Amgen seeks European approval for teprotumumab in thyroid eye disease

Amgen (NASDAQ: AMGN), a global biotechnology leader, has announced its plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for teprotumumab. This treatment, a fully human monoclonal antibody and targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R), is aimed at adults suffering from moderate to severe Thyroid Eye Disease (TED), a serious and potentially vision-threatening autoimmune disease.

Potential First Approved Medicine for TED in the EU

Teprotumumab, if approved, would be the first and only medication authorized for TED in the European Union. This significant development offers hope to patients who currently have limited treatment options, which are often restricted to steroids and invasive surgeries. These methods not only carry significant risks but also fail to address the underlying causes of the disease.

Clinical Studies and Expected Impact

The MAA submission is bolstered by robust clinical evidence from multiple studies, including a Phase 2 study, the confirmatory Phase 3 OPTIC trial, a Phase 4 study, and a Phase 3 clinical trial in Japan. Collectively, these studies, involving 287 patients, demonstrated statistically significant and clinically meaningful improvements in TED symptoms, such as proptosis (eye bulging) and diplopia (double vision). Remarkably, clinical improvements were observed as early as six weeks, with ongoing benefits seen throughout the 24-week treatment period. Teprotumumab has been well-received in terms of safety and efficacy.

Expert and Community Reactions

Jay Bradner, executive vice president of Research and Development and chief scientific officer at Amgen, emphasized the transformative potential of teprotumumab: “This disease is currently managed with steroids and invasive surgeries, both of which carry their own set of risks. Having access to a non-surgical option like teprotumumab that not only treats the signs and symptoms but also targets the underlying cause of the disease represents a major advance for patients.”

Dr. Mario Salvi, MD, founder and head of the Graves Orbitopathy Center at Fondazione IRCCS Ca’ Granda, Milano, highlighted the dire need for such treatments: “People living with Thyroid Eye Disease, a painful and potentially sight-threatening disease, have no approved treatment options in Europe at this time.”

Global Expansion and Regulatory Progress

Already approved for TED in the U.S., Brazil, and the Kingdom of Saudi Arabia under the brand name TEPEZZA, teprotumumab is administered through an intravenous infusion once every three weeks for a total of eight infusions over approximately five months. Beyond Europe, Amgen has also pursued marketing authorization in Great Britain, Canada, and Australia, with an ongoing review in Japan.

As Amgen prepares for the European market launch, teprotumumab stands poised to significantly improve the quality of life for patients suffering from Thyroid Eye Disease, offering a promising new treatment paradigm that combines efficacy with a strong safety profile.