It’s not too often that we see the Food and Drug Administration (FDA) in the US make a decision on a regulatory application ahead of time. If anything, things generally go the other way, with (especially over the last few years) the dreaded three-month delay becoming increasingly prevalent.
This week, however, the agency has bucked this trend and, in doing so, has served to respond (somewhat) to the requests from policymakers in the US to speed up the regulatory and development/approval process.
So, who is involved?
The drug that the agency just approved is called Rhopressa (netarsudil ophthalmic solution) and it is being developed (and now commercialized) by a company called Aerie Pharmaceuticals, Inc. (NASDAQ:AERI). Many reading will likely only be familiar with Aerie but, for those new to the company, it’s a North Carolina, US-based entity that is working to bring a pipeline of treatments to market primarily rooted in conditions and diseases of the eye.
And it is in to this latter category that Rhopressa falls.
The drug is targeting the lowering of elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. Glaucoma is an eye condition where the optic nerve is damaged by the pressure of the fluid inside the eye. An elevated degree of IOP can increase the optic nerve damage and, eventually, can lead to blindness if gone untreated.
So, the development program went relatively well for the company as far as collecting efficacy and safety data was concerned but, leading up to an advisory panel meeting earlier this year, there were some concerns that manufacturing issues may play a role in the FDA’s ultimate decision with regards to the approvability of the asset.
As per the latest decision, however, these concerns have been alleviated.
The FDA has green lighted the drug in its target indication more than two months ahead of its original target PDUFA of February 28.
And what is the market for this sort of asset?
Analysts generally agree that this one could probably make between $500 million and $800 million annually in peak sales. Company management, however, has gone on record to say that it believes this number could rise to $1 billion within five years of approval.
Whichever one is correct, the numbers are still strong for a company that currently holds a market capitalization of $2.14 billion.
So what comes next?
Well, this could be just the beginning of a very strong few months for Aerie. The company has one approval in the bag and is set to submit for a second approval, one based on a combination of the generic latanoprost and the above discussed Rhopressa, called Roclatan, near term. Management expects this application to be with the FDA during the second quarter of next year meaning there is the potential for a final decision before the end of 2018.
Another big mover this week is Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), although this one is for a slightly more unusual reason than the above-mentioned drug approval.
On Monday, Ultragenyx announced that it was going to sell a Priority Review Voucher to Novartis for $130 million. These Priority Review Vouchers are issued by the FDA, generally, on the back of drug approvals and only over the last few years has there really been an active market for them. Prior to this, the company would keep hold of it and could trade it in for an expedited review process as and when it submits for approval for one of its development stage drugs.
More recently, however, we have seen (especially the smaller) companies sell these vouchers to bigger names, essentially cashing them in for operational capital in favor of an expedited review.
Why would a company like Novartis paid $130 million for an expedited review?
Novartis is one of a handful of megacap healthcare companies competing to bring certain assets to market first. The ability to get to market ahead of a competitor dramatically improves the chances of picking up market share and when you are looking at blockbuster drugs like those targeting oncology or diabetes, $130 million is a small price to pay to get a jump on a competitor’s asset.
Of course, it’s something of a gamble for Novartis, since even with expedited review, there is no guarantee that the FDA will approve the asset under investigation.