Paul G. Richardson, MD from Dana-Farber Cancer Institute speaks about FDA Approves Oncopeptides´ PEPAXTO® (melphalan flufenamide) for Patients with Triple-Class Refractory Multiple Myeloma.
The U.S. Food and Drug Administration (FDA) has approved PEPAXTO® (melphalan flufenamide), also known as melflufen during clinical development, in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have rectal bleeding. Based on the HORIZON trial, this indication has been given accelerated approval. PEPAXTO is the first anticancer peptide-drug conjugate that has been approved for the treatment of multiple myeloma.
“While the treatment landscape for multiple myeloma has dramatically improved in recent years, once patients become resistant to existing classes of therapy they can face a very guarded prognosis,” said Paul G. Richardson, MD, Clinical Program Leader and Director of Clinical Research at the Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute. “Research has shown melphalan flufenamide to be a novel and innovative therapeutic option, which is active in refractory disease and has manageable toxicity, with the convenience of being administered by infusion once a month. Based on our findings, melphalan flufenamide is an important addition to the treatment armamentarium, with the potential to meaningfully improve outcomes in an area of important unmet medical need.”
The HORIZON trial involved heavily pre-treated patients with a poor prognosis who were given intravenous PEPAXTO in conjunction with dexamethasone. This multi-center single-arm study looked at 157 patients with relapsed or refractory multiple myeloma, with 97 of them being triple-class refractory and having tried at least four different treatments. The overall response rate for this group of refractory multiple myeloma patients was 23.7 percent, with a median duration of response of 4.2 months. EMD (n=40) was found in 41 percent of the 97 patients, an aggressive and resistant disease with a poor prognosis.
“We are proud to bring forward the first anticancer peptide-drug conjugate approved by the FDA for multiple myeloma,” said Jakob Lindberg, Chief Scientific Officer at Oncopeptides. “PEPAXTO uses innovative technology that links a peptide carrier to a cytotoxic agent. The conjugated agent is a highly lipophilic compound, which allows it to be rapidly distributed into cells. The compound then leverages amino peptidases that are overexpressed in Multiple Myeloma cells, causing the release of the cytotoxic payload.”
PEPAXTO received accelerated approval, which allows for the faster approval of medications that treat severe disorders and meet an unmet medical need using a surrogate endpoint. Continued approval for this indication may be conditional on a confirmatory trial demonstrating and documenting clinical benefit.
PEPAXTO is expected to be commercially available in the United States within two weeks, according to Oncopeptides.