Celgene gets speedy review for thalassaemia drug luspatercept

It’s shaping up to be a landmark year for the community of patients with rare blood disorder beta thalassaemia. The first approval of a gene therapy in the EU has been followed swiftly by news that a Celgene drug could be approved in the US before year-end.

With the ink barely dry on the EMA’s approval notice for bluebird bio’s Zynteglo for transfusion-dependent thalassaemia (TDT), Celgene and partner Acceleron say they have secured an FDA priority review for luspatercept, a red blood cell-boosting drug designed to tackle the anaemia associated with the blood disorder.

The US regulator is due to deliver a verdict on luspatercept in thalassaemia-elated anaemia by 4 December, with a second verdict in anaemia linked to myelodysplastic syndrome (MDS) scheduled for next April in the standard review timeframe.

Luspatercept is an erythroid maturation agent that works by accelerating the maturation of red blood cells, and is also intended for seriously ill thalassaemia patients who require blood transfusions.

The BELIEVE study of the drug in TDT met its primary objective of a reduced need for transfusions compared to placebo, with a similar outcome for the MEDALIST trial in MDS, and prompted Celgene and Acceleron to predict sales of the drug could reach up to $2 billion.

If luspatercept can reach those heights it will be a big help to Celgene’s ambition of reducing its reliance on current cash cow product Revlimid (lenalidomide), which made $9.7 billion in sales last year but has generic competition looming in 2022.

It will also be a comfort to Bristol-Myers Squibb as its $74 billion takeover deal for Celgene rumbles towards the finishing post, despite some investor resistance.

Celgene also said that its marketing application in the EU has also been validated and the EMA review is now underway.

“The acceptance of the luspatercept filings and granting of the US priority review for beta-thalassaemia represent another important step in delivering this novel therapy to patients in need,” said Jay Backstrom, Celgene’s chief medical officer.

“We believe that luspatercept can play a critical role in treating the anaemia associated with these serious blood diseases, and with these milestones achieved we look forward to working closely with the agency to move this therapy toward approval.”