Novartis Reports FDA’s Hold on P-I/II Intrathecal Study Evaluating Zolgensma/AVXS-101 (onasemnogene abeparvovec-xioi) for Spinal Muscular Atrophy Type 2

 Novartis Reports FDA’s Hold on P-I/II Intrathecal Study Evaluating Zolgensma/AVXS-101 (onasemnogene abeparvovec-xioi) for Spinal Muscular Atrophy Type 2

Novartis Reports FDA’s Hold on P-I/II Intrathecal Study Evaluating Zolgensma/AVXS-101 (onasemnogene abeparvovec-xioi) for Spinal Muscular Atrophy Type 2

Shots:

  • The partial hold on clinical study for intrathecal administration of AVXS-101 follows the findings of the pre-clinical study demonstrated dorsal root ganglia (DRG) mononuclear cell inflammation accompanied by neuronal cell body degeneration or loss
  • The partial hold does not have any impact on marketed Zolgensma or AVXS-101 (IV) clinical studies and will remain available in the US. Novartis will work with the FDA to release the partial hold and resume dosing in the AVXS-101 intrathecal study
  • Zolgensma/AVXS-101 is an adeno-associated virus vector-based gene therapy indicated to treat pediatric patients with SMA with bi-allelic mutations in the SMN1 gene in the US

Click here to­ read full press release/ article | Ref: Novartis | Image: Livemint

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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