Russia was the first to decide on Itvisma funding for SMA
Published 05 October 2026
Last update 07 October 2026
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On 29 September, the expert council of the "Circle of Good" foundation approved the inclusion of Itvisma (onasemnogene abeparvovec) in the list of drugs for procurement.
The foundation will be able to provide the drug to children with spinal muscular atrophy (SMA) aged 2 years and older with biallelic mutations in the SMN1 gene who have not previously received pathogenetic therapy.
The drug is not yet registered in Russia.
Itvisma was first authorised in the United States in November 2025, and on 30 June 2026 it received approval for use in the EU for patients with SMA aged 2 years and older.
How Itvisma Differs from Zolgensma
Itvisma contains the same active substance as Zolgensma and is also a single-dose gene replacement therapy.
However, unlike Zolgensma, this form of the drug can be used in patients over two years of age.
The main difference lies in the route of administration.
Zolgensma is administered intravenously, and the dose of the viral vector is calculated depending on body weight. As the patient's weight increases, the required total vector dose and systemic exposure increase, which limits the use of the intravenous form in older patients.
Itvisma is administered once intrathecally — directly into the cerebrospinal fluid. This allows for the use of a significantly lower fixed vector dose that does not depend on body weight, and makes gene replacement therapy possible in older patients.
Evidence Base
The main evidence base for Itvisma was obtained in the randomised Phase III STEER study.
It involved 126 previously untreated patients with SMA aged 2 to 18 years who were able to sit independently but had never walked independently.
At 52 weeks, motor function on the HFMSE scale improved on average by:
— 2.4 points with Itvisma
— 0.5 points in the sham-control group
It is specifically for previously untreated children that the "Circle of Good" has so far approved funding.
At the same time, the registered indications in the US and EU are broader, but the most robust direct evidence of efficacy today has been obtained specifically in the population of previously untreated patients.
European Context
We have previously written about the first joint clinical assessment of Itvisma in the EU: "The first joint assessment of gene therapy in the EU: clinical effect confirmed, but Ireland does not recommend reimbursement at the stated price".
The joint clinical assessment confirmed the clinical effect of the drug but showed continued uncertainty regarding its comparative efficacy against already used treatments for SMA.
Ireland was the first to use the JCA results in a national assessment and did not recommend Itvisma for reimbursement at the price proposed by the manufacturer.
In other European countries, assessment procedures and determination of funding conditions are ongoing.
In Ireland, the HSE is considering a new commercial offer from the manufacturer.
In Germany, the added benefit assessment began on 1 August; publication of results is expected in November, and the final G-BA decision — in January 2027.
In the United Kingdom, Itvisma is undergoing NICE assessment; there is no final funding decision yet.
Why This Matters
Thus, Russia has become the first country to adopt a positive decision on funding Itvisma.
In the context of a limited evidence base, the subsequent collection and analysis of real-world clinical data becomes particularly important.
They will make it possible to assess the long-term efficacy and safety of therapy in Russian patients and to clarify the place of Itvisma in the treatment of SMA.
Sources:
"Circle of Good" Foundation
European Commission — Itvisma
FDA — Itvisma
G-BA — Nutzenbewertung Itvisma
NICE — Itvisma
The foundation will be able to provide the drug to children with spinal muscular atrophy (SMA) aged 2 years and older with biallelic mutations in the SMN1 gene who have not previously received pathogenetic therapy.
The drug is not yet registered in Russia.
Itvisma was first authorised in the United States in November 2025, and on 30 June 2026 it received approval for use in the EU for patients with SMA aged 2 years and older.
How Itvisma Differs from ZolgensmaItvisma contains the same active substance as Zolgensma and is also a single-dose gene replacement therapy.
However, unlike Zolgensma, this form of the drug can be used in patients over two years of age.
The main difference lies in the route of administration.
Zolgensma is administered intravenously, and the dose of the viral vector is calculated depending on body weight. As the patient's weight increases, the required total vector dose and systemic exposure increase, which limits the use of the intravenous form in older patients.
Itvisma is administered once intrathecally — directly into the cerebrospinal fluid. This allows for the use of a significantly lower fixed vector dose that does not depend on body weight, and makes gene replacement therapy possible in older patients.
Evidence BaseThe main evidence base for Itvisma was obtained in the randomised Phase III STEER study.
It involved 126 previously untreated patients with SMA aged 2 to 18 years who were able to sit independently but had never walked independently.
At 52 weeks, motor function on the HFMSE scale improved on average by:
— 2.4 points with Itvisma
— 0.5 points in the sham-control group
It is specifically for previously untreated children that the "Circle of Good" has so far approved funding.
At the same time, the registered indications in the US and EU are broader, but the most robust direct evidence of efficacy today has been obtained specifically in the population of previously untreated patients.
European ContextWe have previously written about the first joint clinical assessment of Itvisma in the EU: "The first joint assessment of gene therapy in the EU: clinical effect confirmed, but Ireland does not recommend reimbursement at the stated price".
The joint clinical assessment confirmed the clinical effect of the drug but showed continued uncertainty regarding its comparative efficacy against already used treatments for SMA.
Ireland was the first to use the JCA results in a national assessment and did not recommend Itvisma for reimbursement at the price proposed by the manufacturer.
In other European countries, assessment procedures and determination of funding conditions are ongoing.
In Ireland, the HSE is considering a new commercial offer from the manufacturer.
In Germany, the added benefit assessment began on 1 August; publication of results is expected in November, and the final G-BA decision — in January 2027.
In the United Kingdom, Itvisma is undergoing NICE assessment; there is no final funding decision yet.
Why This MattersThus, Russia has become the first country to adopt a positive decision on funding Itvisma.
In the context of a limited evidence base, the subsequent collection and analysis of real-world clinical data becomes particularly important.
They will make it possible to assess the long-term efficacy and safety of therapy in Russian patients and to clarify the place of Itvisma in the treatment of SMA.
Sources:"Circle of Good" Foundation
European Commission — Itvisma
FDA — Itvisma
G-BA — Nutzenbewertung Itvisma
NICE — Itvisma
