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Application for O.D.D. for Crofelemer, a rare gastrointestinal pathology

By 12 September 2022October 12th, 2022No Comments

An application for the recognition of orphan drug status to Crofelemer for a rare form of chronic diarrhea (CDD) called microvillary inclusion disease (MVID) was submitted in May to the European Medicines Agency (EMA). It is a very rare and serious intestinal genetic disease, which affects children from birth, condemning them to feed for life through parenteral therapy.
Orphan drugs are drugs intended for the treatment of such rare diseases as to make it difficult to implement a drug development and registration plan. For this reason, incentives have been defined by the European Union for the research, development and marketing of these molecules.
The submission to the EMA is the first phase of the process of recognizing this status. After validation of the submission, the evaluation process will begin which takes approximately 90 days from acceptance.