AskBio, a subsidiary of Bayer AG, has announced that its gene therapy AB-1003 (also known as LION-101) has been granted orphan drug designation by the European Commission (EC) for the treatment of limb-girdle muscular dystrophy (LGMD). This rare disease affects 4.5 people per million worldwide, including over 5,000 people in the EU and US. AB-1003 is an investigational recombinant adeno-associated virus (AAV) based gene therapy being developed as a one-time intravenous (IV) infusion. The decision by the EC follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), and was received through AskBio's EU-based subsidiary BrainVectis. AB-1003 is currently being evaluated in the US in a Phase 1/2 multicenter study.
Orphan drug designation provides special incentives in the EU, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees, as well as 10 years of marketing exclusivity if the medicine is approved for marketing.
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