Celgene & Acceleron Get FDA Nod for Rare Blood Disorder Drug

Celgene Corporation CELG and partner Acceleron Pharma Inc. 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 industry's growth of 1.6%. Shares of Acceleron were up 6.6% on the news of the approval.

 

Celgene has been in news since the beginning of 2019 for its impending merger with Bristol-Myers Squibb Company BMY. The transaction is expected to close by the end of the year.

Zacks Rank & Another Key Pick

Celgene currently carries a Zacks Rank #2 (Buy).

Another top-ranked stock in the biotech space is Vertex Pharmaceuticals Inc. VRTX, which currently carries a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Vertex’s earnings estimates have increased to $4.81 from $4.59 in the past 30 days.

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Bristol-Myers Squibb Company (BMY) : Free Stock Analysis Report
 
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