FDA Grants Priority Review to SpringWorks Therapeutics’ New Drug for NF1 Patients

The Children's Tumor Foundation (CTF) is thrilled to announce that our long-time partner, SpringWorks Therapeutics, has received FDA Priority Review for mirdametinib, a potential new treatment for NF1-associated plexiform neurofibromas. This marks a significant milestone for patients, particularly adults who currently lack approved therapies.

CTF played a key role in the formation of SpringWorks and in driving the drug repositioning strategy that led to this promising development. We are committed to accelerating new treatments and are excited to see this therapy move closer to approval in the U.S. and Europe.

"Plexiform neurofibromas may sit next to or surround vital organs and can cause serious medical complications for patients. While progress has been made, there remains a pressing need for more treatment options, particularly for adults who currently have no approved therapy," said Annette Bakker, PhD, Chief Executive Officer of the Children's Tumor Foundation (CTF) and Board Chair of CTF Europe. "CTF is dedicated to deploying its time, talent, and funding towards accelerating the development of new treatments. We congratulate our long-term partner, SpringWorks, and are very excited to see the first drug repositioning program come so far for NF. We are thrilled that patients in the United States and Europe could soon have a new therapy available to them."

Congratulations to SpringWorks, and here's to continued progress in our fight against NF!

Read the complete press release HERE.