The targeted anticancer drug Pepaxto is included in the NCCN clinical practice guidelines for multiple myeloma in the United States

Oncopeptipes AB is a pharmaceutical company specializing in targeted therapies for refractory blood diseases.

On February 26, 2021, Pepaxto received accelerated approval from the US FDA for the treatment of adult patients with relapsed or refractory multiple myeloma (MM) in combination with dexamethasone.

It is particularly worth mentioning that Pepaxto (melflufen) is the first anti-cancer peptide-drug conjugate (PDC) approved by the US FDA.

NCCN is an alliance of 30 cancer centers in the United States.

Melflufen is activated by aminopeptidase, which is present in all human cells but is overexpressed in many cancers, including myeloma.

Pepaxto's approval is based on the results of the pivotal Phase II HORIZON study.

The results showed that among the 97 patients, the overall response rate (ORR) was 23.

Conclusion: The combination of melflufen and dexamethasone will provide an important treatment option for adult patients with relapsed or refractory MM who are difficult to treat and have a poor prognosis, including three-drug refractory myeloma patients and extramedullary diseases (EMD).

Original source: Oncopeptides Announces National Comprehensive Cancer Network? Adds PEPAXTO? (melphalan flufenamide) to Its Multiple Myeloma Clinical Practice Guidelines