Today, we make history… again! The FDA has officially approved Gomekli (mirdametinib) – the second-ever FDA-approved treatment for NF1 and the first-ever for adults!
This is a game-changing moment for the NF community. For years, patients and families have waited for more treatment options.
Today, that wait gets shorter.
This approval is more than just a win – it’s a result of relentless determination and collaboration. The Children’s Tumor Foundation identified mirdametinib’s potential, ensured its continued development, and partnered with SpringWorks Therapeutics to bring it to this moment. We supported recruitment for clinical trials, worked closely with SpringWorks to deepen their understanding of NF, and never stopped pushing to make this treatment a reality. And now, it is.
But breakthroughs like this don’t happen without YOU. Every patient who participated in a trial, every researcher who pursued new possibilities, and every donor who fueled this work helped make this moment possible. Because of you, NF patients now have another treatment option – and hope for more to come.
“We are excited to celebrate the extraordinary milestone of our partners and long-term friends at SpringWorks for the NF community. This FDA approval shows the power of collaboration to advance innovative science for drugs that may otherwise not have been taken forward,” said Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation. “When industry, researchers, and organizations like ours driving treatment innovation join forces, scientific progress moves faster, and patients gain access to the therapies they need. Every treatment approval is hard-won, built on research, persistence, and partnership. Today, that work delivers a critical new option for NF patients of all ages.”
This is not the first time the NF community has seen a breakthrough like this – and CTF has been instrumental in making it happen. In 2020, CTF helped pave the way for the first-ever FDA-approved treatment for NF, Koselugo (selumetinib), working alongside researchers, industry, and the NF community to ensure its success. That approval, like today’s, was driven by a relentless pursuit of progress, collaboration, and the belief that NF patients deserve options.
Today’s approval of Gomekli marks another huge step forward – and we’re not stopping here.
The future of NF treatments is accelerating – and this is just the beginning.
Read the press release here.
Read the FDA’s announcement here.
Our Celebration!
To mark this milestone, we hosted a special virtual gathering – to come together and celebrate what this means for the NF community.
NF Knowledge Series Webinar: Gomekli (Mirdametinib)
Hear from Christopher Moertel, MD, lead investigator of the ReNeu clinical trial that secured FDA approval for Gomekli (mirdametinib), SpringWorks’ MEK inhibitor drug. Dr. Moertel will discuss the newest FDA-approved treatment for NF1 plexiform neurofibromas.
Friday, February 21
12:00 – 1:00 p.m. EST
This webinar will be recorded.
