US drug regulators have increasingly signaled 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.
American who spent 11 years in prison for Bali “suitcase murder” is freed
...
Read moreDetails
