The company announces that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), has unanimously adopted a positive opinion recommending a full marketing authorization approval (MAA) of Pepaxti in EU.
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 (EEA) countries Iceland, Lichtenstein, and Norway, states the company.
Data from the phase 2 HORIZON study
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 Q4 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.
“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 US Food and Drug Administration (FDA) has now been intensified to achieve a clear path forward also for US patients.”
Efficacy results for triple-class refractory patients who have received at least 3 prior lines of therapies and who had no ASCT or progressed more than 36 months after an ASCT in the HORIZON study.
“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, MD, PhD, Executive Vice President, and Chief Medical Officer. “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.”
As previously disclosed, Oncopeptides has an EIB loan facility. Oncopeptides and EIB are currently in negotiations, to update tranche definitions to reflect the current regulatory situation. In addition, the company is considering additional financing options to capture the opportunities with the upcoming EU-approval. This may include new share issues and other public or private financing options.
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 Q4, 2022, it states. The company is actively considering various options to commercialize the drug, making it available for patients across Europe, and maximizing shareholder value.
Photo of Jakob Lindberg: Oncopeptides