OrphanDrug

The Global Orphan Drug Knowledge Base

Authoritative intelligence on rare drug access, regulatory pathways, and named patient programs across 193 countries.

284

Drugs

Drug Index — Browse A to Z

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

Knowledge Base

Drugs

284

Rare & specialty drug profiles with global availability data

Diseases

9,486

Rare disease index with ICD/MeSH codes and linked drugs

Countries

193

Regulatory frameworks for 193 countries worldwide

Regulatory Pathways

218

Named patient, compassionate use, and early access pathways

Access Programs

214

Structured access programs by type and region

Suppliers

Authorized distributors and access partners

Recently Added Drug Profiles

mitapivat

OrphanFDA Approved (2022), EMA Approved (2022)

loncastuximab tesirine

OrphanFDA Approved (2021), EMA Approved (2022)

vutrisiran

OrphanFDA Approved (2022), EMA Approved (2022)

eplontersen

OrphanFDA Approved (2023), EMA Approved (2024)

eladocagene exuparvovec

OrphanEMA Approved (2022)

avalglucosidase alfa

OrphanFDA Approved (2021), EMA Approved (2021)

efgartigimod alfa

OrphanFDA Approved (2021), EMA Approved (2022), Japan Approved (2022)

bosentan

OrphanFDA Approved