A Message to the Galactosemia Community from the Galactosemia Foundation Board

Dear Galactosemia Families, 

We want to share an update with you about a bio-pharmaceutical company we’ve worked with closely over the past eight years and their development of a treatment being studied for galactosemia.  We also want to speak honestly — with hope, care, and understanding — about what this means for our community. 

Applied Therapeutics, the company who developed govorestat (also known as AT-007), is joining another company called Cycle Pharmaceuticals. Once the sale of the company is finalized, set for early 2026, Cycle will take over all rights to govorestat.  Until that time, Applied Therapeutics is conducting business as usual, with the financial support of Cycle. 

Cycle focuses only on rare diseases and already helps families around the world. They have experience, people, and resources to keep working on treatments for small communities like ours. Cycle has made public statements about their commitment to get govorestat approved by the FDA for people living with galactosemia. 

The medicine is not approved yet; however, we believe Cycle is a great company to move govorestat forward.  There are no guarantees, but as a Foundation we remain hopeful. 

Our Promise to Families 

The Galactosemia Foundation will keep asking questions, sharing updates, and speaking up for families. We are reaching out to Cycle to start a relationship that we hope will result in a productive partnership. We love our community and continue to hold onto hope we will see a treatment for our rare disease in the future.   

Please review the frequently asked questions that follow this update and reach out with any questions (outreach@galactosemia.org). 

With care and commitment,
The Galactosemia Foundation Board 

FAQ: Understanding This Update 

Is there a cure for galactosemia? 

No. There is no cure, but research is continuing.  The treatment being discussed in this letter is not a cure, however, it has shown promise in alleviating some of the symptoms associated with galactosemia through lowering galactitol in patients with galactosemia. 

What is the name of the medicine Applied Therapeutics developed? 

The medicine is named govorestat, also known as AT-007.  It is a medicine developed for both galactosemia and sord deficiency, with ongoing research for other rare conditions. 

Does this mean it will be approved? 

We do not know yet. Approval takes time and there are no promises; however, Cycle has made commitments to working with the FDA to get galactosemia approved. 

When could something happen? 

It could take years. FDA review is slow but important for safety.   

What should families do right now? 

If you are enrolled in the EAP program and have questions, you may reach out to dcaplan@appliedtherapeutics.com. All families should watch for updates from the Foundation and follow all guidelines provided by your medical care team. 

What is the Foundation doing? 

The Foundation is watching closely and keeping open and honest communication with the Applied Therapeutics team. We are also reaching out to start a relationship with Cycle and advocating for our community with the FDA to understand the best way forward. 

Where to Get Support 

You are not alone. Support is available for families living with galactosemia:

• Galactosemia Foundation: Educational resources, family connections, conferences, and updates (www.galactosemia.org)
• Your metabolic clinic or care team: Medical guidance and day-to-day care questions
• Community support: Other families who understand life with galactosemia

The Galactosemia Foundation will continue to share updates as we learn more. Please reach out whenever you need information, support, or connection by visiting our website www.galactosemia.org or emailing us at outreach@galactosemia.org.