# US 12,213,982 B2 · Pharmaceutical compositions comprising sepiapterin and uses thereof > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 12,213,982 B2 - Document ID: US12213982B2 - Status: Granted - Kind: B2 - Application number: 18/235,486 - Filed: 2023-08-18 - Published: 2024-03-28 - Granted: 2025-02-04 - Earliest claimed: 2017-09-01 - Assignees: PTC Therapeutics MP, Inc. - Applicants: PTC Therapeutics MP, Inc. - Inventors: Daniel E. Levy - Web page: https://patentgrep.com/patent/US12213982B2 ## CPC - A61K 9/0095: {Drinks; Beverages; Syrups; Compositions for reconstitution thereof, e.g. powders or tablets to be dispersed in a glass of water; Veterinary drenches (A61K9/0007 takes precedence; eatable gels or foams A61K9/0056; oral mucosa adhesive forms A61K9/006)} - A61K 31/519: ortho- or peri-condensed with heterocyclic rings - A61K 9/14: Particulate form, e.g. powders, {Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles (microspheres A61K9/16; microcapsules A61K9/50; nanocapsules, nanoparticles of the matrix type A61K9/51)} - A61K 9/20: Pills, tablets, {discs, rods (A61K9/0004, A61K9/0007, A61K9/0056, A61K9/0065 take precedence; for reconstitution of a drink A61K9/0095)} - A61K 38/40: Transferrins, e.g. lactoferrins, ovotransferrins - A61K 47/22: Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones - A61K 47/38: Cellulose; Derivatives thereof - A61P 3/00: Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00) - A61P 25/00: Drugs for disorders of the nervous system - A61K 9/1617: {Organic compounds, e.g. phospholipids, fats} - 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 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. ## Claims ### Claim 1 A method of treating a tetrahydrobiopterin-related disorder selected from tetrahydrobiopterin deficiency and phenylketonuria in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition comprising an effective amount of sepiapterin and a pharmaceutically acceptable carrier, wherein the relative amount of antioxidant to sepiapterin is between 0 and 20% wt/wt; wherein the antioxidant, when present, is ascorbic acid, an ester of ascorbic acid, or a salt of ascorbic acid; and wherein from about 2.5 mg sepiapterin per kilogram of body weight of the subject (mg/kg) to about 150 mg/kg is administered. ### Claim 2 (depends on 1) The method of claim 1, wherein from about 40 mg/kg to about 150 mg/kg is administered. ### Claim 3 (depends on 1) The method of claim 1, wherein from about 60 mg/kg to about 120 mg/kg is administered. ### Claim 4 (depends on 1) The method of claim 1, wherein from about 40 mg/kg to about 60 mg/kg is administered. ### Claim 5 (depends on 1) The method of claim 1, wherein from about 2.5 mg/kg to about 20 mg/kg is administered. ### Claim 6 (depends on 1) The method of claim 1, wherein the tetrahydrobiopterin-related disorder is phenylketonuria. ### Claim 7 (depends on 1) The method of claim 1, wherein the tetrahydrobiopterin-related disorder is a tetrahydrobiopterin deficiency. ### Claim 8 (depends on 7) The method of claim 7, wherein the tetrahydrobiopterin deficiency is primary tetrahydrobiopterin deficiency. ### Claim 9 (depends on 1) The method of claim 1, wherein the pharmaceutical composition comprises less than 1.3% of lactoylpterin by weight of the combined amount of sepiapterin and lactoylpterin in the composition. ### Claim 10 (depends on 1) The method of claim 1, wherein the pharmaceutical composition is stable at room temperature for at least 6 months. ### Claim 11 (depends on 1) The method of claim 1, wherein the pharmaceutical composition is formulated as a powder. ### Claim 12 (depends on 1) The method of claim 1, wherein the composition further comprises a dispersant selected from crosslinked polyvinylpyrrolidone, carboxymethylcellulose, croscarmellose sodium, starch, or alginic acid, wherein the pharmaceutical composition comprises 0.5-1.5% dispersant by total weight. ### Claim 13 (depends on 12) The method of claim 12, wherein the dispersant is croscarmellose sodium, wherein the composition comprises 1% croscarmellose sodium by weight. ### Claim 14 (depends on 1) The method of claim 1, wherein the composition further comprises an anti-caking agent or bulking agent selected from colloidal silicon dioxide, microcrystalline cellulose, tricalcium phosphate, microcrystalline cellulose, magnesium stearate, sodium bicarbonate, sodium ferrocyanide, potassium ferrocyanide, calcium ferrocyanide, calcium phosphate, sodium silicate, colloidal silicon dioxide, calcium silicate, magnesium trisilicate, talcum powder, sodium aluminosilicate, potassium aluminum silicate, calcium aluminosilicate, bentonite, aluminum silicate, stearic acid, and polydimethylsiloxane, wherein the composition comprises about 60-80% anti-caking or bulking agent by total weight. ### Claim 15 (depends on 14) The method of claim 14, wherein the anti-caking or bulking agents are microcrystalline cellulose and silicon dioxide, wherein the composition comprises about 63% microcrystalline cellulose by weight and about 6% colloidal silicon dioxide by weight. ### Claim 16 (depends on 1) The method of claim 1, wherein the antioxidant is selected from ascorbic acid, ascorbyl palmitate, ascorbyl myristate, ascorbyl stearate, or sodium ascorbate. ### Claim 17 (depends on 16) The method of claim 16, wherein the antioxidant is ascorbic acid. ### Claim 18 (depends on 1) The method of claim 1, wherein the composition is substantially free of antioxidant. ### Claim 19 (depends on 1) The method of claim 1, wherein the pharmaceutical composition comprises about 25% sepiapterin by total weight.