FDA Alleges Uniqure Misrepresented Request In Rare-Disease Drug Approval
FDA and HHS officials have publicly attacked Uniqure, the biotech company seeking approval for a Huntington’s disease treatment, and accused it of lying about requests made by the FDA for additional studies involving placebo brain surgery, which the company has characterized as unethical. The company says the anonymous FDA statements "are incomplete or entirely incorrect.”
The Wall Street Journal:
Federal Health Officials Attack Rare-Disease Drug, Say Company Lied
Federal health officials, facing criticism from lawmakers for recent rejections of rare-disease drugs, attacked an Amsterdam-based biotech company seeking approval of a Huntington’s disease treatment and accused it of lying. The public criticism of Uniqure by officials at the Food and Drug Administration and Department of Health and Human Services was unusual for agencies that normally shy away from commenting on products still under consideration. (Essley Whyte, 3/5)
Bloomberg:
FDA Push For 10-Hour Fake Brain Surgery Trial Raises Ethical Concerns
Katie Jackson desperately wants a new treatment for Huntington’s disease. Her husband died from the devastating brain disorder. And because the disease runs in families, her three children have a 50% chance of inheriting the condition. She’s pinned her hopes on a cutting-edge gene therapy from UniQure NV. But Jackson says Huntington’s patients have no desire to meet a new demand from the US Food and Drug Administration: to enroll in a new clinical trial where some people will undergo fake brain surgery without getting UniQure’s treatment. (Smith and Langreth, 3/4)
The New York Times:
Ex-Executive At Blood Filter Start-Up Is Charged With Covering Up Deaths
Federal prosecutors charged a former executive of ExThera Medical, a California medical-device manufacturer, with concealing from the government the deaths of two cancer patients who were treated with the company’s blood filter. Dr. Sanja Ilic, ExThera’s former chief regulatory officer, agreed to plead guilty and faces up to three years in prison, the Justice Department said Thursday. Prosecutors also entered into a deferred prosecution agreement with ExThera in which the company admitted that it defrauded and misled the Food and Drug Administration by failing to report the deaths. (Carreyrou, 3/5)
Modern Healthcare:
Novo Nordisk, Eli Lilly Tighten 340B Oversight As Providers Fume
Drugmakers are upping their demands for transparency in the 340B Drug Pricing Program by imposing new requirements that have sparked ire among safety-net providers. Novo Nordisk issued a notice to hospitals and other 340B participants on Monday saying providers will be required to submit comprehensive claims-level data in order to receive discounts on medications starting April 1. A similar Eli Lilly policy took effect last month. (Early, 3/5)
CIDRAP:
GARDP To Explore Animal Antibiotic As A Potential Superbug Treatment
The Global Antibiotic Research and Development Partnership (GARDP) is looking into the potential of an older antibiotic used exclusively in veterinary medicine to treat highly drug-resistant infections in people. GARDP said today that it has acquired all data and licensing rights for apramycin, an aminoglycoside antibiotic introduced in the early 1980s to treat gram-negative bacterial infections in animals. Bought from Swiss drugmaker Juvabis AG after it closed in 2025, the drug has shown potential for human use in preclinical trials. (Dall, 3/5)
The Washington Post:
What Families Need To Know About Antipsychotic Drug Use In Nursing Homes
A man upset over the death of his wife kept a blanket over his head in his Minnesota nursing home. Staff gave him a powerful antipsychotic drug. Another man, in a Philadelphia facility, yelled during two evening shifts. He also got an antipsychotic. And in Michigan, a man was left sitting in his own waste for hours because he was so overmedicated with one of the drugs, he couldn’t hit a call button to summon staff for help. (Rowland, 3/6)
