📋 Tender Overview
Pharming Group N.V. Pharming or the Company EURONEXT Amsterdam PHARMNasdaq PHAR announces that the European Medicines Agencys EMA Committee for Medicinal Products for Human Use CHMP has granted an accelerated assessment for the Marketing Authorisation Application MAA for leniolisib. Leniolisib has been studied for the treatment of activated PI3K delta syndrome APDS a rare primary immunodeficiency in adults and adolescents age 12 or older in the European Economic Area EEA. Pharming is on track and plans to submit its MAA for leniolisib to the EMA in October 2022.Accelerated assessment reduces the timeframe for the CHMP to review an MAA from 210 days to 150 days. The EMA will grant upon request accelerated assessment of an MAA if they decide the product is of major interest for public health and therapeutic innovation. The clinical development for leniolisib includes positive data from a Phase IIIII study of the product which met both its co-primary endpoints in the target patient population of evaluated reduction in lymph node size and correction of immunodeficiency. The primary efficacy results demonstrated clinical efficacy of leniolisib over placebo with a statistically significant reduction from baseline in the log10 transformed sum of product of diameters SPD in the index lymphadenopathy lesions p0.0012 and normalization of immune dysfunction as evidenced by increased proportion of nave B cells from baseline p0.0001. The shrinking of lymphadenopathy lesions and increased proportion of nave B cells are important in patients as they indicate a reduction in APDS disease markers.In the study leniolisib was generally well-tolerated with the majority of reported adverse events in both treatment groups classified as mild. There were no adverse events that led to discontinuation of study treatment there were no deaths and the incidence of serious adverse events SAEs was lower in the leniolisib group than the placebo group. None of the SAEs were suspected to be related to study treatment.The acceptance of an accelerated regulatory review for leniolisib underlines the high unmet need for patients with APDS with the product potentially being the first approved treatment for this rare disease. This is an important milestone for the APDS community and for Pharming and is built on the successful Phase IIIII data which we first reported in February 2022. We remain focused on progressing leniolisib through the regulatory review process with our MAA on track for submission in October of this year as we seek to make this important new product available to immunologists hematologists and their patients in Europe.About Activated Phosphoinositide 3-Kinase 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 PI3K phosphoinositide 3-kinase delta pathway.12 Balanced signaling in the PI3K 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.13 APDS is characterized by severe recurrent sinopulmonary infections lymphoproliferation autoimmunity and enteropathy.45 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.
