The regulator agreed to allow Sangamo Therapeutics to use data to seek accelerated approval for its Fabry gene therapy candidate, eliminating the need for an additional registrational study and potentially shortening the time-to-market by three years.
Sangamo Therapeutics on Tuesday announced that it has aligned with the FDA on an accelerated approval pathway for its investigational Fabry disease gene therapy isaralgagene civaparvovec based on promising data from an ongoing Phase I/II study.
After a “successful” Type B meeting with the regulator, Sangamo can now use estimated glomerular filtration rate (eGFR) slope data—a surrogate marker for kidney function—from the Phase I/II STAAR trial as the “primary basis” to seek accelerated approval for isaralgagene civaparvovec. The company said data to support the accelerated approval pathway will be available in the first half of 2025, and it expects to file the Biologics License Application in the second half of next year.
The FDA also indicated that Sangamo could use data from an extension study, looking at eGFR slope at 104 weeks, to confirm the clinical benefit of isaralgagene civaparvovec.
According to Sangamo, the accelerated approval pathway could speed up the gene therapy’s time-to-market by three years and “avoids the requirement for an additional registrational study to establish clinical efficacy.”
Sangamo CEO Sandy Macrae in a statement said that he is “delighted to have a clear regulatory pathway” for isaralgagene civaparvovec, which could allow the company to “bring this treatment to patients significantly sooner than originally anticipated.”
Analysts, however, seem to be more measured in their assessment of Tuesday’s news, noting key financial challenges for Sangamo. Truist Securities analyst Nicole Germino pointed out in an investor note that “they need a partner to take the program forward to commercial execution.”
In November 2023, Sangamo kicked off a strategic reprioritization initiative to focus its resources on its genomic editing and delivery technologies. As a result, the company deferred investments in its Fabry program, instead opting to search for a development partner for the gene therapy.
While bigger pharma players have shown interest in rare diseases, “gene therapy programs such as sickle cell disease have run into challenges on reimbursement, which has curtailed uptake,” Germino wrote to investors.
H.C. Wainwright analyst Patrick Trucchio was a bit more optimistic in his assessment, however, indicating in an investor note that Sangamo’s promising data from STAAR could “foster a robust collaboration” which may give the company a potential “source of non-dilutive funding for other wholly-owned programs.”
Fabry disease is a rare lysosomal storage disease characterized by the toxic buildup of the lipid globotriaosylceramide, damaging vital organs such as the kidney, heart, stomach and nerves. Patients with Fabry disease typically experience mood disorders, pain, kidney disease, gastrointestinal disturbances and heart failure.
The disease is caused by mutations to the GLA gene, which leads to deficiencies in the activity of the alpha-galactosidase A enzyme. Sangamo’s isaralgagene civaparvovec works by delivering a functional copy of the GLA gene and allowing the liver to restore alpha-galactosidase A activity to healthy levels.
Germino noted that Sangamo “could use a near-term cash infusion to maximize their new pipeline focused on neurological diseases” as the licensing agreement with Roche announced in August 2024 only gives it a cash runway into the first quarter of 2025. Under the deal, Roche’s Genentech paid $50 million in near-term upfront fees and milestone payments to Sangamo to develop novel genomic medicines for neurodegenerative diseases.
