Rare Disease
12 FDA-approved drugs
Recent Approvals
POMBILITI
CIPAGLUCOSIDASE ALFA-ATGA
POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-o...
2023-09-28
AMICUS THERAP US
OPFOLDA
MIGLUSTAT
OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-o...
2023-09-28
AMICUS THERAP US
AUVELITY
BUPROPION HYDROCHLORIDE
major depressive disorder (MDD) in adults...
2022-08-18
AXSOME
YARGESA
MIGLUSTAT
YARGESA is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult pat...
2020-08-06
EDENBRIDGE PHARMS
MIGLUSTAT
MIGLUSTAT
Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult p...
2018-04-17
NAVINTA LLC
DEXTROMETHORPHAN HYDROBROMIDE AND QUINIDINE SULFATE
DEXTROMETHORPHAN HYDROBROMIDE
pseudobulbar affect (PBA)...
2017-10-10
HETERO LABS LTD III
NUEDEXTA
DEXTROMETHORPHAN HYDROBROMIDE
pseudobulbar affect (PBA)...
2010-10-29
AVANIR PHARMS
VPRIV
VELAGLUCERASE ALFA
long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease...
2010-02-26
Takeda
ZAVESCA
MIGLUSTAT
ZAVESCA is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult pat...
2003-07-31
ACTELION
LEVORPHANOL TARTRATE
LEVORPHANOL TARTRATE
the management of pain severe enough to require an opioid analgesic and for which alternative treatm...
2000-03-31
ACERTIS PHARMS
BUTORPHANOL TARTRATE
BUTORPHANOL TARTRATE
the management of pain severe enough to require an opioid analgesic and for which alternative treatm...
1998-08-12
Hikma
BUTORPHANOL TARTRATE PRESERVATIVE FREE
BUTORPHANOL TARTRATE
the management of pain severe enough to require an opioid analgesic and for which alternative treatm...
1997-01-22
Pfizer
Top Companies
Drug Classes
Glucosylceramide Synthase Inhibitor 4
Hydrolytic Lysosomal Glucocerebroside-specific Enzyme 1
Quick Stats
- Total Drugs
- 12
- Companies
- 11
- Drug Classes
- 2
- Latest Approval
- 2023-09-28