Global Blood’s drug might satisfy FDA, but some doctors are wary

A sickle-cell disease drug from Global Blood Therapeutics looks to be on the path to FDA approval, judging by new data released today, but whether the pill’s effects will actually improve patients’ lives remains the subject of debate.

As STAT’s Adam Feuerstein reports, the drug, called voxelotor, significantly increased patients’ hemoglobin levels in a late-stage trial. That, according to Global Blood, should be enough to qualify voxelotor for accelerated FDA approval.

But outside experts cautioned that boosting hemoglobin might not actually address the worst symptom of sickle-cell disease: episodes of severe pain that often land patients in the hospital. Global Blood doesn’t have data on whether voxelotor can do that.

“There is pressure on everybody to pay attention to sickle-cell disease and get more therapeutic options out there, but I don’t think this is a good reason to push through voxelotor’s approval if it doesn’t meet the bar of showing benefit for patients,”  said Dr. Sujit Sheth of Weill Cornell Medicine.

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Is biohacking a crime?

Who came up with the latest CRISPR trick? And did Rage Against the Machine sell out?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s biotech podcast. First, we talk to biohacker Josiah Zayner, perhaps best known for CRISPR'ing himself onstage, about his experience with regulators investigating whether his work amounts to practicing medicine without a license. Then, STAT senior science writer Sharon Begley joins us to explain the twists and turns of the most recent breakthrough in genome editing, one that could offer a preview of the next patent dispute.

You can listen to the episode here. To listen to future episodes, be sure to sign up on iTunes, Stitcher, Spotify, or wherever you get your podcasts.

A blow to the fecal transplant field

Two patients contracted drug-resistant infections after getting fecal microbiome transplants, and one of them has died, according to the FDA.

As STAT’s Kate Sheridan reports, the FDA’s disclosure appears to be the first death publicly attributed to fecal transplant, which is an increasingly popular procedure for people with certain recurring infections. 

The agency isn’t changing its policies with regard to fecal transplant trials, but it cautioned doctors to be mindful of the potential risks and promised to “aggressively monitor clinical trials to ensure patients are protected when safety concerns arise.”

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The importance of photocopier etiquette in biotech

(Elobix)

What you see above is an illustration of how a biotech company called Elobix went from developing a drug for constipation to suing its former partner.

As STAT’s Ed Silverman reports, Elobix saw its relationship with Ferring Pharmaceuticals come unglued after that constipation drug failed in late-stage trials. But the problem wasn’t efficacy, Elobix claims. It was that some Ferring employees didn’t understand how to use a photocopier.

Back in 2013, a Ferring employee photocopied the labeling for the 5mg dose of Elobix's drug but left that paper in the output tray, according to a lawsuit. The next day, a different employee mistook that piece of paper for another copy, one that would go on the 10mg dose, the lawsuit claims. That led to patients in the trial getting the wrong doses, which in turn scrambled the results and cost Elobix millions, according to the suit.

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