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September 4, 2026BiomedicinesOpen Access

Comparative Analysis of SMN2 Splicing Activity and Protein Production Following In Vitro Treatment with the Generic Risdiplam Drug Vapromin® and the Reference Drug Evrysdi®

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Population

Primary dermal fibroblasts derived from 3 spinal muscular atrophy patients and a constructed reporter cell…

Comparison

Generic risdiplam (Vapromin) in vitro treatment vs Reference risdiplam (Evrysdi) in vitro treatment

Design

Preclinical

Key result

In vitro treatment with generic risdiplam (Vapromin) demonstrated comparable biological activity to the reference drug (Evrysdi), with both achieving a 2.5-fold increase in SMN2 full-length transcripts.

Authors

OSO.M. StrizhakovaАПА. С. ПершинYBYana Bahareva

Discussion

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Member takes

Overview

Generic risdiplam demonstrates comparable in vitro splicing activity; leaves open clinical bioequivalence and batch interchangeability in SMA.

Structured PICO

P
Population
In vitro study utilizing primary dermal fibroblasts derived from 3 SMA patients and a reporter cell line to compare the biological activity of generic and reference risdiplam.
I
Intervention
Generic risdiplam (Vapromin) in vitro treatment
C
Comparator
Reference risdiplam (Evrysdi) in vitro treatment
O
Outcome
Increases in SMN protein production, relative transcription level of SMN2 mRNA, and Exon 7 inclusion efficiencysurrogate

The in vitro biological activity of the generic risdiplam Vapromin is comparable to the reference drug Evrysdi in SMA patient-derived fibroblasts and reporter cell lines.

Limitations

  • Did not assess the bioequivalence of the medicinal products for the purpose of comparing biopharmaceutical quality
  • Further research should be conducted to confirm bioequivalence between the two products
  • Did not assess bioequivalence of the medicinal products for the purpose of comparing biopharmaceutical quality

Cite This Study

Strizhakova et al. (2026) studied Spinal muscular atrophy (SMA) (n=3). Vapromin (generic risdiplam) vs. Evrysdi (reference risdiplam) was evaluated on Relative transcription level of SMN2 mRNA and SMN protein production. In vitro treatment with generic risdiplam (Vapromin) demonstrated comparable biological activity to the reference drug (Evrysdi), with both achieving a 2.5-fold increase in SMN2 full-length transcripts.

synapsesocial.com/papers/6a9cc33cc6168cc7c6979da0https://doi.org/10.3390/biomedicines14091994
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Risdiplam in Presymptomatic Spinal Muscular Atrophy2025
  2. 2Safety of Risdiplam in Japanese Patients with Spinal Muscular Atrophy: A 12‑Month Interim Analysis of a Postmarketing Surveillance Study2025
  3. 3Longitudinal efficacy of risdiplam treatment in Chinese children with spinal muscular atrophy2025
  4. 4Overcoming bioanalytical challenges during the development of risdiplam for the treatment of spinal muscular atrophy2025
  5. 5Neurofilaments as Biomarkers of the Efficacy of Risdiplam Treatment in Early SMA Phenotypes Diagnosed by Newborn Screening2025