The U.S. Food and Drug Administration (FDA) has approved the application for orphan drug designation of pembrolizumab for the treatment of recurrent respiratory papillomatosis (RRP), a rare and debilitating condition, caused by the human papillomavirus (HPV). This milestone marks a significant achievement for Yale School of Medicine (YSM) surgeon-scientist and professor of otolaryngology Sara Pai, MD, PhD, who spearheaded the effort.
“This approval is especially noteworthy because it represents a rare instance of an investigator directly submitting an orphan drug application to the FDA, rather than the drug's manufacturer. The move underscores the critical role that surgeon-scientists play in the discovery and delivery of innovative treatments,” said YSM Department of Surgery Chair, Nita Ahuja, MD, MBA.
The FDA's decision is based on the results of a clinical trial that Dr. Pai conducted at Massachusetts General Hospital (MGH). The trial demonstrated that 52% of RRP patients who were treated with pembrolizumab, a commonly used immunotherapy drug for cancer, experienced significant clinical benefits. This trial is notable because it is one of the first to administer pembrolizumab to a non-cancer patient population, showcasing the innovation of the study and potentially expanding the drug’s application beyond cancer treatment.
Dr. Pai expressed optimism about the designation, stating, "I am optimistic that the FDA's endorsement will increase patient access to pembrolizumab nationwide, especially considering the significant clinical benefits observed in the trial."
The orphan drug designation is expected to facilitate further research and expanded availability of pembrolizumab, offering new hope for RRP patients across the country.
Dr. Pai is recognized internationally as an expert in HPV-associated head and neck cancers and cancer immunotherapy. She is leading several clinical immunotherapy trials and has over 120 publications in peer-reviewed journals.