United Therapeutics said Monday that the Food and Drug Administration has cleared it to begin the first clinical trial testing whether organs from gene-edited pigs could provide a viable option for patients in dire need of an organ transplant.
Over the last three years, United and a rival company, eGenesis, have transplanted genetically modified pig hearts or kidneys into several patients, but those procedures were done through compassionate use waivers from the FDA that allow doctors to treat individual patients with no viable alternatives.
Regulators generally ask for more safety and efficacy data in animals to begin a clinical trial, particularly in a fraught and uncharted field like xenotransplantation. Companies pursuing this work have also had to navigate difficult trial design questions, figuring out, for example, how to select patients who were both sick enough to be offered such an unproven intervention but also not so sick that they could not be helped by it.
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