Upstaza (eladocagene exuparvovec)
OrphanATMPExceptional circumstancesAdditional monitoring
- Region
- 🇪🇺 EU
- Active substance
- eladocagene exuparvovec
- Company
- PTC Therapeutics International Limited
- Status
- Authorised
- Therapeutic area
- Amino Acid Metabolism, Inborn Errors
- CHMP opinion
- Thu 19 May 2022
- EU approval
- Mon 18 Jul 2022
- Designations
- Orphan, ATMP, Exceptional circumstances, Additional monitoring
- Application
- EMEA/H/C/005352
- ATC
- A16AB26
- Official record
- www.ema.europa.eu
Indication
Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L amino acid decarboxylase (AADC) deficiency with a severe phenotype (see section 5.1).
Regulatory milestones
No milestones recorded in the last year.
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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.