Aerie Pharmaceuticals has received good news going into its FDA advisory committee meeting. The FDA agreed with the efficacy conclusion Aerie reached to support its bid for approval of glaucoma and ocular hypertension hopeful Rhopressa.
A panel of experts convened by the FDA will take a deep dive into the data and opine on its merits on Friday. The meeting could still throw Aerie a curveball and dent the chances of approval for Rhopressa, which suffered setbacks in the clinic on its way to the FDA filing. But for now investors are celebrating clearing the first hurdle without suffering critical blows.
In a briefing document put together to support the experts’ discussion, the FDA said it concurs with Aerie’s “overall efficacy conclusion that netarsudil 0.02% once-daily is efficacious for the reduction of elevated intraocular pressure (IOP).”
That is a boost for a drug that was on the ropes in 2015 after coming up short in a phase 3 trial. The FDA threw Aerie a lifeline back then by allowing a change to the primary endpoint used in its second phase 3. Aerie duly cleared the new bar to success but would have missed if the FDA had made it stick to the original endpoint.
The briefing document touches on the weaknesses in the data to support Rhopressa, noting that twice-daily doses of timolol are better for patients with baseline IOPs of 25 mmHg and above.
While that may limit use of Rhopressa, it looks unlikely to scuttle the filing altogether. Aerie may face a tougher time when the talk turns to safety.
In one trial, 28% of participants who received daily doses of Rhopressa dropped out because of adverse events. More than half of people who received the drug twice a day dropped out because of adverse events. Two-fifths of patients in that arm dropped out within three months. The adverse event-related dropout rate in the timolol arm was 6%.
Those figures, particularly the dropout rate after three months, prompted the FDA to state that twice-daily doses of Rhopressa were not well tolerated. However, given Aerie’s draft label proposes once-daily dosing, this may have limited effect on the application.
The FDA included Aerie’s draft label and its edits to date in the briefing document. The agency is proposing to cull some lines proposed by Aerie, such as those stating that most cases of conjunctival hyperemia were mild in nature and the IOP-lowering effect increases gradually after initial dosing. The FDA also proposed changing the proportion of patients who suffered conjunctival hyperemia from 54% to 57%. But overall most of the text put forward by Aerie has escaped unscathed.
A panel of experts will discuss these issues on Friday and vote on two questions that ask whether the clinical trials support the efficacy of Rhopressa and whether its efficacy outweighs the risks.