# US 10,441,558 B2 · Therapeutic approaches for treating CMT and related disorders

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- Number: US 10,441,558 B2
- Document ID: US10441558B2
- Status: Granted
- Kind: B2
- Application number: 16/055,710
- Filed: 2018-08-06
- Published: 2018-12-20
- Granted: 2019-10-15
- Earliest claimed: 2007-11-30
- Assignees: Pharnext
- Applicants: Pharnext
- Inventors: Daniel Cohen; Ilya Chumakov; Oxana Guerassimenko; Serguei Nabirochkin
- Web page: https://patentgrep.com/patent/US10441558B2

## CPC

- A61K 31/197: the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid (carnitine A61K31/205)
- A61K 9/0053: {Mouth and digestive tract, i.e. intraoral and peroral administration}
- A61K 9/0095: {Drinks; Beverages; Syrups; Compositions for reconstitution thereof, e.g. powders or tablets to be dispersed in a glass of water; Veterinary drenches (A61K9/0007 takes precedence; eatable gels or foams A61K9/0056; oral mucosa adhesive forms A61K9/006)}
- A61K 9/08: Solutions {(composition of solutions A61K47/00)}
- A61K 9/4866: {Organic macromolecular compounds}
- A61K 31/047: having two or more hydroxy groups, e.g. sorbitol
- A61K 31/395: having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K 31/485: Morphinan derivatives, e.g. morphine, codeine
- A61K 31/7004: Monosaccharides having only carbon, hydrogen and oxygen atoms
- A61P 25/02: for peripheral neuropathies
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

Compositions and methods for the treatment of Charcot-Marie-Tooth disease and related disorders. Also provided are combination therapies for treating this disease by decreasing PMP22 expression in a subject.

## Claims

### Claim 1

A method for treating Charcot-Marie-Tooth (CMT) disease of type 1 A (CMT1A), comprising administering to a subject in need thereof an effective amount of baclofen, or a salt thereof or a sustained release formulation thereof.

### Claim 2 (depends on 1)

The method of claim 1, wherein baclofen is administered at a daily dose of about 2 to about 20 mg.

### Claim 3 (depends on 1)

The method of claim 1, wherein baclofen, or a salt thereof or a sustained release formulation thereof, is administered orally.

### Claim 4 (depends on 1)

The method of claim 1, wherein baclofen, or a salt thereof or a sustained release formulation thereof, is formulated in a unit dosage form.

### Claim 5 (depends on 1)

The method of claim 1, wherein pmp22 expression in Schwann cells of the subject is reduced.

### Claim 6 (depends on 1)

The method of claim 1, wherein the strength or motor performance of the subject is improved.

### Claim 7 (depends on 1)

The method of claim 1, wherein baclofen or a salt thereof is administered orally in a unit dosage form at a daily dose of about 2 to about 20 mg.

### Claim 8 (depends on 1)

The method of claim 1, comprising administering to the subject an effective amount of baclofen, or a salt thereof, optionally in combination with an excipient, wherein none of the following is administered to the subject: D-sorbitol; naltrexone; pilocarpine; methimazole; mifepristone; or ketoprofen; or a salt thereof or prodrug thereof.

### Claim 9 (depends on 8)

The method of claim 8, wherein baclofen is administered orally in a unit dosage form at a daily dose of 2 to 20 mg.