Pharma Knowledge Layer · Global Orphan Drug Access Intelligence
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OrphanDrug covers global rare drug access intelligence for 193 countries.

OrphanDrug

The Global Orphan Drug Knowledge Base

Authoritative intelligence on rare drug access, regulatory pathways, and named patient programs across 195 countries.

280
Drugs
9,491
Diseases
195
Countries
229
Pathways
214
Programs
2
Suppliers

Drug Index — Browse A to Z

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Knowledge Base

Drugs

280

Rare & specialty drug profiles with global availability data

Diseases

9,491

Rare disease index with ICD/MeSH codes and linked drugs

Countries

195

Regulatory frameworks for 195 countries worldwide

Regulatory Pathways

229

Named patient, compassionate use, and early access pathways

Access Programs

214

Structured access programs by type and region

Suppliers

2

Authorized distributors and access partners

Recently Added Drug Profiles

acoramidis
OrphanFDA Approved (2024)
olezarsen
OrphanFDA Approved (2024)
imetelstat
OrphanFDA Approved (2024)
tovorafenib
OrphanFDA Approved (2024)
remestemcel-L
OrphanFDA Approved (2024)
fidanacogene elaparvovec
OrphanFDA Approved (2024)
marstacimab
OrphanFDA Approved (2024)
vanzacaftor/tezacaftor/deutivacaftor
OrphanFDA Approved (2024)
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