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AVROBIO Receives Rare Pediatric Disease Designation from the U.S. FDA for First-in-Class Gene Therapy for Gaucher Disease
AVR-RD-02 has previously received Fast Track status from FDA, orphan drug designation in the U.S. and EU, and ILAP designation from the U.K. MHRA CAMBRIDGE,
About this update from Tectonic Therapeutic, Inc.
[{"type":"text","content":"\n AVR-RD-02 has previously received Fast Track status from FDA, orphan drug designation in the U.S. and EU, and ILAP designation from the U.K. MHRA\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to AVR-RD-02, a first-in-class investigational gene therapy that genetically modifies patients’ own hematopoietic stem cells (HSCs) to treat Gaucher disease, a rare lysosomal disorder that can lead to multiorgan pathology, clinical morbidity and early mortality.\n\nFDA’s Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the company or sold to a third party.\n\nAVROBIO’s Gaucher disease program includes Gaucher disease type 1 and type 3, which collectively impact an estimated 30,000 patients worldwide. The company expects to provide an interim clinical data update for its Phase 1/2 Gaucher disease type 1 clinical trial, as well as an outline of the development and regulatory strategy for its Gaucher disease type 3 program, in Q4 2022.\n\nAbout Gaucher disease\nGaucher disease is a rare, inherited lysosomal disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages enlarged with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) – the current standard of care – people with Gaucher disease typically have a shor...