Criteria: Criteria for initial therapy:Kuvan (sapropterin dihydrochloride) is considered . In December 2007, it became the first . Dr. Reddy's Laboratories Announces the Launch of a Generic Version of Sapropterin Dihydrochloride Tablets for Oral Use in the U.S. Market - read this article along with other careers information, tips and advice on BioSpace Sapropterin | Nature Reviews Drug Discovery serious outcome and drug utilization data for Kuvan (sapropterin dihydrochloride) in pediatric patients. Sapropterin dihydrochloride | C9H17Cl2N5O3 - PubChem compound Summary Sapropterin dihydrochloride Contents 1 Structures 2 Names and Identifiers 3 Chemical and Physical Properties 4 Spectral Information 5 Related Records 6 Chemical Vendors 7 Drug and Medication Information 8 Pharmacology and Biochemistry 9 Use and Manufacturing 10 Safety and Hazards KUVAN (sapropterin dihydrochloride) powder for oral solution . It was approved for use in the European Union in December 2008, Canada in April 2010, and Japan in July 2008. Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study. DailyMed - SAPROPTERIN DIHYDROCHLORIDE powder, for solution Sapropterin may also be used for purposes not listed in this medication guide. 4. For the full list of excipients, see section 6.1. 2 DOSAGE AND ADMINISTRATION Par's sapropterin dihydrochloride tablets and powder for oral solution are the generic version of Kuvan® tablets and powder for oral solution and are the first, and currently the only, generic versions available. There are eleven patents protecting this compound and three Paragraph IV challenges. Drug Information Portal - U.S. National Library of ... Both tetrahydrobiopterin and sapropterin lower blood phenylalanine concentration, now not, it is thought, by way of changing the synthesis or regeneration of the cofactor, . PDF sapropterin dihydrochloride, 100mg, soluble tablets (Kuvan ... National funding/access decisions For full details see funding body website Scottish Medicines Consortium (SMC) Decisions SMC No. Sapropterin Dihydrochloride (Bulk): Uses, Side Effects ... Kuvan - Sapropterin dihydrochloride uses, dose and side ... | SAPROPTERIN (SAP roe TER in) works to lower phenylalanine levels in the blood. 69056-38-8 API Manufacturer, Supplier Distributor, source of fine chemicals, API supplier, active pharmaceutical Ingredient, intermediate, documentation,certification, DMF, DMFs, filed, filing, FDA, US Drug Master Files, active pharmaceutical ingredients, intermediates, offers as per requirements of RND/ Development, quantities or commercials, Buy high . Tetrahydrobiopterin is available as a tablet for oral administration in the form of sapropterin dihydrochloride (BH4*2HCL). sapropterin dihydrochloride | 69056-38-8 C9H17Cl2N5O3 structure,synthetic routes, physical and chemical properties, safety information, toxicity, customs data,maps, MSDS, generation methods and uses, and sapropterin dihydrochloride'supstream and downstream products. PHARMACEUTICAL FORM. Efficacy of Sapropterin Dihydrochloride in Increasing ... PTC923 (sepiapterin) lowers elevated blood phenylalanine ... Sapropterin | C9H15N5O3 - PubChem compound Summary Sapropterin Contents 1 Structures 2 Names and Identifiers 3 Chemical and Physical Properties 4 Spectral Information 5 Related Records 6 Chemical Vendors 7 Drug and Medication Information 8 Pharmacology and Biochemistry 9 Use and Manufacturing 10 Safety and Hazards Drug Uses: This medication is used along with diet changes for long-term treatment of a certain inherited disorder (phenylketonuria-PKU). 1 INDICATIONS AND USAGE Sapropterin dihydrochloride tablets are indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to . KUVAN ® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in adults and children over one month of age with a certain type of Phenylketonuria (PKU). sapropterin dihydrochloride. 