USA: Patients with ultra-rare diseases worry new FDA rules will leave them without treatment

US drug regulators have increasingly signalled a focus on faster approvals and rare diseases, but patients with ultra-rare ailments fear they are falling through the cracks, especially given challenges to conducting clinical trials.

One drug, elamipretide, garnered a narrow recommendation from independent advisers for the US Food and Drug Administration (FDA), but the agency rejected the drug’s application in May and recommended another potential pathway for approval.

Patients and advocates worry about new rules on who may receive the medication during this process, and whether the drug will reach approval before the pharmaceutical company runs out of funding for it.

It underscores the challenges of making progress on rare and ultra-rare diseases while also making sure treatments are safe and effective.

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Source: The Guardian, 6 July 2025