Janssen&Janssen schreef op 6 januari 2022 07:18:
An agreed PIP is the regulatory pathway to market authorization for leniolisib as a treatment of activated phosphoinositide 3-kinase delta syndrome in children
LEIDEN, Netherlands, Jan. 6, 2022 /PRNewswire/ -- 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 Relan, 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".
About the Paediatric Committee (PDCO)
The Paediatric Committee (PDCO) is the European Medicines Agency's (EMA) scientific committee responsible for activities on medicines for children and to support the development of such medicines in the European Union by providing scientific expertise and defining paediatric needs.
About the Paediatric Investigation Plan (PIP)
A paediatric investigation plan (PIP) is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorisation of a medicine for children in Europe. All applications for marketing authorisation for new medicines have to include the results of studies as described in an agreed PIP, unless the medicine is exempt because of a deferral or waiver.
About Activated phosphoinositide 3-kinase d syndrome (APDS)
APDS is an ultra-rare primary immunodeficiency disease that is caused by variants in either of two genes, PIK3CD or PIK3R1. Variants of these genes lead to hyperactivity of the PI3Kd (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3Kd pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Patients with APDS suffer a median 7-year diagnostic delay.6 Because APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7
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