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Last Updated: December 17, 2025

Claims for Patent: 12,350,273

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Summary for Patent: 12,350,273

Title:	Treatment of SMA
Abstract:	The present invention relates to 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one for use in the treatment of spinal muscular atrophy (SMA), its pharmaceutical composition to be used in the treatment of SMA, its methods of treatment thereof.
Inventor(s):	Jean-Paul Pfefen, Heidemarie Kletzl, Lutz Mueller
Assignee:	Hoffmann La Roche Inc
Application Number:	US18/061,985
Patent Claims:	1. A method of treating spinal muscular atrophy (SMA) in a human patient in need thereof, comprising administering to the patient a pharmaceutical composition comprising a compound of formula (I) at a once daily oral dose of 5 mg, wherein the patient has a body weight of more than or equal to 20 kg. 2. The method of claim 1, wherein the patient has a body weight of from 21 kg to 79.5 kg. 3. The method of claim 1, wherein the patient is from 13 to 24 years old. 4. The method of claim 1, wherein the patient is an adult. 5. The method of claim 1, wherein the patient suffers from type II SMA. 6. The method of claim 1, wherein the patient suffers from type III SMA. 7. The method of claim 1, wherein the pharmaceutical composition is administered as an aqueous oral solution. 8. A method of treating spinal muscular atrophy (SMA) in a human patient in need thereof, comprising administering to the patient a pharmaceutical composition comprising a compound of formula (I) at a once daily oral dose of 0.25 mg/kg, wherein the patient has a body weight of less than 20 kg. 9. The method of claim 8, wherein the patient is from 2 to 11 years old. 10. The method of claim 8, wherein the patient suffers from type II SMA. 11. The method of claim 8, wherein the patient suffers from type III SMA. 12. The method of claim 8, wherein the pharmaceutical composition is administered as an aqueous oral solution. 13. The method of claim 8, wherein the patient is more than or equal to 2 years of age. 14. The method of claim 8, wherein the patient suffers from type II SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 15. The method of claim 8, wherein the patient suffers from type III SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 16. The method of claim 8, wherein the patient is more than or equal to 2 years of age, the patient suffers from type II SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 17. The method of claim 8, wherein the patient is more than or equal to 2 years of age, the patient suffers from type III SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 18. The method of claim 1, wherein the patient is more than or equal to 2 years of age. 19. The method of claim 1, wherein the patient is 12 to 25 years of age. 20. The method of claim 1, wherein the patient suffers from type II SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 21. The method of claim 1, wherein the patient suffers from type III SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 22. The method of claim 1, wherein the patient is more than or equal to 2 years of age, the patient suffers from type II SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 23. The method of claim 1, wherein the patient is more than or equal to 2 years of age, the patient suffers from type III SMA, and the pharmaceutical composition is administered as an aqueous oral solution. 24. The method of claim 1, wherein the patient is an adult and suffers from type II SMA. 25. The method of claim 1, wherein the patient is and adult and suffers from type III SMA.

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