The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Bayer’s PI3K inhibitor Aliqopa (copanlisib).
The company stated that the decision was primarily based on follow-up data from the pivotal Phase II study CHRONOS-1, which showed the efficacy of the drug in patients with indolent non-Hodgkin’s lymphoma (iNHL).
The indication is for patients who have received at least two prior therapies, including patients with relapsed or refractory marginal zone lymphoma (MZL).
The company is conducting two additional Phase III studies – CHRONOS-3 and CHRONOS-4 – to confirm the efficacy and safety of Aliqopa in combination with other therapies in iNHL (including MZL) patients who have relapsed following one or more prior therapies.
“The clinical evidence suggests that copanlisib may address an unmet medical need by providing physicians and MZL patients with a therapy in a relapsed setting where there are no approved treatments,” said Dr. Scott Fields, senior VP and head of oncology development at Bayer's Pharmaceutical Division.
“We will continue working closely with the FDA in order to bring copanlisib to these underserved patients as soon as possible.”
The compound is currently not approved by the European Medicines Agency (EMA) or other authorities outside of the US.
The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of drug candidates that treat serious or life-threatening diseases or conditions, and that have shown exceptional results in early stage trials.
The indication, MZL, is an indolent form of non-Hodgkin’s Lymphoma (iNHL) and accounts for about 10% of all non-Hodgkin’s Lymphoma in the US.