FDA has approved Oncopeptides’ PEPAXTO for patients with relapsed or refractory multiple myeloma.

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, states the company in a press release.

“I am immensely proud of the relentless dedication of our organization and development partners around the world who have made this journey possible.”

“When we listed Oncopeptides on Nasdaq Stockholm, we promised to establish melflufen as an attractive treatment option for patients with multi-resistant disease. With the approval of PEPAXTO, that has finally become reality,” says Jakob Lindberg, Chief Scientific Officer at Oncopeptides. “I am immensely proud of the relentless dedication of our organization and development partners around the world who have made this journey possible.”

The first anticancer peptide-drug conjugate approved by the FDA

Oncopeptides will begin promoting PEPAXTO to healthcare professionals across the U.S. immediately and expects a labeled product in distribution centers and specialty pharmacies within approximately two weeks, it states.

PEPAXTO 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.

“Moving ahead, our focus is to further advance PEPAXTO. 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,” says Marty J Duvall, Chief Executive Officer at Oncopeptides.

The HORIZON study

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% 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%), an aggressive and resistant disease associated with a poor prognosis.

Photo of Jakob Lindberg: Oncopeptides