# US 2022/0033876 A1 · Methods of Administering 3,4-Diaminopyridine

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- Number: US 2022/0033876 A1
- Document ID: US20220033876A1
- Status: Published application
- Kind: A1
- Application number: 17/503,148
- Filed: 2021-10-15
- Published: 2022-02-03
- Earliest claimed: 2011-06-30
- Assignees: Serb SA
- Applicants: Serb SA
- Inventors: Marvin R. GAROVOY; Peter E. HAROLDSEN; Donald G. MUSSON
- Web page: https://patentgrep.com/patent/US20220033876A1

## CPC

- C12Q 1/48: involving transferase
- A61K 31/44: Non condensed pyridines; Hydrogenated derivatives thereof
- A61K 38/45: Transferases (2)
- C12Q 1/6883: for diseases caused by alterations of genetic material
- G01N 33/564: for pre-existing immune complex or autoimmune disease {, i.e. systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, rheumatoid factors or complement components C1-C9}
- Y02A 50/30: Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
- C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- 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)
- 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)
- Y02A: TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE

## Abstract

Provided herein are methods of determining NAT acetylation status of a subject with a 3,4-DAP-sensitive disease, methods of selecting a dose of 3,4-DAP or a pharmaceutically acceptable salt thereof adjusted to a subject's acetylation status, methods of administering 3,4-diaminopyridine or a pharmaceutically acceptable salt thereof to a patient in need thereof, and methods of treating 3,4-DAP sensitive diseases.

## Claims

### Claim 1

A method of treating a human patient diagnosed with Lambert-Eaton myasthenic syndrome (LEMS) in need of treatment thereof comprising administering a total daily dose of about 7.5 mg to about 40 mg of 3,4-diaminopyridine (3,4-DAP), or an equivalent amount of a pharmaceutically acceptable salt thereof, to a human patient diagnosed with LEMS having two N-acetyl transferase 2 (NAT2) slow alleles, wherein the total daily dose is optionally provided as a series of divided doses.

### Claim 2 (depends on 1)

The method of claim 1, wherein the two NAT2 slow alleles each comprise one or more of a 282T, 341C, 191A, 481T, 590A, or 857A mutation.

### Claim 3 (depends on 2)

The method of claim 2, wherein the two NAT2 slow alleles each comprise a 282T, a 341C, or a 282T and a 341C mutation.

### Claim 4 (depends on 1)

The method of claim 1, wherein the about 7.5 mg to about 40 mg of 3,4-DAP is provided via an equivalent amount of 3,4-DAP phosphate salt.

### Claim 5 (depends on 1)

The method of claim 1, wherein the total daily dose is provided via up to 5 divided doses per day.

### Claim 6 (depends on 5)

The method of claim 5, wherein the divided dose is provided as one or more scored tablets or portions thereof.

### Claim 7 (depends on 6)

The method of claim 6, wherein the scored tablet comprises about 10 mg of 3,4-DAP, or the equivalent amount of a pharmaceutically acceptable salt thereof.

### Claim 8 (depends on 5)

The method of claim 5, wherein the divided dose is provided as an aqueous suspension.

### Claim 9 (depends on 1)

The method of claim 1, wherein the total daily dose is about 15 mg to about 30 mg of 3,4-DAP, or the equivalent amount of a pharmaceutically acceptable salt thereof.

### Claim 10 (depends on 9)

The method of claim 9, wherein the total daily dose is provided via 3 to 4 divided doses per day.

### Claim 11 (depends on 9)

The method of claim 9, wherein the total daily dose is provided via 2 to 3 divided doses per day.

### Claim 12 (depends on 9)

The method of claim 9, wherein the total daily dose is about 15 mg of 3,4-DAP, or the equivalent amount of a pharmaceutically acceptable salt thereof.

### Claim 13 (depends on 9)

The method of claim 9, wherein total daily dose is about 30 mg of 3,4-DAP, or the equivalent amount of a pharmaceutically acceptable salt thereof.

### Claim 14 (depends on 1)

The method of claim 1, wherein the total daily dose is about 7.5 mg to about 15 mg of 3,4-DAP, or the equivalent amount of a pharmaceutically acceptable salt thereof.

### Claim 15 (depends on 14)

The method of claim 14, wherein the total daily dose is provided via 2 to 3 divided doses per day.

### Claim 16 (depends on 14)

The method of claim 14, wherein the total daily dose is about 7.5 mg of 3,4-DAP, or the equivalent amount of a pharmaceutically acceptable salt thereof.

### Claim 17 (depends on 1)

The method of claim 1, wherein the patient suffers from renal impairment.

### Claim 18

A method of treating a human patient diagnosed with LEMS in need of treatment thereof comprising administering a total daily dose of about 7.5 mg to about 15 mg of 3,4-DAP, or an equivalent amount of a pharmaceutically acceptable salt thereof, as a series of divided doses to a human patient diagnosed with LEMS having two NAT2 slow alleles.

### Claim 19 (depends on 18)

The method of claim 18, wherein the two NAT2 slow alleles each comprise one or more of a 282T, 341C, 191A, 481T, 590A, or 857A mutation.

### Claim 20 (depends on 19)

The method of claim 19, wherein the two NAT2 slow alleles each comprise a 282T, a 341C, or a 282T and a 341C mutation.

### Claim 21

A method of treating a human patient diagnosed with LEMS in need of treatment thereof comprising administering a total daily dose of about 15 mg to about 30 mg of 3,4-DAP, or an equivalent amount of a pharmaceutically acceptable salt thereof, as a series of divided doses to a human patient diagnosed with LEMS having two NAT2 slow alleles.

### Claim 22 (depends on 21)

The method of claim 21, wherein the two NAT2 slow alleles each comprise one or more of a 282T, 341C, 191A, 481T, 590A, or 857A mutation.

### Claim 23 (depends on 22)

The method of claim 22, wherein the two NAT2 slow alleles each comprise a 282T, a 341C, or a 282T and a 341C mutation.

### Claim 24

A method of treating a human patient diagnosed with LEMS in need of treatment thereof comprising administering a total daily dose of 3,4-DAP phosphate salt, which is equivalent to a total daily dose of about 7.5 mg to about 30 mg of 3,4-DAP, as a series of divided doses to a human patient diagnosed with LEMS having two NAT2 slow alleles.

### Claim 25 (depends on 24)

The method of claim 24, wherein the two NAT2 slow alleles each comprise one or more of a 282T, 341C, 191A, 481T, 590A, or 857A mutation.

### Claim 26 (depends on 25)

The method of claim 25, wherein the two NAT2 slow alleles each comprise a 282T, a 341C, or a 282T and a 341C mutation.