Pharma Knowledge Layer · Global Orphan Drug Access Intelligence
OrphanDrug
OrphanDrug
Pharma Knowledge Base
DrugsDiseasesCountriesPathwaysProgramsSuppliersAPI Docs

Navigation

DrugsDiseasesCountriesPathwaysProgramsSuppliersAPI Docs

About

OrphanDrug covers global rare drug access intelligence for 193 countries.

HomeDrugs

Orphan & Rare Drug Database

280 entries
AllABCDEFGHIJKLMNOPQRSTUVWXYZ
Orphan Drugs Only
L-carnitine
Orphan5dFDA Approved, EMA Approved
L-DOPA
FDA Approved
L-Methylfolate
5dFDA Approved
Lanreotide
Orphan Cold Chain14dFDA Approved
laronidase
Orphan Cold Chain14dFDA Approved, EMA Approved
Laser photocoagulation therapy
OrphanFDA Approved
Levetiracetam
3dFDA Approved, EMA Approved
Levodopa/Carbidopa
7dFDA Approved
Lomitapide
OrphanFDA Approved
lonafarnib
Orphan21dFDA Approved, EMA Approved
loncastuximab tesirine
Orphan Cold Chain14dFDA Approved (2021), EMA Approved (2022)
Lorenzo's oil
Lumasiran
Orphan Cold ChainFDA Approved, EMA Approved
luspatercept
Orphan Cold Chain10dFDA Approved, EMA Approved
mannose
Orphan14dInvestigational
mannose-1-phosphate
Orphan Cold Chain30dInvestigational
marstacimab
Orphan Cold ChainFDA Approved (2024)
mavacamten
Orphan14dFDA Approved
mavorixafor
OrphanFDA Approved
mecasermin
Orphan Cold Chain14dFDA Approved, EMA Approved
melatonin
Orphan7dEMA Approved, FDA OTC
Melphalan
7dFDA Approved
Memantine
5dFDA Approved
Mesalamine
7dFDA Approved
Methotrexate
Orphan3dFDA Approved
metoprolol
2dFDA Approved
Metreleptin
Orphan Cold Chain21dFDA Approved
Metronidazole
3dFDA Approved
mexiletine
Orphan14dFDA Approved
Midostaurin
Orphan14dFDA Approved
Page 6 of 10 (280 total)
← PrevNext →
OrphanDrug — Global Pharma Knowledge Base
DrugsDiseasesCountriesPathwaysProgramsSuppliersAPI Docs|Terms of UsePrivacy Policy