Galactosemia community devastated as first treatment fails FDA approval
- The FDA issued a complete response letter on November 27, 2024, denying approval for govorestat, the first investigational treatment for Classic Galactosemia.
- The Galactosemia Foundation, representing the affected community, expressed serious concerns over the lack of treatment options for those facing this devastating disease.
- The foundation is calling for a reconsideration of the FDA's decision, emphasizing the need for patient voices to be heard in the approval process.
On November 27, 2024, in Albany, New York, the Galactosemia Foundation expressed profound disappointment in reaction to the FDA's decision to issue a complete response letter regarding govorestat, marking it as the first potential treatment for Classic Galactosemia. This rare genetic disorder affects the body's ability to metabolize galactose, leading to toxic levels of galactitol in the bloodstream. The foundation highlighted the severe implications of the disease, which can be life-threatening for infants and result in lifelong complications such as cognitive challenges, behavioral issues, and neurological problems. With about 3,300 affected individuals in the U.S. and a mandatory newborn screening in place, the foundation advocates for a re-evaluation of govorestat's approval process, stressing the importance of incorporating patient perspectives in therapeutic assessments. The community feels a tangible opportunity for better quality of life has been lost, emphasizing the urgent need to support patients and families navigating the challenges posed by Classic Galactosemia. The foundation's commitment to providing advocacy and resources underscores the ongoing fight for recognition and treatment options for this ultra-rare condition.