Pharming Group N.V. (“Pharming” or “the Company”) announces that the European Medicines Agency’s (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 II/III 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 (p=0.0012) and normalization of immune dysfunction, as evidenced by increased proportion of naïve B cells from baseline (p<0.0001). The shrinking of lymphadenopathy lesions and increased proportion of naïve 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.
Anurag Relan, Chief Medical Officer of Pharming, commented ” 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 II/III 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.”
