The first ever drug to treat an acute form of dry age-related macular degeneration (AMD) has been approved in the United States.
Pegcetacoplan, also known as Syfovre, has been cleared for use in patients by the Food and Drug Administration (FDA).
Until now there has been no treatment for the severe form of dry AMD called geographic atrophy, a leading cause of blindness typically affecting the over-60s.
Geographic atrophy – seen as part of late-stage AMD, an eye disease that can blur the central vision – is a complex ailment that researchers have spent decades trying to address. Pegcetacoplan’s approval is based on clinical trials which have shown the drug manufactured by Apellis Pharmaceuticals, reduced the condition’s rate of progression in its later-stage.
Whilst not a cure for geographic atrophy, pegcetacoplan’s approval in the US offers a glimmer of hope for the treatment of the devastating disease affecting five million people globally, and could open the door to the drug’s approval in other countries.
A marketing authorisation application for pegcetacoplan is under review by the European Medicines Agency with a decision expected in early 2024. In addition, a marketing application has been submitted to Health Canada, and the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK is currently reviewing the data and evidence on the risks and benefits of the treatment.
Geographic atrophy is a progressive and irreversible form of AMD caused by the growth of lesions, which destroy the retinal cells responsible for sight.
On average, it takes only 2.5 years for geographic atrophy lesions to start impacting the fovea, which is responsible for central vision.
Around 18 million people worldwide are expected to be affected by 2040.
Anjali Shah, MD, an ophthalmologist at Michigan Medicine, the University of Michigan’s academic medical centre, says it is important to note that pegcetacoplan doesn’t reverse or stop progression of geographic atrophy, but may delay further vision loss.
He said: “Based on clinical trials, we estimate that the drug slows the rate of progression by one to three months each year. In other words, if the disease was going to progress to a certain stage of severity in 24 months, it would take 27-30 months while on the drug.”
Dr Shah added: “It’s unclear how long patients need to be on the medication and it’s likely that the duration of treatment will be different for every person, but most people will need to continue treatment for several years. There are currently no drops or pills to treat dry AMD.”
The drug is designed to be injected into the eye every four to eight weeks and according to Dr Shah the clinical trials suggest there’s about a 10% risk of developing wet macular degeneration in the first year of treatment. Wet macular degeneration is a long-lasting eye disorder that causes blurred vision or a blind spot in the central vision. It can be treated, but it would require different injections in the eye.
Injecting medication directly into the eye brings risks, however, the biggest of which is contracting an infection which could cause permanent vision loss. 
Anyone with geographic atrophy can get the injection, but it may only be an ideal treatment for a select group of patients, said Dr Shah.
“In the clinical trials, there was no improvement in vision in the first two years after starting treatment. The studies suggest that there may be some visual benefit over time, but there is currently no data to confirm that,” he explained.
Geraldine Hoad, research manager of the UK-based Macular Society, has described the US development as a “huge milestone” for patients living with dry AMD. But while welcoming the announcement, she added it remains to be seen how much benefit the treatment will provide to patients.
“We hope to have a clearer picture over the coming months and more hope to offer patients diagnosed with this devastating condition,” she said.
Pegcetacoplan is not the only hope for dry AMD sufferers. Iveric Bio based in New Jersey has submitted a New Drug Application (NDA) to the US FDA for Zimura to treat dry AMD. The innovative American biopharmaceutical company expects to hear back in August 2023.
Zimura acts as a blocker of the C5 protein, which is thought to play a key role in the advancement of the condition.
Scientists have reported that the drug decreases the activity which leads to degeneration of retinal cells and can slow the progression of late-stage dry AMD.
AMD falls into two categories – dry and wet – and together affects around 200 million people globally. A person can have either or both types, and dry AMD can suddenly change into the wet kind.
The disease affects the central vision and is the leading cause of irreversible sight loss in older adults.
A clue to those likely to be affected lies in the name, with AMD most commonly seen in people in their 50s and 60s.
While sufferers rarely go blind from AMD, everyday activities like reading, driving, recognising faces, using a computer, preparing meals, watching TV, cleaning and, in some cases, self-care, can become progressively more difficult.
According to the British Medical Journal, there is evidence that those with AMD suffer from higher rates of depression than among community dwelling elderly.
AMD’s exact causes are not known. But it has been linked to smoking, being overweight, eating a diet high in saturated fat, hypertension, and a family history of the disease.
It can often initially go unnoticed by sufferers and will only be picked up during a routine eye test.
Until now there has been no treatment for dry AMD. But regular eye injections and a light treatment called photodynamic therapy can help prevent wet AMD from becoming worse.
