Avrobio Receives Innovation ‘Passport’ From MHRA
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Avrobio an “innovation passport” under a new accelerated approval pathway — the Innovative Licensing and Access Pathway (ILAP) — for its investigational gene therapy for Gaucher disease, a rare genetic disorder that causes a buildup of fatty substances around the spleen and liver.
AVR-RD-02 uses patients’ own hematopoietic stem cells, multipotent cells that can develop into all types of blood cells, to treat the disease, which affects about 30,000 people worldwide.
The ILAP program aims to accelerate the time to market, facilitating patient access to medicines. The Innovation Passport is the first step in the ILAP process.
Avrobio plans to release interim clinical data from its phase 1/2 Gaucher disease type 1 clinical trial in the fourth quarter of this year.