The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with Duchenne muscular dystrophy (DMD) who received Santhera Pharmaceuticals‘ investigational treatment Puldysa (idebenone), Duchenne UK announced in a press release.
This treatment is designed to slow the progression of breathing problems associated with the condition. NICE is currently in a consultation process to determine whether Puldysa should be funded and made available to patients in the U.K. through the National Health Service.
To help NICE understand the impact Puldysa may have on the life of those with DMD and make an informed decision regarding its funding scheme, several patient organizations in the U.K. are urging DMD patients to fill out and submit a survey by Aug. 19 detailing their experiences with the medication.
The survey, which takes approximately 25 minutes to complete, is meant for those with DMD who took Puldysa at some point, as well as for parents of children with the condition who had access to the therapy. The survey was created by Duchenne UK, in partnership with DMD Pathfinders, Joining Jack, Muscular Dystrophy UK, Action Duchenne, Alex’s Wish, and Duchenne Family Support Group.
Puldysa contains idebenone, an artificial small molecule that resembles the antioxidant molecule ubiquinone (also known as coenzyme Q10), which is important for mitochondria, small cell compartments that produce energy.
By mimicking the activity of ubiquinone, idebenone is thought to increase the production of energy inside cells and to protect mitochondria from damage caused by the accumulation toxic oxidant molecules (reactive oxygen species). This is expected to ease the respiratory symptoms caused by DMD.
The medication is currently available free of charge, under the name Raxone, to a small group of DMD patients, ages 10 and older, living in the U.K. through an Early Access to Medicines Scheme (EAMS), which has recently been renewed for another year.
The purpose of the EAMS program is to allow Santhera to gather real-world data on the effectiveness and safety of the therapy — data that will then be used by the company to support its possible approval in Europe.
Santhera submitted a request — in the form of a conditional marketing application — to the European Medicines Agency (EMA) last year for Puldysa to be approved for DMD patients with progressive breathing difficulties who were not taking glucocorticoids. An opinion by the EMA’s Committee for Medicinal Products for Human Use about the therapy’s approval is expected before the end of the year.
The therapy has previously been named a promising innovative medicine by U.K. health authorities, and granted the designations of rare pediatric disease and fast track by the U.S. Food and Drug Administration.
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