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.
