Demystifying Medicine Video



3.7 Million Dollars CAN for a drug? It’s the most expensive medication on the market!! Zolgensma is a single dose medication to relieve the symptoms of Spinal Muscular Atrophy (SMA) in newborns and young children. But how can a drug cost this much? How do scientists even make it? Watch this video to learn about the drug discovery process through the discovery and making of Zolgensma. The drug discovery and manufacturing process is incredibly detailed and extensive.

First, they must Research the specific illness and what known compounds are able to relieve symptoms and potentially lead to a cure. With SMA there is a missing SMN1 gene that leads the child to experience muscle weakness and deterioration. With the knowledge and research gained, the next stage of the drug discovery process is Pre-clinical trials. AAV Therapy was developed as a treatment for SMA and other muscular and nervous system diseases. Specifically, the AAV9 vector can deliver the missing gene to motor neurons in the human body. The success of this medication was tested countless times via the third stage of drug discovery, Clinical Trials. The creators of Zolgensma also held their own clinical trials in children, that they had called START and STR1VE. After all the research and data is collected, the final step is FDA Approval. Zolgensma is currently approved in the United States, Japan and all 27 European nations. Health Canada has yet to approve this drug.

[Please complete our feedback form]




References
  • Atchison, R. W., Casto, B.C., Hammon, W. M. (1966). Electron microscopy of adenovirus-associated virus (AAV) in cell cultures. Virology 29: 353–57 https://www-annualreviews-org.libacce...
  • AveXis. (n.d.). A Caregiver’s Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi). Retrieved from: https://www.zolgensma.com/pdf/a-careg...
  • Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L.R., et al. (2017). Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377: 1713–22. https://www.nejm.org/doi/full/10.1056...
  • Mohs, R. C., & Greig, N. H. (2017). Drug discovery and development: Role of basic biological research. Alzheimer’s and Dementia: Translational Research and Clinical Interventions. Elsevier Inc. https://doi.org/10.1016/j.trci.2017.1...
  • (2020). "Overview of the ZOLGENSMA clinical studies." Retrieved 2020, 2020, from https://www.zolgensma.com/clinical-st....
  • Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., . . . Kaspar, B. K. (2017). Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. N Engl J Med, 377(18), 1713-1722. doi:10.1056/NEJMoa1706198
  • Al-Zaidy, S., et al. (2019). "Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy." Pediatr Pulmonol 54(2): 179-185. Step 4: FDA Drug Review. (2019). Retrieved from https://www.fda.gov/patients/drug-dev...