Pharming receives agreement of Paediatric Investigation Plan and Promising Innovative Medicine designation for leniolisib from UK MHRA
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Pharming Group N.V.
Apr 26, 2022, 01:00 ET
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An agreed Paediatric Investigation Plan (PIP) is the regulatory pathway to market authorization for leniolisib as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in children
A Promising Innovative Medicine (PIM) designation is an early indication that leniolisib is a candidate for the MHRA's Early Access to Medicines Scheme
LEIDEN, Netherlands, April 26, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or the "Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) announces that it has received a positive decision from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) on a Paediatric Investigation Plan (PIP) submission for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor, for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in patients from 1 year of age to less than 18 years of age. The Company also announces that the MHRA has granted Promising Innovative Medicine (PIM) designation to leniolisib for the treatment of APDS.
A PIP is a development plan aimed at ensuring that the necessary data are obtained to support the marketing authorization of a medicine in the pediatric population. All applications for marketing authorization for new medicines in children require the results of studies as described in an agreed PIP, unless the medicine is exempt due to a deferral or waiver. The leniolisib PIP includes two planned global clinical trials in pediatric patients with APDS, the first in children ages 4-11 years and the second in children ages 1-6 years. The Company expects to initiate recruitment for this pediatric program for leniolisib during the second half of 2022.
A PIM designation indicates that a medicinal product is a promising candidate for the MHRA's Early Access to Medicines Scheme (EAMS), which provides pre-market access to products that are intended for the treatment, diagnosis, or prevention of a life-threatening or seriously debilitating condition and have the potential to address an unmet medical need.
Anurag Relan MD, Pharming's Chief Medical Officer, commented:
"We are pleased to have received agreement of this PIP and the PIM designation from the MHRA, these represent important regulatory milestones as we continue to advance leniolisib for the treatment of APDS, a rare, often debilitating, and sometimes fatal condition. The agreed PIP and the PIM designation further support our confidence in the potential of leniolisib to address APDS, as they provide us with a pathway toward marketing authorization in the United Kingdom. Pharming looks forward to continuing to work with regulatory authorities globally to bring leniolisib to patients with this significant unmet medical need as expeditiously as possible."
Leniolisib, which has received Orphan Drug Designation in Europe, received the same designation from the US Food and Drug Administration on January 30, 2018.
As previously announced, Pharming plans to begin submitting global registration filings for leniolisib in the second quarter of 2022 and, subject to approval, launching the treatment in the US in the first quarter of 2023 and starting a series of European launches in the second half of 2023.