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

Orphan Cold Chain30dFDA Approved (2021), Unregistered in India

OrphanFDA Approved

Orphan Cold ChainFDA Approved (2025)

Orphan Cold ChainFDA Approved

Cold ChainFDA Approved

Cold Chain10dFDA Approved

Growth hormone (somatropin)

Orphan Cold Chain14dFDA Approved

Hematopoietic stem cell transplantation

Orphan Cold Chain90dStandard of care

hormone replacement therapy (estrogen/progesterone)

Human Chorionic Gonadotropin (hCG)

Cold ChainFDA Approved

Hydroxychloroquine

hydroxycobalamin

Orphan14dFDA Approved, EMA Approved

Hypertonic saline

Orphan21dEMA Approved

OrphanFDA Approved

imatinib mesylate

Orphan7dFDA Approved, EMA Approved

Orphan Cold ChainFDA Approved (2024)

Orphan Cold Chain14dFDA Approved

10dFDA Approved

Cold Chain10dFDA Approved

Cold Chain14dFDA Approved

Interferon gamma-1b

Orphan Cold ChainFDA Approved

Interferon-gamma

Orphan Cold Chain14dFDA Approved

Intravenous Immunoglobulin (IVIG)

Orphan Cold ChainFDA Approved

3dFDA Approved, EMA Approved

Orphan14dFDA Approved

Page 5 of 10 (280 total)

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