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Jacobus snags pediatric approval of rare autoimmune disease drug based on adult efficacy data

May 7, 2019 10:51 PM UTC

With the pediatric approval of Jacobus' Ruzurgi amifampridine to treat Lambert-Eaton myasthenic syndrome (LEMS) based on clinical data in adults, FDA could be signaling a shift towards greater acceptance of pediatric extrapolation.

Last year, FDA penned draft guidance endorsing pediatric extrapolation for new drugs to treat partial-onset seizures. It has also discussed extending the approach to other indications including heart failure and pulmonary arterial hypertension. Extrapolation can reduce or eliminate the need for clinical trials that are difficult and risky to do, but without which pediatricians are left to guess at appropriate use of drugs in children (see "Pediatric Push")...

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