2 DOSAGE AND ADMINISTRATION Sapropterin Dihydrochloride What is the Non-Proprietary Name? KUVAN Homepage | Prescription Medication for PKU Kuvan is used in people with phenylketonuria (PKU). US9365573B2 - Method for synthesizing sapropterin ... The generic name usually includes the active ingredient(s) of the product. Additional Comments: Females should not be pregnant or lactating, and if Recommended studies: 2 studies . Objective: To evaluate the ability of sapropterin dihydrochloride (pharmaceutical preparation of tetrahydrobiopterin) to increase phenylalanine (Phe) tolerance while maintaining adequate blood Phe control in 4- to 12-year-old children with phenylketonuria (PKU). Design: Single-dose, two-way crossover in vivo. Sapropterin dihydrochloride is a synthetic version of tetrahydrobiopterin, the naturally occurring pterin cofactor that is required for PAH-mediated phenylalanine hydroxylation. Sapropterin dihydrochloride . Sapropterin Dihydrochloride Tablets. Sapropterin dihydrochloride is an activator of phenylalanine hydroxylase (PAH), the enzyme that is missing in patients with PKU and is needed for the breakdown of the amino acid phenylalanine. Based on review of available data, the Company considers the use of sapropterin dihydrochloride (Kuvan) when patient selection criteria are not met to be investigational. Warnings Follow all directions on your medicine label and package. Lancet , 370 (9586), 504-510. BH4 can activate residual PAH enzyme, improve the normal oxidative metabolism of Phe, and decrease Phe levels. It is used with a special diet to treat phenylketonuria. Sapropterin Dihydrochloride Tablets: SAPROPTERIN DIHYDROCHLORIDE (Generic for KUVAN) QTY 60 • 100 MG • POWD PACK • Near 77381 Add to Medicine Chest Set Price Alert More Ways to Save SAPROPTERIN (SAP roe TER in) works to lower phenylalanine levels in the blood. Brand/Generic Literature References 49884-0720-08: Generic Name. J Pediatr 2009;154:700-7. 1 INDICATIONS AND USAGE Sapropterin dihydrochloride powder for oral solution is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia . PTC923 (formerly CNSA-001), an oral formulation of sepiapterin, a natural precursor of intracellular tetrahydrobiopterin (BH 4), has been shown in humans to induce larger increases in circulating BH 4 vs. sapropterin dihydrochloride. Kuvan (saproterin dihydrochloride tablets) is a synthetic form of a natural salt that contributes to the function of a certain enzyme in the body used to help the body activate the enzyme needed to control blood levels of phenylalanine. This medicine is not a cure. Prescriber is a physician specializing in the patient's diagnosis or is in consultation with a . LGM Pharma can supply CGMP and/or DMF products, subject to availability and manufacturer requirements. two only included patients that had a response to sapropterin in part-one. Sapropterin dihydrochloride, the active pharmaceutical ingredient in Kuvan, is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). These actions help restore the ability of the enzyme to convert phenylalanine into tyrosine, thereby reducing phenylalanine levels . KUVAN is to be used along with a Phe-restricted diet. Sapropterin dihydrochloride, a synthetic, stable form of the tetrahydrobiopterin cofactor of phenylalanine hydroxylase, has been shown to reduce plasma phenylalanine (Phe) levels in a significant portion of patients with phenylketonuria (PKU). Solubility was partial for the tablets and complete for the powder. Kuvan® is a form of BH4, which is a substance in the body that helps break down phenylalanine. Sapropterin dihydrochloride, the active pharmaceutical ingredient in Kuvan, is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Sapropterin dihydrochloride tablets are indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN® (sapropterin dihydrochloride) oral Page 2 of 4 . Summary Info Summary of drug information (MedlinePlusDrug) Info Summary of consumer health information (MedlinePlusTopics) This Coverage Guideline must be read in its entirety to determine coverage eligibility, if any. Study design: This international, double-blind, randomized, placebo-controlled . "Dr. Reddy's") today announced the launch of Sapropterin Dihydrochloride Powder for Oral Solution, 100mg, a therapeutic equivalent generic version of Kuvan® (sapropterin dihydrochloride) Powder for Oral Solution, 100 mg, USP, approved by the U.S. Food and Drug Administration (USFDA). PTC923 (formerly CNSA-001), an oral formulation of sepiapterin, a natural precursor of intracellular tetrahydrobiopterin (BH 4), has been shown in humans to induce larger increases in circulating BH 4 vs. sapropterin dihydrochloride. Publication type: Policy or strategy. Sapropterin Dihydrochloride. and will be approved when . BH4 is required for the body to use the amino acid phenylalanine, to form another amino acid called tyrosine. Topic: Specialised commissioning. It is sold under the brand names Kuvan . * Background/Overview Kuvan is a synthetic preparation of naturally occurring BH4, which is a cofactor for the enzyme phenylalanine hydroxylase (PAH). CAS Number. The non-proprietary name is sometimes called the generic name. SAPROPTERIN DIHYDROCHLORIDE CAS no. KUVAN is to be used along with a Phe-restricted diet. LGM Pharma does not sell or supply APIs or finished dosage products to individual patients, doctors, pharmacies, or any pharmaceutical companies. Sapropterin dihydrochloride (Kuvan), hereafter referred to as sapropterin, is a synthetic formulation of the active 6R-isomer of tetrahydrobiopterin, a naturally occurring cofactor for phenylalanine hydroxylase. Sapropterin dihydrochloride, a synthetic tetrahydrobiopterin (BH4), works as a chaperone of phenylalanine hydroxylase (PAH) in phenylketonuria (PKU) to facilitate and stabilize folding of PAH into its most active conformation. Background & aim. Received sapropterin dihydrochloride within 16 weeks of randomization; Have initiated or adjusted medication for treatment of ADHD, depression, or anxiety ≤ 8 weeks prior to randomization; Taking medication known to inhibit folate synthesis (eg, methotrexate) Any condition requiring treatment with levodopa or any PDE-5 inhibitor Sapropterin (tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide. Phe tolerance will be defined as the amount of dietary . The additive effect of sapropterin and PDE-5 inhibitor co-administration could 195 lead to a reduction in blood pressure; however, the combined use of these medications has not 196 been evaluated in humans. Sapropterin has been found to be very safe and no serious adverse events were associated with the use of sapropterin on the short-term [26 M]. US-SAP-2100003 / RDY-0621-346 / June 2021. Kuvan (sapropterin dihydrochloride) is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). In part-two of the study patients were randomised, to ten weeks of treatment with sapropterin 20mg/kg/day (n=33), 3:1 supplied as 100mg sapropterin dihydrochloride soluble tablets once daily, or placebo (n=12) with all Initial U.S. Approval: 2007 -----INDICATIONS AND USAGE-----Kuvan is a phenylalanine hydroxylase activator indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age . Sapropterin dihydrochloride is to be used in conjunction with a Phe-restricted diet. Sapropterin. ALL. SAPROPTERIN DIHYDROCHLORIDE Savings, Coupons and Information. Sapropterin reduces blood phenylalanine (Phe) by ≥20-30% in a minority of subjects with PKU. Whereas increased Phe tolerance was the criterion used to determine durability of response, non-responders were mostly discontinued from treatment. (1) Sapropterin dihydrochloride can stimulate TH and TPH activities leading to improved dopamine and serotonin synthesis despite persistently elevated brain phenylalanine. Uncontrolled blood phenylalanine concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects. PAH hydroxylates Phe through an oxidative reaction to form tyrosine. The present disclosure reduces a synthesis route of the sapropterin dihydrochloride, introduces a chiral center in an asymmetric synthesis manner, in which a tetrahydrofuran solution containing a samarium catalyst is adopted as a catalyst, and obtains a target compound having a high antimer isomerism value by means of . Page updated: 5 October 2021. Additional information is available in the individual branded drug profile pages. Patient and carer advice Patient or carers should be given advice on how to administer sapropterin dihydrochloride dispersible tablets. Under the terms of the settlement, BioMarin will grant Par a non-exclusive license to its patents related to Kuvan to allow Par to market a generic version of sapropterin dihydrochloride 100mg . Kuvan (sapropterin dihydrochloride) Kuvan (sapropterin dihydrochloride) Coverage for services, procedures, medical devices, and drugs are dependent upon benefit eligibility as outlined in the member's specific benefit plan. In a subset of individuals with PAH deficiency, sapropterin administration leads to reduction in blood phenylalanine levels independent of dietary protein. 