Estimated Spending on Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa.

ABSTRACT:

Importance

New gene therapies can offer substantial benefits to patients, particularly those with rare diseases who have few therapeutic options. In May 2023, the US Food and Drug Administration (FDA) approved the first topical gene therapy, beremagene geperpavec (B-VEC), for treating both autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa (DEB). However, FDA approval was based on limited data in patients with autosomal dominant disease, even though they comprise approximately 50% of all DEB cases.

Objective

To estimate projected spending in the US on B-VEC therapy for treating autosomal recessive and autosomal dominant DEB.

Design, setting, and participants

This economic evaluation used data from the National Epidermolysis Bullosa Registry to

SUBMITTER: Raymakers AJN 

PROVIDER: S-EPMC10831624 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature