The UK’s Medicines and Healthcare products Regulatory Agency is increasingly granting drugmakers access to its exclusive Innovative Licensing and Access Pathway, with Cambridge, MA-based Avrobio announcing Tuesday the addition of its investigational gene therapy that uses patients’ own hematopoietic stem cells to treat a rare disease.
Under the pathway, which in early 2021 began awarding so-called “passports” to the five-month, accelerated review pathway, patients could potentially gain access to rare disease treatment more quickly.
As of June 22, MHRA says it has received 120 total Innovation Passport applications, and 71 (59%) have been awarded, while 10 (8%) have not been awarded. The top three areas of interest currently are oncology, neurology and respiratory therapies, the regulator said.
Avrobio, which saw its stock price rise by almost 20% on Tuesday (but the price was down more than 97% over the last five years), still has a steep hill to climb to gain market access.
The company said in a statement Tuesday that sometime this quarter it expects to provide an interim clinical data update for its Phase I/II Gaucher disease type 1 clinical trial, as well as an outline of the development and regulatory strategy for its Gaucher disease type 3 program.
The company’s development program encompasses Gaucher disease types 1 and 3, which the company says collectively impact an estimated 30,000 patients worldwide. The current standard of care, enzyme replacement therapy, still typically means a shortened life expectancy and the potential for debilitating symptoms.
But Avrobio has run into issues with its early development programs. It halted development of its initial lead gene therapy to treat Fabry disease last January after dropping plans for the therapy’s accelerated approval once the FDA fully approved Sanofi’s Fabry disease drug Fabrazyme.
Still, early data from February 2021 on the gene therapy for Gaucher disease showed that the first patient saw around a 50% reduction on a key metric from where they were on enzyme replacement therapy.
