BRIEF—'Breakthrough' status for Gilead's magrolimab

Gilead Sciences today announced that the US Food and Drug Administration has granted Breakthrough Therapy designation for magrolimab, a first-in-class, investigational anti-CD47 monoclonal antibody for the treatment of newly-diagnosed myelodysplastic syndrome (MDS).

Gilead gained rights to magrolimab along with its $4.9 billion acquisition of Forty Seven that completed in April this year.

MDS is a type of cancer caused by poorly formed or dysfunctional blood cells in the bone marrow. Around 15,000 people are diagnosed with MDS in the USA each year, and no new treatments have been approved in 14 years.

The average survival rate for those with lower-risk MDS is six years and approximately 18 months for those with higher-risk MDS.

Magrolimab is currently being studied in the double-blind, placebo-controlled, randomized Phase III ENHANCE trial in previously untreated higher risk MDS.

The trial will evaluate the safety and efficacy of magrolimab, in combination with azacitidine, as measured by CR and duration of CR.

Magrolimab is an investigational agent and has not been approved anywhere globally. Its safety and efficacy have not been established.