Summary:
The FDA has selected Regeneron’s DB-OTO gene therapy for genetic hearing loss as one of nine drugs to receive expedited review under its new Commissioner’s National Priority Voucher (CNPV) program, following promising trial results showing significant and sustained hearing improvements in children.

Key Takeaways:

  • DB-OTO demonstrated clinically meaningful hearing improvements in 11 of 12 CHORD trial participants, with three achieving normal hearing levels.
  • The CNPV program accelerates FDA review from up to a year to just 1–2 months through a streamlined, multidisciplinary process.
  • The selection highlights genetic hearing loss as a national medical priority and reflects the FDA’s focus on expediting transformative treatments.

The U.S. Food and Drug Administration has announced nine experimental medicines that will receive vouchers for expedited FDA review under the new Commissioner’s National Priority Voucher (CNPV) pilot program. “Each recipient has a product with significant potential to address a major national priority, such as meeting a large unmet medical need,” according to an FDA press release. One of the nine drugs chosen to receive a voucher is Regeneron Pharmaceuticals’ DB-OTO for treating genetic hearing loss. 

Regeneron recently announced updated data for their investigational gene therapy DB-OTO for profound genetic hearing loss due to variants of the otoferlin (OTOF) gene. These latest results from the pivotal CHORD trial show 11 out of 12 participants have experienced clinically meaningful hearing improvements, including 3 who achieved normal hearing levels. Additionally, eight participants with longer follow-up showed stability or continued improvement in their hearing, and among three who completed speech assessments, all showed significant improvement.

New DB-OTO Results Show Sustained Improvements for Children with Profound Genetic Hearing Loss

“One of our core goals is to deliver more cures and meaningful treatments—especially ones that have an outsized impact on our most pressing national priorities,” says FDA Commissioner Marty Makary, MD, MPH. “We must modernize the review process and try new approaches to meet the needs of the American people.”

Regeneron’s Gene Therapy DB-OTO Trial Shows Promising Hearing Improvement

The new CNPV process accelerates the standard 10- to 12-month timeline by convening a multidisciplinary team of physicians and scientists for a team-based review, interacting frequently with the sponsor to clarify questions, and completing review of the application concurrently, according to the FDA. Once all streamlined review steps are complete, the team will convene for a 1-day “tumor board style” meeting. The timeline for this new process is a total of only 1 to 2 months. 

The FDA expects to announce another group of CNPV recipients for a fast-tracked drug review process in the coming months.

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