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FDA approves expanded use of BMS’ Reblozyl in blood disorders

Drug treats anaemia in adults with lower-risk MDS with ring sideroblasts

BMS building

Bristol-Myers Squibb’s red blood cell-boosting drug Reblozyl has been approved for treating anaemia in people with myelodysplastic syndromes in the US, opening up a larger market for the drug.

The FDA has cleared Reblozyl (luspatercept) for anaemia in adults with lower-risk MDS with ring sideroblasts – an uncommon form of the cancer characterised by red blood cell with iron-packed mitochondria that form a ring around the nucleus.

The approved label is for use of the drug in patients who have failed standard red blood cell-boosting therapy with erythropoiesis stimulating agent (ESA) drugs like erythropoietin, and have needed transfusions of at least two units of red blood cells over the prior eight weeks.

Reblozyl got the go-ahead from the FDA last November for anaemia in patients with beta thalassaemia who require regular blood transfusions, becoming the first erythroid maturation agent to be cleared by the regulator. It is also under regulatory review in Europe for this indication with a verdict due shortly.

The drug was originated by Acceleron and licensed to Celgene, featuring as one of the company’s five late-stage pipeline drugs with blockbuster sales potential before Celgene’s $74bn takeover by BMS, along with just-approved multiple sclerosis drug Zeposia, CAR-Ts liso-cel and ide-cel and Inrebic (fedratinib) for myelofibrosis.

MDS is a much bigger market than beta thalassaemia, and Reblozyl’s opportunity in the latter could also be affected by the availability of bluebird bio’s recently-launched one-shot thalassaemia gene therapy Zynteglo.

There are around 1,000 to 1,500 people with thalassemia in the US who require blood transfusions, but more than 20,000 MDS patients who could qualify for treatment with Reblozyl, according to Acceleron’s chief executive Habib Dable.

Analysts predicts the drug could hit peak sales of about $2bn, with MDS accounting for two-thirds or more of that revenue estimate.

Filing for MDS was based on the phase 3 MEDALIST trial, which showed that around a third of patients became transfusion independent with Reblozyl treatment, compared to 11% of those who received a matched placebo.

Ahead of the coronavirus outbreak, BMS and Acceleron had announced plans to start a phase 3 trial of Reblozyl in myelofibrosis patients with anaemia requiring blood transfusions who are on JAK inhibitor treatment, after positive phase 2 data was reported at last year’s ASH congress.

Shortly after, however, BMA said it was suspending all new clinical trial sites and reining in new patient recruitment until at least 13 April, which could impact that trial, called INDEPENDENCE.

That deadline looks quite likely to be extended as COVID-19 continues to escalate in the US and Europe.

Article by
Phil Taylor

6th April 2020

From: Research



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