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

Currently, Russian-manufactured generic products are being actively implemented into the clinical practice in the Russian Federation, including spinal muscular atrophy treatment. This article presents five clinical cases with various types of spinal muscular atrophy treatment with generic Russian nusinersen, both naive and those who had previously received therapy with the reference drug nusinersen and continued therapy with generic nusinersen. The trend in motor activity in patients during treatment, assessed by motor development scales, is presented. In all of 5 described clinical cases both the positive effect and favorable safety profile of the Russian-manufactured generic nusinersen were demonstrated.

Compliance with ethical standards. All patients or their legal representatives signed voluntary informed consent.

Contributions:
Kuzenkova L.M. — concept and design of the review, writing the text, editing;
Uvakina E.V. — concept and design of the review, writing the text, editing;
Serebrennikova E.B. — writing the text, editing;
Karpovich E.I. — writing the text, editing;
Samofal T.A. — writing the text, editing;
Lapochkin O.L. — writing the text, editing;
Smirnova A.A. — writing the text, editing;
Golenko A.A. — writing the text, editing.
All co-authors — responsibility for the integrity of all parts of the manuscript and approval of its final version.

Funding. The study had no sponsorship.

Conflict of interest. The authors declare no conflict of interest.

Received: July 22, 2025
Accepted: August 5, 2025
Published: August 20, 2025

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

2. Clinical Guidelines: 5q Proximal Spinal Muscular Atrophy, Children; 2023. Available at: https://cr.minzdrav.gov.ru/schema/780_1 (in Russian)

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): 80–8. https://doi.org/10.17650/2073-8803-2024-19-2-80-88 (in Russian)

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

5. Highlights of prescribing information: Spinraza (nusinersen) injection, for intrathecal use. Available at: 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. Available at: https://lk.regmed.ru/Register/EAEU_SmPC (in Russian)

7. New Data at Cure SMA 2021 Highlight the Long-Term Efficacy of SPINRAZA® (nusinersen) and Biogen’s Commitment to Innovation in SMA Therapy. Available at: https://investors.biogen.com/news-releases/news-release-details/new-data-cure-sma-2021-highlight-long-term-efficacy-spinrazar дата обращения 15.06.2025

8. General characteristics of the medicinal product Avrisdi LP-No.(008925)-(RG-RU); 2025. Available at: https://lk.regmed.ru/Register/EAEU_SmPC (in Russian)

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

10. General characteristics of the medicinal product Zolgensma LP-No.(001462)-(RG-RU); 2022. Available at: https://lk.regmed.ru/Register/EAEU_SmPC (in Russian)

11. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. Available at: https://fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-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. https://doi.org/10.32364/2225-2282-2025-4-5 https://elibrary.ru/diezhm (in Russian)

13. General characteristics of the medicinal product Lantesens LP-No.(005199)-(RG-RU); 2024. Available at: https://lk.regmed.ru/Register/EAEU_SmPC (in Russian)

14. Federal Law on the Circulation of Medicines No. 61; 2010. (in Russian)

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 Russian)

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 Russian)

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®. Own data of JSC Generium. (in Russian)

18. According to art. 27.1 of Federal Law No. 61 and the published list of interchangeable drugs on the website of the State Register of Medicines. Available at: http://grls.rosminzdrav.ru/Forum//Files/243339/%D0%9F%D0%B5%D1%80%D0%B5%D1%87%D0%B5%D0%BD%D1%8C_(%D0%BC%D0%B0%D1%80%D1%82_2025).xls (in Russian)

19. Based on data from the Xmark Analytics pharmaceutical data collection and analysis system on movement and balances of medicinal products. Available at: https://app.analytics.xmark.pro/login (accessed 15.06.2025) (In Russian)