After the Food and Drug Administration offered only a standard review to Onyx Pharmaceuticals' (Nasdaq: ONXX) multiple myeloma treatment carfilzomib, it appeared the advisory committee meeting would focus mostly on whether the drug was treating an unmet need.
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 (Nasdaq: IMGN) T-DM1 because the agency didn't believe the phase 2 trial enrolled a population without any other options.
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 (NYSE: SNY) semuloparin before the voting question about the benefit-to-risk ratio.
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 (Nasdaq: CELG) Revlimid and Velcade, which is sold by Takeda Pharmaceutical and Johnson & Johnson (NYSE: JNJ). The FDA's standard will likely be higher, especially if the agency believes the patients have other options.
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.
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