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OrphanDrug covers global rare drug access intelligence for 193 countries.

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Drug Database

126 entries
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Orphan Drugs Only
Acitretin
Orphan3dFDA Approved
alglucosidase alfa
Orphan Cold Chain7dFDA Approved, EMA Approved
alpha-L-iduronidase
Orphan Cold Chain14dFDA Approved, EMA Approved
ambrisentan
Orphan10dFDA Approved
Amnioreduction
OrphanStandard of care
Amphotericin B liposomal
Orphan Cold Chain5dFDA Approved, EMA Approved
Anakinra
Orphan Cold Chain14dFDA Approved
Arsenic trioxide
OrphanFDA Approved
asciminib
Orphan7dFDA Approved, EMA Approved
Ataluren
Orphan21dEMA Approved
atidarsagene autotemcel
Orphan Cold Chain90dEMA Approved
avalglucosidase alfa
Orphan Cold Chain21dFDA Approved (2021), EMA Approved (2021)
Azacitidine
Orphan Cold Chain5dFDA Approved
baricitinib
Orphan14dFDA Approved, EMA Approved
belumosudil
Orphan10dFDA Approved
Belzutifan
Orphan14dFDA Approved
Beremagene geperpavec
Orphan Cold Chain14dFDA Approved
Berotralstat
Orphan14dFDA Approved
Betaine anhydrous
Orphan21dFDA Approved
bosentan
Orphan10dFDA Approved
Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine)
Orphan Cold Chain2dFDA Approved
Brentuximab vedotin
Orphan Cold Chain7dFDA Approved
brivaracetam
Orphan10dFDA Approved
Busulfan
Orphan14dFDA Approved
C1 esterase inhibitor (human)
Orphan Cold Chain7dFDA Approved
Carboplatin
Orphan7dFDA Approved
carglumic acid
Orphan14dFDA Approved, EMA Approved
Carnitine
Orphan7dFDA Approved, EMA Approved
Cenegermin
Orphan Cold Chain21dFDA Approved
cerliponase alfa
Orphan Cold Chain30dFDA Approved, EMA Approved
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