The Food and Drug Administration (FDA)’s Dermatologic and Ophthalmic Drugs Advisory Committee voted in favor of the approval of teprotumumab (Horizon Therapeutics) for the treatment of thyroid eye disease.
Teprotumumab is an investigational fully human monoclonal antibody that works by inhibiting the insulin-like growth factor 1 receptor (IGF-1R). The Biologics License Application (BLA) included data from the phase 3 OPTIC trial, which evaluated the safety and efficacy of teprotumumab in patients with moderate to severe thyroid eye disease, as well as positive results from a phase 2 trial. Integrated, pooled efficacy data from the phase 2 and phase 3 trials showed that treatment with teprotumumab led to significant reductions in inflammation, proptosis and diplopia in patients with active thyroid eye disease.
As for safety, adverse events that occurred at a higher rate in the teprotumumab group included muscle spasms, hearing impairment, gastrointestinal complaints, and infection. In addition, as teprotumumab is an insulin-like growth factor-1 receptor inhibitor, it has the potential to interfere with glucose regulation, especially in those with diabetes. As such, the FDA panel noted in meeting documents that some patients may require additional insulin to maintain glycemic control.
“Today’s unanimously positive vote marks a significant step towards the first FDA-approved treatment for patients with [thyroid eye disease], a vision-threatening autoimmune disease,” said Timothy Walbert, chairman, president and chief executive officer, Horizon. “We believe that teprotumumab has the potential to address a significant unmet need for these patients and we look forward to working with the FDA as it completes its review of our application.”
Although not bound by the committees’ recommendations, the FDA does take them into consideration when making decisions on approval. A Prescription Drug User Fee Act date of March 8, 2020 has been set for the application.
For more information visit fda.gov.