The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the treatment of Krabbe disease, a severe form of pediatric leukodystrophy that is typically fatal by age two. As there is currently no approved treatment for this condition, this IND approval marks a large step in the right direction for patients.
About Krabbe Disease
Krabbe disease, also called globoid cell leukodystrophy, is a rare disorder that causes the destruction of myelin in the brain and nervous system. It can range in severity between affected individuals, but it is typically fatal by age two. Symptoms include fever, difficulties feeding, vomiting, loss of head control, developmental delays, irritability, unexplained crying, muscle spasms, and declines in alertness. These effects are all the result of a mutated gene that is inherited in an autosomal recessive pattern. This gene is responsible for an enzyme called galactocerebrosidase (GALC), and when there is not enough of it, it allows fats to accumulate. This buildup then results in the destruction of myelin. Unfortunately, there is no cure for this condition. Treatment is symptomatic.
FBX-101 is an adeno-associated viral (AAV) gene therapy, and it is the result of nearly 20 years of Krabbe disease research. Through a hematopoietic stem cell transplant, this drug gives the body a working copy of the GALC gene, therefore increasing life span, treating peripheral and central neuropathy, and correcting behavioral impairments.
The clearance of the IND came after approval from the Institutional Review Board and the Institutional Biosafety Committee. Not only does this mean that clinical trials of FBX-101 can move forward, but it also means that Forge Biologics can expand from development to gene therapy manufacturing.
This approval is also exciting for Krabbe disease patients and their families, as it may lead to a viable treatment option. You can read more about FBX-101 and its IND here.