Pharming receives agreement of Paediatric Investigation Plan and Promising Innovative Medicine designation for leniolisib from UK MHRA
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
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.
"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
Leniolisib, which has received Orphan Drug Designation in
As previously announced,
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that affects approximately one to two people per million. Also known as PASLI, it is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3Kδ 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 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.
Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger specifically activating AKT and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and PI3Kγ are expressed primarily in cells of hematopoietic origin. The central role of PI3Kẟ in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3Kẟ is a valid and potentially effective therapeutic target for several immune diseases.
To date, leniolisib has been well tolerated during both a Phase 1 first-in-human trial in healthy subjects and a Phase II/III registration-enabling study.
This press release contains forward-looking statements, including with respect to timing and progress of
1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.
2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.
4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.
5. Maccari ME, et al. Front Immunol. 2018;9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21.
7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338
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