Therapy Areas: Devices
Pharming initiates Phase III trial in Japan for leniolisib in APDS treatment
9 August 2023 -

Biopharmaceutical company Pharming Group N.V. (Euronext Amsterdam:PHARM) (NASDAQ:PHAR) announced on Wednesday that the first patient has been enrolled in its Phase III clinical trial in Japan for leniolisib, a potential treatment for activated phosphoinositide 3-kinase delta syndrome (APDS).

The single-arm, open-label study will include patients aged 12 and above, aiming to assess safety, tolerability and efficacy through a 12-week course of leniolisib. Data from the trial will be used in a future registration application for approval by Japan's Pharmaceuticals and Medical Devices Agency (PMDA).

Leniolisib received orphan drug designation (ODD) in Japan in May 2023. This provides additional support and resources for research and development, facilitating its progress towards potential approval.

The drug received regulatory approval from the US Food and Drug Administration (FDA) for the treatment of APDS in patients aged 12 years and older in March 2023 and was commercially launched under the brand name Joenja in the US in April 2023.