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FDA approval summary for lonafarnib (Zokinvy) for the treatment of Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies.
Suzuki, Mari; Jeng, Linda J B; Chefo, Solomon; Wang, Yan; Price, Dionne; Li, Xiaohui; Wang, Jie; Li, Ruo-Jing; Ma, Lian; Yang, Yuching; Zhang, Xinyuan; Zheng, Nan; Zhang, Ke; Joseph, David B; Shroff, Hitesh; Doan, Jenny; Pacanowski, Michael; Smpokou, Patroula; Donohue, Kathleen; Joffe, Hylton V.
Affiliation
  • Suzuki M; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD. Electronic address: mari.suzuki@fda.hhs.gov.
  • Jeng LJB; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Chefo S; Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Wang Y; Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Price D; Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Li X; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Wang J; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Li RJ; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Ma L; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Yang Y; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Zhang X; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Zheng N; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Zhang K; Office of New Drugs, Office of Immunology and Inflammation, Division of Pharmacology/Toxicology for Immunology and Inflammation, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Joseph DB; Office of New Drugs, Office of Immunology and Inflammation, Division of Pharmacology/Toxicology for Immunology and Inflammation, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Shroff H; Office of Pharmaceutical Quality, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Doan J; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Pacanowski M; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Smpokou P; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Donohue K; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
  • Joffe HV; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
Genet Med ; 25(2): 100335, 2023 02.
Article in 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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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Progeria Type of study: Guideline Limits: Humans Country/Region as subject: America do norte Language: En Journal: Genet Med Journal subject: GENETICA MEDICA Year: 2023 Type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Progeria Type of study: Guideline Limits: Humans Country/Region as subject: America do norte Language: En Journal: Genet Med Journal subject: GENETICA MEDICA Year: 2023 Type: Article