Waldemarus
- A committee of the European Medicines Agency (EMA) granted accelerated assessment for Pharming's (NASDAQ:PHAR) potential application seeking approval of leniolisib to treat patients aged 12 years and older with activated phosphoinositide 3-kinase (PI3K) delta syndrome (APDS).
- The company said it plans to submit the marketing authorization application (MAA) to the EMA in October.
- Pharming noted that an accelerated assessment reduces the timeframe for review by the EMA's Committee for Medicinal Products for Human Use (CHMP) to 150 days from 210 days.
- The company said clinical development for leniolisib includes positive data from a phase 2/3 trial.
- 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, the company said in an Aug. 1 press release.