Had Pfizer’s Freda Lewis-Hall not stepped in, SpringWorks’ rare disease treatment may never have reached patients. Pharmas can act now to help find the next Gomekli.
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. Behind every shelved compound is a patient running out of time, a parent searching for answers, a community hoping that someone inside a truly innovative company will fight for them.
When someone inside a company decides to champion an abandoned therapy, everything can change.
Enter Dr. Freda Lewis-Hall. She began her career on the frontlines of psychiatric care before rising to senior leadership roles across major biopharma companies and ultimately becoming chief medical officer at Pfizer.
Freda’s actions should be the expectation, not the exception.
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 her own scientists who still believed in the compound, and 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. With support from CTF and believers across Pfizer and beyond, SpringWorks advanced mirdametinib from the shelf to the clinic and eventually to patients.
Because one of Pfizer’s C-suite leaders stepped up, a once-forgotten compound became Gomekli, the first treatment approved for adults with NF1. For these patients, who had waited years for hope, the February 2025 approval of Gomekli, which shrinks inoperable tumors, changed everything.
We should celebrate Freda’s courage and urge more pharma executives to follow her example. Freda’s actions should be the expectation, not the exception.
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.
Too often, there’s no process and no champion to revisit shelved compounds or partner with groups ready to carry them forward. While they sit idle, patients wait for treatments that could already exist. Every day of hesitation costs lives.
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.
What’s missing isn’t capability, it’s conviction. There are countless C-suite leaders like Freda who never lose sight that beyond endpoints are real patients who can benefit from once forgotten assets. They just need a pathway to act.
Companies can start by creating formal review processes, transparent reporting systems and standardized data filings for shelved assets. They should also empower their scientists to ask hard questions: What promising treatments are we leaving unused? Who could bring them to patients?
Partnering with patient-centric research foundations led by pharma veterans is a pragmatic pipeline strategy. They know the science, the community and the urgency—and their mandate is to deliver treatments.
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.