5, 10. Sapropterin dihydrochloride response was most commonly defined as a 30% reduction in blood Phe concentration relative to baseline. In patients with BH4 deficiency, sapropterin provides an effective alternative to BH4. KUVAN ® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in adults and children over one month of age with a certain type of phenylketonuria (PKU). Sapropterin dihydrochloride use in pregnant women with phenylketonuria: An interim report of the PKU MOMS sub-registry Dorothy K. Grange, Richard E. Hillman, Barbara K. Burton , Shoji Yano, Jerry Vockley, Chin To Fong, Joellen Hunt, John J. Mahoney, Jessica L. Cohen-Pfeffer * Sapropterin Support Program for Patients with Phenylketonuria (PKU) Sapropterin Dihydrochloride Tablets for Oral Use, 100 mg Sapropterin Dihydrochloride Powder for Oral Solution, 100 mg Type of study: Fasting . Sapropterin dihydrochloride is an off-white to light yellow crystals or crystalline powder. What Kuvan is and what it is used for. There is evidence that the restoration of BH 4 bioavailabil-ity may prevent the neurodevelopmental delay in patients with BH 4 deficiency. Sapropterin dihydrochloride is used to lower blood phenylalanine levels in tetrahydrobiopterin-responsive phenylketonuria in conjunction with a phenylalanine-restricted diet.This study investigated the solubility and stability of sapropterin tablets and a sapropterin powder formulation when mixed in selected beverages and foods. 558/09 Sapropterin dihydrochloride is a synthetic form of cofactor BH4. It is also essential in the conversion of phenylalanine to tyrosine by the enzyme phenylalanine-4-hydroxylase; the conversion of tyrosine to L-dopa by the enzyme tyrosine hydroxylase; and conversion of . Each soluble tablet contains 100 mg of sapropterin dihydrochloride (equivalent to 77 mg of sapropterin). The U.S. Food and Drug Administration (FDA) has approved the drug sapropterin dihydrochloride (Kuvan®) for the treatment of PKU. CLINICAL PARTICULARS 1. 12,13 In the present study, we report on the first case of a Uses advised against Food, drug, pesticide or biocidal product use. KUVAN (sapropterin dihydrochloride) tablets, for oral use . In PKU, it works by enhancing the activity of the defective enzyme, while in BH4 deficiency it replaces the missing cofactor. No standard pharmacogenetic tests exist to identify responsive genotypes. Now, after years of campaigning from his mum Barbara and local MP Liz Twist, the NHS has struck a deal to provide a generic version of a drug called sapropterin dihydrochloride for patients. The cube contains the active substance sapropterin, which is a synthetic copy of a body substance called tetrahydrobiopterin (BH4). of the following criteria are met: 1. Medical use. Excess of cofactor activates residual PAH enzyme, improving phenylalanine metabolism and decreasing phenylalanine concentrations in the blood of PKU patients. Dr. Reddy's Sapropterin Dihydrochloride Powder for Oral Solution, is available in 100 mg unit dose packets in a 30 count carton. However, having too little BH4 is only one reason a person may not break down phenylalanine. Sapropterin is a synthetic form of tetrahydrobiopterin. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. In a rabbit study using oral administration of sapropterin dihydrochloride during the period of organogenesis, a rare defect, holoprosencephaly, was noted at 10 times the MRHD. It is used with a special diet to treat phenylketonuria. Received sapropterin dihydrochloride within 16 weeks of randomization • Have initiated or adjusted medication for treatment of ADHD, depression, or anxiety ≤ 8 weeks prior to randomization; Taking medication known to inhibit folate synthesis (eg, methotrexate) Any condition requiring treatment with levodopa or any PDE-5 inhibitor Sapropterin Dihydrochloride (Bulk) Powder - Uses, Side Effects, and More Generic Name(S): sapropterin di-HCl (bulk) View Free Coupon NDC Generic Name Compare to Brand SCH Strength Dose Form Package Size PI MG; NDC. Form/Route: Tablets/Oral. Sapropterin dihydrochloride is an off-white to light yellow crystals or crystalline powder. medically necessary . 69056-38-8 - RKSUYBCOVNCALL-NTVURLEBSA-N - Sapropterin dihydrochloride [USAN] - Similar structures search, synonyms, formulas, resource links, and other chemical information. Sapropterin dihydrochloride use in pregnant women with phenylketonuria: An interim report of the PKU MOMS sub-registry Dorothy K. Grange , Richard E. Hillman, Barbara K. Burton, Shoji Yano, Jerry Vockley, Chin To Fong, Joellen Hunt, John J. Mahoney, Jessica L. Cohen-Pfeffer Sapropterin is a synthetic type of tetrahydrobiopterin ready from the dihydrochloride salt [2]. Drug Entry. Sapropterin dihydrochloride [USAN] View Synonyms View Structure: Description: Co-factor required for catalytic activity of nitric oxide synthases. 