DRUG: Lunsotogene parvec
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Entry
D13347 Drug
Name
Lunsotogene parvec (USAN/INN);
Lunsotogene parvec-cwha;
Otarmeni (TN)
Efficacy
Otoferlin production promotor
Disease
Sensorineural hearing loss (molecularly confirmed biallelic variants in the OTOF gene) [DS:
H00605
]
Type
Gene therapy product
Comment
Treatment of OTOF-related congenital hearing loss due to congenital auditory neuropathy
Target
OTOF [HSA:
9381
] [KO:
K19949
]
Brite
New drug approvals in the USA [
br08319.html
]
Cellular and gene therapy products
D13347
New drug approvals in the USA, Europe and Japan [
br08328.html
]
Approval dates by FDA, EMA and PMDA
D13347
Other DBs
CAS:
2907748-12-1
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