In December, the U.S. Food and Drug Administration (FDA) approved the first gene therapy, Waskyra, for the rare condition Wiskott-Aldrich syndrome, which affects the immune system. What’s even more remarkable is that it’s also the first FDA-approved cell or gene therapy—which are notoriously expensive—sponsored by a nonprofit. “Rare conditions have become less and less attractive for industry, because the value proposition from a commercial standpoint is pretty limited,” says Ilaria Villa, CEO of Fondazione Telethon, the Italian charity behind the drug. The organization was founded more than 30 years ago by the families of people affected by muscular dystrophy with the goal of collecting funds from TV marathon donations to finance research for rare conditions. Since then, the group has invested heavily in turning their decades of funded research into medicines.
The approval of Waskyra is a positive sign for their model. But it’s uncharted territory for a nonprofit like theirs. “We have to keep the balance between making the product as [accessible] as possible to the patients, but at the same time, containing the costs,” says Villa. “We have to find a better approach to make those products available, sustainable, and scalable over time.”