Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM/NASDAQ: PHAR) announces that a positive decision has been made by the European Medicines Agency (EMA) on the Paediatric Investigation Plan (PIP) for leniolisib, a phosphoinositide 3-kinase (PI3K) inhibitor, currently in development for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS).
The ongoing registration-enabling Phase II/III study has enrolled patients ages 12 years and older. Since APDS also affects younger children, Pharming, as part of the agreed PIP, has developed a clinical plan to include children as young as one year old in future studies.
For the registration of new medicines in Europe, biopharmaceutical companies are required to provide a PIP which outlines the strategy for investigation of a new medicinal product in the paediatric population. The positive PIP opinion from the Paediatric Committee (PDCO) is an endorsement of the clinical program to evaluate the safety and efficacy of leniolisib in patients from 1 year of age to less than 18 years of age with APDS; and the subsequent positive PIP decision of EMA thus paves the way for the potential submission of a Marketing Authorisation Application (MAA) in Europe for leniolisib in the treatment of APDS in adults and adolescents in 2022.
Upon successful completion of the agreed PIP, leniolisib would be eligible for up to an additional two years of marketing exclusivity in the EU, on top of the ten-year EU market exclusivity after market approval as result of its EU Orphan Drug Designation.
Pharming remains on track to announce top-line data from the Phase II/III registration enabling clinical trial of leniolisib for the treatment APDS in Q1 2022.
Chief Medical Officer of Pharming, Anurag Reland, commented:
“We are pleased to have received a positive PIP decision from EMA and accomplish this important regulatory milestone as we continue to advance leniolisib for the treatment of APDS in Europe as well as globally. The approval of the PIP further supports our confidence in the potential of leniolisib to address this orphan disease and population and provides us with a pathway towards marketing approval in Europe, in parallel with our US regulatory strategy. Moving forward, we look forward to continuing to work with the regulatory authorities to bring leniolisib to the European market as expeditiously as possible”.