Adam Feuerstein has an important list of companies (the “Gnarly Nine”) that will be going to the FDA soon. That process is always important for the companies involved, but these will serve as indicators of how the agency is dealing with the pressures for lowering the standards for drug approval. We don’t have a new FDA commissioner yet, of course, and who knows when someone is actually going to be nominated, what they think, or what even phylum they’re from, honestly, but pretty much all the names that have been floated have leaned in that direction.
One really out-there example is PTC Therapeutics, whose Ataluren has failed pretty much every test it’s been put to in the clinic (most recently for some forms of cystic fibrosis). The company has filed anyway, which is an unusual move, and I’ll let the legal language in a recent press release speak on that subject. I was going to excerpt this, but it’s pretty much all one sentence, so here goes (emphasis added):
PTC’s actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to PTC’s scientific approach and general development progress; the outcome of ongoing or future clinical studies in Translarna and PTC’s other product candidates; expectations for regulatory approvals; PTC’s ability to meet existing or future regulatory standards with respect to Translarna; the sufficiency of PTC’s cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; PTC’s ability to maintain its marketing authorization of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in the European Economic Area (EEA), including whether the European Medicines Agency (EMA) determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; PTC’s ability to enroll, fund, complete and timely submit to the EMA the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open label extension; PTC’s ability to resolve the matters set forth in the Refuse to File letter it received from the United States Food and Drug Administration (FDA) in connection with its New Drug Application (NDA) for Translarna for the treatment of nmDMD, including whether filing the NDA over protest with the FDA will result in a timely or successful review of the NDA, and whether PTC will be required to perform additional clinical and non-clinical trials or analyses at significant cost; the eligible patient base and commercial potential of Translarna and PTC’s other product candidates; PTC’s ability to commercialize and commercially manufacture Translarna in general and specifically as a treatment for nmDMD; the outcome of pricing and reimbursement negotiations in those territories in which PTC is authorized to sell Translarna for the treatment of nmDMD; and the factors discussed in the “Risk Factors” section of PTC’s most recent Quarterly Report on Form 10-Q as well as any updates to these risk factors filed from time to time in PTC’s other filings with the SEC. You are urged to carefully consider all such factors.
Yeah, that last sentence is pretty sound advice. Some of risk factors themselves are pretty gnarly themselves. It’s a little rich of them to suggest that the FDA might not give them a review on schedule if they file their NDA at them, if that’s indeed what they’re suggesting, but the risk of it not being successful is very substantial indeed. In fact, I don’t really see any way the compound can be approved under current standards of efficacy, so PTC seems to be hoping that those standards will have changed by then.
But hey, all kinds of things might change. The former governor of my home state of Arkansas, Mike Huckabee, was on Twitter last night telling us all how to do it: “Take on 4 big diseases that cost the BULK of $$: Cancer, heart disease, Alzheimers, diabetes; find CURES like we did with polio in 50’s.” I just can’t tell you what that advice is worth, because mere scientific notation is not sufficient to denote its cash value. Times ten to the minus what, I wonder? Gov. Huckabee has probably been too busy going off the rails over the years (a full-time job) to notice that the biopharma business has actually been pouring countless billions of dollars into just those very diseases. And he might not realize that coming up with a vaccine for a virus whose only host is humans, while not easy, is still a lot easier than going after cancer (which is not just one disease, but thousands). He might also consider that while we knew that the polio virus caused polio, we still have no certainty about the cause of Alzheimer’s, despite decades of intense research. Perhaps it is a hard problem, as opposed to bloviating on Twitter?