Pepaxto Approved to Treat Multiple Myeloma

February 26, 2021 – The U.S. FDA has approved Pepaxto® (melphalan flufenamide), manufactured by Oncopeptides, for use in conjunction with dexamethasone to treat relapsed or refractory multiple myeloma (MM) in adults who have received at least four prior lines of therapy. Under the indication, the patient’s disease must be refractory (resistant to treatment) to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.

Multiple myeloma is a cancer that develops in the bone marrow, where blood cells are made. It affects plasma cells, which are a type of blood cell that produces antibodies to fight infection. In MM, the cancerous cells crowd out healthy cells in the bone marrow, weakening the body’s immune system and producing harmful abnormal proteins. Treatment options for triple-class refractory MM – which is disease that has not responded adequately to three different types of therapy – are limited.

In a clinical study, Pepaxto plus dexamethasone delivered an overall response rate of 23.7%. with a 4.2- month median duration of response. The study population consisted of patients diagnosed with an uncommon form of MM known as extramedullary disease (EMD), in which the cancer is found outside of the bone marrow. EMD is both aggressive and difficult to treat.

Recommended dosing is 40mg of Pepaxto administered via a 30-minute intravenous infusion on Day 1 of each 28-day cycle until disease progression or unacceptable toxicity occur. Dosing adjustments may be required if the patient experiences an adverse reaction.

The FDA granted Pepaxto accelerated approval, meaning continued approval may be contingent on verification and description of clinical benefit in confirmatory trials. Oncopeptides has launched the product at a wholesale acquisition cost (WAC) of $9,500 per 20mg vial.