# US 10,307,371 B2 · Rapamycin for the treatment of lymphangioleiomyomatosis

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- Number: US 10,307,371 B2
- Document ID: US10307371B2
- Status: Granted
- Kind: B2
- Application number: 15/803,005
- Filed: 2017-11-03
- Published: 2018-07-19
- Granted: 2019-06-04
- Earliest claimed: 2014-02-11
- Assignees: AI Therapeutics, Inc.
- Applicants: AI Therapeutics, Inc.
- Inventors: Thomas Armer; Lawrence S. Melvin, Jr.; Jonathan M. Rothberg; Henri Lichenstein
- Web page: https://patentgrep.com/patent/US10307371B2

## CPC

- A61K 9/0075: {for inhalation via a dry powder inhaler [DPI], e.g. comprising micronized drug mixed with lactose carrier particles}
- A61K 9/145: {with organic compounds}
- A61K 31/436: the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
- A61P 11/00: Drugs for disorders of the respiratory system
- A61K 47/26: Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
- 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

The present invention relates to methods and compositions for treating lymphangioleiomyomatosis in a human subject in need of such treatment. The methods comprise administering to the subject via inhalation an aerosol composition comprising rapamycin or a prodrug or derivative (including analog) thereof.

## Claims

### Claim 1

A method for treating Lymphangioleiomyomatosis in a human subject in need of such treatment, the method comprising administering to the subject by inhalation a pharmaceutical dry powder composition comprising microparticles of sirolimus, particles of a carrier, and one or more optional excipients, wherein the microparticles of sirolimus have a mean diameter of from 1 to 5 microns, and wherein the sirolimus is the only therapeutic agent in the composition.

### Claim 2 (depends on 1)

The method of claim 1, wherein the carrier is selected from the group consisting of arabinose, glucose, fructose, ribose, mannose, sucrose, trehalose, lactose, maltose, starches, dextran, mannitol, lysine, leucine, isoleucine, dipalmitylphosphatidylcholine, lecithin, polylactic acid, poly (lactic-co-glutamic) acid, and xylitol, or mixtures of any of the foregoing.

### Claim 3 (depends on 1)

The method of claim 1, wherein the administration achieves a respirable dose of from 5 to 500 micrograms of sirolimus is delivered to the lung.

### Claim 4 (depends on 1)

The method of claim 1, wherein the administration is once daily.

### Claim 5 (depends on 1)

The method of claim 1, wherein the administration achieves a blood trough level of sirolimus of less than 2 ng/ml 12 or 24 hours after administration.

### Claim 6 (depends on 1)

The method of claim 1, wherein the amount of sirolimus in the pharmaceutical dry powder composition is from 10 to 250 micrograms.

### Claim 7 (depends on 1)

The method of claim 1, wherein the amount of sirolimus in the pharmaceutical dry powder composition is from 20 to 100 micrograms.