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AVROBIO Granted ILAP Designation from U.K. MHRA for First-in-Class Gene Therapy for Gaucher Disease
ILAP intended to accelerate regulatory review process and facilitate patient access in U.K. for seriously debilitating and life-threatening diseases
About this update from Tectonic Therapeutic, Inc.
[{"type":"text","content":"\nILAP intended to accelerate regulatory review process and facilitate patient access in U.K. for seriously debilitating and life-threatening diseases\n\nAVR-RD-02 has previously received Fast Track status from U.S. FDA and orphan drug designation in the U.S. and EU\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.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) to investigational AVR-RD-02, a first-in-class gene therapy that uses patients’ own hematopoietic stem cells to treat Gaucher disease, a rare lysosomal disorder that can lead multiorgan pathology, clinical morbidity and early mortality.\n\nThe Innovation Passport is the first step in the ILAP process, triggering the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and National Health Service (NHS) England, to chart a roadmap for regulatory and development milestones with the goal of early patient access in the U.K.\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 ILAP\nThe U.K. Medicines and Healthcare products Regulatory Agency (MHRA) launched ILAP at the start of 2021 in order to accelerate the development and access to promising medicines in the early stages of development. The pathway, part of the UK’s plan to attract life sciences development in the post-Brexit era, features enhanced input and interactions with MHRA and other stakeholders. Other benefits of ILAP include access to a range of development tools, such as the potential for a 150-day accelerated Marketing Authorization Application (MAA) assessment, rolling review and a continuous benefit risk assessment. More information about ILAP can be found here.\n\nAbout Gaucher disease\nGaucher disease is a rare, inherited lysosomal...