1. Pink Sheet
  2. >>Product Reviews
  3. >>EU CHMP

EMA Considers Whether Intrathecal Zolgensma Deserves Fast Tracking

 
• By Neena Brizmohun

Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.

Preparation of patient before performing spinal anaesthesia or Lumbar puncture
An intrathecal formulation of Zolgensma has been developed for use in older patients (Shutterstock)
Key Takeaways
  • Novartis has asked the European Medicines Agency to grant an accelerated assessment for its planned EU marketing application for OAV101 IT, an intrathecal formulation of its gene therapy, Zolgensma, that targets older patients with spinal muscular atrophy (SMA).
  • New safety and efficacy data supports the use of OAV101 IT in a broad population of patients with SMA aged two to under 18 years old.
  • An approval for the intrathecal formulation is expected boost the sales of Zolgensma.

Novartis could soon learn whether the European Medicines Agency will agree to fast track its review of the company’s planned EU marketing application for an intrathecal formulation of the

OAV101 IT (onasemnogene abeparvovec) targets older patients, who are not eligible for treatment with intravenous (IV) Zolgensma, which was approved in

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from EU CHMP

EU Moment Of Truth For Obe-Cel And 10 Other New Medicines

 
• By Neena Brizmohun

Autolus’s CAR T therapy obe-cel is among the medicines that are this week due for an opinion by the European Medicines Agency on whether they should be marketed in the EU.

Blenrep’s Revival Gains Steam: EMA Decision Imminent As Japan Approves New Regimen

 
• By Neena Brizmohun

The European Medicines Agency is due to decide whether GSK’s previously approved multiple myeloma drug that was withdrawn from the market in 2022 should be approved again for use in combination with BorDex or PomDex. Meanwhile, Japan today became the second major regulator to approve the Blenrep combinations.

New EU Filings

 
• By Neena Brizmohun

Brensocatib, Insmed's investigational treatment for non-cystic fibrosis bronchiectasis, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

New EU Filings

 
• By Neena Brizmohun

Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

More from Pathways & Standards

Life After Loper: Vaping Case Shows US FDA Retains Considerable Deference

 
• By Craig Bleifer, Royce DuBiner, Max Bogost, Michael Brody, and Allyson Maur

A US Supreme Court ruling in favor of the agency in an e-cigarette case has implications for drugs and other medical products as the justices decided not to disturb the FDA’s ‘change in position’ authority, while also creating a complex new landscape.

US FDA Legislative Lead Calls For User Fee Reforms As Renewal Talks Near

 
• By Sarah Karlin-Smith

Deputy FDA Commissioner Grace Graham acknowledged the importance of user fees, while also calling for restructuring. She also said part of MAHA's mission is to reexamine uses of drugs not supported by data.

US FDA Adcomm To Consider If Genentech’s Columvi Study Generalizable To US

 
• By Sue Sutter

Patients from Asia saw a differential treatment effect versus non-Asian regions in the Phase III multiregional study, with an adverse survival trend seen in the latter group, the FDA said about the lymphoma drug.