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2 March, 2026 |
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We've got an update to today's story on uniQure's interactions with the FDA, with comments from a senior FDA official who shared more of the agency's thinking about why it told the biotech it would have to run a placebo-controlled trial, and what that would mean for the company. |
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Drew Armstrong |
Executive Editor, Endpoints News
@ArmstrongDrew
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by Max Bayer
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HHS Secretary Robert F. Kennedy Jr. named two new members to a key CDC vaccine panel less than three weeks before it’s set to convene. Sean Downing and Angelina Farella were appointed to the Advisory Committee on Immunization Practices in an announcement Friday. Their joining comes as the group is set to discuss Covid-19 vaccine injuries and long Covid at a meeting later this month. Downing is a Sarasota, FL-based physician described by HHS as having more than two decades of experience in primary care. He opened a concierge primary care practice in 2019 and has
provided medical services to a Florida nonprofit, Second Heart Homes, that provides housing and wrap-around care to unhoused people. | |
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Matt Kapusta, uniQure CEO |
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by Max Gelman
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Will Huntington’s disease patients be willing to risk a 12-hour sham brain surgery if it means a gene therapy for their fatal condition might ultimately be approved? That’s the implied question at the heart of uniQure’s dispute with the FDA, as an increasing number of rare disease companies have come under heightened — and often
contradictory — regulatory scrutiny. Under FDA Commissioner Marty Makary, the agency has trumpeted efforts to accelerate rare disease drug approvals, while at the same time rejecting such treatments and insisting that they be tested in clinical trials that may not be feasible. | |
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Paul Chaplin, Bavarian Nordic CEO |
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by Anna Brown
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Following the collapse of a $3 billion bid for Bavarian Nordic by private equity firms in November, CEO Paul Chaplin is stepping down after more than three decades at the company due to "personal reasons." Chaplin, who has been CEO of the Danish vaccine maker since 2014, will continue in his role until the end of this year, before relocating to Australia with his family or until a successor is appointed. The board has started its hunt for a new CEO, a Monday press release states. Bavarian’s stock was down about 2.84% Monday on the Copenhagen stock exchange. In November, the company’s chairman Luc Debruyne stepped down, following the breakdown of Nordic Capital and Permira’s potential $3 billion takeover of Bavarian. The firms pulled out after the bid failed to muster up enough support from the vaccine maker's shareholders. | |
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by Kyle LaHucik
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United Therapeutics said its pulmonary arterial hypertension drug ralinepag succeeded in a Phase 3 study, setting the drugmaker up for an FDA approval request this year and a potential launch in 2027. Pulmonary arterial hypertension is a form of high blood pressure |
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