May 19, 2023

The need-to-know this morning

Terry Horgan, the 27-year-old patient with Duchenne muscular dystrophy who died eight days after receiving a CRISPR therapy custom-built for him, likely suffered a fatal innate immune response to the virus used to deliver the treatment, investigators concluded. STAT reporter Jason Mast has more here.
The Food and Drug Administration is convening an advisory panel today to review a treatment for NASH from Intercept Pharmaceuticals. You can watch a webcast of the proceedings here.

Regulatory

FDA advisory panel recommends Pfizer's maternal RSV vaccine, but has some concerns

An FDA advisory committee yesterday unanimously agreed that regulators ought to approve Pfizer's maternal RSV vaccine, though they did express some safety concerns.

The panel of vaccine experts voted 14-0 that the inoculation was able to effectively prevent severe disease in the infants of people who were vaccinated while pregnant. But the panelists voted 10-4 on safety, with some believing that the data there were insufficient.

Abrysvo, as the vaccine is called, could potentially prevent tens of thousands of hospitalizations of young children in the U.S. alone. The vaccine was shown to reduce the risk of severe lower respiratory tract disease caused by RSV by 82% at three months after birth, and 69% at six months.

patents

Supreme Court sides with Sanofi, not Amgen, on patent case

Sanofi won a patent battle with Amgen that escalated all the way to the Supreme Court. In their ruling yesterday, the justices unanimously agreed that Amgen shouldn't have been granted certain vaguely worded patents for its cholesterol drug, Repatha.

The lawsuit, which has been closely watched by the biopharma industry since 2014, will essentially prevent other companies from using overly expansive patents to prevent competitors from launching similar drugs. Amgen had initially filed the lawsuit to try and win millions from Sanofi for purportedly infringing on its Repatha patents.

"Amgen manufactured a purported problem in this case," Arti Rai, a Duke law professor specializing in intellectual property, told STAT. "The Supreme Court took the bait when it (agreed to review the case), but thankfully, refused to take the bait in its ultimate decision. Once one understands the science, and I think the court actually did at some level, the case is not that challenging."

marketing

Reflecting on the ethics of Viagra

Erectile dysfunction met its match 25 years ago: Viagra was approved by the FDA in March 1998. As one might imagine, the ethics of testing and marketing such a medication had to be handled delicately. At the time, Pfizer paid former Republican presidential candidate Bob Dole to speak of his own sexual impotence, aiming to help dispel shame and stigma. Arthur Caplan, head of the Division of Medical Ethics at NYU, writes for STAT about how he helped the drugmaker navigate these thorny issues.

"My most important advice, which I think is evident in the advertising campaign, was that the pill needed to target a disease," he writes. "'Impotence' was too vague a concept. A disease category would make it easier for men to overcome their shame and look for help."

podcast

Does the FTC have it out for pharma?

What does it take to make an RSV vaccine? And how do you say "setting cash on fire" in Dutch? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Jared Holz, biotech strategist at Mizuho Securities, joins us to explain why federal regulators are suing to block Amgen's $28 billion merger with Horizon Therapeutics and what it means for the drug industry. We also discuss the latest on Sarepta Therapeutics and its gene therapy for Duchenne muscular dystrophy, a turnaround story in the making at the Belgian drug maker Galapagos, and the effort to secure approval for a maternal RSV vaccine.

Listen here.

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