# US 2021/0275639 A1 · Compositions for the treatment of sarcopenia or disuse atrophy

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- Number: US 2021/0275639 A1
- Document ID: US20210275639A1
- Status: Published application
- Kind: A1
- Application number: 17/261,314
- Filed: 2019-07-19
- Published: 2021-09-09
- Earliest claimed: 2018-07-20
- Assignees: Association Institut De Myologie; Inserm (Institut National De La Sante Et De La Recherche Medicale); Sorbonne Universite
- Applicants: Association Institut De Myologie; Inserm (Institut National De La Santé Et De La Recherche MéDicale); Sorbonne Universite
- Inventors: France PIETRI-ROUXEL; Sestina FALCONE
- Web page: https://patentgrep.com/patent/US20210275639A1

## CPC

- A61K 38/1841: {Transforming growth factor [TGF]}
- A61K 38/177: {Receptors; Cell surface antigens; Cell surface determinants}
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 21/06: Anabolic agents (androgens A61P5/26)
- G01N 33/74: involving hormones {or other non-cytokine intercellular protein regulatory factors such as growth factors, including receptors to hormones and growth factors}
- A61K 38/1875: {Bone morphogenic factor; Osteogenins; Osteogenic factor; Bone-inducing factor}
- G01N 2333/475: Assays involving growth factors
- G01N 2800/2878: Muscular dystrophy
- 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
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)

## Abstract

The present invention relates to a substance that activates the GDF5 pathway, for use in a method for the treatment of age-related sarcopenia or disuse atrophy.

## Claims

### Claim 1

A GDF5 pathway-activating substance for use in a method for the treatment or prevention of sarcopenia or disuse atrophy.

### Claim 2

A GDF5 pathway-activating substance for use in treating or preventing muscle weakness in a myopathy or neuromuscular disease, alone or in combination with a treatment of said myopathy or neuromuscular disease.

### Claim 3 (depends on 1)

The substance for use according to claim 1, wherein the substance is selected from compounds that increase the activity of GDF5 or compounds that increase the expression of GDF5.

### Claim 4 (depends on 1)

The substance for use according to claim 1, wherein the substance is recombinant human GDF5.

### Claim 5 (depends on 1)

The substance for use according to claim 1, wherein the substance is recombinant human CaVβ1-E or a vector encoding human CaVβ1-E.

### Claim 6 (depends on 1)

The substance for use according to claim 1, wherein the substance is administered to a subject aged 50 years or older, in particular 55 years or older, in particular 60 years or older, more particularly 65 years or older, even more particularly 70 years or older, such as 75 years or older or even 80 years or older.

### Claim 7 (depends on 1)

The substance for use according to claim 1, wherein the substance is administered via the oral, nasal, intravascular, intramuscular, intraperitoneal route, transdermal or subcutaneous route.

### Claim 8 (depends on 1)

The substance for use according to claim 1, wherein the substance is administered on a regular basis, such as on a monthly basis, in particular on a weekly basis, or more particularly on a daily basis.

### Claim 9 (depends on 1)

The substance for use according to claim 1, wherein the treatment of sarcopenia results in an increase of muscle mass and/or function, an increase in physical performance or mobility, and/or an increase in muscle strength.

### Claim 10

A pharmaceutical composition comprising a GDF5 pathway-activating substance and a pharmaceutically acceptable carrier.

### Claim 11 (depends on 10)

The pharmaceutical composition according to claim 10, wherein the substance is recombinant human GDF5.

### Claim 12

A GDF5 pathway-activating substance, for use as a medicament.

### Claim 13 (depends on 12)

The substance for use according to claim 12, wherein said substance is recombinant human GDF5.

### Claim 14

A method for the diagnosis of sarcopenia in a subject, comprising determining the level of GDF5 in a biological sample of said subject.