Pharma Knowledge Layer · Global Orphan Drug Access Intelligence

Pharma Knowledge Base

Orphan & Rare Drug Database

280 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

Orphan14dFDA Approved, EMA Approved

diazoxide choline

OrphanFDA Approved (2025)

diethylcarbamazine

7dFDA Approved (limited availability)

Digoxin Immune Fab

Orphan Cold Chain7dFDA Approved

10dEMA Approved

Cold Chain7dFDA Approved, EMA 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

emollients with keratolytics

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

Orphan Cold Chain14dFDA Approved

Cold Chain30dFDA Approved, Limited Global Availability

eribulin mesylate

Orphan Cold Chain14dFDA Approved

Orphan Cold Chain14dFDA Approved

3dFDA Approved, EMA Approved

Cold Chain10dFDA Approved

Orphan Cold ChainFDA Approved

OrphanFDA Approved

fidanacogene elaparvovec

Orphan Cold ChainFDA Approved (2024)

Orphan Cold ChainFDA Approved

Orphan Cold Chain10dFDA Approved

Follitropin alfa

Cold ChainFDA Approved

Page 4 of 10 (280 total)

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