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.
