Here Are the Drugs That Just Broke the FDA Approval Record

Mark Collins  |


Over the last twelve months or so, a considerable amount of pressure has been placed on the US Food and Drug Administration (FDA) to speed up its review time for in-development drugs. The idea that a large amount of opportunity to treat diseases exists but that – at the same time – patients and the healthcare system in the US are unable to gain access to this opportunity because it's taking the agency too long to process applications for approval has led to this pressure and the FDA has said on numerous occasions that it is trying to alter its processes to overcome the issue.

We're now closing in on the end of the year and – finally – it seems as thought the agency has started to do exactly that.

This week, we've seen three major FDA approvals, all of which have come ahead of their expected PDUFA dates and all of which have, collectively, pushed the agency through and beyond its previous record level of approvals in any on year.

Here's a look at the three approvals in question.

The first came on Monday, when the FDA approved a drug called Rhopressa (netarsudil ophthalmic solution). This one was being developed by Aerie Pharmaceuticals, Inc. (AERI) in a target indication of the lowering of elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension and was initially slated for review on February 28, 2018, meaning the FDA has beaten its review target by more than two months.

When patients get glaucoma it occurs as a result of damage to their optic nerve and it's generally caused by IOP. The more damaged the nerve gets, however, the more IOP the patient experiences, meaning the condition gets increasingly serious over time.

Aerie was able to prove with a pretty substantial late stage dataset that its drug can reduce IOP and – in turn – should be able to halt (or even reverse) the severity of the glaucoma in question.

Next up, Spark Therapeutics Inc (ONCE).

The drug approved in this instance was green lighted on Tuesday (after hours) and is called Luxturna. This one is especially interesting as it's a gene therapy and it's targeting a condition that causes blindness in (in most cases) children. The condition arises from the child receiving two mutated genes, one fro each parent, and with Luxturna, Spark takes a functional version of the gene and inserts it into the patient in question. This functional version then starts to produce functional versions of the protein that they otherwise wouldn't be able to and – in turn – can halt the progression towards blindness.

The original PDUFA for this asset was slated for January 12, 2018 and not only does it fall in line with the FDA's efforts to speed up the review process, it also aligns with the agency's Commissioner, Scott Gottlieb, that gene therapies could form the basis of the next major wave of treatment types in the US.

Finally, Exelixis, Inc. (EXEL).

This one's not quite as interesting as the previous two from a scientific perspective but that far from negates its importance, both from the standpoint of the company and the population its designed to treat.

The drug in question is called CABOMETYX (cabozantinib) and Exelixis has been trying to get it approved in tablet form for the treatment of patients with advanced renal cell carcinoma (RCC). First line treatment, that is. It's already approved as a second line therapy but this second line billing somewhat limits its potential to generate revenues for the company.

And as per this week, the first line therapy approval is in place.

This one was initially slated to go up for review on February 15, 2018, meaning the FDA has come in around one month ahead of schedule.

DISCLOSURE: Disclosure: the author holds no positions in any of the stocks mentioned in this piece.


The views and opinions expressed in this article are those of the authors, and do not represent the views of equities.com. Readers should not consider statements made by the author as formal recommendations and should consult their financial advisor before making any investment decisions. To read our full disclosure, please go to: http://www.equities.com/disclaimer.

Companies

Symbol Name Price Change % Volume
AERI Aerie Pharmaceuticals Inc. 19.01 0.08 0.42 836,359 Trade
EXEL Exelixis Inc. 16.23 -0.28 -1.70 2,223,334 Trade
ONCE Spark Therapeutics Inc. 111.15 0.34 0.31 398,399 Trade

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