FDA News

FDA prioritizes thyroid eye disease medication review

The FDA on Monday granted priority review of the investigational medication teprotumumab for treatment of thyroid eye disease, also known as Graves’ orbitopathy according to a press release from Horizon Therapeutics.

“Priority review for the teprotumumab [biologics license application] is another positive step toward our goal to make a difference in the lives of people who are living with active [thyroid eye disease] — a painful, debilitating and vision-threatening rare disease,” Timothy Walbert, chairman, president and CEO of Horizon Therapeutics, said in the release. “The accelerated review timeline is particularly important given that there is no FDA-approved medical treatment for [thyroid eye disease], and the window of time for treatment is limited before patients experience potentially long-term, permanent damage to their eyes.”

The FDA’s decision was based on results from the phase 3 OPTIC trial as well as data from the phase 2 trial, according to the release.

As Endocrine Today previously reported, in phase 3 of OPTIC, proptosis was reduced by 2.82 mm over 24 weeks for adults with thyroid eye disease who took teprotumumab. In comparison, those assigned a placebo had a reduction of 0.54 mm. Adverse events were mostly mild or moderate: 31.7% of those taking teprotumumab reported muscle spasms, 14.6% reported infusion-related reactions, 9.8% reported diarrhea and 9.8% reported hearing impairment.

Diplopia and clinical activity scores were also reduced, and quality of life was improved for those taking teprotumumab during the trial, according to the release.

Endocrine Today also reported that there was an 82.9% proptosis response rate at week 24 for those taking teprotumumab vs 9.5% for those taking placebo (P < .001) based on phase 2 results for the trial.

By putting the medication on the priority review track, the FDA has reduced the review time for teprotumumab from 10 months to 6 months. An approval decision is expected in March 2020, according to the release. by Phil Neuffer

Disclosure: Walbert reports he is chairman, president and CEO of Horizon Therapeutics.

The FDA on Monday granted priority review of the investigational medication teprotumumab for treatment of thyroid eye disease, also known as Graves’ orbitopathy according to a press release from Horizon Therapeutics.

“Priority review for the teprotumumab [biologics license application] is another positive step toward our goal to make a difference in the lives of people who are living with active [thyroid eye disease] — a painful, debilitating and vision-threatening rare disease,” Timothy Walbert, chairman, president and CEO of Horizon Therapeutics, said in the release. “The accelerated review timeline is particularly important given that there is no FDA-approved medical treatment for [thyroid eye disease], and the window of time for treatment is limited before patients experience potentially long-term, permanent damage to their eyes.”

The FDA’s decision was based on results from the phase 3 OPTIC trial as well as data from the phase 2 trial, according to the release.

As Endocrine Today previously reported, in phase 3 of OPTIC, proptosis was reduced by 2.82 mm over 24 weeks for adults with thyroid eye disease who took teprotumumab. In comparison, those assigned a placebo had a reduction of 0.54 mm. Adverse events were mostly mild or moderate: 31.7% of those taking teprotumumab reported muscle spasms, 14.6% reported infusion-related reactions, 9.8% reported diarrhea and 9.8% reported hearing impairment.

Diplopia and clinical activity scores were also reduced, and quality of life was improved for those taking teprotumumab during the trial, according to the release.

Endocrine Today also reported that there was an 82.9% proptosis response rate at week 24 for those taking teprotumumab vs 9.5% for those taking placebo (P < .001) based on phase 2 results for the trial.

By putting the medication on the priority review track, the FDA has reduced the review time for teprotumumab from 10 months to 6 months. An approval decision is expected in March 2020, according to the release. by Phil Neuffer

Disclosure: Walbert reports he is chairman, president and CEO of Horizon Therapeutics.