UniQure and FDA Diverge on AMT-130 Approval Pathway
On November 3, 2025, uniQure briefly announced that it no longer shares the same view as the FDA regarding the next steps for its drug AMT-130. This comes just weeks after promising data from ongoing clinical trials generated significant excitement.
Background on AMT-130 and the Regulatory Goal
UniQure aimed to use the clinical trial data to file a Biologics Licensing Application (BLA) to bring AMT-130 to market as a disease-modifying treatment for Huntington’s disease (HD). A BLA is the formal request to the FDA to approve a biologic drug, which is derived from living organisms such as proteins, cells, or genetic material.
AMT-130 qualifies as a biologic because it employs a harmless virus to deliver genetic instructions that reduce huntingtin protein levels in brain cells.
Timeline and Recent Developments
- The company planned to submit the BLA in early 2026.
- Until June 2025, uniQure and the FDA were aligned on this plan.
- However, within the last five months, the FDA's position has shifted, creating a misalignment.
Current Status and Outlook
The details of uniQure’s recent meeting with the FDA remain unclear since official minutes have not been released or shared with uniQure. This leaves only a broad overview of the updated situation.
“Unfortunately, we only have a 30,000 foot view of the update from uniQure’s meeting with the FDA, as minutes from that meeting haven’t been released or given to uniQure yet.”
The impact of this disagreement on AMT-130’s path forward is yet to be determined.
Author's summary: UniQure and the FDA now differ on the approval process for AMT-130, halting plans for a Biologics Licensing Application despite earlier trial success and prior regulatory alignment.