FDA approval summary for lonafarnib (Zokinvy) for the treatment of Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies.
Genet Med
; 25(2): 100335, 2023 02.
Article
em En
| MEDLINE
| ID: mdl-36507973
ABSTRACT
The U.S. Food and Drug Administration recently approved lonafarnib as the first treatment for Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies. This approval was primarily based on a comparison of patients with HGPS treated with lonafarnib in 2 open-label trials with an untreated patient cohort. With up to 11 years of follow-up, it was found that the lonafarnib treated patients with HGPS had a survival benefit of 2.5 years compared with the untreated patients with HGPS. This large treatment effect on the objective endpoint of mortality using a well-matched comparator group mitigated potential sources of bias and together with other evidence, established compelling evidence of a drug effect with benefits that outweighed the risks. This approval is an example of U.S. Food and Drug Administration's regulatory flexibility for a rare disease while ensuring that standards for drug approval are met.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Progéria
Tipo de estudo:
Guideline
Limite:
Humans
País como assunto:
America do norte
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article