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

OrphanFDA Approved

fidanacogene elaparvovec

Orphan Cold ChainFDA Approved (2024)

Orphan Cold ChainFDA Approved

Orphan Cold Chain10dFDA Approved

Orphan Cold Chain30dFDA Approved (2021), Unregistered in India

OrphanFDA Approved

Orphan Cold ChainFDA Approved (2025)

Orphan Cold ChainFDA Approved

Growth hormone (somatropin)

Orphan Cold Chain14dFDA Approved

Hematopoietic stem cell transplantation

Orphan Cold Chain90dStandard of care

hydroxycobalamin

Orphan14dFDA Approved, EMA Approved

Orphan21dEMA Approved

OrphanFDA Approved

imatinib mesylate

Orphan7dFDA Approved, EMA Approved

Orphan Cold ChainFDA Approved (2024)

Orphan Cold Chain14dFDA Approved

Interferon gamma-1b

Orphan Cold ChainFDA Approved

Interferon-gamma

Orphan Cold Chain14dFDA Approved

Intravenous Immunoglobulin (IVIG)

Orphan Cold ChainFDA Approved

Orphan14dFDA Approved

Orphan5dFDA Approved, EMA Approved

Orphan Cold Chain14dFDA Approved

Orphan Cold Chain14dFDA Approved, EMA Approved

Laser photocoagulation therapy

OrphanFDA Approved

OrphanFDA Approved

Orphan21dFDA Approved, EMA Approved

loncastuximab tesirine

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

Orphan Cold ChainFDA Approved, EMA Approved

Orphan Cold Chain10dFDA Approved, EMA Approved

Orphan14dInvestigational

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