Pharma Knowledge Layer · Global Orphan Drug Access Intelligence
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OrphanDrug covers global rare drug access intelligence for 193 countries.

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Orphan & Rare Drug Database

23 entries
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Orphan Drugs Only
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
Mifepristone
OrphanFDA Approved
miglustat
Orphan14dFDA Approved, EMA Approved
minoxidil
5dFDA Approved
Mipomersen
Orphan Cold ChainFDA Approved
mirdametinib
OrphanFDA Approved (2025)
mitapivat
Orphan10dFDA Approved (2022), EMA Approved (2022)
mycophenolate mofetil
3dFDA Approved, EMA Approved
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