Pharming receives Orphan Drug Designation from the European Commission for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS)
- Leniolisib is in a phase II/III registration-enabling study for the treatment of APDS, an ultra-rare and debilitating disease with no approved treatment
- If approved, the drug is expected to reach the market H2 2022
LEIDEN, Netherlands, Oct. 21, 2020 /PRNewswire/ -- Pharming Group N.V. (Euronext Amsterdam: PHARM) today announced that the European Commission has granted orphan drug designation for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicine Agency (EMA). Leniolisib was previously granted Orphan Drug Designation by the US Food and Drug Administration (FDA) in January 2018 for "the treatment of Activated PI3Kδ Syndrome (APDS) or p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency (PASLI)".
The European Commission orphan drug designation provides certain regulatory procedural and financial incentives including, but not limited to, product market exclusivity for ten years in the EU following regulatory approval. To qualify, an investigational drug must be intended to treat a life-threatening or chronically debilitating condition that affects fewer than five in 10,000 people in the EU, and where the treatment provides a significant benefit to those affected by the condition or no satisfactory treatment is available.
Sijmen de Vries, Chief Executive Officer of Pharming, commented:
"We are pleased to have received orphan drug designation from the European Commission, an important milestone in the development of leniolisib for the treatment for APDS, an ultra-rare and debilitating disease. With no currently approved treatment, leniolisib has the potential to address a significant unmet need for patients with APDS. Leniolisib is currently being studied in a registration-enabling Phase II/III trial and remains, subject to regulatory approval, on track to launch in H2 2022."
About Activated Phosphoinositide 3-kinase Delta Syndrome (APDS)
Activated phosphoinositide 3-kinase-delta (PI3Kδ) syndrome (APDS) is caused by mutations in the gene PIK3CD (Type 1 APDS) or PIK3R1 (Type 2 APDS) that activate PI3Kδ. Synonyms for Type 1 and Type 2 APDS are PASLI-CD and PASLI-R1, respectively. PASLI is the acronym for p110δ-activating mutation causing senescent T cells, lymphadenopathy, and immunodeficiency.