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The Real-Time Oncology Review (RTOR) Pilot Program for New Molecular Entities Approvals

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FDA Guidance for Human Gene Therapy for Hemophilia A & B

[/vc_column_text][vc_column_text]Early phase

According to the FDA gene therapy recommendations, the development of G.T. for haemophilia is similar to that of other biologics, needing proof of safety and feasibility and bioactivity and preliminary effectiveness. It is recommended that potential sponsors contact FDA review personnel early in the development process, as each clinical research must be evaluated on a case-by-case basis.

Hemophilia A is a blood clotting disorder that affects the blood vessels of the body. Due to qualitative variations in assay results from one-stage clotting (O.C.) and chromogenic (C.S.) assays, it’s not always viable to extrapolate recombinant factor VIII levels to those acquired by users of G.T. products. For example, C.S. tests show more significant factor activity than O.C. assays in haemophilia A treated with recombinant B-domain-deleted factor VIII preparations. On the other hand, O.C. tests indicate that patients who receive G.T. products with a B-domestic-deleted factor VIII transgene have more excellent factor activity.[/vc_column_text][vc_column_text]Late phase

The FDA advises using a within-subject Non-inferiority (N.I.) trial to show that well-controlled research was undertaken to provide adequate data to support the G.T. product’s marketing application. If a conventional approval route is selected, Annualized Bleeding Rate should be used as the critical endpoint for showing therapeutic benefit (ABR). Factor activity might be a surrogate endpoint if early phase trial findings support an expedited approval strategy. Remember that any differences in factor test findings from different assay techniques must be resolved for an accelerated route. A target factor activity level must be determined within the average population’s factor activity range.

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References

Draft Guidance for Industry: Human Gene Therapy for Hemophilia, dated July 2018

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/

Guidances/CellularandGeneTherapy/UCM610801.pdf

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