EMA CHMP adopts positive opinion for teprotumumab (Tepezza, Amgen) marketing authorisation application

In phase 3 trials, teprotumumab was successful in reducing instances of proptosis. Image credit: ©Pickoloh – stock.adobe.com

During its April meeting, the European Medicines Agency (EMA) announced that the Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for the first severe thyroid eye disease therapy. Teprotumumab (Tepezza, Amgen) received a positive recommendation in response to its marketing authorisation application (MAA). According to a summary from the EMA, it will be available as a 500 mg powder concentrate for solution for infusion intended for the treatment of moderate to severe thyroid eye disease (TED), also known as Grave disease, in adult patients.¹ A subsequent news release from the EMA noted that teprotumumab is the first treatment available in Europe for the treatment of severe TED.²

Teprotumumab is a monoclonal antibody which binds to the insulin-like growth factor 1 receptor, inhibiting its activity. By blocking the autoimmune activation of orbital fibroblasts, the therapeutic can stop the progression of TED.¹ Benefits of the product were demonstrated over the course of three randomised, placebo-controlled phase 3 trials, which included a total of 225 patients with active TED. A separate phase 3 trial, focused on 62 patients with chronic TED, demonstrated reduction in protrusion of the eyeball from the eye socket (proptosis).¹

“After 24 weeks, patients treated with Tepezza experienced a significant reduction (-2 to -2.3 mm) in protrusion of the eyeball from the eye socket (proptosis) and in the Clinical Activity Score (CAS), a standard tool to evaluate inflammatory signs and symptoms of TED, compared to patients treated with placebo,” the EMA noted.² “The reduction in proptosis was smaller (-1.5mm) in patients with chronic TED.”

The most common adverse effects experienced by patients treated with teprotumumab included muscle spasms, hyperglycaemia, fatigue, nausea, headache, diarrhoea and alopecia. Additionally, evidence from clinical trials indicates that teprotumumab may cause severe, possibly permanent, hearing impairment. Teprotumumab may also pose risks for foetal development, according to pre-clinical data. According to the EMA, additional risk management measures will be implemented to mitigate these risks.

The European Commission will issue a decision on whether to grant an EU-wide marketing authorisation. The EMA notes that the Commission typically issues an opinion within 67 days from the adoption of the CHMP opinion; under these parameters, a final decision is anticipated to be announced by 1 July.¹

References

1. Summary of opinion (initial authorisation), Tepezza teprotumumab. European Medicines Agency Committee for Medicinal Products for Human Use. Published April 25, 2025. Accessed April 30, 2025. https://www.ema.europa.eu/en/documents/smop-initial/chmp-summary-positive-opinion-tepezza_en.pdf

2. First treatment against severe thyroid eye disease. News release. European Medicines Agency Committee for Medicinal Products for Human Use. Published April 25, 2025. Accessed April 30, 2025. https://www.ema.europa.eu/en/news/first-treatment-against-severe-thyroid-eye-disease