Gilead gets FDA breakthrough therapy status for magrolimab in MDS

Gilead Sciences has been given breakthrough therapy designation for magrolimab from the US Food and Drug Administration (FDA) for the treatment of myelodysplastic syndrome (MDS) in newly diagnosed patients.

Magrolimab is an investigational monoclonal antibody, which has been developed to act against CD47 and macrophage checkpoint inhibitor. It is being developed in various hematologic and solid tumour malignancies that include myelodysplastic syndrome.

According to Gilead Sciences, myelodysplastic syndrome is a type of cancer that results from poorly formed or dysfunctional blood cells in the bone marrow.

Every year, nearly 15,000 people are diagnosed with the condition in the US. In the last 14 years, no new treatments have been approved, said the company.

The FDA breakthrough therapy status helps to accelerate the development and regulatory review of investigational drugs for serious or life-threatening conditions. The designation is granted based on preliminary clinical evidence to investigational drugs that have the potential to significantly improve clinical outcomes compared with available treatments.

Magrolimab was given the designation based on the positive data from an ongoing phase 1b trial in which it is being evaluated in combination with azacitidine. The participants of the early-stage trial are those with intermediate, high and very high-risk myelodysplastic syndrome and are yet to get any treatment for their condition.

Gilead Sciences chief medical officer Merdad Parsey said: “The Breakthrough Therapy designation recognizes the potential for magrolimab to help address a significant unmet medical need for people with MDS and underscores the transformative potential of Gilead’s immuno-oncology therapies in development.”

Currently, the anti-CD47 monoclonal antibody is being evaluated in the ENHANCE trial, which is a double-blind, placebo-controlled, randomised phase 3 study in previously untreated higher risk myelodysplastic syndrome.

The late-stage trial will assess the safety and efficacy of the investigational drug in combination with azacitidine, as measured by complete remission and duration of complete remission.

Magrolimab also holds FDA fast track designation for the treatment of myelodysplastic syndrome, acute myeloid leukemia (AML), diffuse large B-cell lymphoma (DLBCL), and follicular lymphoma.

It had also secured FDA orphan drug designation for myelodysplastic syndrome and acute myeloid leukemia. A similar drug designation is held by the drug candidate from the European Medicines Agency for acute myeloid leukemia.

Recently, Gilead Sciences signed a deal worth about $21bn to acquire Immunomedics, a developer of antibody-drug conjugates.