Teprotumumab confers long-term benefits in thyroid eye disease
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Adults with active thyroid eye disease assigned the human monoclonal antibody teprotumumab experienced improvements in disease outcomes that persisted nearly 1 year after receiving the last trial dose, new study data show.
“These findings indicate that teprotumumab provided clinical benefits nearly 1 year after the last dose in people with thyroid eye disease participating in a placebo-controlled clinical trial,” Roger A. Dailey, MD, FACS, professor of ophthalmology, oculofacial plastic surgery, and dermatology at Oregon Health & Science University’s Casey Aesthetic Facial Surgery Center (CAFSC), told Healio. “These data indicate that all patients completing the long-term evaluation had at least some improvement in one or more of the major outcomes under analysis.”
As Healio previously reported, the FDA in January approved teprotumumab-trbw (Tepezza, Horizon Therapeutics) for the treatment of adults with thyroid eye disease, marking the first drug approved for the condition. Teprotumumab, an insulin-like growth factor I receptor inhibitor, is a fully human monoclonal antibody developed to address a substantial unmet need for patients with thyroid eye disease. The drug blocks the inflammatory/autoimmune pathophysiology that underlies thyroid eye disease.
In a phase 2 trial, Dailey and colleagues assessed data from 42 adults with active, moderate to severe thyroid eye disease assigned to the teprotumumab arm of the randomized controlled study (mean age, 52 years; 66.7% women; 85.7% white; 73.8% nonsmokers). Participants in the drug arm received teprotumumab for 24 weeks, with the last dose given at week 21. Researchers followed the cohort for 48 weeks after treatment ended. Within the cohort, 37 participants completed the study treatment, with 36 completing the off-treatment follow-up period and evaluated at week 72 for any improvement from their baseline measurements for proptosis, clinical activity score and diplopia. The data were presented as part of the Academy of Managed Care Pharmacy (AMCP) Nexus 2020 Virtual Meeting.
Researchers found that 100% of participants in the teprotumumab arm experienced improvement in at least one or more of the signs and symptoms of thyroid eye disease 51 weeks after initiating treatment; most improved without other thyroid eye disease medications or surgical procedures. Four patients received other therapies (corticosteroids and/or orbital decompression surgery) during follow-up and were counted as improved in the study.
Additionally, 86.2% of treated participants who met proptosis response criteria at week 24 maintained some proptosis improvement compared with baseline at week 72. Of patients with baseline diplopia, 70% had an improvement of at least one grade and 70% of treated participants experienced disease inactivation at 72 weeks, defined as a clinical activity score of 0 or 1.
There were no new safety concerns noted in the analysis.
“This study assessed how many of the trial participants who received teprotumumab experienced any improvement from their baseline measurement in proptosis, diplopia or clinical activity score, collectively and individually over a long period after drug discontinuation,” Dailey said. “All participants experienced some improvement in one or more of the outcomes and 70% demonstrated disease inactivation, as measured by inflammation score of 0 or 1 point at the end of the follow-up.”
Dailey said the latest data should give hope to patients with debilitating symptoms of thyroid eye disease.
“It is encouraging to see teprotumumab improved signs and symptoms up to 1 year after the last dose, especially improvements in the more difficult problems to manage nonsurgically, like proptosis and diplopia,” Dailey said.
Dailey said additional data from the OPTIC-X trial will be presented at a future medical congress.
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Douglas RS, et al. Assessing long-term response to teprotumumab in thyroid eye disease. Presented at: AMCP Nexus 2020 Virtual Meeting; Oct. 19-23, 2020.
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