First-ever drug for Stargardt shows promise

The first ever treatment for Stargardt disease has been shown to slow the progression of retinal damage by 36% in a major clinical trial.

Professor Michel Michaelides, consultant ophthalmologist at Moorfields Eye Hospital and professor at University College London's Institute of Ophthalmology, shared the latest progress in our most recent My Macular and Me webinar as he revealed the results ‘exceeded expectations’.

“36% slowing of atrophy progression is genuinely incredible,” he said.

How does the treatment work?

At the webinar, professor Michaelides explained how the orally administered tablet, known as tinlarebant, works by reducing the formation of harmful substances that accumulate in the retina and contribute to its deterioration.

The Phase 3 DRAGON trial involved 104 patients aged between 12 and 20, who received either the medication or a placebo over a period of two years.

Researchers found that retinal damage progressed 36% more slowly in patients taking tinlarebant, which taken once daily, compared with those receiving the placebo.

However, despite the encouraging results, the trial did not demonstrate that the treatment helped patients retain better vision over the two-year period.

Professor Michaelides explained that this was not unexpected, as “the rate of decline on the vision chart is incredibly slow.”

During the webinar's question and answer session, he explained how visual acuity tests were not always an accurate way to assess people’s functional vision and said he believed slowing retinal damage could help patients retain useful vision for longer.

He also suggested that, if the drug is approved, diagnosing and treating Stargardt disease before symptoms develop could become increasingly important.

He said: “If you're going to take a medication that's about slowing progression, well, if you start it before you've had any progression, you're definitely getting most bang for your buck.”

He suggested that the availability of a treatment could eventually lead to Stargardt disease being included in newborn genetic screening in the UK.

“Once there's a therapy available, it will be appropriate to do genetic testing even before patients have symptoms,” professor Michaelides said.

But, while he said the results were exciting, he highlighted that the drug would not stop the disease altogether.

He added: “Of course, it's slowing progression, it's not stopping progression, so it's not that, one will retain vision forever,” he said.

Who could benefit from the treatment?

While the DRAGON trial focused on younger patients, Professor Michaelides said he believed the treatment could eventually benefit a much wider age group, but he said it was unlikely to help those in the very advanced stages of the condition.

“I believe for the majority of people who have Stargardt disease, this will be relevant to them,” he said.

Further studies are planned with children aged between three and 11 whose condition has been confirmed through genetic testing.

This research will focus primarily on the safety of the medication in younger children, while also investigating whether it could slow the progression of the disease.

The treatment was generally well tolerated during the DRAGON trial, although around a third of participants experienced difficulties seeing in the dark or adjusting to changes in lighting. Some also reported altered colour vision or flashing lights.

Professor Michaelides acknowledged that side effects could become more significant when the treatment is used outside of a clinical trial.

“There is no doubt when it gets into the real world there may be more people who find those side effects troublesome. I fully accept that may well be the case,” he said.

But, as other therapies become available professor Michaelides was confident that  patients who experience side effects would soon have alternative options.

What happens next?

Pharmaceutical company Belite Bio announced in October that it had submitted an application for tinlarebant to be approved in the UK, alongside applications in Singapore and Switzerland.

If approved it would become the first authorised treatment for Stargardt disease in the UK, although a further assessment of its cost-effectiveness would be needed before it could be made available on the NHS.

The medication is also under regulatory review in the US and Japan, with the US Food and Drug Administration expected to make a decision by 12 February 2027.

The drug is also being trialled for geographic atrophy, otherwise known as late-stage dry AMD, through a separate phase three PHOENIX trial.