2 FDA Decisions You Can't Afford to Miss This Week


Last week we focused on five can't-miss earnings reports. This week, we're going to turn the tables back to the Food and Drug Administration and focus on a PDUFA decision and an FDA panel review slated for this week.

Veloci-Raptor time?
First up is Raptor Pharmaceuticals with Procysbi (previously known as RP-103), its oral delayed and extended-release medication to treat nephropathic cystinosis. In trials, Procysbi proved to be non-inferior to the only other FDA-approved treatment for nephropathic cystinosis, known as Cystagon from Mylan .

As my Foolish colleague Keith Speights has already pointed out, both Procysbi and Cystagon are two different forms of cysteamine bitartate. However, Cystagon comes with the need to be taken four times daily (meaning sleep schedules can and will get interrupted) and has a slew of other potential side effects. Procysbi, on the other hand, needs only to be taken twice daily and has milder side effects.

From an ease-of-use standpoint, the drug looks like a slam-dunk for an approval, but the FDA will have to be able to determine that the side effects are diminutive compared to the benefits Procysbi can deliver. If Raptor can get Procysbi approved it would give the company its first revenue generating drug, as well as peak sales potential in the U.S. of $60 million according to JMP Securities. This PDUFA decisions has already been pushed back 90 days to review additional data, so this will certainly be a name to watch on Tuesday.

Will AVEO be on target?
The other FDA decision that should have the attention of biotech investors this week is AVEO Pharmaceuticals' Tivozanib, which is set to go before the FDA's review panel on Thursday.

Tivozanib is an oral, once-daily treatment for metastatic renal cell carcinoma that works by blocking three VEGR-receptors that are critical for blood vessel growth development for solid renal cell tumors, and is being targeted as a first-line treatment. In trials, AVEO and its partner Astellas Pharma went head-to-head against Onyx Pharmaceuticals' Nexavar and delivered good, but not groundbreaking, results. In its TIVO-1 study, released last year, Tivozanib met its primary progress-free survival endpoint and delivered a median PFS of 11.9 months compared with the median PFS of just 9.1 months for Nexavar. The difference was even more pronounced in treatment-naive patients where the PFS difference grew to 12.7 months as compared to 9.1 months for Nexavar. However, overall survival rates for AVEO's drug actually were slightly lower than Nexavar over the course of the study.

Regardless of what the FDA panel has to say about Tivozanib, it could be an uphill struggle given that seven drugs are currently approved by the FDA to treat renal cell carcinoma. In addition to the aforementioned Nexavar, Pfizer's Sutent is a lead drug in metastatic renal cell carcinoma, delivering $1.24 billion in sales -- a 9% year-over-year increase -- in 2012.Even if Tivozanib makes it through the gauntlet, it could have a hard time swaying physicians away from these two commonly prescribed medications.

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The article 2 FDA Decisions You Can't Afford to Miss This Week originally appeared on Fool.com.

Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.Try any of our Foolish newsletter services free for 30 days. We Fools don't all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

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