Claims for Patent: 12,472,172

Summary for Patent: 12,472,172

Title:	Lomitapide for use in methods of treating hyperlipidemia and hypercholesterolemia in pediatric patients
Abstract:	Provided herein are methods of treating hyperlipidemia or hypercholesterolemia in pediatric patients with lomitapide or a pharmaceutically acceptable salt thereof.
Inventor(s):	Ruth NALLEN, Tracy CUNNINGHAM, Mark SUMERAY
Assignee:	Amryt Pharmaceuticals Inc
Application Number:	US18/489,542
Patent Claims:	1. A method of treating homozygous familial hypercholesterolemia (HoFH) in a pediatric patient aged 5 to 10 years in need thereof, comprising administering lomitapide or a pharmaceutically acceptable salt thereof to the patient, wherein said administration comprises: (i) administering an initial dose of 2 mg per day of lomitapide, or a pharmaceutically acceptable salt thereof, to the patient for about 8 weeks; (i) titrating the initial dose to a second dose, wherein the second dose is 5 mg per day of lomitapide, or pharmaceutically acceptable salt thereof, and wherein the second dose is administered for about 4 weeks; (iii) titrating the second dose to a third dose, wherein the third dose is 10 mg per day of lomitapide, or pharmaceutically acceptable salt thereof, and wherein the third dose is administered for about 4 weeks; and (iv) titrating the third dose up to a maximum daily dose of 20 mg per day of lomitapide, or pharmaceutically acceptable salt thereof. 2. The method of claim 1, wherein the administration provides a reduction in the patient's low-density lipoprotein (LDL-C) compared to prior to the administration. 3. The method of claim 1, wherein the administration provides a reduction in the patient's LDL-C to less than 135 mg/dL. 4. The method of claim 1, wherein the administration provides a reduction in the patient's total cholesterol (TC), apolipoprotein B (apo B), non-high-density lipoprotein cholesterol (non-HDL-C), very low-density lipoprotein cholesterol (VLDL-C), triglyceride, and high-density lipoprotein cholesterol (HDL-C) compared to prior to the administration. 5. The method of claim 1, wherein the patient is on a stable lipid-lowering therapy. 6. The method of claim 1, further comprising administering to the patient vitamin E, alpha-linolenic acid, linoleic acid, eicosapentaenoic acid, and docosahexaenoic acid. 7. The method of claim 1, wherein the lomitapide or pharmaceutically acceptable salt thereof is a lomitapide mesylate. 8. The method of claim 7, wherein the lomitapide mesylate is encapsulated in a capsule. 9. The method of claim 1, wherein the patient has at least 2 mutant alleles at the LDLR, apo B, PCSK9, or LDLRAP1 gene locus. 10. The method of claim 1, wherein the patient has an untreated LDL-C level of greater than 500 mg/dL and cutaneous or tendon xanthoma before 10 years of age or untreated LDL-C level consistent with heterozygous familial hypercholesterolemia in both parents. 11. The method of claim 1, wherein the patient has a treated LDL-C level of at least 300 mg/dL and cutaneous or tendon xanthoma before 10 years of age or untreated LDL-C level consistent with heterozygous familial hypercholesterolemia in both parents. 12. The method of claim 1, wherein the patient has no documented cardiovascular disease and LDL-C level of greater than 160 mg/dL prior to the administration. 13. The method of claim 1, wherein the patient has established cardiovascular disease and LDL-C level of greater than 130 mg/dL prior to the administration. 14. A method of treating homozygous familial hypercholesterolemia (HoFH) in a pediatric patient aged 11 to 15 years in need thereof, comprising administering lomitapide or a pharmaceutically acceptable salt thereof to the patient, wherein said administration comprises: (i) administering an initial dose of 2 mg per day of lomitapide, or a pharmaceutically acceptable salt thereof, to the patient for about 4 weeks; (ii) titrating the initial dose to a second dose, wherein the second dose is 5 mg per day of lomitapide, or pharmaceutically acceptable salt thereof, and wherein the second dose is administered for about 4 weeks; (iii) titrating the second dose to a third dose, wherein the third dose is 10 mg per day of lomitapide, or pharmaceutically acceptable salt thereof, and wherein the third dose is administered for about 4 weeks; and (iv) titrating the third dose to a fourth dose, wherein the fourth dose is 20 mg per day of lomitapide, or pharmaceutically acceptable salt thereof, and wherein the fourth dose is administered for about 4 weeks; and (v) titrating the fourth dose up to a maximum daily dose of 40 mg per day. 15. The method of claim 14, wherein the patient is on a stable lipid-lowering therapy. 16. The method of claim 14, further comprising administering to the patient vitamin E, alpha-linolenic acid, linoleic acid, eicosapentaenoic acid, and docosahexaenoic acid. 17. The method of claim 14, wherein the lomitapide or pharmaceutically acceptable salt thereof is a lomitapide mesylate. 18. The method of claim 17, wherein the lomitapide mesylate is encapsulated in a capsule. 19. The method of claim 14, wherein the patient has at least 2 mutant alleles at the LDLR, apo B, PCSK9, or LDLRAP1 gene locus. 20. The method of claim 14, wherein the patient has an untreated LDL-C level of greater than 500 mg/dL and cutaneous or tendon xanthoma before 10 years of age or untreated LDL-C level consistent with heterozygous familial hypercholesterolemia in both parents. 21. The method of claim 14, wherein the patient has a treated LDL-C level of at least 300 mg/dL and cutaneous or tendon xanthoma before 10 years of age or untreated LDL-C level consistent with heterozygous familial hypercholesterolemia in both parents. 22. The method of claim 14, wherein the patient has no documented cardiovascular disease and LDL-C level of greater than 160 mg/dL prior to the administration. 23. The method of claim 14, wherein the patient has established cardiovascular disease and LDL-C level of greater than 130 mg/dL prior to the administration. 24. A method of treating hyperlipidemia in a pediatric patient aged 5 to 10 years in need thereof, comprising administering lomitapide or a pharmaceutically acceptable salt thereof to the patient, wherein said administration comprises: (i) administering an initial dose of 2 mg per day of lomitapide, or a pharmaceutically acceptable salt thereof, to the patient for about 8 weeks; (ii) titrating the initial dose to a second dose, wherein the second dose is 5 mg per day of lomitapide, or pharmaceutically acceptable salt thereof, and wherein the second dose is administered for about 4 weeks; (iii) titrating the second dose to a third dose, wherein the third dose is 10 mg per day of lomitapide, or pharmaceutically acceptable salt thereof, and wherein the third dose is administered for about 4 weeks; and (iv) titrating the third dose up to a maximum daily dose of 20 mg per day of lomitapide, or pharmaceutically acceptable salt thereof.