Itvisma

Opinion

EMA has issued an opinion on this medicine

onasemnogene abeparvovec

Overview

On 23 April 2026, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Itvisma, intended for the treatment of spinal muscular atrophy (SMA). As Itvisma is an advanced therapy medicinal product, the CHMP positive opinion is based on an assessment by the Committee for Advanced Therapies.

The applicant for this medicinal product is Novartis Europharm Limited.

Itvisma will be available as a 1.2 x 10¹⁴ vector genomes solution for injection. The active substance of Itvisma is onasemnogene abeparvovec, a gene therapy (ATC code: M09AX09). Onasemnogene abeparvovec is a non-replicating recombinant viral vector designed to introduce a functional copy of the survival motor neuron gene (SMN1) in the transduced cells to address the monogenic root cause of SMA. By providing an alternative source of SMN protein expression in motor neurons, it is expected to promote the survival and function of transduced motor neurons.

The benefit of Itvisma is an improvement in motor function in children from 2 years of age with SMA who have bi-allelic mutation in the SMN1 gene. The most common side effects with Itvisma include upper respiratory tract infection, pyrexia, vomiting, headache and increased hepatic enzymes.

The full indication is:

Itvisma is indicated for the treatment of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene in patients 2 years of age and older.

Treatment with Itvisma should be initiated and administered in clinical centres and supervised by a physician experienced in the management of patients with SMA.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.

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Note: This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained

CHMP summary of positive opinion for Itvisma

Adopted Reference Number: EMADOC-1829012207-48071

Product details

• Name of medicine:
  Itvisma
• Active substance:
  onasemnogene abeparvovec
• International non-proprietary name (INN) or common name:
  onasemnogene abeparvovec
• Therapeutic area (MeSH):
  Muscular Atrophy, Spinal
• Anatomical therapeutic chemical (ATC) code:
  M09AX09
• EMA product number:
  EMEA/H/C/006498
• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation applicant:
  Novartis Europharm Limited
• Opinion adopted:
  23/04/2026
• Opinion status:
  Positive