The Readout Damian Garde & Meghana Keshavan

Calls grow for an investigation into Aduhelm’s approval

Former health secretary Donna Shalala called for a federal investigation into the FDA’s polarizing approval of Biogen’s Aduhelm, citing STAT’s reporting that regulators were far more closely aligned with the company than previously disclosed.

Speaking to STAT, Shalala said that “when you see a report like this, you have to investigate it. ... You've got to send in the Office of Inspector General.” Shalala joins a chorus of advocates and academics demanding answers on the close relationship between Billy Dunn, head of the FDA’s neuroscience division, and Biogen executives in the run-up to the approval of the drug, called Aduhelm. Public Citizen cited STAT’s reporting in a renewed call for an OIG investigation, as did Yale University public health professor Gregg Gonsalves.

But external pressure alone can’t force the OIG into action. The office sets its own agenda and sincerely values its independence, one former OIG attorney said.

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Your guide to biotech’s biggest binaries

Will FibroGen’s anemia pill pass a crucial heart-safety review? Can Cytokinetics make M&A dreams come true? And will a different Waksal get a drug approved?
We’re likely going to learn all of that over the next three months, as STAT’s quarterly scorecard helpfully lays out. Still smarting from a data-alteration scandal, FibroGen brings its anemia pill roxadustat in front of an FDA advisory panel on July 15, where its cardiovascular side effect profile will be closely scrutinized. The readout of a mid-stage study for a heart disease drug from Cytokinetics could ignite a wave of takeout speculation. And Kadmon Holdings, run by Harlan Waksal, brother of Sam, is hoping for good news in August when the FDA announces an approval decision on Kadmon's treatment for graft-versus-host disease.

There will also be market-moving updates from Seres Therapeutics, Biogen, Provention Bio, and many more.

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Regenerating heart tissue works in pigs, which is good news for people

An experimental gene therapy helped pigs regenerate cardiac muscle after damage from a heart attack, a preliminary finding that could bode well for translation into humans.

As STAT’s Megan Molteni reports, scientists sought to remove a genetic brake that stops cardiac tissue from regenerating, attaching three short snippets of RNA to a viral vector in hopes of silencing that gene and healing the heart. They induced heart attacks in pigs and injected the gene therapy into the border between dead and damaged tissue.

The result: After three months, pigs that received the treatment had more proliferating cardiac cells and less scarring than their control counterparts. On one measure of heart function, the treated pigs showed a 14.3% improvement.

Read more.

Aduhelm would be cost-effective at a 95% discount

That’s according to the Institute for Clinical and Economic Review, a nonprofit that measures the value of new medicines, which just released a new assessment of Biogen’s recently approved treatment for Alzheimer’s disease.

As STAT’s Ed Silverman reports, ICER contends that Aduhelm would be cost-effective if priced between $3,000 and $8,400 per year, substantially below Biogen’s list price of $56,000. In its review, ICER cited “insufficient” evidence that Aduhelm benefits patients.

ICER has no regulatory power, but its conclusions are frequently cited in fights between drug makers and payers. The group’s latest word on Aduhelm represents its final report. Next, ICER will hold a meeting July 15 to discuss the clinical evidence and hear testimony from patients, neurologists, and Biogen, before voting on the net benefits of the drug and its long-term cost-effectiveness.

Read more.

More reads

  • Washington’s antitrust push could limit Amazon, Google’s health care ambitions. (STAT+)
  • CureVac COVID-19 vaccine records only 48% efficacy in final trial readout. (Reuters)
  • 12 lessons Covid-19 taught us about developing vaccines during a pandemic. (STAT)
  • Moderna locks up Spikevax name in Europe, joins Pfizer's Comirnaty in wait for official brand approval in U.S. (FiercePharma)
Correction: Yesterday's edition misstated the lead indication of Cerevel Therapeutics' drug. It is schizophrenia. 

Thanks for reading! Until tomorrow,

Thursday, July 1, 2021


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