Intelligence · INN directory
Rare Disease Drugs Directory
Orphan and rare disease therapies, enzyme replacements, and gene therapies for ultra-rare indications.
Quick answer
Rare disease drugs: NovaPharmaNews indexes 7 pharmaceutical drugs in this therapeutic segment with regulatory status, pipeline programs, patents, and congress coverage cross-links. Orphan and rare disease therapies, enzyme replacements, and gene therapies for ultra-rare indications.
Recently updated
FDA-approved
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dextromethorphan hydrobromide
NUEDEXTA Amneal Pharma Europe Ltd
N07XX59 -
dextromethorphan polistirex
DEXTROMETHORPHAN POLISTIREX Amneal Pharma Europe Ltd
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butorphanol tartrate
BUTORPHANOL TARTRATE
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levallorphan tartrate
LORFAN
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levorphanol tartrate
LEVO-DROMORAN
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miglitol
MIGLITOL
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pancreas powder
ENZEPI
A09AA02
Frequently asked questions
How many rare disease drugs are in the directory?
7 drug profiles match this therapeutic area filter based on class, mechanism, target, and INN metadata.
How are drugs assigned to a therapeutic area?
Classification uses WHO therapeutic class, mechanism of action, molecular target, and INN pattern matching against curated oncology, metabolic, CNS, rare disease, and immunology taxonomies.
Where does regulatory data come from?
Profiles combine FDA Drugs@FDA, EMA EPAR references, ClinicalTrials.gov pipeline links, and editorial enrichment. See our data sources page for attribution and update cadence.