Preview

Archives of Pediatrics and Pediatric Surgery

Advanced search

First experience of generic Russian-manufactured nusinersen treatment in pediatric SMA patients

https://doi.org/10.66825/2949-4664-apps-3-4-36-42

Abstract

Generic drugs manufactured in Russia are increasingly being used in clinical practice. This article describes the experience of physicians in the Moscow Oblast of Russia in transitioning children with spinal muscular atrophy (SMA) from the reference formulation of nusinersen to its generic equivalent. We present a clinical case involving a child with type 2 SMA who initially received pathogenetic therapy with the reference nusinersen and was subsequently switched to the Russian-manufactured generic formulation. Improvements in motor function observed during treatment, as assessed by standardized functional motor scales, are reported. As a result, the Russian generic nusinersen demonstrated a favorable safety profile.

About the Authors

A. A. Smirnova
Research Clinical Institute of Childhood
Russian Federation

Aleksandra A. Smirnova, Cand. Sci. (Med.), Neurologist, Head, 2nd Psychoneurological department

24A, bldg. 1, Kominterna str., Mytishchi, 141009


Competing Interests:

The authors declare no conflict of interest.



M. A. Shvedova
Research Clinical Institute of Childhood
Russian Federation

Marina A. Shvedova, Neurologist, Head, Department of neurology and Medical Rehabilitation

24A, bldg. 1, Kominterna str., Mytishchi, 141009


Competing Interests:

The authors declare no conflict of interest.



References

1. Clinical Guidelines: 5q Proximal Spinal Muscular Atrophy, Chil-dren; 2023. (In Russ.). https://cr.minzdrav.gov.ru/schema/780_1.

2. Artemieva S.B., Kuzenkova L. M., Ilyina E. S., Kursakova Yu.A., Kolpakchi L. M., Sapego E.Yu., et al. The efficacy and safe-ty of Nusinersen within the expanded access program in Russia. Nervno-myshechnye bolezni. 2020;10(3):35–41. (In Russ.). doi: 10.17650/2222-8721-2020-10-3-35-41. https://elibrary.ru/ouh-juq.

3. The path from theory to practice in the diagnosis and treatment of patients with spinal muscular atrophy. Russkii zhurnal detskoi nevrologii. 2024;19(2):80–88. (In Russ.). doi: 10.17650/2073-8803-2024-19-2-80-88.

4. Biogen’s press release. https://investors.biogen.com/news-releases/news-release-details/us-fda-approves-biogens-spinrazatm-nusinersen-first-treatment (In Russ.).

5. Highlights of prescribing information: Spinraza (nusinersen) injection, for intrathecal use. https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/209531lbl.pdf.

6. General characteristics of the medicinal product Spinraza LP-No. (005833)-(RG-RU); 2024. https://lk.regmed.ru/Regis-ter/EAEU_SmPC (In Russ.).

7. New Data at Cure SMA 2021 Highlight the Long-Term Efficacy of SPINRAZA(r) (nusinersen) and Biogen’s Commitment to Innovation in SMA Therapy. https://investors.biogen.com/news-releases/news-release-details/new-data-cure-sma-2021-high-light-long-termefficacy-spinrazar (available at: 15.06.2025).

8. General characteristics of the medicinal product Avrisdi LP-No. (008925)-(RG-RU); 2025. https://lk.regmed.ru/Regis-ter/EAEU_SmPC (In Russ.).

9. FDA Approves Oral Treatment for Spinal Muscular Atrophy. https://fda.gov/news-events/press-announcements/fda-ap-proves-oral-treatment-spinal-muscular-atrophy

10. General characteristics of the medicinal product Zolgensma LP-No. (001462)-(RG-RU); 2022. (In Russ.). https://lk.regmed.ru/Regis-ter/EAEU_SmPC.

11. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. https://fda.gov/news-events/press-an-nouncements/fda-approves-innovative-gene-therapy-treat-pediat-ricpatients-spinal-muscular-atrophy-rare-disease.

12. Komarova E.A., Kotov A. S. Pathogenetic therapy in children with spinal muscular atrophy. Russkii Meditsinskii Zhurnal. 2025;(4):26–30. doi: 10.32364/2225-2282-2025-4-5. (In Russ.). https://elibrary.ru/diezhm.

13. General characteristics of the medicinal product Lantesens LP-No. (005199)-(RG-RU); 2024. (In Russ.). https://lk.regmed.ru/Regis-ter/EAEU_SmPC.

14. Federal Law on the Circulation of Medicines No. 61; 2010. (In Russ.).

15. Decision of the Council of the Eurasian Economic Commission No. 78 «On the Rules for Registration and examination of medicines for medical use”; 2016. (In Russ.).

16. Decision of the Council of the Eurasian Economic Commission No. 85 «On approval of the Rules for conducting Bioequivalence studies of medicines within the framework of the Eurasian Economic Union”; 2016. (In Russ.).

17. Expert report of the Federal State Budgetary Institution «Scientific Center for Expertise of Medical Devices” of the Ministry of Health of the Russian Federation on the assessment of safety, efficacy and quality of the medicinal product Lantesens(r). Own data of JSC Generium. (In Russ.).

18. Mailman M. D. et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet. Med. 2002;4(1):20–26.

19. Calucho M. et al. Correlation between SMA type and SMN2 copy number revisited: An analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases. Neuromuscul. Disord. Elsevier. 2018;28(3):208–215.

20. Russian Pharmaceutical Market; 2025. (In Russ.). https://delprof.ru/press-center/open-analytics/farmatsevticheskiy-rynok-rossii/.


Review

For citations:


Smirnova A.A., Shvedova M.A. First experience of generic Russian-manufactured nusinersen treatment in pediatric SMA patients. Archives of Pediatrics and Pediatric Surgery. 2025;3(4):36-42. (In Russ.) https://doi.org/10.66825/2949-4664-apps-3-4-36-42

Views: 20

JATS XML


Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 License.


ISSN 2949-4664 (Print)
ISSN 3033-6783 (Online)