For patients living with rare and neglected diseases, the next innovative treatment may already be sitting in a drawer at a pharmaceutical company.
Pfizer once shelved an experimental cancer drug. Then the Children’s Tumor Foundation got in touch. The two organizations recognized that the compound could fight tumors caused by a rare genetic disorder, so Pfizer licensed it to a new spin-off, SpringWorks Therapeutics. SpringWorks developed the compound into Gomekli, a drug that now shrinks tumors in patients with this condition.
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The bet paid off. Last spring, the drug was approved by the FDA and EMA, and Merck acquired SpringWorks for $3.4 billion — one of the biggest biotech deals of 2025.
Thousands of medicines are waiting
Across the pharmaceutical industry and academia, it is estimated that more than 5,000 potential shelved drugs have been discontinued for reasons unrelated to safety or efficacy.
Each represents a potential therapy for conditions that, in many cases, have no approved treatment. Industry participants 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 carefully matching them with qualified entrepreneurial actors can benefit both patients and investors.
No company can allocate resources to develop all promising medicines, especially those aimed at very small patient populations.
It takes an extraordinary amount of time and money to bring a medicine to market — and that’s risky. Even after entering clinical trials, nine out of ten potential drugs fail.
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Companies need to prioritize potential drugs that fit their strategy and offer the greatest return — which means many promising therapies never move forward.
The impact is greater for patients with rare diseases. Of the approximately 7,000 rare and neglected diseases with known molecular causes, only about 500 have an approved treatment. Families facing these diagnoses cannot wait decades for something new to be invented from scratch.
That’s why the nonprofit organization one of us runs, the Children’s Tumor Foundation — dedicated to NF (group of genetic conditions – neurofibromatosis and schwannomatosis) — convinced Pfizer to create SpringWorks. Since then, the foundation has identified about 30 more shelved medications that could help patients with the same group of symptoms.
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Reviving shelved assets isn’t just good medicine — it’s good business. SpringWorks turned discarded compost into a multibillion-dollar company. Licensing even a fraction of the estimated pipeline could boost a new wave of biotech startups.
Building a market for forgotten medicines
Unlocking this potential requires two things: a functioning marketplace for shelved assets and a well-structured collaboration plan.
At this time, there is no unified catalog of discontinued medicines nor a shared system for evaluating their potential. The underlying data exists — but industry engagement is needed to validate and organize it.
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From there, a “connection” system could bring asset holders closer to biotechnology companies, philanthropic research organizations and investors capable of taking the medicine forward.
SpringWorks has proven what is possible. With the right infrastructure, this doesn’t have to be the exception.
The opinions expressed in Fortune.com opinion pieces are solely those of the authors and do not necessarily reflect the opinions and beliefs of Fortune.
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