For many of us, the New Year brings resolutions; a hopeful promise to do things better next time. For biotechs, it brings new data; data that can sink or save a product, a division, and even a company. Refusal of approval doesn't necessarily exclude a next time, but clinical costs and lost profits might. Also, the way FDA and other regulatory bodies lean generally decides the fate of a research project's funding.
Should Old Acquaintance Be Forgot
It's no secret that the FDA prefers new applications of known, approved drugs. A longer clinical history is perhaps the most valuable asset a "new" drug (or device or biologic) can have—it certainly helps with the safety part of the application. Except, of course, when that clinical history is bad news.
Consider Orexigen's Contrave (naltrexone + bupropion), which recently received a positive review from the FDA's expert panel (though they still advised more cardiovascular monitoring). Its competitors, Vivus' Qnexa (phentermine + topiramate) and Arena's lorcaserin, were refused approval earlier this year for lack of safety and efficacy data.
Breaking down the compounds, Contrave is made up of two drugs already in use for several years in treating addiction; Qnexa has the misfortune of containing phentermine, a stimulant that was half of the infamous "Fen-Phen" combo; and lorcaserin is both a new molecular entity and also structurally similar to fenfluramine (the "Fen" part of the duo).
Cleaning up for 2011, the FDA also withdrew approval for weight-loss drug Meridia (sibutramine, related to the other amines) after 13 years on the market.
As a result of the October withdrawal of Meridia, there is only one FDA-approved long-term weight-loss drug: Orlistat. Its loneliness at the top, combined with its embarrassing side effects and potential liver problems, give the FDA a very strong motive to tilt the scales toward approval of another anti-obesity drug, even when some of the efficacy benchmarks remain unfulfilled.
It's also been a bad year for personalized genetic testing. From being a $30 over-the-counter deal in Walgreens to qualifying as a "medical device" for the purposes of regulation, the appearance and disappearance of consumer genetic testing served as an excellent example of how much regulatory guidance is needed for a nascent field such as this.
On the flip side, rapid HIV tests, HIV drugs, and anticancer drugs have benefited from being pioneers in enormously hurting fields. Mylotarg (an antibody for treating acute myeloid leukemia) was granted accelerated approval in 2000. It was, however, pulled from the market in June after continued studies found no significant benefit to taking the drug.Similarly mAb therapeutic Avastin lost its breast cancer approval last year; it's a bad day for antibodies.
Clearly, being too new is a liability as well, even when playing by the FDA's rules. Provenge, the innovative anticancer vaccine approved this year, received an initial refusal on its first run through the approval gauntlet. Clearly, the ideal product has a long, clean clinical record combined with a novel application in the FDA's latest field of interest; so how does that relate to 2011?
What to Expect
With the latest round of funding and FDA initiatives, challenges such
as obesity, unprecendented medicines like gene therapy or nanotech-based drugs, and personalized medicine have secured regulators' interest. This increased attention can only help new technologies bridging unmet medical needs.
The annual critiques of the FDA's device and drug approvals have come out, mostly emphasizing the agency's onerous evidence requirements and unpredictable behavior. It seems, however, that the agency is slowly coming around to better communication—the FDA may finally publish its new rules on advertising using social networks and other informal channels.
At the same time, the FDA has promised to adapt so it can address new technologies—the "too new" of 2010 might be "on track" in 2011. Together with the social engineering that FDA intends to do, it would be reasonable to expect a friendlier reception of lifestyle treatments (anti-obesity, smoking cessation, and other public health issues) and diagnostics (personal sequencing, disease biomarkers, and pathogen detection).
All of these topics have received attention in the FDA's latest initiative and most have forces outside of the FDA also pushing for their adoption. Personal sequencing, with a head start on consumer deployment and proven technology, seems to be well positioned for approval in the coming years.
A complication is the approval of Provenge (a ~$93,000 prostate cancer treatment) around the same time that Avastin is losing its breast cancer approval (up to $100,000/year). This is certain to bring about some controversy, and it pokes holes in claims that the FDA makes decisions based on treatment affordability. How the ripples of these decisions will affect future policy is hard to tell—perhaps accelerated approval will become even harder to obtain.