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.

Click to continue reading on OncLive.