The FDA approved Horizon Therapeutics’ Tepezza (teprotumumab-trbw) for the treatment of adults with thyroid eye disease (TED), a rare condition where the muscles and fatty tissues behind the eye become inflamed, causing the eyes to be pushed forward and bulge outwards (proptosis). The approval represents the first drug approved for the treatment of thyroid eye disease.
“Today’s approval marks an important milestone for the treatment of thyroid eye disease. Currently, there are very limited treatment options for this potentially debilitating disease. This treatment has the potential to alter the course of the disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, non‑surgical treatment option,” Wiley Chambers, MD, deputy director of the Division of Transplant and Ophthalmology Products in the FDA’s Center for Drug Evaluation and Research, said in an agency news release. “Additionally, thyroid eye disease is a rare disease that impacts a small percentage of the population, and for a variety of reasons, treatments for rare diseases are often unavailable. This approval represents important progress in the approval of effective treatments for rare diseases, such as thyroid eye disease.”
Tepezza is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R) that is administered to patients once every three weeks for a total of eight infusions.
Thyroid eye disease is associated with the outward bulging of the eye that can cause a variety of symptoms such as eye pain, double vision, light sensitivity or difficulty closing the eye. This disease impacts a relatively small number of Americans, with more women than men affected. Although this condition impacts relatively few individuals, thyroid eye disease can be incapacitating. For example, the troubling ocular symptoms can lead to the progressive inability of people with thyroid eye disease to perform important daily activities, such as driving or working.
“Today is a great day for people living with thyroid eye disease, a rare, vision-threatening disease that previously had no FDA-approved treatment options,” Timothy Walbert, chairman, president and chief executive officer, Horizon, said in a company news release. “The TED community has gone far too long without an FDA-approved therapy, and we are grateful to the people living with TED and physicians who partnered with us on the clinical development program that led to today’s approval of Tepezza. This also marks the early approval of Horizon’s first Biologics License Application – a key step in our evolution to an innovation-focused biopharma company, developing new medicines for debilitating diseases with few or no treatment options.”
During a conference call with investors, Mr. Walbert discussed the planned pricing strategy for Tepezza.
“We expect the average net realized price for a 6-month course of therapy for Tepezza to be approximate $200,000. This assumes a 75 to 80 percent adherence rate for the course of therapy, and typical rebates and discounts acquired by various stakeholders, as well as typical financial assistance that Horizon provides to eligible patients. The list price per vial for Tepezza is $14,900,” Mr. Walbert said.
“The potential for Tepezza to significantly change the lives of many people living with thyroid eye disease underscores the compelling value it offers. We’ve had meaningful conversations with payers as well as team members of the patient, advocacy, and physician community, regarding this value in relation to pricing. These factors have all informed our pricing decision, which also reflects a significant investment made to develop and make an approved treatment available to thyroid eye disease patients,” Mr. Walbert added.
Tepezza was approved based on the results of two studies (Study 1 and 2) consisting of a total of 170 patients with active thyroid eye disease who were randomized to either receive Tepezza or a placebo. Of the patients who were administered Tepezza, 71% in Study 1 and 83% in Study 2 demonstrated a greater than 2 millimeter reduction in proptosis (eye protrusion) as compared to 20% and 10% of subjects who received placebo, respectively.
The most common adverse reactions observed in patients treated with Tepezza are muscle spasm, nausea, alopecia (hair loss), diarrhea, fatigue, hyperglycemia (high blood sugar), hearing loss, dry skin, dysgeusia (altered sense of taste) and headache. Tepezza should not be used if pregnant, and women of child-bearing potential should have their pregnancy status verified prior to beginning treatment and should be counseled on pregnancy prevention during treatment and for 6 months following the last dose of Tepezza.
The FDA granted this application priority review, in addition to fast track and breakthrough therapy designation. Additionally, Tepezza received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases or conditions. Development of this product was also in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and efficacy of products for use in rare diseases or conditions.