In their review of Aerie Pharmaceuticals' product candidate Rhopressa (netarsudil ophthalmic solution) 0.02%, the members of Dermatologic and Ophthalmic Drugs Advisory Committee of the FDA provided a favorable vote. On the two points that the FDA Advisory Committee members considered, they responded as follows:
1. Do the clinical trials support the efficacy of netarsudil ophthalmic solution for reducing elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension?
Results: (10-0) There were ten yes votes and zero no votes.
2. Does the efficacy of netarsudil ophthalmic solution, demonstrated in the clinical trials, outweigh the safety risks identified for the drug product?
Results: (9-1) There were nine yes votes and one no vote.
In addition, there was general discussion on suggestions regarding the draft product labeling proposed by the FDA, which will ultimately be determined based on follow-on discussions between Aerie and the FDA.
The goal date for the FDA to take action under the Prescription Drug User Fee Act (PDUFA) is February 28, 2018. The FDA is not bound by the Advisory Committee's guidance, but takes its advice into consideration when reviewing investigational medicines.
“We are delighted with the outcome of the FDA’s Advisory Committee, and we now have our sights set on the February 28 PDUFA date. We believe there are significant unmet needs in the treatment of patients with open-angle glaucoma or ocular hypertension, and we are very excited about the prospects for Rhopressa,” Vicente Anido, Jr., PhD, Chairman and Chief Executive Officer at Aerie, said in a company news release.
Rhopressa is a novel eye drop that the company believes, if approved, would become the only once-daily product available that, based on Aerie’s preclinical and clinical studies to date, specifically targets the trabecular meshwork. Preclinical and clinical studies have also demonstrated that Rhopressa lowers episcleral venous pressure, which contributes approximately half of IOP in healthy subjects. Further, based on Aerie’s preclinical studies, Rhopressa may provide an additional mechanism that reduces fluid production in the eye and therefore lowers IOP. Biochemically, the active ingredient in Rhopressa, netarsudil, has been shown in Aerie studies to inhibit both Rho kinase (ROCK) and norepinephrine transporter (NET). Recent preclinical studies have also shown that Rhopressa may have disease-modifying properties, including an anti-fibrotic effect of netarsudil on trabecular meshwork cells and the potential to increase perfusion of the trabecular meshwork.
The results of two phase 3 registration trials (Rocket 2 and Rocket 1) for Rhopressa were included in the NDA (new drug application) submission to the FDA in February 2017. There were two additional phase 3 registration trials for Rhopressa, named Rocket 3 and Rocket 4. Rocket 3 was a small 12-month safety-only study in Canada that was not necessary for the NDA submission and for which enrollment has been discontinued. Rocket 4, which was successfully completed in April 2017, was designed to provide adequate 6-month safety data for regulatory filing purposes in Europe, and was also not necessary for the NDA submission. The 90-day efficacy results from Rocket 4 and Mercury 1, the initial phase 3 registration trial for Aerie product candidate Roclatan (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%, were also included in the Rhopressa NDA submission as supportive.