[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients

Core Viewpoint - Roche has implemented new dosing restrictions for Elevidys™ (delandistrogene moxeparvovec) for non-ambulatory Duchenne muscular dystrophy (DMD) patients due to two cases of fatal acute liver failure, leading to a reassessment of the benefit-risk profile as unfavorable for this patient group [1][2][4][7]. Group 1: Dosing Restrictions - Effective immediately, non-ambulatory patients will no longer receive Elevidys in commercial settings, and dosing for these patients in clinical trials is paused until additional risk mitigation measures are established [1][7]. - The new restrictions do not affect ambulatory DMD patients, where the benefit-risk ratio remains positive [2][7]. Group 2: Clinical Context - The two fatal cases of acute liver failure occurred among approximately 140 non-ambulatory patients treated with Elevidys globally [4]. - European regulators have requested temporary clinical holds on several Elevidys studies following the incidents [4]. Group 3: Product Overview - Elevidys is the first approved gene therapy for DMD, designed to deliver new instructions to cells to produce dystrophin, aiming to slow disease progression [10][11]. - The treatment has been approved in eight territories, including Bahrain, Brazil, and Japan, and Roche collaborates with Sarepta Therapeutics for its commercialization [5][11]. Group 4: Disease Background - Duchenne muscular dystrophy is a rare genetic disease primarily affecting males, with a prevalence of 1 in 5,000 boys born worldwide, leading to progressive muscle weakness and loss of mobility [3][12]. - The average life expectancy for individuals with Duchenne is only 28 years, highlighting the critical need for effective treatments [12][16].