Immunome recently announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for varegacestat, a once-daily oral gamma-secretase inhibitor (GSI) for adults living with desmoid tumors.
Why is this milestone important?
For patients and families navigating this rare and often unpredictable disease, continued progress in research and drug development is critical. While regulatory review is still ahead, this submission reflects growing momentum across the field and reinforces the importance of collaboration between researchers, industry, and the patient community.
What happens next?
The FDA will now conduct an initial review over the next 60 days to determine whether to accept the application for formal review. If accepted, a comprehensive, multidisciplinary evaluation of the therapy’s safety, efficacy, and manufacturing quality typically follows over the next 6 to 10 months. This process may include inspections and, in some cases, an advisory committee review.
The Desmoid Tumor Research Foundation (DTRF) is deeply grateful to the Immunome team and everyone who helped bring this moment closer. We look forward to the progress ahead for people living with desmoid tumors.
“The varegacestat NDA submission marks an important milestone for Immunome. It reflects the strength of the RINGSIDE dataset and the commitment of the team advancing this program,” said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Immunome. “We believe varegacestat has the potential to provide adults with progressing desmoid tumors with a meaningful new oral treatment option, and we are grateful to the patients, families, investigators and study site teams whose participation made this submission possible.”
Read Immunome’s full press release below:
