RARE Daily

U.K. MHRA Grants ILAP Designation to Avrobio for First-in-Class Gene Therapy for Gaucher Disease

October 18, 2022

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) to Avrobio’s 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 to early mortality.

ILAP is intended to accelerate regulatory review process and facilitate patient access in the United Kingdom for seriously debilitating and life-threatening diseases, and 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 assessment, rolling review and a continuous benefit risk assessment.

The 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, the Scottish Medicines Consortium and National Health Service England, to chart a roadmap for regulatory and development milestones with the goal of early patient access in the U.K.

Gaucher disease is characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages enlarged with these fatty substances are called Gaucher cells and 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–the current standard of care–people with Gaucher disease typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life.

Avrobio’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 the fourth quarter of 2022.

AVR-RD-02 has previously received Fast Track status from the U.S. Food and Drug Administration and Orphan Drug designation in the U.S. and EU.

Author: Rare Daily Staff

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