The Food and Drug Administration has approved a drug that the manufacturer calls the first ever for the most common form of orbital eye disease in the U.S.
Dublin-based Horizon Therapeutics said Tuesday that the FDA had approved Tepezza (teprotumumab-trbw) for thyroid eye disease, or TED. TED is associated various thyroid conditions, most commonly Graves’ disease, and has a higher prevalence in men than in women – 16 per 100,000 women, compared with three per 100,000 men, according to research.
“Currently, there are very limited treatment options for this potentially debilitating disease,” FDA Division of Transplant and Ophthalmology Products Director Wiley Chambers said in a statement. “This treatment has the potential to alter the course of disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, non-surgical treatment option.”
Shares of the company were down 2.4% Thursday morning.
TED causes bulging of the eyes that can make it difficult for patients to close their eyes and result in eye pain, double vision and light sensitivity.
The approval was based on results from Phase II and Phase III clinical trials. Published results of the Phase II study showed that among 87 patients, 43% of 42 patients receiving Tepezza had a clinical response, defined as a reduction of at least two points in a score indicating active ophthalmopathy and a two millimeter or greater reduction in eye bulging after 24 weeks. Meanwhile, 4% of 45 patients receiving placebo showed a response. In the Phase III study, 83% of 41 patients receiving the drug showed a two-millimeter or greater reduction in eye bulging, compared with 9.5% of 42 patients in the placebo arm.
In an email, a Horizon spokesperson noted that the drug’s list price will be $343,000, or $14,900 per vial, with a full treatment lasting six months and including about 23 vials. The company expects a rate of compliance of 75-80%.
Other drugs are also in development for TED and Graves’ disease, according to ClinicalTrials.gov. These include Immunovant’s RVT-1401, a monoclonal antibody in Phase IIa and IIb studies of respectively eight and 77 patients with Graves’ disease taking place in Canada, the U.S. and Europe. Academic studies are also underway, investigating the use of the painkiller celecoxib and the statin drugs atorvastatin and simvastatin.
Photo: bandit2523, Getty Images
