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Yale Surgeon-Scientist Secures FDA Orphan Drug Designation for Pembrolizumab in Rare Respiratory Disease

Sara Pai, MD, PhD, has achieved FDA orphan drug designation for pembrolizumab to treat recurrent respiratory papillomatosis (RRP), a rare and debilitating condition. This designation, based on an investigator-initiated clinical trial conducted by Dr. Pai at Massachusetts General Hospital, highlights significant clinical benefits, with 52% of treated patients showing marked improvement. Notably, this marks a rare instance of an investigator directly submitting the application to the FDA rather than the drug’s manufacturer. The designation is expected to enhance patient access and drive further research into this promising therapy.

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