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The Readout Damian Garde & Meghana Keshavan

In Aduhelm, ALS advocates see unfair treatment

The FDA approved Biogen’s controversial Alzheimer’s drug this week through a special pathway that evaluates a drug based on surrogate endpoints, rather than proving that it has a direct clinical benefit. It's the same kind of approach ALS advocates would like to see the FDA apply to experimental treatments for that disease.

But, as STAT's Nicholas Florko writes, they're unlikely to get it anytime soon. FDA drug center chief Patrizia Cavazzoni told reporters the science just isn’t there yet for such an approval in ALS. Patient advocates are frustrated.

The FDA is "not focused enough on the needs of people with ALS and not following its own guidance,” Neil Thakur, the chief mission officer of the ALS Association, told STAT.

Read more.

A big possible advance with CAR-T

CAR-T is typically used as a cancer treatment when patients have exhausted all other options. A new study, however, suggests it might have a role in an earlier phase of care.

Results from a randomized clinical trial comparing Breyanzi, a cellular therapy from Bristol Myers Squibb, against the standard of care for large B-cell lymphoma found that the CAR-T therapy was superior.

The news was released in a press release, with few details on the study or outcome, STAT’s Matthew Herper writes. But if the results hold up, it would mean a major step forward for CAR-T therapy. Carl June, one of the earliest to draw attention for CAR-T therapy, called the result “a big deal.”

Read more.

Vertex shelves protein 'corrector' drug

Vertex Pharmaceuticals said yesterday it would shelve an experimental drug for alpha-1 antitrypsin deficiency, STAT’s Adam Feuerstein writes.

Although the drug, called VX-864, was deemed safe and was incrementally more effective than placebo, it wasn’t enough to benefit patients, the company said. On the upside, Vertex said that the protein “corrector” drug’s mechanism of action seems to work — so it plans to push one or two more of these types of drugs into human studies in 2022. 

“This is the first time we have, or anyone has, demonstrated proof of biologic activity using a small molecule corrector approach in AATD, so I see this as a significant de-risking event that will allow us to move forward with the program,” Vertex CEO Reshma Kewalramani told STAT.

Read more.

Was the FDA right to approve Aduhelm?

What’s a fair price for a debatable drug? And what does this have to do with ’80s HIV activism? 

We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. We’re devoting this entire episode to the FDA’s polarizing decision to approve Aduhelm, a controversial Biogen treatment for Alzheimer’s disease. First, we’ll break down what happened and why it’s such a big deal, and then we’ll talk about the broader implications for science, medicine, and the drug industry as a whole. Finally, we’ll zoom out explore how the FDA got to this moment in a conversation with Yale University professor and global health activist Gregg Gonsalves. 

Listen here

More reads 

  • FDA accuses company of distributing unapproved Covid test and using falsified data (STAT)
  • A CRISPR treatment for blood diseases shows curative promise in small study (STAT)

Thanks for reading! More next week,

Damian

Friday, June 11, 2021

STAT

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