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Pharmaceutical compositions comprising sepiapterin and uses thereof

A composition, the technology of mepterin, applied in the direction of drug combination, medical preparations containing active ingredients, medical preparations with non-active ingredients, etc., can solve problems such as nerve movement defects and reduce the formation of neurotransmitters

Pending Publication Date: 2020-08-04
PTC医疗MP公司
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Reduced Tyr synthesis and impaired Tyr and tryptophan hydroxylase activity lead to reduced neurotransmitter formation and subsequent neuromotor deficits

Method used

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  • Pharmaceutical compositions comprising sepiapterin and uses thereof
  • Pharmaceutical compositions comprising sepiapterin and uses thereof
  • Pharmaceutical compositions comprising sepiapterin and uses thereof

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0242] Example 1. Preparation of a pharmaceutical composition comprising mepterin and an antioxidant

[0243] The process related to the manufacture of a pharmaceutical composition comprising mepterin and less than 10% by total weight of an antioxidant is as follows:

[0244] 1. Put mepterin (0.34 kg), ascorbic acid (0.071 kg), microcrystalline cellulose (0.85 kg), croscarmellose sodium (0.14 kg) and colloidal silicon dioxide (0.085 kg) separately Screen through a 140 mesh sieve.

[0245] 2. Mix colloidal silicon dioxide with microcrystalline cellulose and pass the combined material through an 80 mesh screen.

[0246] 3. Put the sifted material from step 2 into the V-blender.

[0247] 4. Add the filtered croscarmellose, ascorbic acid and mepterin to the V-blender.

[0248] 5. Blend the contents of the V-blender for at least 10 minutes.

[0249] 6. Pass the mixture through a 140-mesh sieve.

[0250] 7. Store the mixture at -20°C.

Embodiment 2

[0251] Example 2. Preparation of Pharmaceutical Compositions Comprising Mepterin and Antioxidants in an Administration Vehicle

[0252] The composition prepared in Example 1 (180 mg / kg methotrexate) was added to an amount of Medisca® oral mix (2.5% (w / w) in water) sufficient to produce a metopterin concentration of 58.3 mg / mL glycerol, 27% (w / w) sucrose).

Embodiment 3

[0253] Embodiment 3. In vivo pharmacokinetic analysis of the pharmaceutical composition of the present invention

[0254] A total of 18 CD-1 mice were divided into 2 groups as summarized below. Each test article was evaluated at one dose level (180 mg / kg). All animals in each group received a single oral (PO) administration of one dose level of the test or control article. Doses (mg / kg) were calculated based on animal body weight. There were nine male mice in each group. Group 1 was given mepterin added to a citrate buffer solution consisting of 750 mg sodium metabisulfite, 639 mg sodium citrate, 2.69 g anhydrous citric acid, and 750 mg ascorbic acid in 300 mL sterile water . Then 1.5 grams of carboxymethylcellulose was added and stirred until dissolved. Mepterin (180 mg / kg) was added to the buffer to give a concentration of 27 mg / mL. Administer group 1 mice at a dose volume of 6.67 mL / kg. The preparation prepared in Example 2 was administered to the second group. Admi...

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Abstract

The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and / or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.

Description

[0001] Background of the invention [0002] Primary tetrahydrobiopterin deficiency (PBD) results from deficiency of GTP cyclohydrolase I (GTP-CH), 6-pyruvyl-tetrahydropterin synthase (PTPS), or mepterin reductase (SR) (impaired tetrahydrobiopterin (BH4) biosynthesis), or by defects in BH4 recycling (pterin-4a-methanolamine dehydratase (PCD) or dihydropteridine reductase (DHPR) deficiency) . PBD accounts for 1-3% of all cases of hyperphenylalaninemia (HPA), while nearly all remaining cases are due to phenylalanine hydroxylase deficiency. [0003] BH4 is an essential cofactor for phenylalanine hydroxylase, tyrosine hydroxylase, tryptophan hydroxylase, fatty acid glyceryl ether oxygenase, and nitric oxide (NO) synthase. [0004] In PBD, impaired phenylalanine (Phe) to tyrosine (Tyr) hydroxylation leads to HPA. Reduced Tyr synthesis and impaired Tyr and tryptophan hydroxylase activity lead to reduced neurotransmitter formation and subsequent neuromotor deficits. [0005] Phenoty...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/519A61K9/20
CPCA61K47/22A61K9/0095A61K47/38A61K31/519A61P3/00A61K9/1617A61P25/00A61K9/14A61K9/20A61K38/40
Inventor D.E.勒维
Owner PTC医疗MP公司
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