Health
Family faces regulatory and trial hurdles seeking Batten disease gene therapy
A child diagnosed with rare Batten disease faces hurdles accessing an experimental treatment, CNBC reports.
The short version
- A child diagnosed in infancy with Batten disease is facing barriers accessing an experimental gene therapy.[CNBC]
- The therapy was previously returned by Amicus Therapeutics to Nationwide Children's Hospital following FDA trial demands.[CNBC]
- Neela Therapeutics is discussing a new clinical trial with the FDA, but the child may be excluded due to taking another medication.[CNBC]
Key facts
- Wheeler Stecker was diagnosed with Batten disease at four weeks old.[CNBC]
- Amicus Therapeutics relinquished its rights to the CLN3 Batten disease gene therapy back to Nationwide Children's in 2024.[CNBC]
- Former Amicus CEO John Crowley stated the FDA required a five-year study with at least 50 patients and a placebo arm in late 2021.[CNBC]
- Neela Therapeutics is in talks with the FDA on a trial design delivering the therapy into patients' spinal cords and eyes.[CNBC]
What remains uncertain
- Whether Wheeler will be permitted into Neela Therapeutics' upcoming trial remains uncertain due to his use of miglustat.[CNBC]
Sources
Outlet counts describe coverage, not independent confirmation. Reports may share a wire service or original source.