Novartis and partner ThromboGenics have reported positive phase III data on Jetrea, a new drug which could become the first approved treatment for the eye disorder vitreomacular adhesion (VMA).
VMA is a progressive, age-related condition that can lead to compromised vision and eventually central blindness, and affects around 300,000 people in Europe.
In patients with VMA the jelly-like substance in the centre of the eyeball or vitreous adheres too strongly to the retina at the rear of the eye, causing a 'pulling' effect that distorts vision. In time, this pulling can lead to the formation of holes in the macula, the central region of the retina, leading to loss of vision entirely in that area.
At the moment the only treatment for advanced VMA is a vitrectomy, a surgical procedure which involves the removal of the vitreous, so a pharmacological intervention which removes the need for such an invasive procedure would be welcomed by patients and their doctors.
Jetrea (ocriplasmin) is a recombinant form of human plasmin that dissolves the protein links that form between the vitreous and macula, separating them non-surgically. The drug is given via a single injection into the eyeball.
Two phase III trials of Jetrea have now been published in the New England Journal of Medicine (NEJM) and show that the drug resolved 27 per cent of VMAs within 28 days, compared to 10 per cent in the placebo group.
By the end of a six-month follow-up period, 18 per cent of the Jetrea group needed surgery, compared to 27 per cent of the placebo group.
Jetrea has already been filed for approval as a treatment for VMA in the US and EU on the strength of the data, and in July an FDA advisory committee voted unanimously in favour of the drug's approval in the US.
The US regulator is due to deliver its verdict on the application in October, and Novartis and ThromboGenics have said they are also hopeful of EU approval by the end of the year.
Belgium-based ThromboGenics licensed non-US rights for Jetrea to Alcon in March in return for $99m in upfront fees and up to $400m in milestone payments.
Analysts have predicted that peak sales of the drug could come in the $600m-$1bn range, with the potential to increase further the drug secures additional indications such as diabetic retinopathy (DR), age-related macular degeneration (AMD) and stroke.