STAT+: Pharmalittle: We’re reading about a Huntington’s gene therapy, Novo’s challenges, and more

STAT PlusNewsletterPharmalot Pharmalittle: We’re reading about a Huntington’s gene therapy, Novo’s challenges, and more New White House drug pricing deals might not amount to much, at least not immediately Manage alerts for this article Email this article Share this article By Andrew JosephSept. 2, 2026 Europe Correspondent Andrew Joseph[email protected]Andrew covers the biopharma industry, scientific research, and public health across the continent. You can reach Andrew confidentially on Signal at drewqjoseph.71.

Hello there again from STAT’s London outpost, with Andrew Joseph here filling in for Mr. Pharmalot. Typing that out, I just misspelled my own last name, so it does in fact appear to be Wednesday.

Let’s just get to the headlines … The drug pricing deals the White House signed with drug companies this week might not amount to much, at least not immediately, STAT explains. That’s because they chiefly apply to Medicaid, which by law gets the lowest prices of any U.S. payer, while exempting companies from mandatory price reductions that are being considered in Medicare.

STAT also lays out some of the biggest outstanding questions about the new deals. Advertisement UniQure has submitted its experimental Huntington’s gene therapy to the Food and Drug Administration for an accelerated approval review, the company announced. The move is notable because the FDA, under officials who have since left the agency, earlier told the company it couldn’t submit the therapy based on its Phase 1/2 trial, which showed that the medicine could slow the progression of the fatal neurodegenerative disease.

The FDA reversed that stance in June. UniQure said it had also applied for approval to the U.K.’s regulatory agency. STAT+ Exclusive Story Already have an account?

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