Onasemnogene abeparvovec

Onasemnogene abeparvovec
Gene therapy
Target geneSMN1
VectorAdeno-associated virus serotype 9
Clinical data
Trade namesZolgensma
Other namesAVXS-101, onasemnogene abeparvovec-xioi
AHFS/Drugs.comProfessional Drug Facts
License data
Pregnancy
category
Routes of
administration		Intravascular
ATC code
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
DrugBank
UNII
KEGG

Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA),[6][7] a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein.[6] It works by providing a new copy of the SMN gene that produces the SMN protein.[6]

SMA stems from an SMN1 gene mutation, causing SMN protein deficiency vital for motor neuron survival. Onasemnogene abeparvovec, a biologic drug utilizing AAV9 virus capsids containing an SMN1 transgene, is administered to motor neurons, boosting SMN protein levels. Common side effects include vomiting and elevated liver enzymes, while more severe reactions involve liver issues and low platelet count.[6][8]

Developed by AveXis and acquired by Novartis, onasemnogene abeparvovec gained various FDA designations and approvals globally. Controversies included data manipulation concerns and delayed reporting to regulatory agencies. Onasemnogene abeparvovec's price is high, earning it the title of the world's most expensive medication at the time of commercial approval.[10] This has later been exceeded by other gene therapies like Hemgenix. Japan negotiated a lower price for Zolgensma for its public healthcare system.[11][12]

  1. ^ a b "Zolgensma". Therapeutic Goods Administration (TGA). 10 March 2021. Archived from the original on 9 September 2021. Retrieved 8 September 2021.
  2. ^ a b "AusPAR: Onasemnogene abeparvovec". Therapeutic Goods Administration (TGA). 22 April 2021. Archived from the original on 9 September 2021. Retrieved 8 September 2021.
  3. ^ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
  4. ^ "Summary Basis of Decision (SBD) for Zolgensma". Health Canada. 23 October 2014. Archived from the original on 30 May 2022. Retrieved 29 May 2022.
  5. ^ "Zolgensma 2 x 1013 vector genomes/mL solution for infusion - Summary of Product Characteristics (SmPC)". (emc). 30 June 2020. Archived from the original on 28 October 2020. Retrieved 30 July 2020.
  6. ^ a b c d e "Zolgensma- onasemnogene abeparvovec-xioi kit full prescribing information". DailyMed. 24 May 2019. Archived from the original on 19 November 2019. Retrieved 18 November 2019.
  7. ^ a b Cite error: The named reference FDA Zolgensma was invoked but never defined (see the help page).
  8. ^ a b "Zolgensma EPAR". European Medicines Agency (EMA). 24 March 2020. Archived from the original on 17 July 2020. Retrieved 30 July 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  9. ^ "Zolgensma Product information". Union Register of medicinal products. Retrieved 3 March 2023.
  10. ^ Cite error: The named reference pharmanews18401 was invoked but never defined (see the help page).
  11. ^ Cite error: The named reference :1 was invoked but never defined (see the help page).
  12. ^ Cite error: The named reference :2 was invoked but never defined (see the help page).