AVRO - AVROBIO granted FDA's orphan drug designation for metabolic disorder therapy

• The shares of gene therapy company AVROBIO ( NASDAQ: AVRO ) jumped ~9% in the pre-market Monday after the company announced that the FDA awarded the orphan drug designation for AVR-RD-05, a gene therapy candidate for mucopolysaccharidosis type II (MPSII), or Hunter syndrome.
• MPSII belong to a group of inherited metabolic disorders collectively called mucopolysaccharidosis. The rare lysosomal disorder primarily affects young boys.
• With its orphan drug designation, the FDA aims to offer financial incentives to drug developers targeting rare diseases and conditions.
• In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers of orphan drugs can claim seven years of marketing exclusivity upon regulatory approval of the treatment.
• In 2023, AVROBIO ( AVRO ) plans to start a Phase 1/2 clinical trial for Hunter syndrome in collaboration with the University of Manchester, UK.

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