Pharma Knowledge Layer · Global Orphan Drug Access Intelligence

Pharma Knowledge Base

Orphan & Rare Drug Database

146 entries

All A B C D E F G H I J K L M N O P Q R S T U V W X Y Z

Orphan Drugs Only

Orphan3dFDA Approved

OrphanFDA Approved (2024)

alglucosidase alfa

Orphan Cold Chain7dFDA Approved, EMA Approved

alpha-L-iduronidase

Orphan Cold Chain14dFDA Approved, EMA Approved

Orphan10dFDA Approved

OrphanStandard of care

Amphotericin B liposomal

Orphan Cold Chain5dFDA Approved, EMA Approved

Orphan Cold Chain14dFDA Approved

Arsenic trioxide

OrphanFDA Approved

Orphan7dFDA Approved, EMA Approved

Orphan21dEMA Approved

atidarsagene autotemcel

Orphan Cold Chain90dEMA Approved

avalglucosidase alfa

Orphan Cold Chain21dFDA Approved (2021), EMA Approved (2021)

Orphan Cold ChainFDA Approved (2024)

Orphan Cold Chain5dFDA Approved

Orphan14dFDA Approved, EMA Approved

Orphan10dFDA Approved

Orphan14dFDA Approved

Beremagene geperpavec

Orphan Cold Chain14dFDA Approved

Orphan14dFDA Approved

Betaine anhydrous

Orphan21dFDA Approved

Orphan10dFDA Approved

Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine)

Orphan Cold Chain2dFDA Approved

Brentuximab vedotin

Orphan Cold Chain7dFDA Approved

Orphan10dFDA Approved

Orphan14dFDA Approved

C1 esterase inhibitor (human)

Orphan Cold Chain7dFDA Approved

Orphan7dFDA Approved

Orphan14dFDA Approved, EMA Approved

Orphan7dFDA Approved, EMA Approved

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