Two late-stage trials of Novartis and partner ThromboGenics' injectable eye drug ocriplasmin suggest that it could be an effective treatment for patients with vitreomacular adhesion (VMA), a condition that affects than 300,000 patients in Europe.
VMA, including vitreomacular traction (VMT) and macular holes, is an age-related progressive, debilitating eye disease that can eventually cause visual distortion, loss in visual acuity, and central blindness.
In patients with the condition, the jelly-like substance in the center of the eye (vitreous) sticks in an abnormally strong way and pulls on the retina, potentially leading to the development of macular holes.
To date, the only option for patients with the condition is to undergo surgery. But data from the studies - involving 652 patients and published in the New England Journal of Medicine - indicate that patients could be a step closer to getting access to a pharmaceutical therapy.
Ocriplasmin - a recombinant, truncated form of human protein (plasmin) - works by dissolving the proteins that link the vitreous to the macula at the center of the retina, thus relieving the traction detaching the vitreous from the retina.
The late-stage studies showed that, 28 days after a single administration of the drug, resolution of VMA was observed in 26.5% of patients compared to 10.1% in the placebo group, and this statistically significant difference was maintained through six months of observation, Novartis said.
"Results from the Phase III program with ocriplasmin are significant as they demonstrate the potential for using an enzymatic approach to resolve vitreomacular adhesion," noted Prof Dr Peter Stalmans, Department of Ophthalmology, University Hospitals, Leuven, Belgium.
"This represents a real advance for patients living with vitreomacular adhesion who currently only have the option of surgery at a later stage of the disease," he added.
Novartis' ophthalmic unit Alcon snapped up the non-US rights to the drug for the treatment of symptomatic vitreomacular adhesion from Belgian drugmaker ThromboGenics in a deal worth 375 million euros back in March this year.
Ocriplasmin is currently under review with the European Medicines Agency, and, in July, the US Food and Drug Administration's Dermatologic and Ophthalmic Drugs Advisory Committee backed the drug for the treatment of symptomatic VMA, voting 10 to 0 that its benefits outweigh the potential risks in this setting.