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

OrphanFDA Approved

Orphan14dFDA Approved, EMA Approved

Orphan Cold ChainFDA Approved

OrphanFDA Approved (2025)

Orphan10dFDA Approved (2022), EMA Approved (2022)

mycophenolate mofetil

3dFDA Approved, EMA Approved

N-acetylcysteine

5dFDA Approved, EMA Approved

Orphan21dFDA Approved

Orphan Cold ChainFDA Approved

Orphan Cold Chain7dUnregistered in India

Occupational and Physical Therapy Support

14dStandard of Care

Orphan Cold Chain10dFDA Approved

Orphan Cold ChainFDA Approved (2024)

Orphan Cold Chain14dFDA Approved

Onasemnogene abeparvovec

Orphan Cold Chain14dNot registered in India

3dFDA Approved, EMA Approved

Orphan14dFDA Approved

Orphan Cold ChainFDA Approved

Cold ChainFDA Approved

phacoemulsification surgery

14dStandard surgical procedure

physical therapy and orthotic management

Standard of Care

Page 7 of 10 (280 total)

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