Horizon Therapeutics has had its drug Tepezza, which is the first ever treatment for thyroid eye disease, approved by The US Food and Drug Administration (FDA).
Tepezzav was approved on the results of two randomised studies which included a total of 170 patients. Of the participants administered with the drug, 71% in the first study and 83% in the second study exhibited a greater than 2-millimetre reduction in proptosis, as compared to 20% and 10% in respective studies who received the placebo.
The approval has been heralded as an important milestone in the treatment of thyroid eye disease.
“Currently, there are very limited treatment options for this potentially debilitating disease,” Dr Wiley Chambers, deputy director of the Division of Transplant and Ophthalmology Products in the FDA’s Center for Drug Evaluation and Research, said.
“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.
“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.”
The treatment was approved as part of the FDA’s Priority Review system, as well as Fast Track and Breakthrough Therapy Designation. Its development was also supported by the FDA’s Orphan Drug program, which provides grants for clinical studies towards the safety and efficacy of products for rare diseases and conditions.
“This approval is meaningful to our organisation because we are committed to helping patients with vision impairment and those who are at significant risk,” Mr Jeff Todd, president and CEO of Prevent Blindness, said.
Horizon Therapeutics will now conduct a post-marketing study to assess the treatment’s safety among a wider population. The drug is not yet available in Australia.