FDA approves Oncopeptides’ Multiple Myeloma Therapy

Rare Daily Staff

The U.S. Food and Drug Administration approved Oncopeptides’ Pepaxto in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma.

The approval was for patients 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.

Multiple myeloma is a cancer that impacts plasma cells, a type of white blood cell that produces antibodies to help fight infection. Multiple myeloma causes cancer cells to accumulate in the bone marrow. Approximately 7 per 100,000 Americans are diagnosed with multiple myeloma each year, making it a rare disease. A growing subset of this population is becoming triple-class refractory. This means that their disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. 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, and there is currently no cure.

Pepaxto (melflufen) is the first anticancer peptide-drug conjugate approved by the FDA. The product has been granted accelerated approval based on the phase 2 HORIZON study in relapsed or refractory multiple myeloma.

The HORIZON study evaluating intravenous melflufen in combination with dexamethasone, included heavily pre-treated patients with a poor prognosis. This multi-center single arm study evaluated 157 patients with relapsed or refractory multiple myeloma, of whom 97 were triple-class refractory and had received at least four prior lines of treatment. The Overall Response Rate for the patients within this group of patients with refractory multiple myeloma was 23.7 percent and the Median Duration of Response was 4.2 months. Furthermore, melflufen in combination with dexamethasone demonstrated activity in a subset of patients with Extra Medullary Disease (41 percent), an aggressive and resistant disease associated with a poor prognosis.

Marty Duvall, CEO of Oncopeptides, called the accelerated approval of Pepaxto an important milestone for the company.

“Moving ahead, our focus is to further advance Pepaxto,” he said. “We look forward to receiving top line data from the phase 3 OCEAN-study in relapsed refractory multiple myeloma, in the second quarter. The comparative study with pomalidomide, is designed to support a future supplementary New Drug Application to expand the label.”

Photo: Marty Duvall, CEO at Oncopeptides