FDA Gives Breakthrough Therapy Designation to Myelodysplastic Syndrome Drug

The FDA has granted a breakthrough therapy designation to magrolimab for the treatment of patients with newly diagnosed myelodysplastic syndrome (MDS), according to an announcement from Gilead Sciences, Inc.¹

The designation was based on data from an ongoing phase 1b trial (NCT03248479) that is evaluating the first-in-class, investigational anti-CD47 monoclonal antibody in combination with azacitidine in treatment-naïve patients with high and very high-risk MDS, which showed that the regimen was well tolerated and effective in this patient population.

“The breakthrough therapy designation recognizes the potential for magrolimab to help address a significant unmet medical need for [patients] with MDS and underscores the transformative potential of Gilead’s immuno-oncology therapies in development, Merdad Parsley, MD, PhD, chief medical officer of Gilead Sciences, stated in a press release.

Magrolimab was developed to inhibit recognition of CD47 by the SIRPα receptor on macrophages; by doing this, the agent is able to block the signal utilized by cancer cells to evade ingestion by the macrophages.

In the early-phase trial, investigators set out to evaluate the safety and efficacy of magrolimab in combination with azacitidine in patients with untreated higher-risk MDS. A total of 39 patients with a median age of 70 years received treatment with the combination. Twenty-eight percent of patients had intermediate cytogenetic risk and the majority, or 64%, were considered to be poor risk. Moreover, 31% of participants had therapy-related MDS and 13% of patients had TP53-mutant disease.

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