By Annette Bakker
The tragedy of rare disease is not only measured in lives lost—it’s also measured in lives that could have been saved, if only abandoned discoveries had been given another chance.
Promising experimental therapies that could change or even save lives are often sidelined by companies, not because they failed but because they fell off the priority list.
When someone inside a company decides to champion an abandoned therapy, everything can change.
Enter Dr. Freda Lewis-Hall — former chief medical officer at Pfizer.
Years ago at Pfizer, a compound called mirdametinib had shown early promise for treating neurofibromatosis type 1 (NF1), a rare genetic disorder that causes painful tumors to grow along nerves. But like many experimental therapies, it was on the brink of being shelved due to a shift in the company’s priorities.
That could’ve been the end of the story if not for Freda. As CMO, she was willing to listen to patient organizations like the Children’s Tumor Foundation (CTF)—which I lead—that urged the company to give the therapy another chance.
Freda saw potential, took action and found a path forward—outside Pfizer’s pipeline, but with its backing. She helped launch SpringWorks Therapeutics, a spinout company created to give sidelined compounds a second life.
Because one of Pfizer’s C-suite leaders stepped up, a once-forgotten compound became Gomekli, the first treatment approved for adults with NF1.
Across the industry, hundreds of promising compounds are waiting for their own Freda to pull them off the shelf. These aren’t failures. Many have passed initial safety testing and shown signs of real efficacy. Yet they’ve been set aside because they didn’t fit a portfolio, or the market seemed too small, or resources shifted elsewhere.
To be clear, no one faults the companies; business realities are inevitable. But paralysis isn’t. Leadership means finding ways to turn dormant science into real solutions.
Freda’s repositioning of Gomekli proves that doing the right thing isn’t just lifesaving, it’s also smart business. Selling or licensing shelved compounds unlocks value that benefits everyone: patients, investors and companies themselves.
Pfizer, for example, will receive royalties in SpringWorks, which was recently acquired by Merck KGaA for $3.4 billion—a validation of what’s possible when companies choose collaboration over complacency.
It’s time to make stories like this far more common. At CTF, we’ve identified around 30 shelved drugs across companies that could hold promise for neurofibromatosis and related tumors alone. Scale that across more than 7,000 rare diseases—95% of which still lack an FDA-approved treatment—and the potential impact is staggering.
Patient-centric research foundations like the CTF, Myhre Syndrome Foundation and many others are ready to help. We know which compounds deserve another look and which partners can support spinouts, and in many cases we have investors eager to engage. We’re even developing a clearinghouse for companies to list deprioritized compounds and connect with potential developers, turning idle assets into active opportunities.
Every company has its gatekeepers and doers. The future of rare disease treatment will be written by who we choose to back and partner with. My organization stands ready to meet the moment and work with future “Fredas,” the savvy pharma decisionmakers, the life science executives who care deeply about the patients the industry’s well-connected movers and shakers. But we need them to come forward. The next Gomekli is out there.
Together, we can grow the Freda movement and turn courage into care.
Annette Bakker, PhD, is the Chief Executive Officer of the Children’s Tumor Foundation. This piece originally ran in BioSpace.
