FLT201 Drug Wins Orphan Drug Designation From FDA

Release Date: 02-Oct-2021

Both the European Commission (EC) and FDA have granted Orphan Drug Designation for FLT201 for the treatment of Gaucher disease. FLT201 is an investigational liver-directed AAV gene therapy for the treatment of Gaucher disease Type 1. FLT201 contains a liver-specific promoter and a GBA1 sequence, proprietary GCase variant 85, which has a 20-fold longer half-life at lysosomal pH conditions than wild-type GCase protein.


Orphan Drug Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (“COMP”). It is assigned to medicines intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating and affects fewer than five in 10,000 people in the EU. It provides several benefits like financial incentives for companies to develop and market therapies, including market exclusivity, protocol assistance and fee reductions.


The US FDA Orphan Drug Designation Program gives orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the US and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

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