The FDA previously granted the therapy — now in an enrolling clinical trial in the U.S. — orphan drug and rare pediatric disease status; all aim to support and speed its development. Fast track also offers the possibility of priority review should it come up for regulatory approval.
“FDA Fast Track designation for PR001 for the treatment of neuronopathic Gaucher disease underscores the significant unmet medical need for this devastating condition, for which there are no currently approved therapies,” Asa Abeliovich, MD, PhD, Prevail’s founder and CEO, said in a press release.
Mutations in the GBA1 gene underlie all forms of Gaucher disease. PR001 is designed to provide a healthy version of GBA1 to patients’ nerve cells, restoring production of the beta-glucocerebrosidase enzyme that is damaged in these patients, leading to a toxic buildup of large molecules inside cells.
Neuronopathic Gaucher includes types 2 and 3, and typically appears early in life. Type 2 Gaucher begins in infancy and features rapid neurodegeneration. Type 3 is usually milder and progresses more slowly.
The mainstay of Gaucher treatment is enzyme replacement therapy (ERT), which provides patients with a form of the glucocerebrosidase enzyme they are deficient in or lacking. But these therapies currently can only benefit people with non-neuronopathic forms, or type 1 — and those with type 3 — disease, because ERTs cannot cross the blood-brain barrier and reach the brain.
“We believe PR001 has the potential to serve as a much-needed therapeutic option for these patients,” Abeliovich added.
An open-label Phase 1/2 clinical trial, called PROVIDE (NCT04411654), will evaluate PR001 in up to 15 children, newborns to age 2, with type 2 Gaucher. Recruitment is underway at NYU Medical Center, with sites in California, Minnesota, and Pennsylvania expected to open soon. Contact and site information is available here.
All enrolled be treated with a single administration of PR001 given via intracisternal injection, or an injection directly into the cisterna magna, an area above the spinal canal containing spinal fluid.
The study’s primary goals are the safety and tolerability of the treatment, as seen over five years of follow-up, and evidence of its immunogenicity (ability to induce an immune response) at years two and three post-treatment.
Secondary outcomes include measures of efficacy, like changes in cognitive abilities and motor skills throughout the first year, and again at three years, after the treatment is given.
Prevail announced earlier this year that, under a compassionate use program, a patient with type 2 Gaucher was treated with PR001.