Zolgensma (onasemnogene abeparvovec)
OrphanPRIMEATMPConditionalAdditional monitoring
- Region
- 🇪🇺 EU
- Active substance
- onasemnogene abeparvovec
- Company
- Novartis Europharm Limited
- Status
- Authorised
- Therapeutic area
- Muscular Atrophy, Spinal
- CHMP opinion
- Thu 26 Mar 2020
- EU approval
- Mon 18 May 2020
- Designations
- Orphan, PRIME, ATMP, Conditional, Additional monitoring
- Application
- EMEA/H/C/004750
- ATC
- M09AX09
- Official record
- www.ema.europa.eu
Indication
Zolgensma is indicated for the treatment of: patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
Regulatory milestones
No milestones recorded in the last year.
Same substance in other markets
- 🇪🇺 Itvisma Authorised · Novartis Europharm Limited
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.