Part I: How a Shelved Drug Became a Lifeline for NF Patients
At the Children’s Tumor Foundation (CTF), we believe that no promising treatment should sit on a shelf while patients wait. That’s why we played a pivotal role in reviving a once-abandoned drug—now known as Gomekli—to bring hope to families affected by NF1 with plexiform neurofibromas (PN). Through advocacy, collaboration, and an unwavering commitment to patients, we worked to ensure that this potential treatment didn’t fade into obscurity. Our efforts helped connect Pfizer’s discontinued MEK inhibitor with SpringWorks Therapeutics, leading to the drug’s development and, ultimately, FDA approval.
“We couldn’t have done this without CTF,” Jim Cassidy, SpringWorks’ chief medical officer, told BioSpace.
This story is more than just a drug’s journey—it’s a testament to the power of patient-driven research and the partnerships that make scientific breakthroughs possible. Read the full article on how Gomekli went from a “ghost drug” to an approved treatment for NF1-PN.
Read more on BioSpace: How SpringWorks Took Pfizer’s Rare Disease Ghost Drug and Got It to Patients
Part II: Unlocking the Potential of Shelved Drugs for Rare Diseases
The journey of Gomekli from an abandoned compound to an FDA-approved treatment for NF1 with plexiform neurofibromas (PN) is just one example of how patient-driven organizations like the Children’s Tumor Foundation (CTF) are reshaping the future of rare disease drug development. But Gomekli is not an isolated case—there are countless potential treatments hidden in pharmaceutical pipelines, waiting for the right advocates and innovators to bring them to patients.
“This is one drug, but it’s definitely not the last one,” Annette Bakker told BioSpace. “And what we actually really hope is that this may trigger a motivation for some other companies.”
This is where strategic partnerships and creative problem-solving come in. By identifying promising yet overlooked compounds, patient groups, researchers, and biotech companies can work together to breathe new life into stalled drug programs. The model that helped bring Gomekli to patients is one that can—and should—be replicated across other rare diseases.
How can more organizations mine biopharma’s storerooms for potential treatments? What does it take to turn a shelved asset into a life-changing therapy? The next article in this series explores exactly that.
