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

Orphan Cold Chain21dFDA Approved

cerliponase alfa

Orphan Cold Chain30dFDA Approved, EMA Approved

OrphanFDA Approved (2025)

Orphan7dFDA Approved

OrphanFDA Approved

copper histidinate

Orphan Cold Chain14dFDA Approved (Orphan)

Orphan Cold ChainFDA Approved

Orphan Cold Chain14dFDA Approved

Orphan10dFDA Approved

cyclosporine ophthalmic

Orphan5dFDA Approved

OrphanFDA Approved

cysteamine bitartrate

Orphan Cold Chain14dFDA Approved

OrphanFDA Approved

Orphan5dFDA Approved, EMA Approved

Orphan7dFDA Approved

delandistrogene moxeparvovec-rokl

Orphan Cold ChainFDA Approved

Orphan7dFDA Approved

Orphan14dFDA Approved, EMA Approved

diazoxide choline

OrphanFDA Approved (2025)

Digoxin Immune Fab

Orphan Cold Chain7dFDA Approved

Orphan Cold Chain14dFDA Approved, EMA Approved

Orphan7dFDA Approved

efgartigimod alfa

Orphan Cold Chain14dFDA Approved (2021), EMA Approved (2022), Japan Approved (2022)

eladocagene exuparvovec

Orphan Cold Chain45dEMA Approved (2022)

Orphan10dFDA Approved, EMA Approved

Orphan Cold Chain21dFDA Approved (2023), EMA Approved (2024)

Orphan Cold Chain14dFDA Approved

eribulin mesylate

Orphan Cold Chain14dFDA Approved

Orphan Cold Chain14dFDA Approved

Orphan Cold ChainFDA Approved

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