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JCA report published

Onasemnogene abeparvovec, intrathecal (Itvisma): EU joint clinical assessment

Patients 2 years and older with 5q spinal muscular atrophy and a bi-allelic SMN1 mutation

  1. Regulatory review
  2. CHMP opinion
  3. EU authorised
  4. JCA report published

Overview

Company
Novartis
Therapy area
Neurology & neuromuscular · Spinal muscular atrophy
Indication
Patients 2 years and older with 5q spinal muscular atrophy and a bi-allelic SMN1 mutation
EC marketing authorisation
30 Jun 2026
JCA report published
7 Sep 2026
JCA assessor
NCPE, Ireland
Co-assessor
HAS, France
Pivotal evidence
STEER (randomised phase 3 vs sham) and STRENGTH (single-arm, treatment-experienced)
01

Scope

From the published report

The JCA report set out 12 PICOs across 6 populations.

Six populations after the claimed indication was narrowed to patients aged 2 and over, which removed two PICOs. Comparators: nusinersen, risdiplam, best supportive care, intravenous onasemnogene abeparvovec and individualised treatment.

JCA report on the European Commission website

02

Evidence

How the evidence covered the scope

Comparative evidence informed 7 of the 12 PICOs. Pro breaks down how the evidence covered each one, and what it means for later submissions.

Included in Pro
03

Countries

National translator and outlook

Ireland and France lead the joint assessment. Pro explains what the JCA means for the national route in each of 31 markets, with 6 national records already in place.

See JCA Pulse ProIncluded in Pro

Next milestones

  • 23 Nov 2026G-BA written comments close · Germany · source
  • End Jan 2027G-BA resolution expected · Germany · source
  • 2 Nov 2026G-BA assessment published · Germany · source
  • 18 Sep 2026NT Council: wait for TLV assessment · Sweden · source
  • 9 Sep 2026HSE CPU: company proposal under review · Ireland · source
  • 7 Sep 2026JCA report published · EU · source
  • 25 Nov 2026NICE guidance expected (ID6556) · United Kingdom · source

Sources

Last checked 5 November 2026. Compiled from public sources; check them for the latest position. Forecasts are Evidaxia’s independent view, not official assessments.

From Evidaxia