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Top 30 Orphan Drugs in Global Pharmaceutical Industry:--

Sep 14, 2024

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Here's a list of top 30 orphan drugs to consider developing in India:

1. Hydroxyurea (sickle cell disease)

2. Miglustat (Gaucher's disease)

3. Imiglucerase (Gaucher's disease)

4. Agalsidase alfa (Fabry disease)

5. Laronidase (MPS I)

6. Idursulfase (Hunter syndrome)

7. Elaprase (Hunter syndrome)

8. Naglazyme (MPS VI)

9. Aldurazyme (MPS I)

10. Fabrazyme (Fabry disease)

11. Cerezyme (Gaucher's disease)

12. Myozyme (Pompe disease)

13. Lumizyme (Pompe disease)

14. Kalydeco (cystic fibrosis)

15. Orkambi (cystic fibrosis)

16. Spinraza (spinal muscular atrophy)

17. Zolgensma (spinal muscular atrophy)

18. Brineura (CLN2 disease)

19. Mepsevii (MPS VII)

20. Vimizim (MPS IV A)

21. Kanuma (LAL deficiency)

22. Strensiq (HPP)

23. Crysvita (XLH)

24. Luxturna (inherited blindness)

25. CAR-T cell therapies (various cancers)

26. Tafamidis (ATTR amyloidosis)

27. Voretigene neparvovec (inherited blindness)

28. Defibrotide (VOD)

29. Ravulizumab (aHUS)

30. Eculizumab (aHUS)


These orphan drugs address rare diseases with:


- High unmet medical needs

- Limited treatment options

- Potential for market exclusivity

- Government incentives for development


Please note that this list is not exhaustive, and it's essential to research the Indian market, competition, and regulatory requirements before selecting an orphan drug to develop.

Sep 14, 2024

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