Govorestat, First and Only Potential Treatment for Classic Galactosemia, Does Not Receive FDA Approval

ALBANY, N.Y., November 27, 2024 – The Galactosemia Foundation, a non-profit charitable organization that advocates for people with Galactosemia and their families, issued the following statement in response to the U.S. Food and Drug Administration (FDA)’s decision to issue a complete response letter (CRL) for govorestat for the treatment of individuals with Classic Galactosemia. Govorestat is the first and only investigational treatment to be evaluated for Galactosemia, a rare genetic disease that can be life-threatening for newborns and can cause severe lifelong complications including challenges with daily living, behavioral symptoms, cognition, fine motor skills, and tremor.

Nicole Casale, president of the Galactosemia Foundation, stated:

The Galactosemia community is devastated by the FDA’s decision to not approve the first and currently only potential treatment for this ultra-rare, life-altering disease. Individuals facing this devastating disease need a treatment now. We urge the FDA to reconsider their decision on behalf of those facing this disease, their family members who love and care for them, and future generations who may be impacted by this genetic condition.

Our community has witnessed marked improvements in the lives of our children and loved ones who have been treated with govorestat via the clinical study or the expanded access program. Because of this, we have advocated for years for the FDA to consider the patient voice when evaluating this therapy, which represents a tangible opportunity to change the course of disease and enable those with Galactosemia to live fuller, more independent lives.

The Galactosemia Foundation is committed to supporting the community during this difficult time by providing accurate and timely information, as well as resources for emotional support and advocacy. We will continue to advocate for our voices to be heard and for a path forward.

About Classic Galactosemia

Galactosemia is a rare genetic metabolic disease that affects the body’s ability to convert galactose (a sugar contained in milk, including a mother’s milk) to glucose (a different type of sugar) in the metabolic pathway. Galactose is found in foods but it also produced by the body. In Classic Galactosemia, galactose is is not metabolized properly and is converted into a substance called galactitol. Galactitol is highly toxic, which means it’s harmful to the body.

Toxic galactitol can build up in the blood, tissues, and organs, including the brain. There is evidence that toxic galactitol is responsible for the neurological complications that people with Galactosemia may experience including challenges with daily living skills, behavior, cognition, fine motor skills, speech, tremor and seizures. There are approximately 3,300 patients with Galactosemia in the U.S. and 80-100 new births per year, and approximately 4,400 patients with Galactosemia in the EU and approximately 120 new births per year. Newborn screening for Galactosemia is mandatory in the U.S. and most E.U. countries

About the Galactosemia Foundation

Galactosemia Foundation Inc. is a non-profit charitable organization that advocates for. people with galactosemia and their families. Founded in November 1985, Galactosemia Foundation helps provide families information about galactosemia and facilitates networking between families, clinicians, and researchers. Our mission and objectives include education, support, and advocacy for those affected by galactosemia. For more information about the Galactosemia Foundation and its board of directors, please visit galactosemia.org, and follow the Galactosemia Foundation on Facebook, Instagram, X (Twitter), and LinkedIn.

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Jodie Solari
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