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…
News
GC Biopharma and Novel Pharma plan to initiate a Phase 1 clinical trial this year to test the safety and tolerability of GC1130A, an investigational enzyme replacement therapy that the companies are developing for Sanfilippo syndrome type A. The announcement follows the U.S. Food and Drug Administration…
Over the next two decades, the economic burden of Sanfilippo syndrome in the U.S. is expected to exceed $2 billion, according to research that the National Institutes of Health (NIH) called “the first disease burden value estimate” ever done for the rare disease. “Our results suggest that [Sanfilippo…
Most children with Sanfilippo syndrome type A who received the one-time gene therapy UX111 early in life in a clinical trial continue to show cognitive development gains beyond the age where patients usually start regressing. These improvements were associated with reductions in heparan sulfate (HS) in the children’s cerebrospinal fluid…
Developing biomarkers may help support accelerated federal approval for treatments in Sanfilippo syndrome and related diseases. That was the subject of an in-person and virtual workshop last month by the Reagan-Udall Foundation for the Food and Drug Administration that discussed primary disease activity biomarkers and focused on…
The European Medicines Agency (EMA) has granted orphan drug status to GC1130A, an investigational brain-penetrant enzyme replacement therapy that’s in preclinical testing for Sanfilippo syndrome type A. GC1130A, which is being developed by GC Biopharma with Novel Pharma, was also awarded…
An experimental enzyme replacement therapy (ERT), called JR-441, for Sanfilippo syndrome type A has been designated an orphan drug by the U.S. Food and Drug Administration, the treatment’s developer, JCR Pharmaceuticals, has announced. The designation is given to therapies being developed for conditions affecting fewer than 200,000 people…
Note: This story was updated Dec. 11, 2023, to correct that Kyowa Kirin has entered into an agreement to acquire Orchard Therapeutics, which will be finalized next year. The story also corrected that OTL-200, Libmeldy, is under priority review by the U.S. Food and Drug Administration with a review date…
JCR Pharmaceuticals is now recruiting children and adolescents with Sanfilippo syndrome type A for a Phase 1/2 clinical trial testing its enzyme replacement therapy JR-441 at a single location in Hamburg, Germany. The first patient in the trial recently was dosed, according to a press release from…
A primary goal of this year’s World Sanfilippo Awareness Day, observed annually on Nov. 16, is to get people talking about Sanfilippo syndrome, a rare disorder thought to affect 1 in 70,000 people. “World Sanfilippo Awareness Day is about spreading awareness and sparking conversations globally about Sanfilippo syndrome,…
Recent Posts
- Being a Sanfilippo parent means facing impossible decisions
- UX111 gene therapy shows long-term benefit in Sanfilippo type A: Trial data
- Our respite caregiver provides much-needed help
- Study says U.S. Sanfilippo societal cost will top $2B over 20 years
- Bone marrow transplant helps hand-eye coordination in Sanfilippo child
- The support system that allows my special needs child to thrive
- Enzyme therapy directly into the brain tested for Sanfilippo type D
- I’m finally seeing a therapist, 3 years after my daughter’s diagnosis
- Tralesinidase alfa ERT for Sanfilippo type B children put on fast track
- Educating others about Sanfilippo syndrome, one bite at a time