TL;DR
The recent decision by the Medicines and Healthcare products Regulatory Agency (MHRA) to decline approval for Syfovre (pegcetacoplan) as a treatment for geographic atrophy (GA) has significant implications for those affected by this advanced form of age-related macular degeneration (AMD).
This ruling has sparked concern among patients, healthcare professionals, and advocacy groups who had hoped for a breakthrough in treating this progressive and debilitating condition. Despite the setback, the rejection sheds light on the current state of treatment options and the need for further research in tackling GA.
What is geographic atrophy?
Geographic atrophy is an advanced and irreversible form of dry AMD that results in the gradual breakdown of retinal cells, leading to severe vision loss.
Unlike wet AMD, which can be managed with anti-VEGF injections, there is currently no approved treatment available for GA. As a result, individuals diagnosed with the condition often experience a decline in their ability to perform daily tasks such as reading, driving, and recognising faces.
Statistics indicate that GA is a significant contributor to vision impairment and blindness. The condition is responsible for approximately 26% of cases of legal blindness, making it a major public health concern.
Moreover, the prevalence of GA increases with age, affecting around 1.3% of individuals aged 65 and older, with the rate rising to 6.7% in those aged 80 and above.
Given that the ageing population is steadily growing, these figures are expected to increase in the coming years, further underscoring the urgent need for an effective treatment.
Why was Syfovre rejected?
Syfovre, developed by Apellis Pharmaceuticals, was intended to be the first available treatment for GA. The drug works by targeting complement proteins, which play a role in the immune system and are believed to contribute to retinal cell degeneration in GA. Clinical trials showed that Syfovre could slow the progression of the disease, providing a potential means of delaying the loss of vision.
However, the MHRA determined that, despite Syfovre’s ability to slow retinal deterioration, the evidence provided did not sufficiently demonstrate improvements in patients’ overall visual function and quality of life.
This means that, while the drug showed promise in delaying the worsening of GA, it was not proven to make a meaningful difference in how well patients could see and function on a daily basis.
This decision is consistent with the European Medicines Agency’s earlier recommendation to deny marketing authorisation for Syfovre across the European Union. The EMA raised similar concerns regarding the clinical significance of the drug’s benefits, citing that the improvements observed in trials did not translate into measurable gains in visual performance for patients.
The Macular Society’s response and future research efforts
Despite the setback, the Macular Society remains committed to supporting those affected by GA and continuing to push for better treatment options. Dr Peter Bloomfield, director of research at the Macular Society, acknowledged the disappointment that many patients may feel following the MHRA’s decision. However, he emphasised that this rejection does not mark the end of the road for potential GA treatments.
Several other experimental treatments for GA are currently in various stages of research and development. Many of these treatments focus on targeting different pathways involved in retinal degeneration, with some showing promising early results.
Ongoing clinical trials in the UK and internationally are exploring new therapies, including gene therapy and regenerative medicine approaches, to halt or even reverse the damage caused by GA. The Macular Society continues to advocate for increased investment in research and is actively funding studies aimed at understanding the underlying causes of GA.
Key takeaways
The MHRA’s decision to reject Syfovre is a significant moment in the ongoing search for an effective treatment for geographic atrophy. While it represents a hurdle for patients and researchers alike, it also highlights the importance of continued efforts to develop new therapies that not only slow disease progression but also improve visual function and quality of life.
For the thousands of people in the UK affected by GA, the journey towards an approved treatment continues. In the meantime, ongoing research, clinical trials, and advocacy efforts provide hope that better options will become available in the future. Until then, those living with GA can benefit from the support and resources offered by organisations like the Macular Society as they navigate the challenges of vision loss.
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