EMA accepts filing for AEB 1102 in arginase 1 deficiency
Immedica Pharma AB announced that the company's Marketing Authorisation Application for AEB 1102 (pegzilarginase) has been successfully validated by the European Medicines Agency (EMA) for the treatment of patients with Arginase 1 Deficiency (ARG1-D)
The application is supported by positive results from the international phase III study (CAEB1102-300A, also named PEACE), investigating the safety and efficacy of pegzilarginase.
PEACE (Pegzilarginase Effect on Arginase 1 deficiency Clinical Endpoints) is an international, randomized, double-blind, placebo-controlled trial that enrolled 32 patients aged 2 years and older with arginase 1 deficiency in the United States, Canada and Europe (NCT03921541). PEACE was designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction from baseline. Secondary endpoints include clinical outcome assessments focused on multiple mobility assessments, in addition to safety and pharmacokinetics. Patients were randomized on a two-to-one basis to receive weekly infusions of pegzilarginase or placebo for the double-blind 24-week treatment period.
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