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UniQure’s Huntington’s Disease Therapy Approval Blocked by FDA Amid Regulatory Concerns

March 7, 2026
UniQure announced on Monday that its efforts to secure approval for a Huntington’s disease treatment from the Food and Drug Administration (FDA) remain obstructed, marking another hurdle for a therapy that previously seemed poised for approval. In a recent meeting with the FDA, UniQure learned that regulators remain unconvinced that the data from its completed…

UniQure Faces Setback as FDA Prefers Randomized Trial for Huntington’s Treatment

March 7, 2026
UniQure’s stock experienced a decline on Monday following unfavorable feedback from the FDA regarding its Huntington’s disease program, AMT-130. The agency indicated that the current application lacks sufficient data to support its approval, specifically expressing a preference for a randomized clinical trial to validate the treatment’s efficacy. This development underscores the FDA’s stringent requirements for…

Kennedy adds 2 new members to CDC’s vaccine panel ahead of delayed meeting

March 7, 2026
The Centers for Disease Control and Prevention (CDC) has appointed two new members to its Advisory Committee on Immunization Practices (ACIP) as it prepares for a postponed meeting. The new appointees, Dr. Sean G. Downing, a specialist in internal medicine and pediatrics from Florida, and Dr. Angelina Farella, a pediatrician, bring valuable expertise to the…

FDA Approves Ascendis Pharma’s Weekly Yuviwel for Paediatric Dwarfism

March 7, 2026
The U.S. Food and Drug Administration (FDA) has granted approval for Ascendis Pharma’s Yuviwel, a weekly treatment for pediatric patients with growth hormone deficiency due to dwarfism. This approval introduces a significant advancement in the management of this condition, providing a more convenient dosing schedule compared to the existing daily injection option, Voxzogo by BioMarin.…

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