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Ganaxolone for use in treating genetic epileptic disorders

A technology of ganaxolone and epilepsy, applied in the field of ganaxolone for the treatment of hereditary epilepsy

Pending Publication Date: 2020-08-21
MARINUS PHARMA INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, some drugs, such as gabapentin, pregabalin, oxcarbazepine, and tiagabine, appear to be effective only for focal seizures in epileptic seizures

Method used

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  • Ganaxolone for use in treating genetic epileptic disorders
  • Ganaxolone for use in treating genetic epileptic disorders
  • Ganaxolone for use in treating genetic epileptic disorders

Examples

Experimental program
Comparison scheme
Effect test

specific Embodiment approach

[0527] The following examples of formulations according to the invention should not be construed as limiting the invention in any way, but merely as samples of the various formulations described herein.

example 1

[0530] A 50 mg / ml ganaxolone suspension was prepared having the composition listed in Table 1 below:

[0531] Table 1: Composition of 50mg / ml Ganaxolone Suspension

[0532]

[0533]

[0534] Table 2 shows the function of the excipients used in the 50 mg / ml ganaxolone suspension.

[0535] Table 2: Summary of Component Functions for 50mg / ml Ganaxolone Suspension

[0536]

[0537] The oral bioavailability of 50 mg / ml ganaxolone suspension depends on the rate and extent of nanoparticle drug dissolution in the relevant physiological environment. The method and specification for granulation shaping are designed to ensure that the ganaxolone drug product does not exhibit aggregation after dispersion in simulated gastrointestinal fluids.

[0538] Figure 3B A summary of the key steps in the suspension manufacturing process applicable to the 50 mg / ml ganaxolone suspension is provided.

[0539] The dispersion nanomilling process was used to reduce the particle size of ganax...

example 2

[0546] Ganaxolone capsules (225 mg) were prepared with the ingredients listed in Tables 4 and 5 below:

[0547] Table 4: Composition of 225 mg Ganaxolone Capsules IR Beads

[0548]

[0549] Table 4: Composition of 225 mg Ganaxolone Capsules IR Beads

[0550] Element grade %w / w Ganaxolone GMP 45.06 Hydroxypropylmethylcellulose (Pharmacoat 603) USP / EP 10.28 Sodium dodecyl sulfate USP / EP / NF 0.70 Sodium Methylparaben USP 0.26 sodium benzoate USP / EP 0.20 Citric acid, anhydrous USP / EP 0.39 Sodium chloride USP / EP 1.03 30% Simethicone Emulsion, (Dow Corning Q7-2587) USP 0.11 Sucrose - Ultrafine Granules EP / NF 23.04 polyethylene glycol 3350 NF / EP 1.08 polyethylene glycol 400 NF / EP 0.54 Polysorbate 80 NF / EP, JP 0.65 Microcrystalline Cellulose Balls, Grade: CP-305 NF / EP 16.65 total 100.0

[0551] Table 5 summarizes the function of the ingredients used in th...

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PUM

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Abstract

The disclosure provides a method of treating a mammal having a genetic epileptic disorder, comprising chronically administering a pharmaceutically acceptable pregnenolone neurosteroid to a mammal having a genetic epileptic disorder in an amount effective to reduce the seizure frequency in the mammal. In certain preferred embodiments, the mammal is a human patient who has a CDKL5 genetic mutation.In certain preferred embodiments, the patient has a low endogenous level of a neurosteroid(s). In certain preferred embodiments, the pregnenolone neurosteroid is ganaxolone.

Description

[0001] Cross References to Related Applications [0002] This application claims priority to U.S. Provisional Application No. 62 / 584,403, filed November 10, 2017, the disclosure of which is hereby incorporated by reference in its entirety for all purposes. [0003] Background of the invention [0004] Infantile epileptic encephalopathy and rare pediatric epilepsy are conditions with high unmet medical need. These conditions include PCDH19-associated epilepsy, CDKL5 deficiency (CDD), Dravet syndrome, Lennox-Gastaut syndrome (LGS), continuous sleep waves during sleep (CSWS), status epilepticus during sleep (ESES), and other intractable Sexual and refractory genetic epileptic conditions clinically similar to PCDH19-associated epilepsy, CDKL5 deficiency, Dravet syndrome, LGS, CSWS, and ESES. [0005] PCDH19-associated epilepsy is a severe and rare epilepsy syndrome that primarily affects women. The disorder is caused by inherited mutations in the protocadherin 19 (PCDH19) gene lo...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/57A61P25/08C12Q1/6883
CPCA61K9/0019A61K9/0095A61K31/565A61K9/4866A61K9/4858A61K9/485A61P25/08A61K47/38A61K31/57A61K9/08A61K47/34A61K47/24A61K47/12A61K47/26
Inventor L·K·马索卡J·拉帕莱南
Owner MARINUS PHARMA INC