FDA

The FDA’s insider view on Aerie’s glaucoma drug: Yes on efficacy, but…

Aerie CEO Vicente Anido

The FDA had some encouraging words for Aerie’s new glaucoma drug Rhopressa, but there’s a significant caveat that might come back to haunt the biotech as it hunts an approval.

In the internal review offered up by the agency two days ahead of the mission-critical panel discussion and vote, FDA insiders give the data a thumbs up on efficacy, but note that the comparator therapy was better than the drug in the more severely afflicted patients included in their Phase III studies.

Looking over the data from three Phase III studies, the FDA reviewers stated:

(W)e concur with the Applicant’s overall efficacy conclusion that netarsudil 0.02% once-daily (QD) (also referred to as netarsudil QD) is efficacious for the reduction of elevated intraocular pressure (IOP). However, netarsudil QD is less efficacious compared to the comparator, timolol ophthalmic solution 0.5% twice daily (BID), for subjects with higher maximum baseline IOP (≥ 25 mmHg).

Investors didn’t seem to mind the issue, at least at first glance. Aerie’s stock shot up 14% as the review made the rounds.

The FDA’s note on efficacy immediately drew the attention of Mizuho’s Difei Yang. But Yang says the suggested label doesn’t restrict an OK to mild to moderate patients, paving the way to widespread use. Yang notes:

We view FDA’s modifications to Aerie’s proposed language to be very minor. Additionally, we are encouraged by the language used in the indication and usage section where there is no mention of Rhopressa being used for mild to moderate glaucoma patients only. The current proposed label reads as follows, “RHOPRESSA (netarsudil ophthalmic solution) 0.02% is a Rho kinase inhibitor indicated for the reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension.” We believe this to be a best case scenario for Rhopressa.

Aerie, though, still has to clear a bar on the manufacturing side. Its drug is manufactured at the Bausch + Lomb manufacturing facility in Tampa, which recently failed to measure up to federal standards for the second time. The Valeant company, though, followed up with a vow that it will soon have its house in order, encouraging investors as short sellers hovered about in hopes of a setback.

A bullish Aerie CEO Vicente Anido has already touted Rhopressa as a likely blockbuster able to earn more than $1 billion a year, even though the biotech had to get the FDA’s permission to change the endpoint for its second late-stage study of Rhopressa in order to avoid back-to-back failures.

Aerie is also two for two on its pivotal Phase III studies for Roclatan, a combo drug that combines the generic latanoprost to Rhopressa.


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