The FDA approved a new infused specialty therapy last week, Pepaxto (melphalan flufenamide) from Oncopeptides AB, for patients with relapsed or refractory multiple myeloma following four or more lines of therapy. It is the first anticancer peptide-drug conjugate approved by the FDA.
Multiple myeloma is a cancer of plasma cells, a type of white blood cell which produces antibodies to help fight infection. Multiple myeloma causes cancer cells to accumulate in the bone marrow.
Approximately 7 per 100,000 Americans per year are diagnosed with multiple myeloma, making it a rare disease. However, the number of patients diagnosed with multiple myeloma is growing and the number of cases diagnosed annually is expected to almost double in 20 years. The average age for diagnosis is 70 years of age, and there is currently no cure.
Pepaxto is administered intravenously over 30 minutes on Day 1 of each 28-day cycle until disease progression or until unacceptable toxicity. Pepaxto is administered in combination with dexamethasone 40 mg orally or intravenously on Days 1, 8, 15 and 22 of each cycle.
Pricing for Pepaxto has not yet been confirmed.
Oncopeptides will release Pepaxto in the U.S. this month. They also announced that the product will be available through both wholesalers and specialty pharmacies. They did not specify whether they will implement a specialty pharmacy limited distribution program for this infused product. Given the small US patient population an LD program is likely.
FDA approves Oncopeptides’ PEPAXTO® ( melphalan flufenamide) for patients with relapsed or refractory multiple myeloma
STOCKHOLM, Feb. 26, 2021 /PRNewswire/ — Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, today announced that the U.S. Food and Drug Administration, FDA, has approved PEPAXTO® (melphalan flufenamide, also known as melflufen), in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.
The product has been granted accelerated approval based on the phase 2 HORIZON study in relapsed or refractory multiple myeloma.
“The accelerated approval of PEPAXTO in the US is an important……….
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