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

Orphan3dFDA Approved

OrphanFDA Approved (2024)

Cold Chain14dFDA Approved

Cold Chain7dFDA Approved, EMA Approved

Alendronate Sodium

7dFDA Approved, EMA Approved

alglucosidase alfa

Orphan Cold Chain7dFDA Approved, EMA Approved

2dFDA Approved, EMA Approved

alpha-L-iduronidase

Orphan Cold Chain14dFDA Approved, EMA Approved

Orphan10dFDA Approved

OrphanStandard of care

Amphotericin B liposomal

Orphan Cold Chain5dFDA Approved, EMA Approved

Orphan Cold Chain14dFDA Approved

Angiotensin-Converting Enzyme Inhibitors (Enalapril)

2dFDA Approved, EMA Approved

Arsenic trioxide

OrphanFDA Approved

Artificial tears

Artificial tears (Hypromellose)

Orphan7dFDA Approved, EMA Approved

1dFDA Approved, EMA Approved

Orphan21dEMA Approved

atidarsagene autotemcel

Orphan Cold Chain90dEMA Approved

atropine sulfate

avalglucosidase alfa

Orphan Cold Chain21dFDA Approved (2021), EMA Approved (2021)

Orphan Cold ChainFDA Approved (2024)

Orphan Cold Chain5dFDA Approved

3dFDA Approved, EMA Approved

Orphan14dFDA Approved, EMA Approved

Orphan10dFDA Approved

Orphan14dFDA Approved

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