Details for Patent: 9,433,624
✉ Email this page to a colleague
Title: | Methods and compositions for the treatment of metabolic disorders |
Abstract: | The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated. |
Inventor(s): | Oppenheimer; Daniel I. (Castro Valley, CA), Kakkis; Emil D. (Novato, CA), Price; Fredric D. (Bedford, NY), Dorenbaum; Alejandro (Mill Valley, CA), Moser; Rudolf (Schaffhausen, CH), Groehn; Viola (Dachsen, CH), Egger; Thomas (Kempthal, CH), Blatter; Fritz (Reinach, CH) |
Assignee: | BIOMARIN PHARMACEUTICAL INC. (Novato, CA) |
Filing Date: | Nov 16, 2011 |
Application Number: | 13/297,644 |
Claims: | 1. A method for treating a subject suffering from hyperphenylalaninemia (HPA) due to BH4-responsive phenylketonuria (PKU) comprising administering to said subject tetrahydrobiopterin (BH4) or pharmaceutically acceptable salt thereof at a daily dose of 10 mg/kg to 20 mg/kg, wherein the administering is multiday, oral, and only once per day. 2. The method of claim 1, wherein said subject is administered BH4 for at least 7 days. 3. The method of claim 1, wherein the subject is administered BH4 for at least 2 weeks. 4. The method of claim 1, wherein said subject is administered BH4 for at least 6 weeks. 5. The method of claim 1, wherein said BH4 is administered as a crystallized form stable for at least 3 months at 40.degree. C. and 75% relative humidity. 6. The method of claim 5, wherein said crystallized form of BH4 comprises at least 99.5% pure (6R)-5,6,7,8-tetrahydrobiopterin. 7. The method of claim 1, further comprising administering to said subject a protein-restricted diet. 8. The method of claim 1, wherein said subject is pregnant, is an infant of 0 to 3 years, or has a plasma phenylalanine concentration of greater than 600 .mu.M prior to treatment with BH4. 9. The method of claim 1, wherein said BH4 is in the form of a tablet. 10. The method of claim 1, wherein said BH4 is dissolved in a liquid. 11. The method of claim 1, wherein the BH4 is administered at a dose of about 10 mg/kg. 12. The method of claim 1, wherein the BH4 is administered at a dose of about 20 mg/kg. |