Ultragenyx Pharmaceutical Inc. has provided additional information regarding its UX111 gene therapy program for Sanfilippo Type A and the Complete Response Letter (CRL) it received from the U.S Food & Drug Administration (FDA) in July.
They created a document outlining what a CRL is, key facts about the UX111 CRL, and next steps in the process to resolve the FDA’s observations.
In the document, Ultragenyx describes as CRL as “The FDA’s way of saying ‘revise and resubmit’ – [noting that] aspects of the application need to be updated before FDA can complete its review and make a decision. The details of a CRL vary across drug applications.”
The document notes that “While the FDA has not yet fully reviewed or made any determinations regarding the clinical trial data, the FDA did not raise concerns about the quality of product manufactured to date, safety, or effectiveness of the gene therapy.”
The company continues to offer assurances that they believe all of the FDA’s observations “are readily addressable and our priority is to resolve them so that we can resubmit the BLA as soon as possible for review.”
Ultragenyx lists in the document the following steps are it foresees ahead:
- Ultragenyx prepares for a meeting with the FDA by developing a briefing document and agenda, and then submits a request for a meeting.
- The FDA schedules the meeting.
- The meeting with FDA takes place with the goal to agree on the planned resolution of the observations.
- Once we have alignment with the FDA on what will be included in the application, Ultragenyx plans to resubmit the BLA.
- The FDA will re-review the BLA. This step typically takes the FDA up to 6 months to review and make a decision.
Download and read the entire document from Ultragenyx.
Learn more about the UX111 gene therapy program for Sanfilippo Type A.