The filing with the EMA comes a few weeks after Pharming filed leniolisib for approval in the US, setting up potential approvals for what would be the Netherlands-based biotech’s second product on both sides of the Atlantic.
APDS is an ultra-rare disease with no approved therapies. It causes severe immune deficiency and a risk of blood cancers and only affects around 1-2 people per million. But Pharming has an opportunity to be first to market in an indication with hardly any competition coming through the industry pipeline.
