneficial for patients with NTD -thalassemia.
  • In conclusion, luspatercept is efficacious and safe for treating patients with NTD -thalassemia.

European Hematology Association - Above and BEYOND: Luspatercept is Efficacious and Well-Tolerated in Patients with Non-Transfusion-Dependent β-Thalassemia

Retrieved on: 
Friday, June 11, 2021
Medical specialties, Anemias, Clinical medicine, Blood, Transfusion medicine, Luspatercept, Thalassemia, Beta thalassemia, Transfusion-dependent anemia

Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.

Key Points: 
  • Despite their transfusion-independence, patients with NTD -thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life.
  • There are currently no approved therapies for the management of anemia in NTD -thalassemia.
  • Luspatercept is a drug that is approved for the treatment of anemia in transfusion-dependent -thalassemia and may also be beneficial for patients with NTD -thalassemia.
  • In conclusion, luspatercept is efficacious and safe for treating patients with NTD -thalassemia.

Bristol Myers Squibb and Acceleron Present First Results from Phase 2 BEYOND Study of Reblozyl® (luspatercept-aamt) in Adults with Non-Transfusion Dependent (NTD) Beta Thalassemia

Retrieved on: 
Friday, June 11, 2021
Research, Clinical trials, Cardiology, Biotechnology, Health, Pharmaceutical, General Health, Science, Oncology, Health, Clinical research, Clinical medicine, Luspatercept, Thalassemia, Beta thalassemia, Placebo-controlled study, Placebo

BEYOND is a Phase 2, randomized, double-blind, placebo-controlled multi-center study to determine the efficacy and safety of Reblozyl versus placebo in adults with non-transfusion dependent (NTD) beta thalassemia.

Key Points: 
  • BEYOND is a Phase 2, randomized, double-blind, placebo-controlled multi-center study to determine the efficacy and safety of Reblozyl versus placebo in adults with non-transfusion dependent (NTD) beta thalassemia.
  • 89.6% of patients in the Reblozyl arm remained transfusion free at weeks 1-24 versus 67.3% of patients in the placebo arm (P=0.0013).
  • Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration.
  • A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta ()-Thalassemia (BEYOND).

Acceleron Announces Presentations on REBLOZYL® (luspatercept-aamt) at the 2021 European Hematology Association Virtual Congress

Retrieved on: 
Wednesday, May 12, 2021
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Clinical medicine, Health, Luspatercept, Medicine, Beta thalassemia, Primary myelofibrosis, Pulmonary hypertension, Hematologic disease, Thalassemia, PAH, REBLOZYL (luspatercept-aamt), Acceleron, REBLOZYL (LUSPATERCEPT-AAMT), ACCELERON

Acceleron\xe2\x80\x99s 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.\nAcceleron focuses its research, development, and commercialization efforts in pulmonary and hematologic diseases.

Key Points: 
  • Acceleron\xe2\x80\x99s 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.\nAcceleron focuses its research, development, and commercialization efforts in pulmonary and hematologic diseases.
  • In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension (PAH), having reported positive topline results of the PULSAR Phase 2 trial.
  • REBLOZYL is part of a global collaboration partnership with Bristol Myers Squibb.
  • The Companies co-promote REBLOZYL in the United States and are also developing luspatercept for the treatment of anemia in patient populations of myelodysplastic syndromes, beta-thalassemia, and myelofibrosis.\nFor more information, please visit www.acceleronpharma.com .

Acceleron Announces First Quarter 2021 REBLOZYL® Net Sales

Retrieved on: 
Thursday, April 29, 2021
Biotechnology, Pharmaceutical, Health, Companies, Bristol Myers Squibb, Life sciences, Acceleron Pharma, Luspatercept, Squib, Health care

b'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 net sales of REBLOZYL\xc2\xae(luspatercept-aamt) as reported by its global collaborator, Bristol Myers Squibb, were approximately $112 million for the first quarter ended March 31, 2021.\nAcceleron expects to report royalty revenue of approximately $22.4 million from net sales of REBLOZYL in the first quarter ended March 31, 2021.

Key Points: 
  • b'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 net sales of REBLOZYL\xc2\xae(luspatercept-aamt) as reported by its global collaborator, Bristol Myers Squibb, were approximately $112 million for the first quarter ended March 31, 2021.\nAcceleron expects to report royalty revenue of approximately $22.4 million from net sales of REBLOZYL in the first quarter ended March 31, 2021.
  • In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension (PAH), having reported positive topline results of the PULSAR Phase 2 trial.
  • REBLOZYL is part of a global collaboration partnership with Bristol Myers Squibb.
  • Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn .\nThis press release contains forward-looking statements about the Company\'s financial results.

Acceleron to Webcast First Quarter 2021 Financial Results on May 6, 2021

Retrieved on: 
Monday, April 26, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Orphan drugs, Hypertension, Acceleron Pharma, Pulmonary hypertension, PAH, Luspatercept, Pulmonary artery, Health care, Health, Branches of biology

b'Acceleron Pharma Inc. (Nasdaq:XLRN) today announced it will host a webcast and conference call on Thursday, May 6, 2021 at 5:00 p.m. EDT to discuss its first quarter 2021 financial results.\nThe webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company\xe2\x80\x99s website at www.acceleronpharma.com .

Key Points: 
  • b'Acceleron Pharma Inc. (Nasdaq:XLRN) today announced it will host a webcast and conference call on Thursday, May 6, 2021 at 5:00 p.m. EDT to discuss its first quarter 2021 financial results.\nThe webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company\xe2\x80\x99s website at www.acceleronpharma.com .
  • In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension (PAH), having reported positive topline results of the PULSAR Phase 2 trial.
  • REBLOZYL is part of a global collaboration partnership with Bristol Myers Squibb.
  • Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn .\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210426005098/en/\n'