FDA and EMA Join Forces on Treatment of Gaucher Disease in Children - - BioPharm International

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FDA and EMA Join Forces on Treatment of Gaucher Disease in Children


FDA and the European Medicines Agency (EMA) released a draft joint proposal to facilitate research in the development of new drugs to treat Gaucher disease in children. The proposal, the result of consultation with various groups of stakeholders, facilitates an agreement between an FDA Pediatric Study Plan and an EMA Paediatric Investigation Plan and addresses the development of drugs for rare diseases in a reduced timeframe in a limited number of patients.

A number of drugs are under development for the treatment of Gaucher disease, a rare condition where specific chemical substances (glucocerebrosides) accumulate in certain cells throughout the body. The disease causes multiple symptoms, including neurological symptoms. According to EMA, there is a high need for new routes of treatment administration to reduce the treatment burden for children with the disease.

The joint FDA/EMA proposal suggests the extrapolation of efficacy from adults to children, including the use of modeling and simulation approaches, and multi-arm, multi-company clinical trials to determine the safety and efficacy of several new medicines and, at the same time, to reduce the number of children involved in the trials.

“The complementary approach proposed would not only facilitate the investigation of new medicines for rare diseases in children but also optimize the number of patients recruited, thereby reducing the burden on children and families,” explains Jordi Llinares Garcia, head of EMA’s Product Development Scientific Support Department, in a press release.

The agencies encourage sponsors who wish to incorporate these proposed approaches in their development plan to contact either EMA or FDA for scientific advice.

Source: European Medicines Agency

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