FDA, Regulatory

Celgene, Acceleron submit FDA application for billed blockbuster luspatercept — will they get priority review?

Celgene and their partners at Acceleron are counting on their red blood cell boosting drug to win a speedy review as they submitted a marketing application for the biologic on Friday. For the big biotech — tarnished by a poor performance under CEO Mark Alles — the drug is one of the top five in its pipeline, critical for the mighty $74 billion Bristol-Myers Squibb takeover.

The drug, luspatercept, has been developed to manage anemia resulting from myelodysplastic syndrome (MDS) as well as beta thalassemia and has inspired peak sales estimates of $2 billion plus.

Luspatercept, which is designed to work by blocking signals that reduce red blood cell counts, was submitted for approval on Friday, on the basis of late-stage data presented last December at ASH. In patients with MDS, 38% of patients hit the primary endpoint: at least eight weeks without a need for a red blood cell transfusion. The key secondary goal — 12 weeks without transfusion — was met by 28% of the patient studied.

Acceleron $XLRN also tested their drug successfully as a means to reduce regular transfusions in beta thalassemia, although bluebird’s $BLUE one-shot gene-therapy LentiGlobin — already under regulatory review — may render luspatercept obsolete in this patient population.

For Celgene $CELG the drug provides an opportunity to model life beyond its cash-cow Revlimid and move past a series of setbacks in its late-stage pipeline, making it a rosier acquisition for Bristol-Myers. Partner Acceleron can benefit from the establishment of a commercial group with co-promotion rights in the United States, as they work on putting together new data in frontline low-risk MDS, which could significantly expand their market opportunity.

Yaron Werber

Cowen’s Yaron Werber modeled the drug will get a standard FDA review, but said he sees a good probability that the US agency will grant the treatment priority review, expediting approval to December 2019. A decision on the priority review is expected by June 4, he said.

The companies also plan to submit a marketing application to the European Medicines Agency in the second quarter of 2019, they added.


The best place to read Endpoints News? In your inbox.

Comprehensive daily news report for those who discover, develop, and market drugs. Join 47,300+ biopharma pros who read Endpoints News by email every day.

Free Subscription

Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

Visit Endpoints Careers ->