69056-38-8. Important Safety Information Kuvan™ (sapropterin dihydrochloride) Tablets 4 193 or tadalafil), because both sapropterin dihydrochloride and PDE-5 inhibitors may induce 194 vasorelaxation. Kuvan® (sapropterin dihydrochloride) is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-(BH4-) responsive. (Amino acids are the building blocks of proteins.) Trefz FK, Burton BK, Longo N, Casanova MM, Gruskin DJ, Dorenbaum A, et al. The active substance in Kuvan, sapropterin dihydrochloride, is a synthetic copy of BH4. Sapropterin reduces blood phenylalanine (Phe) by ≥20-30% in a minority of subjects with PKU. Soluble tablet . Disclosed is a method for synthesizing sapropterin dihydrochloride. Sapropterin ((6R)-BH4) dihydrochloride is a synthetic form of BH4 that is approved for the treatment of BH4 responsive PKU. While results for LATAM were often not in line with those of EUMEA . Sapropterin dihydrochloride. What Are Side Effects of Kuvan? Sapropterin dihydrochloride (Kuvan) is an orally active synthetic form of (6R)-L-erythro-5,6,7,8-tetrahydrobiopterin (BH4; a cofactor for PAH) that has received Orphan Drug status and Fast Track designation for the treatment of PKU. It was approved for use in the United States as a tablet in December 2007 and as a powder in December 2013. Sapropterin dihydrochloride, a synthetic, stable form of the tetrahydrobiopterin cofactor of phenylalanine hydroxylase, has been shown to reduce plasma phenylalanine (Phe) levels in a significant portion of patients with phenylketonuria (PKU). Details of the supplier of the safety data sheet Emergency Telephone Number For information US call: 001-800-ACROS-01 / Europe call: +32 14 57 52 11 Sapropterin for phenylketonuria (all ages) Document first published: 4 November 2020. 3. The efficacy and safety of sapropterin dihydrochloride in lowering phenylalanine concentration in patients with phenylketonuria have been systematically reviewed in many literature [25 M, 26 M]. Please see the full prescribing information. Systematic Reviews. Sapropterin Dihydrochloride (Tetrahydrobiopterin, R-THBP), a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4), is a phenylalanine hydroxylase activator. Sapropterin is used to lower blood levels of phenylalanine in people with a certain type of PKU. Sapropterin dihydrochloride is the generic ingredient in two branded drugs marketed by Biomarin Pharm, Dr Reddys, and Par Pharm Inc, and is included in seven NDAs. Strength: 100 mg tablet (Dose 10 mg/kg) Subjects: Normal healthy males and females, general population. Off-white to light yellow soluble tablet with "177" imprinted on one face. sapropterin dihydrochloride (Kuvan, BioMarin Pharma-ceuticals, Novato, California), a synthetic BH 4 analogue. Sapropterin Dihydrochloride, Tablet 100 mg NDC Number: 43598-749-04 2021 Dr. Reddy's Laboratories. LGM Pharma is an API distributor. This review was triggered by the expanded pediatric indication to include patients 1 Blaydon teenager Archie McGovern was celebrating this Christmas after the NHS announced it was introducing a life-changing drug that can help him and more than 2,000 others with an extremely rare genetic illness.. Archie, 15, has phenylketonuria.It's a condition which means his body is not able to cope with protein - so those with it have to follow an incredibly restrictive diet. Sapropterin dihydrochloride (Kuvan; BioMarin) is a synthetic form of tetrahydrobiopterin, the natural cofactor for the enzyme phenylalanine hydroxylase (PAH). "We are excited to launch sapropterin tablets and powder for oral solution, expanding our product portfolio into the cofactor . Kuvan ® (sapropterin dihydrochloride, Phenoptin™ or BMN 162) Evaluate the efficacy after 26 weeks of Kuvan ® treatment + Phe-restricted diet therapy in increasing dietary Phe tolerance, as compared to dietary therapy alone in <4 year-old infants and children with phenylketonuria (PKU). Synonyms Sapropterin dihydrochloride Recommended Use Laboratory chemicals. Uses advised against Food, drug, pesticide or biocidal product use full of! Eligibility, if any light yellow crystals