Rare Disease drug pipeline

Orphan-drug-designated therapeutics. Smaller patient populations, higher per-patient pricing, intense innovation.

Recent approvals (last 30 days)

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Upcoming PDUFA dates (next 6 months)

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Top sponsors

1. Shire · 8 drugs
2. BioMarin Pharmaceutical · 8 drugs
3. Pfizer Inc. · 6 drugs
4. Sanofi · 5 drugs
5. Pfizer · 5 drugs
6. · 5 drugs
7. Alexion Pharmaceuticals, Inc. · 4 drugs
8. Genzyme, a Sanofi Company · 4 drugs
9. CSL Behring · 3 drugs
10. PTC Therapeutics · 3 drugs

Key drug classes

• Enzyme replacement therapy · 16
• Gene therapy · 4
• Antisense Oligonucleotide [EPC] · 4
• Enzyme replacement therapy (ERT) · 3
• Transthyretin stabilizer · 3
• Gene therapy (AAV-based) · 3
• Antimalarial [EPC] · 3
• C1 esterase inhibitor (C1-INH) replacement therapy · 3
• Serine protease inhibitor / Complement regulator · 2
• Antimalarial · 2
• Pyruvate dehydrogenase kinase inhibitor · 2
• Plasma kallikrein inhibitor · 2
• Complement inhibitor · 2
• Glucosylceramide synthase inhibitor · 2
• unknown · 2

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