Stay Informed
Advocating for a Treatment
YOUR STORY MATTERS!
Last year, Applied Therapeutics received a Complete Response Letter (CRL) stating the FDA could not approve govorestat in its current form.
Applied Therapeutics remains committed to the galactosemia community and is continuing to seek a path forward with the FDA.
They responded to the warning letter within the required timeline and plan to request a meeting with the FDA to discuss the CRL and resubmission options.
In response, we provided the following statement:
“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.”
While Applied Therapeutics continues to work on a path forward with the FDA, our community is waiting to see how we can support. In the meantime, continuing to share your story, as a patient, caregiver, or rare disease advocate, can make a difference. Talk about the burden of disease and the difference a treatment would mean for you and your family. Your story matters.
Advocate Now!
We know advocacy can be time consuming and overwhelming, but it is more important than ever that our community shares their personal experience with galactosemia and the need for a treatment.
It is going to take our entire community to help us get the attention of the FDA and let them know that we have waited long enough for treatment. There are many ways you can advocate.
Our community has been busy advocating for a treatment. Here is a look back at some of the things we’ve accomplished.
Created a Petition
The galactosemia community urges FDA to incorporate Voice of the Patient in review of potential first treatment for patients with life-altering rare disease. Please spread the word by signing and sharing our Change.org petition.
The Hill article
Submitted an article to The Hill written by our VP Brittany Cudzilo: The FDA is ignoring patients in clinical trials
Voice of the Patient Report
Read the Voice of the Patient Report which gives patient and caregiver perspectives on disease burden and unmet treatment needs in galactosemia.
Shared our stories on Social Media
