Thumbs Up/Down

Thumbs Up/Thumbs Down: The FDA damaged itself badly with the Sarepta OK

Endpoints assesses the big biopharma R&D stories of the week, with a little added commentary on what they mean for the industry.

thumb down  Califf’s decision to bow to Janet Woodcock on Sarepta hurts the entire biotech industry.

Janet Woodcock’s decision to override the caustic internal criticism of Sarepta’s weak and at times misleading case for its Duchenne drug eteplirsen has damaged the FDA in ways that will leave a mark for years to come. Overruling her colleagues – including senior FDA officials — to provide an accelerated approval this week for this application sends a clear message to biotech: If you can muster enough public support for a campaign – which includes an erroneous study for bait — you should proceed full steam ahead. To his credit, FDA commissioner Robert Califf posted his own review of the case as well as the heated objections of two top regulators, including the current acting chief scientist. Not to his credit, Califf chose to stand down in “deference” to Woodcock’s intransigence, but not before chiding Sarepta for promoting a study that raised false hopes in the patient community, which swallowed it all hook, line and sinker. He was also none too happy that Sarepta stayed focused on its lobbying campaign rather than the clinical work that needed to be done. I sincerely hope that eteplirsen does everything that advocates devoutly believe it does, without presenting the dangers cited for a “scientifically elegant placebo.” And let’s hope patients aren’t left to cover any of the $300,000 annual cost. Payers, and ultimately society, can and should do that now. And what happens next? Does the FDA now slap back the next company to use similar tactics, to try and reestablish old boundaries? Or does it lower its standards permanently, encouraging more large demonstrations and dodgy data to back weak applications? That’s a position the FDA should never have been put in. And that is on Janet Woodcock and Robert Califf.

thumb The immuno-oncology revolution will cause lots of pain, but it will be worth it.

We are living at the start of the immuno-oncology revolution, and it’s been a real privilege covering the explosion of deals and collaborations that are being struck now. Every day it seems brings one, or two, new I/O pacts. This week there was iOmx’s startup round to fund the hunt for new checkpoint inhibitors. Genentech struck a potentially groundbreaking partnership with BioNTech on using messenger RNA to develop personalized cancer vaccines. With the three leading checkpoints on the market and more coming, the potential to partner on new combos is driving billions of dollars in fresh investments. True, anything this hot will breed an excess of alternatives. Investors will get burned. The shakeout will have to occur eventually and not everyone will succeed. Many, most, will fail or fall by the wayside. But it’s been fun watching the spotlight grow bigger and more intense every week. I was chatting with another CEO at a US startup in this field earlier in the week, and he believes that we’re still just at the beginning of this i/o era. This will go on for another 20 years, he says. And he’s likely right. What a great time to be in biotech.

thumb down Setbacks happen. It’s time for the big companies to be more transparent about them.

Why do so many Big Pharma companies have such a hard time talking about a setback? In recent times Amgen never missed a beat when it came to touting their kidney disease drug Parsabiv (etelcalcetide), and then the FDA unexpectedly rejected it and the big biotech shut down, adopting the turtle defense in refusing to even attempt to explain it. Novartis likes to slip bad news into quarterly reports, including a line item on the recent rejection of one of its biosimilars. And this week J&J could only acknowledge, after I contacted them, that the FDA had placed a hold on one of its cancer drugs. They declined to explain the serious adverse events cited on that forced regulators’ hands. I’ve said it before and no doubt will have plenty of occasions to do it again, but companies running drug studies have a responsibility to explain these snafus in some detail. They owe it to their investors, the people who sign up for all of their clinical studies and the scientists trying to make safe progress in the same field. There’s far too little transparency in drug development. At a time when the industry is looking for some understanding from the public on the cost of innovation, big players should also recognize that they can be leaders in owning up to what can go wrong in developing a new drug. It won’t hurt as much as they evidently think it will.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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