IP Library › Granted Patent US 12,419,937
Granted Patent B2
US 12,419,937 · App. 17/699,910 · Granted Sep 23, 2025

High concentration alpha-glucosidase compositions for the treatment of Pompe disease

Inventors: Kenneth Valenzano (East Brunswick, NJ); John Crowley (Princeton, NJ); Richie Khanna (Somerset, NJ); John Flanagan (East Windsor, NJ)
Assignee: Amicus Therapeutics, Inc.
A61K38/47A61K9/0019A61K31/445A61K31/7008A61K47/22C12N9/2408C12N9/96C12Y302/0102
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Quick Facts
Patent No.
US 12,419,937
App. No.
17/699,910
Granted
Sep 23, 2025
Kind
B2
Abstract

The present application provides for compositions comprising high concentrations of acid α-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.

Claims (25)

1. A method of treating Pompe disease in a subject comprising administering

(i) a first composition comprising n-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof, and

(ii) a second composition comprising acid α-glucosidase, wherein the acid α-glucosidase is formulated at a concentration of about 5 mg/ml to about 80 mg/mL in the second composition, and

wherein the n-butyl-deoxynojirimycin and the acid α-glucosidase is administered in an amount effective for treating Pompe disease in the subject.

2. The method of claim 1 , wherein the acid α-glucosidase is administered in an amount of about 5 mg/kg to about 50 mg/kg.

3. The method of claim 2 , wherein the acid α-glucosidase is administered in an amount of about 10 mg/kg to about 40 mg/kg.

4. The method of claim 3 , wherein the acid α-glucosidase is administered in an amount of about 15 mg/kg to about 25 mg/kg.

5. The method of claim 4 , wherein the acid α-glucosidase is administered in an amount of about 20 mg/kg.

6. The method of claim 1 , wherein the second composition further comprises a buffer.

7. The method of claim 6 , wherein the buffer is selected from the group consisting of citrate buffer, acetate buffer, bicarbonate buffer, phosphate buffer, and combination thereof.

8. The method of claim 1 , wherein the acid α-glucosidase is formulated at a concentration of about 15 mg/kg in the second composition.

9. The method of claim 1 , wherein at least one of the first composition and the second composition further comprises an excipient.

10. The method of claim 1 , wherein the first composition is administered orally, and the second composition is administered intravenously.

11. A method of treating Pompe disease in a subject comprising administering

(i) a first composition comprising n-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof, and

(ii) a second composition comprising acid α-glucosidase,

wherein the acid α-glucosidase is formulated at a concentration of about 15 mg/mL in the second composition; and

wherein the acid α-glucosidase is administered in an amount of about 5 mg/kg to about 50 mg/kg.

12. The method of claim 11 , wherein the acid α-glucosidase is administered in an amount of about 15 mg/kg to about 25 mg/kg.

13. The method of claim 12 , wherein the acid α-glucosidase is administered in an amount of about 20 mg/kg.

14. The method of claim 11 , wherein the second composition further comprises a buffer.

15. The method of claim 14 , wherein the buffer is selected from a group consisting of citrate buffer, acetate buffer, bicarbonate buffer, phosphate buffer, and combinations thereof.

16. The method of claim 11 , wherein the acid α-glucosidase is administered in an amount of about 10 mg/kg to about 40 mg/kg.

17. The method of claim 11 , wherein at least one of the first composition and the second composition further comprises an excipient.

18. The method of claim 11 , wherein the first composition is administered orally, and the second composition is administered intravenously.

Assignments (5)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 20, 2023
From: VALENZANO, KENNETH; CROWLEY, JOHN; KHANNA, RICHIE; FLANAGAN, JOHN
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 062441/0259 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 11, 2022
From: MITCHELL, THOMAS A; CORL, JONAS D.
To: INC., WAVEFRONT R
Reel/Frame 060476/0813 →
Continuity (7)
Continuation 16580541 · Sep 24, 2019
Continuation 16015556 · Jun 22, 2018
Division 15192137 · Jun 24, 2016
Continuation 14379131
Provisional Application 61750718 · Jan 9, 2013
Provisional Application 61607920 · Mar 7, 2012
Related Publication 20220347272A1 · Nov 3, 2022
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