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BMS scores EU okay for first-in-class anaemia drug Reblozyl

Drug is one of five of Celgene’s late-stage pipeline drugs with blockbuster potential

Bristol-Myers Squibb

Bristol-Myers Squibb has chalked up another key approval stemming from its $74bn takeover of Celgene. The EMA has given the go-ahead to anaemia therapy Reblozyl, the first in a new class of erythroid maturation agents (EMAs), which is billed as a future blockbuster.

Reblozyl (luspatercept) has been approved in the EU to treat anaemia in selected patients with rare blood disorder beta thalassemia or myelodysplastic syndrome (MDS), a form of blood cancer.

The drug is one of five late-stage pipeline drugs from Celgene that BMS said had blockbuster potential when it announced the Celgene deal, along with myelofibrosis therapy Inrebic (fedratinib), Zeposia (ozanimod) for multiple sclerosis and CAR-T therapies liso-cel for lymphoma and ide-cel for multiple myeloma.

Originally developed by Acceleron as ACE-536 and licensed by Celgene in 2011, Reblozyl was approved by the FDA for anaemia associated with beta thalassaemia last year, and for the MDS indication in April.

Analysts have predicted that the drug could eventually reach sales of more than $2bn, with anaemia associated with MDS making up around two-thirds of those peak revenues.

Specifically, the EMA has approved the drug for adult patients who have low- and intermediate-risk MDS with ring sideroblasts – a form of the cancer characterised by red blood cells with mitochondria that form a characteristic ring around the nucleus.

MDS patients are eligible for the drug if they are dependent on blood transfusions, and either cannot tolerate or don’t response to erythropoietin (EPO) drugs.

In the case of thalassaemia, the EU regulator has cleared Reblozyl for adult patients who are also dependent on blood transfusions, which can require frequent and lengthy hospital visits which can pose additional health risks and affect patients’ quality of life, according to BMS.

The approval in MDS is based on the phase 3 MEDALIST trial, which showed that around a third of previously transfusion dependent patients were transfusion independent over weeks 1-48 of the trial, as opposed to just 11% in the placebo arm.

The drug’s effects in thalassaemia – from the BELIEVE trial – were not quite so impressive but still showed a significant improvement over placebo, cutting the rate of transfusions, although no patients were able to achieve transfusion independence.

The beta thalassemia market could prove tougher to crack because bluebird bio’s newly approved gene therapy Zynteglo offers patients a cure in a single treatment.

Celgene and Acceleron have further plans for Reblozyl’s development, with trials on the go in EPO-naïve, lower-risk MDS patients, non-transfusion-dependent beta-thalassemia, and myelofibrosis.

Article by
Phil Taylor

29th June 2020

From: Regulatory

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