Why hemophilia gene therapies haven't taken off

After four decades and billions of dollars of research, science has finally produced gene therapies for hemophilia, the inherited disorder that stops blood from properly clotting. And yet patients, some of whom eagerly awaited one-time treatments, have been loath to try them.

As STAT's Jason Mast reports, the issue, for once, isn't the price. While Hemgenix, a hemophilia B gene therapy marketed by CSL Behring, costs $3.5 million, and Roctavian, BioMarin Pharmaceutical's treatment for the more common hemophilia A, goes for $2.9 million, insurers have been happy to pay. Current treatments for the disease can easily run over $1 million a year.

Instead, the problem is that current treatments are so effective. Hemlibra, a weekly drug from Roche, has allowed 23-year-old Noah Frederick to live without giving much thought to his hemophilia, giving him little reason to consider gene therapy. And he's hardly alone: Only a handful of have been treated with Hemgenix since its 2022 approval, and just three people have received Roctavian since it became available last summer.

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Biotech

Geron's resurgence might have been short-lived

Geron Corporation, a company that has largely toiled in biotech obscurity since the 1990s, rocketed back to relevance earlier this year with news that its treatment for a group of chronic blood disorders succeeded in a late-stage trial. But that corporate comeback might not hold up to scrutiny.

In documents published yesterday, FDA reviewers picked apart Geron's data from a trial enrolling patients with myelodysplastic syndromes, or MDS. The study met its primary goals, but it's not clear the magnitude and duration of the drug's benefits outweigh its risks, FDA reviewers wrote, and there's no evidence the treatment modified the course of disease or improved patients' symptoms.

The documents sent Geron's share price down as much as 15% yesterday, casting doubt on how the drug will fare before a panel of FDA advisers at a meeting scheduled for Thursday. The agency is expected to make a decision on Geron's drug by June 16.

Research

What Amylyx's failure means to ALS patients

Last week's news that Amylyx Pharmaceuticals' treatment for ALS failed in a pivotal study was a devastating development for many patients with the disease. But after countless disappointments in the search for effective medicines, people with ALS said they haven't lost hope.

As STAT's Annalisa Merelli reports, the immediate aftermath brought concerns that Amylyx would remove its drug, Relyvrio, from the market, people with ALS said, leaving patients with one fewer treatment option. Some feared that Amylyx's setback, the latest in a string of disappointments in ALS research, would discourage other companies from investing in the disease. But as long as that work goes on, there is always hope, patients said.

"We keep going," said Katrina Byrd, who has ALS. "With our emancipated bodies, broken hearts, and broken spirits, we stand together and demand to be a part of the ALS drug development process. If we fail, we get up again and again."

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Washington

Biden's plan for biosimilars

Tucked somewhere in President Biden's $130.7 billion budget proposal for 2025 is a provision that would let pharmacists fill prescriptions for brand-name biologics with biosimilars without consulting physicians, a change the administration says would lower drug costs.

As STAT's John Wilkerson and Sarah Owermohle report, the request is among a handful of budget proposals with implications for health policy. And, like everything else in a presidential budget, it's more of a wish than an actionable policy goal.

But even if Congress nixed the so-called interchangeability provision, there's no guarantee it would significantly increase the adoption of biosimilars. The convoluted system through which most Americans get their drugs often creates incentives for formularies to choose brand-name biologics over biologics, even if the latter has a lower sticker price.