Vitreomacular adhesion is an age-related progressive, debilitating eye disease, often leading to blindness; standard of care is "watchful waiting" or surgery
Phase III results show that ocriplasmin significantly (p<0.001) resolved vitreomacular traction and closed macular holes compared to placebo
For the majority of patients with resolution of vitreomacular adhesion after ocriplasmin administration, the resolution was achieved within seven days
Alcon, the eye care division of Novartis, acquired the commercialization rights to ocriplasmin outside the United States from ThromboGenics
Basel, August 15, 2012 - Novartis announced today, that the New England Journal of Medicine (NEJM) published the results of two Phase III studies of the investigational eye treatment ocriplasmin. The studies including 652 patients found that ocriplasmin, significantly resolved vitreomacular traction and closed macular holes compared to placebo in patients with vitreomacular adhesion. Vitreomacular adhesion (VMA), including vitreomacular traction (VMT) and macular holes, is an age-related progressive, debilitating eye disease that may lead to visual distortion, loss in visual acuity and central blindness. More than 300,000 patients suffer from this disease in Europe alone.
"Results from the Phase III program with ocriplasmin are significant as they demonstrate the potential for using an enzymatic approach to resolve vitreomacular adhesion. 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," said Peter Stalmans, Department of Ophthalmology, University Hospitals, Leuven, Belgium. "The majority of patients who achieved resolution of their vitreomacular adhesion after a single intravitreal injection of ocriplasmin showed this positive outcome within the first seven days."
The results of these Phase III trials, which both met their primary endpoints, demonstrate the efficacy of ocriplasmin which, if approved, could be the first pharmaceutical therapy to treat patients with vitreomacular adhesion. The ocriplasmin Phase III program was conducted by Alcon’s partner ThromboGenics, NV.
In patients with VMA, the vitreous (jelly-like substance in the center of the eye that is surrounded by a membrane) adheres in an abnormally strong way to the retina (light-sensitive layer of tissue at the back of the eye), which can lead to traction (’pulling’) on the retina, causing symptoms including impaired vision. Further unresolved traction may lead to the development of macular holes and central blindness . The only treatment option for progressed VMT and macular holes is a surgical procedure called vitrectomy (removal of the vitreous from the eyeball). In cases where VMT and macular holes are not severe enough to require surgery, the standard of care is "watchful waiting" .
Ocriplasmin, a recombinant, truncated form of human protein (plasmin), works by dissolving the proteins that link the vitreous to the macula (center of the retina), relieving the traction and resulting in posterior detachment of the vitreous from the retina.
Alcon, a division of Novartis, acquired the commercialization rights to ocriplasmin outside the United States from the Belgian biopharmaceutical company ThromboGenics, who retains the rights to commercialize the drug in the US. As the global leader in eye care, Alcon is dedicated to bringing innovative eye treatments to patients with unmet medical needs.
"The data from these studies provide a basis for worldwide regulatory filings," said Kevin Buehler, Division Head, Alcon. "With Alcon’s extensive commercial capabilities, geographic footprint and strong relationships with retinal specialists and ophthalmologists, we are well positioned to bring this innovative treatment to patients outside the United States, once it is approved."
Ocriplasmin is currently under review with the European Medicines Agency (EMA) and was accepted for review by the EMA in October 2011. In July 2012, the US Food and Drug Administration (FDA) Dermatologic and Ophthalmic Drugs Advisory Committee issued a positive recommendation supporting approval of ocriplasmin in the United States.
About the Studies
Study 006 and Study 007 are two multicenter, randomized, double-blind, placebo-controlled Phase III studies to test the efficacy and safety of a single intravitreal injection of ocriplasmin in patients with VMT and macular holes. One half and one third of the patients enrolled in Study 006 and Study 007, respectively, received placebo; all other patients received a single intravitreal injection of ocriplasmin (125 microgram). The primary endpoint for the studies was the percentage of eyes with nonsurgical resolution of vitreomacular adhesion at day 28, determined by optical coherence tomography (OCT) imaging.
After 28 days, following a single administration of ocriplasmin, resolution of vitreomacular adhesion was observed in 26.5% of patients compared to 10.1% in the placebo group, (p<0.001). This statistically significant difference was maintained through six months of observation. For those patients with resolution of vitreomacular adhesion after ocriplasmin administration, resolution was achieved in the majority of patients within seven days. By the end of the six months observation period, fewer patients required a vitrectomy in the ocriplasmin treated group, compared to placebo (17.7% vs. 26.6% respectively, p=0.02).
In the ocriplasmin group, ocular adverse events occurring in the studied eye were transient and mild in severity, the most common adverse event being self-reported vitreous floaters (in 68.4% of patients compared to 53.5% in the placebo group). In the group treated with ocriplasmin, the incidence of any serious ocular adverse event was 7.7%, compared to 10.7% in the placebo group, (p=0.26).