FDA Finally OKs Twice-Rejected Melanoma Drug

The U.S. Food and Drug Administration (FDA) has given accelerated approval to vusolimogene oderparepvec, also known as RP1, a genetically modified oncolytic viral therapy. This combination drug is intended for treating adults with advanced cutaneous melanoma that has progressed despite treatment with a drug known as nivolumab (Opdivo). The FDA's approval comes after a series of regulatory setbacks, with the drug being rejected twice prior to this decision.

The approval is based on data from a single-arm phase II IGNYTE study involving 140 patients with advanced melanoma. Patients in the study experienced an objective response rate of 24% with a median duration of response of 14.1 months. The FDA's Acting Director, Karim Mikhail, emphasized the urgency of this development, stating that the new tool provides oncologists with better options for patients who have exhausted other treatments.

Despite FDA concerns about the reliability of the IGNYTE study's results, an FDA advisory committee voted 10-3 to recognize the study's efficacy. The panel noted that while the methods used in the study had limitations, they still supported the conclusion that the trial demonstrated clinically meaningful results. The drug's approval includes warnings for potential adverse effects such as fatigue, infections, and immune-mediated events.

The prescribing information for RP1 also cautions about the risks of accidental exposure and herpetic infection or reactivation.