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

sunscreen (broad spectrum SPF 50+)

surgical repair

Standard of Care

tacrolimus ophthalmic

10dOff-label use

Orphan10dFDA Approved

tamoxifen citrate

Orphan7dFDA Approved, EMA Approved

Orphan Cold ChainFDA Approved

5dFDA Approved, EMA Approved

Timolol maleate

Cold Chain7dFDA Approved, EMA Approved

Orphan14dFDA Approved

Topical corticosteroids

Total Parenteral Nutrition

Cold Chain5dFDA Approved

OrphanFDA Approved (2024)

Orphan Cold Chain21dFDA Approved

OrphanFDA Approved

OrphanFDA Approved (2025)

Orphan14dFDA Approved

Orphan21dFDA Approved, EMA Approved

Ursodeoxycholic acid

Page 9 of 10 (280 total)

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