CHMP Recommends EU Approval of Oncopeptides’ Pepaxti for Patients with Refractory Multiple Myeloma

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has unanimously adopted a positive opinion recommending a full marketing authorization approval of Oncopeptide’s Pepaxti in the European Union for patients with triple class refractory multiple myeloma.

Photo: Jakob Lindberg, CEO of Oncopeptides

The European Commission (EC) will make a legally binding decision based on the EMA recommendation within 60 days. Once granted by EC, the marketing authorization is valid in all EU member states, as well as in the European Economic Area countries Iceland, Lichtenstein, and Norway.

Multiple myeloma is a cancer that originates in plasma cells, a type of white blood cells which produce antibodies to help fight infection and causes the cancer cells to accumulate in the bone marrow. Patients with multiple myeloma may have symptom-free periods, but the disease always relapses, and patients may become refractory to all available treatment options due to mutations and/or clonal evolution of the tumor cells. A growing subset of patients are triple-class refractory, and develop disease refractory to immunomodulatory drugs, proteasome inhibitors, and CD38- targeting monoclonal antibodies. These patients have a very short expected overall survival.

Pepaxti inhibits proliferation and induces apoptosis of hematopoietic and solid tumor cells. It shows synergistic cytotoxicity in combination with dexamethasone in melphalan resistant and non-resistant multiple myeloma cell lines.

“Pepaxti helps patients with multiple myeloma, an incurable hematologic cancer. Today’s positive CHMP opinion confirms that Pepaxti provides benefit to these patients and is foundational for the future of Oncopeptides and our development pipeline,” says Jakob Lindberg, CEO of Oncopeptides. “Based on the scientific evaluation by EMA, our dialogue with the U.S. Food and Drug Administration has now been intensified to achieve a clear path forward also for U.S. patients.”

The positive opinion is based on data from the phase 2 HORIZON study and is supported by data from the randomized controlled phase 3 OCEAN study, which was utilized as confirmatory study. No specific post-marketing commitments were issued. Oncopeptides intends to submit a type II variation in the fourth quarter of 2022 to enable access to earlier lines of treatment for patients with relapsed refractory multiple myeloma (RRMM).

Pepaxti is indicated, in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

“EMA´s assessment of Pepaxti corroborates our scientific conclusion that the overall survival result in the OCEAN study constitutes a case of true survival heterogeneity which is reflected in the indication statement in accordance with the agency´s guidelines,” says Klaas Bakker, executive vice president and chief medical officer of Oncopeptides. “In addition, EMA confirms that there are no toxicological safety signals in both studies and there is a positive benefit risk profile in the indicated patient population. The non-transplanted, often older patient population, which represents the largest group of RRMM patients, particularly benefits from treatment with Pepaxti.”

Oncopeptides will advance market access activities after an approval by the European Commission, to pave the way for a successful launch of Pepaxti in Germany in the fourth quarter of 2022.

Pepaxto has been granted accelerated approval in the United States, in combination with dexamethasone, for treatment of adult patients with relapsed or refractory multiple myeloma.

Author: Rare Daily Staff