The US Food and Drug Administration (FDA) yesterday approved ocriplasmin intravitreal injection (Jetrea, ThromboGenics, Inc) for the treatment of symptomatic vitreomacular adhesions (VMAs), the agency announced today.
"Today’s approval represents a significant advancement in treatment for patients with symptomatic VMA," said Edward Cox, MD, MPH, director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research. "Those with this sight-threatening disease now have a nonsurgical treatment option."
The decision follows a July 26 advisory committee meeting at which the committee voted unanimously to recommend approval of ocriplasmin for the treatment of VMAs and also voted 7 to 3 to recommend approval for the treatment of macular holes. Today's FDA announcement does not mention macular holes.
Richard Spaide, MD, a clinical correspondent for the American Academy of Ophthalmology and a retina-macular specialist with Vitreous-Retina-Macula Consultants of New York City, commented on the development for Medscape Medical Newsin an email interview.
"Ocriplasmin offers the opportunity to treat symptomatic vitreomacular adhesion and traction without the need for vitrectomy. There are a number of potential research opportunities to evaluate if release of vitreous adhesions would change the clinical course of a variety of diseases such as diabetic macular edema and choroidal neovascularization, and to evaluate if release of traction influences treatment response," Dr. Spaide said.
K. Bailey Freund, MD, a clinical correspondent for the American Academy of Ophthalmology, also commented on the approval in a telephone interview with Medscape Medical News. Dr. Freund is a retina specialist with Vitreous-Retina-Macula Consultants of New York City and a clinical associate professor of ophthalmology at New York University School of Medicine in New York City.
"I'm not aware of anything that's gone into late-phase clinical trials similar to ocriplasmin," said Dr. Freund.
The advisory committee relied on 2 studies that enrolled a combined total of 652 patients: 464 patients were randomly assigned to receive a single intravitreal injection of ocriplasmin 125 μg, and 188 patients received a placebo injection. Patient assessments were conducted at baseline, on injection day, and 7, 14, 28, 90, and 180 days postinjection. Investigators could recommend vitrectomy at any time if they felt the patient required it.
A total of 26.5% of the patients in the ocriplasmin group met the primary end point of nonsurgical VMA resolution at day 28 compared with 10.1% of those in the placebo group.
The percentage of patients with total posterior vitreous detachment was higher in the ocriplasmin group (13.4%) than in the placebo group (3.7%; odds ratio [OR], 4.27; 95% confidence interval [CI], 1.89 - 11.32; P < .001).
The percentage of eyes with nonsurgical closure of macular hole was higher in the ocriplasmin group (40.6%) than in the placebo group (10.6%; OR, 5.94; 95% CI, 20.9 - 21.01; P < .001).
The percentage of patients who required vitrectomy was lower in the ocriplasmin group (17.7%) than in the placebo group (26.6%; OR, 0.61; 95% CI, 0.40 - 0.94; P = .02).
Overall, the percentage of patients who experienced best-corrected visual acuity improvement of 3 or more lines on an eye chart was higher in the ocriplasmin group (12.3%) than in the placebo group (6.4%; OR, 2.09; 95% CI, 1.08 - 4.41; P = .02).
For patients who did not require vitrectomy, the corresponding values were 9.7% vs 3.7%.
Risk-Benefit Concerns
The incidence of ocular adverse events (conjunctival hemorrhage, vitreous floaters, photopsia, and injection-related pain) was higher in the ocriplasmin group (68.4%) than in the placebo group (53.5%;P < .001).
The incidence of more serious ocular adverse events including retinal tear or detachment was 7.7% in the ocriplasmin group compared with 10.7% in the placebo group (P = .26).
"The majority of patients who receive this drug will not get benefit from the drug, and I think that that needs to be very clearly stated. If the drug is approved, it must be very clearly stated in labeling...even though there is efficacy benefit over risk," voting committee member Lynn Gordon, MD, PhD, a professor of ophthalmology at Jules Stein Eye Institute and associate dean of diversity affairs at David Geffen School of Medicine at the University of California, Los Angeles, said at the July 26 meeting.
"You'll have to be careful counseling patients and say that, 'this is definitely worth a try, and could potentially avoid a much more invasive procedure, but the chances are, based on the results of the trial...you're still going to need surgery,' " Dr. Freund said.
"The eyes that it worked best on were very specific types...eyes without epiretinal membrane, eyes with small holes. You should look at this data, and it's probably not appropriate to inject it on the other types of situations that were studied," Dr. Freund added.
Dr. Spaide said ocriplasmin is likely to be expensive. "The costs related to developing the drug and doing the clinical trials [have] to be covered. The costs of the drug are likely to temper the 'let's give the drug and see what happens' approach for certain diseases," said Dr. Spaide.
He believes insurance companies will approve reimbursement for treatment of VMAs and macular holes, but not for other uses like diabetic macular edema, where it might be helpful. "Insurance companies may hold off on approving the costs of the drug until better data is available. The drug labeling may be restricted to cover only a select list of conditions at first. I think the reimbursement issues are going to be interesting," Dr. Spaide noted.
More information on today's announcement is available on the FDA Web site.
Dr. Freund has disclosed no relevant financial relationships. Dr. Spaide receives royalties from Topcon and is a consultant to Topcon, Optos, and Thrombogenics.
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