Celgene and Acceleron Pharma have won a first-in-class FDA approval for Reblozyl® (luspatercept-aamt), an erythroid maturation agent that has won the agency’s first authorization to treat anemia in adults with beta thalassemia who require regular red blood cell (RBC) transfusions.

Reblozyl is designed to work by regulating late-stage red blood cell maturation to help patients reduce their RBC transfusion burden. Until now, treatment options for anemia associated with beta thalassemia have consisted mainly of RBC transfusions—a potential contributor to iron overload, which can cause organ damage among other serious complications.

Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia, Celgene and Acceleron added.

“Today’s approval is an important milestone and underscores our continued commitment to patients with hematology disorders,” said Nadim Ahmed, president, global hematology and oncology for Celgene—which is set to be acquired by Bristol-Myers Squibb (BMS) for $74 billion, a deal announced in January.

Reblozyl is one of five later clinical-stage candidates whose combined approvals have been expected to generate more than $15 billion annually for the combined company, and thus were called “potential blockbusters” by Celgene chairman and CEO Mark Alles when the BMS acquisition became public.

Reblozyl is also the first Acceleron pipeline candidate to gain FDA authorization: “We’re thrilled that Acceleron’s first approved medicine is one with the potential to help patients with beta thalassemia, who have been in need of new treatments for this lifelong disease,” stated Acceleron’s president and CEO Habib Dable.

Pricing for Reblozyl has been set at $3,441 per 25 mg vial, according to Acceleron. The drug is expected to reach the market as early as next week.

$25M upfront in 2011

Acceleron and Celgene agreed to co-develop Reblozyl in 2011 by expanding a three-year-old collaboration to include the drug, then an Acceleron pipeline candidate called ACE-536. At the time, Celgene agreed to pay Acceleron $25 million upfront, and up to $217 million in payments tied to achieving development, regulatory, and commercial milestones.

The FDA approved Reblozyl following a Priority Review, based on positive results from the pivotal, Phase III BELIEVE trial (NCT02604433), designed to assess the safety and efficacy of Reblozyl as an anemia treatment in adults with beta thalassemia who require regular RBC transfusions. “Regular” was defined as 6–20 RBC units per 24 weeks, with no transfusion-free period greater than 35 days during that period. A total of 336 patients were randomized 2:1 to receive either Reblozyl (224 patients) or placebo (112 patients) at a starting dose of 1.0 mg/kg by subcutaneous injection every 21 days for up to 48 weeks.

According to Celgene and Acceleron, the BELIEVE trial achieved clinically meaningful and statistically significant improvement in the primary endpoint. In the Reblozyl arm, 48 patients (21.4%) achieved a ≥33% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) during weeks 13–24 after randomization, compared to five patients (4.5%) in the placebo arm.

Reblozyl also met the BELIEVE trial’s key secondary endpoints, including transfusion burden reduction of at least 33% (with a reduction of at least 2 units), during weeks 37 to week 48, which was achieved in 44 patients (19.6%) in the Reblozyl arm and four patients (3.6%) in the placebo arm.

Reblozyl remains under FDA review 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 FDA has set a target decision date under the Prescription Drug User Fee Act (PDUFA) of April 4, 2020.

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