An independent advisory committee for the US Food and Drug Administration (FDA) today unanimously recommended the injectable drug ocriplasmin from the Belgium-based company ThromboGenics as an effective treatment for the age-related eye disorder known as symptomatic vitreomacular adhesion (VMA).
Currently, individuals diagnosed with VMA are offered either surgery or a ‘wait-and-see’ approach as their only options. “It’s always good to have a medication on the market [for eyes] that is injectable because if it works, then the patient avoids surgery,” Mayo Clinic retinal surgeon Sophie Bakri told Nature Medicine during a phone interview between scheduled surgeries in Rochester, Minnesota. Bakri was not involved in ThromboGenics’ clinical trials, but she has kept an eye on ocriplasmin’s progress.
VMA usually occurs in older adults—the average age in the clinical trials was 70 years—when the colorless jelly between the lens and the retina begins to shrink and forms an unusually strong adhesion to the retina. Over time, that pulling force can distort vision, causing straight lines to appear wavy, general blurriness and possibly a macular hole, resulting in complete loss of vision in the eye’s center. Ocriplasmin works by breaking apart the protein structure that attaches to the surface of the retina.
Maureen Kearny, a patient with VMA who participated in a phase 3 clinical trial site in Lawrenceville, New Jersey, told panel members during the public comment portion of the meeting that the next day after receiving her injection, “I was able to read the newspaper because the print was no longer distorted.” Kearny was one of the estimated 500,000 people in the US and EU combined who suffer from VMA, according to estimates from ThromboGenics.
But despite the mostly positive reception, advisory panel members did mention points of concern. One of those top worries was voiced by Wiley Chambers, director of transplant and ophthalmology products in the Center for Drug Evaluation and Research of the FDA in Rockville, Maryland. “After accounting for all patients whose vision decreased for reasons we understand, we still have a group of patients who experienced a 15-letter loss [in vision measurement] some time during the trial for which there is no immediate explanation,” Chambers said.
During phase 3 clinical trials for its drug, ThromboGenics also tested how a standard dose—125 micrograms administered only once—affects macular holes that can result from the disease. On this secondary study endpoint, the advisory committee expressed doubt. However, the panel decided no further studies would be necessary prior to the FDA’s final decision on the drug for the main indication of VMA, slated for 17 October.
While the FDA usually follows the advice of its advisory panels, it is not bound by those endorsements. Ocriplasmin, which may carry the trade name Jetrea, has also applied for approval in Europe. So the team at ThromboGenics is holding its breath on two continents for the moment.
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