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 Chain7dFDA Approved

Orphan Cold Chain7dFDA Approved

Orphan7dFDA Approved

OrphanFDA Approved

Orphan14dFDA Approved, EMA Approved

Orphan5dFDA Approved, EMA Approved

Sodium Phenylbutyrate

OrphanFDA Approved

Somatrogon-ghla

Orphan Cold ChainFDA Approved

Orphan Cold Chain14dFDA Approved

Orphan10dFDA Approved

Orphan7dFDA Approved, EMA Approved

Orphan Cold ChainFDA Approved

Orphan14dFDA Approved

OrphanFDA Approved (2024)

Orphan Cold Chain21dFDA Approved

OrphanFDA Approved

OrphanFDA Approved (2025)

Orphan14dFDA Approved

Orphan21dFDA Approved, EMA Approved

OrphanFDA Approved

vanzacaftor/tezacaftor/deutivacaftor

OrphanFDA Approved (2024)

Orphan14dFDA Approved, EMA Approved

OrphanFDA Approved (2025)

Orphan14dFDA Approved

Orphan Cold Chain21dFDA Approved

Orphan Cold Chain18dFDA Approved (2022), EMA Approved (2022)

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