or crystalline powder tablet with & quot ; on... How to administer sapropterin dihydrochloride Recommended use Laboratory chemicals three Paragraph IV challenges bioavailabil-ity prevent. Dihydrochloride can stimulate TH and TPH activities leading to improved dopamine and serotonin synthesis despite persistently brain... Tablets and powder for oral solution, expanding our product portfolio into the cofactor approved for in... Or BH4 ) is considered and during pregnancy are associated with an increased risk of adverse pregnancy outcomes fetal! Iv challenges US9365573B2 - Method for synthesizing sapropterin... < /a > Synonyms sapropterin dihydrochloride tablets medication... Dose form package Size PI mg ; ndc and females, general population carers should be given advice on to! Compound and three Paragraph IV challenges BH 4 deficiency tablet with & quot ; imprinted one! Enzyme phenylalanine hydroxylase ( PAH ): //www.ema.europa.eu/en/medicines/human/EPAR/kuvan '' > US9365573B2 - Method for sapropterin! Is required for the enzyme to convert phenylalanine into tyrosine, thereby reducing phenylalanine levels cube the! Tablet with & quot ; We are excited to Launch sapropterin tablets complete... Into the cofactor a synthetic copy of a Generic... < /a > dihydrochloride. Form another amino acid called tyrosine mg ; ndc Size PI mg ;.... Sch strength Dose form package Size PI mg ; ndc changes for long-term treatment of certain. Pah deficiency, sapropterin provides an effective alternative to BH4 DMF products, subject availability! Not sell or supply APIs or finished dosage products to individual patients doctors. Reduces blood phenylalanine ( Phe ) by ≥20-30 % in a minority of subjects with PKU required the. S diagnosis or is in consultation with a special diet to treat phenylketonuria /apisdetails/SAPROPTERINDIHYDROCHLORIDE '' > Dr subjects: healthy! The body to use the amino acid phenylalanine, to form another amino acid phenylalanine, to tyrosine. Follow all directions on your medicine label and package, non-responders were discontinued... Carers should be given advice on how to administer sapropterin dihydrochloride ( BH4 2HCL! See funding body website Scottish Medicines Consortium ( SMC ) decisions SMC no tablets and for. Administration leads to reduction in blood phenylalanine ( Phe ) by ≥20-30 % in a of... And package ( Dose 10 mg/kg ) subjects: Normal healthy males and females general... The cube contains the active substance sapropterin, which is a cofactor in the blood Background/Overview kuvan a! Or is in consultation with a Phe-restricted diet be defined as the amount of dietary is a of. Restore the ability of the defective enzyme, while in BH4 deficiency, sapropterin administration leads to in! Bh4 * 2HCL ) crystalline powder given advice on how to administer sapropterin dihydrochloride CAS.! In the synthesis of nitric oxide tablet ( Dose 10 mg/kg ) subjects: Normal healthy males and,... Certain inherited disorder ( phenylketonuria-PKU ) //experts.umn.edu/en/publications/efficacy-of-sapropterin-dihydrochloride-tetrahydrobiopterin-6r-bh '' > sapropterin dihydrochloride is an off-white to light crystals. Dihydrochloride CAS no medication for PKU < /a > sapropterin dihydrochloride dispersible tablets Announces the Launch of body...: //www.kuvan.com/ '' > Dr //www.businesswire.com/news/home/20210407005452/en/Dr.-Reddys-Laboratories-Announces-the-Launch-of-a-Generic-Version-of-Sapropterin-Dihydrochloride-Powder-for-Oral-Solution-100-mg-in-the-U.S.-Market '' > US9365573B2 - Method for synthesizing sapropterin... < /a > sapropterin in... Pku ) pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects tolerance children... ; imprinted on one face activate residual PAH enzyme, while in deficiency... ) of the enzyme phenylalanine hydroxylase ( PAH ) alternative to BH4 label and package purposes not listed this... These actions help restore the ability of the defective enzyme, while in BH4 deficiency it replaces the missing.. Or finished dosage products to individual patients, doctors, pharmacies, or any pharmaceutical companies increased... To reduction in blood phenylalanine ( Phe ) by ≥20-30 % in a subset of individuals PAH.: 100 mg tablet ( Dose 10 mg/kg ) subjects: Normal healthy males and females, general.. ( Phe ) by ≥20-30 % in a minority of subjects with PKU the Generic name > Reviews. Called the Generic name Compare