Recently, Applied Therapeutics has issued a press release announcing while the ACTION-Galactosemia Kids trial data did not quite meet statistical significance, they believe it has demonstrated compelling evidence of clinical benefit and a favorable safety profile in patients with Galactosemia. Applied Therapeutics believes there is compelling evidence of clinical efficacy and plans to move toward registration of govorestat for Galactosemia. They plan to request a meeting with the FDA to discuss a potential New Drug Application (NDA) submission. The trial will be unblinded and placebo patients will be crossed to active treatment.
The Galactosemia Foundation has shared qualitative data on the patient journey and burden of disease with the FDA on several occasions, including an externally led patient-focused drug development meeting held in September 2022, patient and caregiver focus groups and direct outreach to the agency.
We know advocacy can be time consuming and overwhelming, but it is more important than ever that our community rally behind this important cause. Here are several ways you can advocate and tools to help you communicate your message and support our community.
But 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.
Email us outreach@galactosemia.org: Share your experience with the clinical trial and/or your experiences with galactosemia and why you believe we need a treatment now. We will use quotes on social media. Please include a photo of your child/family if possible!
Share your story with a group of passionate passion advocates who believe the patient voice should be central to healthcare and barriers to access should be removed. https://patientsrisingstories.org/get-involved/your-patient-story/
In a few sentences or less, respond to the prompt: “What a treatment for galactosemia would mean for my family” through a simple smartphone video. Answers should be specific/tangible examples, such as “more time to participate in after-school activities” or “less struggling with math” or “the ability for my child to make friends because she can remember their names.” Please email your videos to us at outreach@galactosemia.org and we will create a video collage for social media.
Post about your galactosemia journey on your social media platforms. For tips and ideas for getting started, see #ThisIsGalactosemia storytelling resource [add link]. Use #PaveTheWayFDA and #ThisIsGalactosemia, and tag FDA.
Share your story, as well as other stories from the galactosemia community, on your social media platforms. Feel free to use the text, photos, and videos you created for the “share your story” prompts above! Use #PaveTheWayFDA and #ThisIsGalactosemia, and tag FDA.
Follow the Galactosemia Foundation on social media and share the posts on your personal social media channels.
Ask your friends, family, neighbors, and acquaintances to sign the Change.org petition.
