(Boursier.com) — Pharma Group NV announced that the U.S. Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kd) syndrome (APDS) in adult and pediatric patients 12 years of age and older . The FDA has evaluated the New Drug Application (NDA) for Joenja.
under priority review and approved the drug based on the results of a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial that evaluated efficacy and safety in 31 patients aged 12 years and older, diagnosed with APDS syndrome. Data from a long-term, open-label, extension clinical trial, in which 38 patients received Joenja for a median duration of two years, were also submitted as part of the regulatory application.
The most common adverse reactions in the clinical trial (incidence greater than 10%) were headache, sinusitis and atopic dermatitis. With the approval of Joenja as a treatment for a rare pediatric disease, the FDA granted Pharming a Priority Review Voucher (“PRV”).
Examples of forward-looking statements may include statements regarding the timing and progress of Pharming’s preclinical studies and clinical trials for its product candidates, Pharming’s clinical and business prospects, and Pharming’s expectations regarding its working capital requirements and cash resources, which are subject to a number of risks, uncertainties and assumptions, including, but not limited to, the scope, progress and expansion of Pharming’s clinical trials and expansion clinical trials and Pharming clinical trials.
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