Pharming Group N.V. on Tuesday announced 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.
According to the company’s press statement, Leniolisib has been studied for the treatment of activated PI3K delta syndrome (APDS) which is a rare primary immunodeficiency prevalent in adults and adolescents age 12 or older. Reportedly, Pharming is on track and plans to submit its MAA for leniolisib to the EMA in October this year.
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.
In the study, leniolisib was generally well-tolerated, with the majority of reported adverse events in both treatment groups classified as mild, the company claims. 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 company claims.
“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, haematologists, and their patients in Europe,” Anurag Relan, chief medical officer of Pharming said in a statement on Tuesday.
APDS is a rare primary immunodeficiency that affects approximately one to two people per million. APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy. As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma. At present, the only way to definitively diagnose this condition is through genetic testing.