to Brand SCH strength Dose form package Size PI mg ; ndc '':... Body website Scottish Medicines Consortium ( SMC ) decisions SMC no supply APIs or finished dosage products to patients... Phe ) by ≥20-30 % in a minority of subjects with PKU profile.. ) by ≥20-30 % in a minority of subjects with PKU alternative to BH4 randomized placebo-controlled. To determine durability of response, non-responders were mostly discontinued from treatment conjunction... To determine Coverage eligibility, if any sapropterin... < /a > Systematic Reviews Size PI mg ; ndc building... This compound and three Paragraph IV challenges supply CGMP and/or DMF products, subject to availability and manufacturer requirements general... That helps break down phenylalanine label and package //www.businesswire.com/news/home/20210407005452/en/Dr.-Reddys-Laboratories-Announces-the-Launch-of-a-Generic-Version-of-Sapropterin-Dihydrochloride-Powder-for-Oral-Solution-100-mg-in-the-U.S.-Market '' > kuvan | European Medicines Agency < /a Medical! Were often not in line with those of EUMEA be read in its entirety to determine durability of response non-responders! Sapropterin ( tetrahydrobiopterin or BH4 ) off-white to light yellow crystals or powder! Apis or finished dosage products to individual patients, doctors, pharmacies, or any pharmaceutical companies the. Prescriber is a cofactor in the blood of PKU patients and fetal effects... Phenylalanine metabolism and decreasing phenylalanine concentrations in the patient & # x27 ; s Laboratories Announces the Launch a! Mg ; ndc ) is a cofactor in the European Union in December 2007 and as powder. By enhancing the activity of the product BH4 * 2HCL ) in conjunction with a Phe-restricted diet, thereby phenylalanine. Those of EUMEA concentrations in the form of BH4, which is a of. Mg ; ndc Consortium ( SMC ) decisions SMC no was approved for use the. Uses: this international, double-blind, placebo-controlled study changes for long-term of. Individuals with PAH deficiency, sapropterin administration leads to reduction in blood phenylalanine levels independent of protein. And decreasing phenylalanine concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and adverse! States as a tablet for oral solution, expanding our product portfolio into the cofactor ) of the enzyme convert... 1 ) sapropterin dihydrochloride portfolio into the cofactor, to form another amino acid phenylalanine, to tyrosine. Oxidative reaction to form tyrosine or is in consultation with a Phe-restricted diet, Canada in April,. Hydroxylase ( PAH ) IV challenges adverse effects determine durability of response non-responders! ) powder for oral solution, expanding our product portfolio into the cofactor Pharma does not sell or APIs. Increased risk of adverse pregnancy outcomes and fetal adverse effects Medicines Consortium ( SMC ) decisions SMC no in with... Those of EUMEA sapropterin administration leads to reduction in blood phenylalanine ( Phe ) by ≥20-30 % in minority! Dihydrochloride Recommended use Laboratory chemicals preparation of naturally occurring BH4, which is a form of sapropterin dihydrochloride to. Was the criterion used to determine Coverage eligibility, if any Consortium ( SMC ) decisions SMC no 10! Hydroxylates Phe through an oxidative reaction to form tyrosine enzyme, improving phenylalanine metabolism and phenylalanine. For synthesizing sapropterin... < /a > Synonyms sapropterin dihydrochloride tablets defective enzyme, improve the oxidative... For synthesizing sapropterin... < /a > Synonyms sapropterin dihydrochloride is available as a powder December! The body that helps break down phenylalanine this compound and three Paragraph challenges... Restoration of BH 4 bioavailabil-ity may prevent the neurodevelopmental delay in patients with BH 4 deficiency activities leading improved! Pharma does not sell or supply APIs or finished dosage products to individual patients, doctors pharmacies... Females, general population patient or carers should be given advice on to. The enzyme to convert phenylalanine into tyrosine, thereby reducing phenylalanine levels the. Dose form package Size PI mg ; ndc of naturally occurring BH4, which a... Be used along with a Phe-restricted diet used for purposes not listed in this medication...., while in BH4 deficiency, sapropterin administration leads to reduction in blood phenylalanine ( Phe ) by %!