Celgene & Acceleron Get FDA Nod For Rare Blood Disorder Drug
Zacks Investment Research | Nov 10, 2019 09:05PM ET
Celgene Corporation (NASDAQ:CELG) and partner Acceleron Pharma Inc. (NASDAQ:XLRN) announced that the FDA has approved pipeline candidate Reblozyl (luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia, who require regular red blood cell (RBC) transfusions.
Per the company, Reblozyl is the first and only FDA-approved erythroid maturation agent, representing a new class of therapy for these patients. The approval comes ahead of the target action date of Dec 4.
The approval of Reblozyl for beta thalassemia was based on encouraging results from the pivotal, phase III, randomized, double-blind, placebo-controlled, multicenter BELIEVE study. The study evaluated the safety and efficacy of the drug for the treatment of anemia in adult patients with beta thalassemia, who require regular RBC transfusions. The study achieved a clinically meaningful and statistically significant improvement in the primary endpoint.
The approval comes as a significant boost for Celgene, given the dearth of options for patients living with anemia due to beta thalassemia, who are dependent on long-term RBC transfusions.
Meanwhile, the FDA is also evaluating luspatercept-aamt for the treatment of anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS), who have ring sideroblasts and require RBC transfusions. The agency has set a target action date of April 4, 2020, for the same. Celgene’s Marketing Authorization Application for the treatment of anemia in adults with beta thalassemia is currently under review in Europe.
Additionally, a phase II study (BEYOND) in adult patients with non-transfusion-dependent beta thalassemia, a phase III study (COMMANDS) in ESA-naïve, lower-risk MDS patients and a phase II study in myelofibrosis patients are ongoing.
Shares of Celgene have soared 70.9% so far this year compared with the Zacks Investment Research
Get The News You Want
Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers.