After the Food and Drug Administration offered only a standard review to Onyx Pharmaceuticals'
Cancer drugs like carfilzomib, which will go by the trade name Kyprolis if approved, typically get a priority review, which offers a quicker six-month review instead of the standard 10 months, because the drugs offer a substantial advance over what's currently available.
Onyx needs the FDA to believe it's better than anything else available because it's seeking an accelerated review, which requires less data -- in this case a single-arm study without any control arm to compare to -- but is only available for drugs that treat patients without any other option. The FDA, for instance, didn't even accept the application for an accelerated approval for Roche and ImmunoGen's
Instead of dwelling on the unmet-need issues, the agency seems to be more focused on the overall risk-benefit ratio for carfilzomib. There's only one question the committee will vote on, essentially whether the benefits outweigh the risks for this patient population. Having just one voting question isn't that unusual, but there are no discussion questions before that, which is a bit unusual. The morning session, for instance, has a non-voting question about the population that should be given Sanofi's
Carfilzomib provided a 22% response rate, but the agency appears worried about the drug's causing heart problems in some patients, which appears to have killed some patients. In general, cancer drugs are given a free pass on side effects if they work well because the alternative to not treating is usually death. Does it really matter whether the drug is killing the patients or whether the disease is? The focus should be on the 22% of patients that do respond.
But, coming full circle, Onyx is asking for approval based on a phase 2 trial testing patients that failed or couldn't take Celgene's
For investors to have confidence that carfilzomib will be approved, the voting question needs to go substantially in Onyx's favor tomorrow. Without a discussion question beforehand, the panel of outside experts will be swayed by the FDA's and Onyx's presentations and how they field questions from the panel. Check back Thursday for a recap.
The biggest binary event, the presidential election, is around the corner. Check out the Fool's new free report "These Stocks Could Skyrocket After the 2012 Presidential Election," in which you'll get ideas for companies that can benefit from each candidate's platform. Get your free copy by clicking here.
Fool contributor Brian Orelli holds no position in any company mentioned. Click here to see his holdings and a short bio. The Motley Fool owns shares of Johnson & Johnson. Motley Fool newsletter services have recommended buying shares of Johnson & Johnson and ImmunoGen and creating a diagonal call position in Johnson & Johnson. The Motley Fool has a disclosure policy. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. Try any of our Foolish newsletter services free for 30 days.