PHAR - Pharming's leniolisib review converted to standard from accelerated as EMA seeks more data

A panel of the European Medicines Agency (EMA) converted the review of Pharming's ( NASDAQ: PHAR ) drug leniolisib to standard review from accelerated assessment as the committee sent questions to the company, including requesting more data.

The company's marketing authorization application (MAA) for leniolisib was validated for evaluation under an accelerated assessment by the EMA's Committee for Medicinal Products for Human Use (CHMP) in October 2022.

The company is seeking approval of leniolisib to treat patients aged 12 years and older with activated phosphoinositide 3-kinase (PI3K) delta syndrome (APDS), a rare primary immunodeficiency.

The Netherlands-based company said it received the list of questions from EMA which included a request to submit updated data from an ongoing long-term extension study collected after the interim analysis included in the original MAA.

Pharming expects that the CHMP will issue its opinion on the leniolisib MAA in H2 2023.

"We are continuing to work with EMA through the MAA review process and remain dedicated to seeking regulatory approval of leniolisib within the European Economic Area. In the US, the FDA's priority review of the leniolisib NDA remains on track, with a PDUFA goal date of March 29, 2023," said Pharming CEO Sijmen de Vries.

The FDA had granted priority review to leniolisib in September 2022.

APDS is a rare immunodeficiency disease that affects 1 to 2 people per million and is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells, according to the company.

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