Pharming’s Leniolisib Wins EU Orphan Drug Status

Pharming Group has received an Orphan Drug designation from the European Commission for leniolisib as a treatment for a rare, genetic immunodeficiency disease known as activated phosphoinositide 3-kinase delta syndrome (APDS).

APDS patients often have low numbers of white blood cells, which leaves them vulnerable to infections.

The Dutch drugmaker is developing leniolisib under license from Novartis and is currently evaluating the treatment in a phase 2/3 trial.

Pharming is hoping to launch the product in 2022. In the EU, Orphan Drug status grants market exclusivity for 10 years. The drug received an Orphan Drug designation from the FDA in 2018, giving it exclusivity for seven years.