FDA puts Sanfilippo A enzyme replacement therapy on fast track

New designation comes after agency OK of Phase 1 trial for GC1130A

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Fireworks are seen over the hands of a clinician and a patient standing side by side and both giving a double thumbs up.

GC1130A, an experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type A, has been put on the fast track by the U.S. Food and Drug Administration (FDA), its developers GC Biopharma and Novel Pharma have announced.

“We are pleased with the FDA’s decision to grant fast track designation for GC1130A, especially given the lack of approved treatments for Sanfilippo syndrome,” the companies stated in a press release.

The FDA awards fast track status to experimental medications that have shown the potential to address major unmet needs in the treatment of serious diseases. The goal of granting the designation is to speed the development of potentially important new treatments.

It gives GC and Novel, as the therapy’s co-developers, perks like more frequent communication with the FDA during the drug development process. The companies said the new designation will allow them “to accelerate the development of this new drug, bringing hope to patients and families affected by Sanfilippo syndrome.”

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FDA fast track status follows orphan drug designations in US, Europe

The new designation comes shortly after the FDA cleared GC and Novel to launch a Phase 1 clinical trial that will test the safety of GC1130A in patients with Sanfilippo type A. The companies said they are now gearing up to launch the trial, which is expected to take place at sites in Korea, Japan, and the U.S.

In addition to its new fast track status, the FDA has granted GC1130A rare pediatric disease and orphan drug designations, which provide economic incentives to companies that are working to develop treatments for rare disorders. GC1130A also has been named an orphan drug in Europe.

Sanfilippo type A, the most common and usually most severe form of the disease, is caused by mutations in the gene that provides instructions to make an enzyme called N-sulphoglucosamine sulphohydrolase. This enzyme is needed to break down a molecule called heparan sulfate; without a working version of the enzyme, heparan sulfate builds up to toxic levels and damages brain cells, ultimately driving disease symptoms.

GC1130A contains a lab-made, functional version of this enzyme. The therapy, which is administered directly into fluid-filled cavities within the brain, aims to replace the dysfunctional enzyme in Sanfilippo A patients, allowing clearance of toxic heparan sulfate to combat disease progression. Experiments in animal models have indicated that the therapy reduces heparan sulfate buildup and helps normalize behavior as intended.