Amgen's TEPEZZA Approved as First Targeted Treatment for Moderate-to-Severe Thyroid Eye Disease in UK

In an important development for healthcare in the UK, Amgen has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for TEPEZZA® (teprotumumab). This marks a significant milestone as it becomes the first therapy specifically approved for treating adults with moderate-to-severe Thyroid Eye Disease (TED).

Thyroid Eye Disease is an autoimmune condition affecting approximately 50,000 individuals in the UK. It is a progressive disorder that can potentially threaten vision and leads to symptoms such as eye bulging, double vision, pain, redness, and swelling around the eyes. The disease can have a profound impact on patients' quality of life, leading to anxiety, depression, and concerns about appearance, which may affect their social interactions and professional lives.

Dr. Tony Patrikios, Executive Medical Director of Amgen UK & Ireland, highlighted the significance of this authorisation, noting that TED can severely impact patients by affecting their vision and causing discomfort and pain, which in turn can lead to a loss of self-confidence. The approval of teprotumumab offers a novel treatment option and underscores Amgen’s dedication to aiding patients with serious and previously underserved conditions.

The UK authorisation of teprotumumab is backed by various clinical studies, including the pivotal Phase 3 OPTIC trial. This large-scale study involved 83 participants and demonstrated a significant improvement in reducing eye bulging, a primary symptom of TED. Results showed that 83% of patients treated with teprotumumab experienced at least a 2mm reduction in proptosis after 24 weeks, compared to only 10% in the placebo group.

Dr. Jimmy Uddin, a Consultant Ophthalmologist and Oculoplastic Surgeon, pointed out the challenges faced by TED patients in obtaining prompt diagnosis and treatment, as the condition is often misdiagnosed as more common eye conditions. Typically, existing treatments address only general inflammation without targeting the underlying causes of TED. Teprotumumab represents a new option for patients, specifically addressing the drivers of the disease.

The safety profile of teprotumumab has been extensively reviewed, with data drawn from multiple clinical trials. Common side effects include muscle spasms, diarrhea, hearing impairment, alopecia, hyperglycemia, fatigue, nausea, and headache. Despite these potential side effects, the introduction of teprotumumab provides a promising alternative for those with limited options.

Beyond the regulatory approval, Amgen plans to collaborate with the National Institute for Health and Care Excellence (NICE) to facilitate reimbursement for eligible patients. This initiative demonstrates the company's commitment to ensuring that patients have access to this innovative treatment.

Thyroid Eye Disease primarily affects individuals with Graves' disease but stands as a distinct disorder caused by an autoimmune reaction. It involves antibodies that activate signaling pathways leading to inflammation and tissue expansion around the eyes. Early detection and treatment are crucial, as the condition can cause irreversible damage, including blindness.

Teprotumumab, a monoclonal antibody, targets the insulin-like growth factor-1 receptor (IGF-1R), an essential component in the disease's pathogenesis. By blocking this receptor, teprotumumab helps to alleviate symptoms such as eye bulging, offering relief and improved quality of life for those affected by TED.

Amgen's role as a biotechnology pioneer, focusing on innovation and patient care, continues to be evident with this new treatment option. Their ongoing work in the UK contributes to advancements in healthcare, addressing diverse medical needs with cutting-edge solutions.