The drug will evaluate the safety, tolerability and efficacy of a single IV infusion of gene therapy in adult subjects.

Asklepios BioPharmaceutical, Inc., a wholly owned and independently operated subsidiary of Bayer AG, announced that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy (LGMD).

AB-1003 is a novel investigational recombinant adeno-associated virus (AAV) based gene therapy currently being developed as a one-time intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I/R9), a disease subtype affecting 4.5 people per million worldwide, including more than 5,000 people in the EU and U.S.

The EC decision 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 being investigated in the U.S. in a Phase 1/2 multicenter study that will evaluate the safety, tolerability and efficacy of a single IV infusion of gene therapy in adult subjects with genotypically confirmed LGMD2I/R9.

“The EC orphan drug designation for AB-1003 is an important recognition of the unmet medical need in LGMD, which has no approved therapy,” said Sheila Mikhail, co-Founder & CEO, AskBio.

“The burden of this rare form of muscular dystrophy on patients and their families is significant, and this decision supports our efforts to potentially bring a new therapeutic option to people in the EU living with the 2I/R9 type of this devastating disease,” said Mikhail.

The EC grants orphan drug designation for medicinal products intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000 in the EU, provided there is no other satisfactory treatment option, or the medicine can be of significant benefit to those affected by a specific condition.

The designation provides special incentives in the EU, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees. In addition, if the medicine is approved for marketing, this designation will provide 10 years of marketing exclusivity.

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