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# Support for Hemophilia Gene Therapy Guidance
- URL: https://www.fdaweb.com/support-for-hemophilia-gene-therapy-guidance/
- Published: 2018-12-12T12:00:00.000Z
- Updated: 2026-09-15T00:50:24.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5143027

BioMarin says it commends FDA for providing recommendations for stakeholders developing human gene therapy products to treat hemophilia. The company’s [comment](https://www.regulations.gov/document?D=FDA-2018-D-2238-0029&ref=fdaweb.com) says that overall it supports the recommendations in the agency’s draft guidance as a positive step toward providing regulatory clarity for developing these novel products. The comment letter addresses the importance of patient-focused development, traditional approval, and maintaining the regulatory standard for accelerated approval.

In its [comment](https://www.regulations.gov/document?D=FDA-2018-D-2238-0028&ref=fdaweb.com), the American Society of Gene & Cell Therapy says the agency’s recommendations “are generally welcomed and will provide clarity for development of gene therapy products for hemophilia.” The society also provides specific line-by-line comments.

Bayer [says](https://www.regulations.gov/document?D=FDA-2018-D-2238-0027&ref=fdaweb.com) the FDA definition of gene therapy includes gene editing, but the draft guidance does not specifically address gene editing. “It would be important to clarify whether any of the components or the combination of the different components of gene editing … would need to be tested in vitro or in nonclinical studies,” the letter says.