For patients living with rare and neglected diseases, the next breakthrough treatment might already be sitting in a pharmaceutical company’s filing cabinet.
Pfizer once shelved an experimental cancer drug. Then the Children’s Tumor Foundation came knocking. The two organizations recognized the compound could fight tumors caused by a rare genetic disorder, so Pfizer licensed it to a new spin-off, SpringWorks Therapeutics. SpringWorks advanced it into Gomekli, a medicine that now shrinks tumors in patients with that condition.
The bet paid off. Last year, in spring, the drug was approved by the FDA and EMA and Merck acquired SpringWorks for $3.4 billion — one of 2025’s largest biotech deals.
Thousands of drugs are waiting
Across pharma and academia, an estimated 5,000+ shelved drug candidates were discontinued for reasons unrelated to safety or efficacy. Each represents a potential therapy for conditions that, in many cases, have no approved treatment at all. Industry stakeholders have a unique opportunity to collaborate on identifying these compounds. Aligning these assets with capable and motivated partners will benefit both drug developers and patients — and matching them carefully with capable entrepreneurial stakeholders could benefit both patients and investors.
No company can commit the resources to develop every promising drug, especially those designed for very small patient populations. It takes an extraordinary amount of time and money to bring a drug to market — And it’s risky. Even after entering clinical trials, nine in 10 candidate medicines fail. Companies must prioritize candidates that fit their strategy and offer the strongest return — which means many promising therapies never move forward.
The stakes are highest for rare disease patients. Of the roughly 7,000 identified rare and neglected diseases with known molecular causes, only about 500 have an approved treatment. Families facing those diagnoses can’t wait decades for something new to be invented from scratch.
That’s why the nonprofit that one of us runs, the Children’s Tumor Foundation — which is dedicated to NF (family of genetic conditions – neurofibromatosis and schwannomatosis) — convinced Pfizer to create SpringWorks. The foundation has since identified roughly 30 more shelved drugs that could help patients with the same family of conditions.
Reviving shelved assets isn’t just good medicine — it’s good business. SpringWorks turned a discarded compound into a multibillion-dollar company. Licensing even a fraction of the estimated pipeline could fuel a new wave of biotech startups.
Building a market for forgotten drugs
Unlocking that potential requires two things: a functioning market for shelved assets, and a well-thought-out plan for collaboration.
Right now, there’s no unified catalog of discontinued drugs or shared system for evaluating their potential. The underlying data exists — but industry buy-in is needed to validate and organize it.
From there, a “matchmaking” system could pair asset owners with biotech firms, research philanthropies, and investors equipped to take the drug forward.
SpringWorks proved what’s possible. With the right infrastructure, it doesn’t have to be the exception.
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