
Onasemnogene Abeparvovec-xioi: World’s most expensive and approved therapy for spinal muscular atrophy
Author(s) -
Aslam Pathan,
Vinod Bairagi
Publication year - 2021
Publication title -
neuropharmac journal
Language(s) - English
Resource type - Journals
ISSN - 2456-3927
DOI - 10.37881/1.612
Subject(s) - spinal muscular atrophy , sma* , medicine , food and drug administration , disease , pediatrics , intensive care medicine , pharmacology , mathematics , combinatorics
On May 24, 2019, the U.S. Food and Drug Administration approved Onasemnogene Abeparvovec-xioi (Zolgensma), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. It is priced in the United States at $2.1m (£1.6m; €1.9m) the world’s most expensive drug. Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure. Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival. This review summarized the clinical guidelines for the Onasemnogene Abeparvovec-xioi use and may be useful to healthcare professionals.