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New Drug Approval Expands Options for Patients with Triple-Class Refractory Multiple Myeloma

Update: October 25, 2021

After discussions with the U.S. Food and Drug Administration (FDA), the manufacturer withdrew Pepaxto from the U.S. market on October 22, 2021. While results from the HORIZON study were positive, a second phase 3 study called OCEAN showed conflicting results.

While this is disappointing news, the priority of the FDA is to ensure approved treatments are effective and safe. Today’s news reflects the scientific rigor FDA applies to these decisions. When a second phase 3 clinical trial provides conflicting results from earlier trials, researchers and regulators must review all data thoroughly to make a decision that is in the best interests of patients.

LLS is committed to funding pioneering science to improve the outlook for patients with multiple myeloma. Last year alone, LLS committed $31.7 million to myeloma research including immunotherapy, genomics and personalized medicine.

Rye Brook, N.Y., February 26, 2021 – The U.S. Food and Drug Administration (FDA) today announced approval of melphalan flufenamide, also known as melflufen (Pepaxto), in combination with dexamethasone for the treatment of adults with multiple myeloma whose disease has returned or worsened following prior treatment with four different classes of drugs.

Today’s approval of melflufen was based on results from HORIZON, a pivotal phase 2 study. Melflufen demonstrated a 23.7% overall response rate (ORR) in patients with so-called “triple-class refractory multiple myeloma” or RRMM. The responses in melflufen-treated patients were durable and often deepened with prolonged treatment, suggesting that patients may benefit from staying on treatment for as long as possible. 

Despite the availability of new treatments, multiple myeloma will progress in most patients. In recent years, a new subset of patients with RRMM has been identified. The current approaches to the treatment of RRMM are limited and generally have short-lived effectiveness.

Multiple myeloma is among The Leukemia & Lymphoma Society’s primary research initiatives. LLS currently has over 30 active projects (over a $40M commitment) in multiple myeloma focused on laboratory and clinical research. They include world-class researchers at Dana Farber Cancer Institute, MD Anderson Cancer Center and the University of Pennsylvania who are looking at ways to provide safe and effective targeted therapies and to adapt CAR T-cell immunotherapy for patients with multiple myeloma.

Today’s approval of melflufen is an encouraging advancement for a patient population with significant unmet medical need who have already been treated with the three major classes of myeloma drugs.