The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for Amgen’s Tepezza (teprotumumab) as the first licensed treatment for adults with moderate-to-severe thyroid eye disease (TED).

TED affects around 50,000 people in the UK, causing eye bulging, double vision, pain and swelling, and significantly impacting patients’ quality of life.

TED can lead to anxiety and depression, with patients often struggling with self-confidence and their ability to carry out daily tasks. Teprotumumab, an immunotherapy targeting the insulin-like growth factor-1 receptor (IGF-1R), is the first treatment designed to address the underlying drivers of the disease rather than generalised inflammation.

Dr Tony Patrikios, executive medical director at Amgen UK & Ireland said: “The marketing authorisation for teprotumumab as the first therapy specifically licensed for thyroid eye disease (TED) in the UK marks a step forward for the patient community.”

He added: “TED can negatively affect patients’ lives impacting vision, causing eye pain, making everyday tasks difficult and causing a loss of self-confidence. This authorisation introduces a new alternative treatment option and reinforces Amgen’s commitment to supporting eligible patients with serious, underserved conditions.”

The authorisation is backed by multiple clinical studies, including the phase 3 OPTIC trial, which showed that 83% of patients with active TED experienced a reduction in proptosis (eye bulging) of at least 2mm compared to just 10% in the placebo group.

Amgen is working with the National Institute for Health and Care Excellence (NICE) to secure reimbursement for teprotumumab to ensure access for all eligible patients in the UK.

UCB has announced that its developmental treatment DoxTM, a combination of doxecitine and doxribtimine, has been granted Promising Innovative Medicine (PIM) designation by the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK.

TK2d is a rare genetic disorder caused by a mutation in the TK2 gene. This leads to mitochondrial DNA depletion, resulting in severe and progressive muscle weakness.  Many affected individuals lose mobility, require respiratory support, and need assistance with basic functions such as eating.

The MHRA has granted PIM designation for DoxTM as a treatment for paediatric and adult patients with symptom onset before 12 years. The designation signals that the therapy may qualify for Early Access to Medicines (EAMS), which allows patients with life-threatening or seriously debilitating conditions to access treatments before full regulatory approval.

Professor Robert McFarland, Professor of Paediatric Mitochondrial Medicine & Consultant Paediatric Neurologist, Newcastle University, said: “Mitochondrial diseases are rare and life-limiting, seriously impacting the lives of patients and their families. Tk2D can be difficult to diagnose and is hard to treat. Improving the quality of life for people living with this disease is a real priority and I openly welcome access to all new treatments that may make a difference to the lives of these patients and their loved ones.”

Nadeem Aurangzeb, Head of Rare and Epilepsy, UCB UK & Ireland, added: “TK2d is a debilitating genetic disease, which has a devastating impact on the lives of those living with it as well as their families, friends and loved ones.

“We are delighted that the MHRA has recognised the potential of this developmental treatment to positively impact the lives of those with the condition in the UK.”

UCB has confirmed that it will now apply for EAMS status to provide early patient access to the treatment.