Sapropterin Dihydrochloride
Generic Name: sapropterin dihydrochloride
Brand Names:
Sapropterin Dihydrochloride
11 DESCRIPTION Sapropterin dihydrochloride is an orally administered Phenylalanine Hydroxylase activator (or PAH activator). Sapropterin dihydrochloride, the active pharmaceutical ingredient in sapropterin dihydrochloride powder for oral solution, is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Sapropterin dihydrochloride is a white to pale yellow color powder.
Overview
11 DESCRIPTION Sapropterin dihydrochloride is an orally administered Phenylalanine Hydroxylase activator (or PAH activator). Sapropterin dihydrochloride, the active pharmaceutical ingredient in sapropterin dihydrochloride powder for oral solution, is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Sapropterin dihydrochloride is a white to pale yellow color powder.
Uses
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 (HPA) due to tetrahydrobiopterin-(BH4-) responsive Phenylketonuria (PKU). Sapropterin dihydrochloride powder for oral solution is to be used in conjunction with a Phe-restricted diet. Sapropterin dihydrochloride powder for oral solution is a phenylalanine hydroxylase activator 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).
Dosage
2 DOSAGE AND ADMINISTRATION All patients with PKU who are being treated with sapropterin dihydrochloride powder for oral solution should also be treated with a Phe-restricted diet, including dietary protein and Phe restriction. ( 2.1 ) Starting Dosage • Pediatric patients 1 month to 6 years : The recommended starting dosage of sapropterin dihydrochloride powder for oral solution is 10 mg/kg administered orally once daily. ( 2. 2) • Patients 7 years and older: The recommended starting dosage of sapropterin dihydrochloride powder for oral solution is 10 to 20 mg/kg administered orally once daily. ( 2. 2) Dosage Adjustment • Doses of sapropterin dihydrochloride powder for oral solution may be adjusted in the range of 5 to 20 mg/kg taken once daily. ( 2.
Side Effects
6 ADVERSE REACTIONS Most common adverse reactions (≥4%) are: headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, and nasal congestion. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Annora Pharma Private Limited at 1-866-495-1995, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to the rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. PKU Clinical Studies The safety of sapropterin dihydrochloride was evaluated in 7 clinical studies in patients with PKU (aged 1 month to 50 years) [see Clinical Studies (14) ].
Interactions
7 DRUG INTERACTIONS Table 4 includes drugs with clinically important drug interactions when administered with sapropterin dihydrochloride and instructions for preventing or managing them. Table 4: Clinically Relevant Drug Interactions Levodopa Clinical Impact Sapropterin dihydrochloride may increase the availability of tyrosine, a precursor of levodopa. Neurologic events were reported postmarketing in patients receiving sapropterin and levodopa concomitantly for a non-PKU indication [see Warnings and Precautions (5.5) ]. Intervention Monitor patients for a change in neurologic status.
Warnings
5 WARNINGS AND PRECAUTIONS • Hypersensitivity Reactions Including Anaphylaxis : Sapropterin dihydrochloride is not recommended in patients with a history of anaphylaxis to sapropterin dihydrochloride; discontinue treatment in patients who experience anaphylaxis and Initiate appropriate medical treatment. Continue dietary Phe restrictions. ( 5.1 ) • Upper Gastrointestinal Mucosal Inflammation : Monitor patients for signs and symptoms of these conditions including esophagitis and gastritis. ( 5.2 ) • Hypophenylalaninemia : Pediatric patients younger than 7 years treated with sapropterin dihydrochloride doses of 20 mg/kg per day are at increased risk for low levels of blood Phe compared with patients 7 years and older. 4 CONTRAINDICATIONS None. None. ( 4 )
Pregnancy
8.1 Pregnancy Risk Summary Available data from pregnancy safety studies, pharmacovigilance, and published case reports with sapropterin dihydrochloride use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes (see Data) . Uncontrolled blood phenylalanine concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects (see Clinical Considerations) .
Storage
16 HOW SUPPLIED/STORAGE AND HANDLING Sapropterin Dihydrochloride Powder for Oral Solution Supplied as off-white to yellow powder supplied in unit dose packets as follows: 100 mg sapropterin dihydrochloride per packet: Carton of 30 unit dose packets NDC 31722-047-30 Single unit dose packet NDC 31722-047-01 Carton of 1 unit dose packet NDC 31722-047-31 500 mg sapropterin dihydrochloride per packet:...
Frequently Asked Questions
What is Sapropterin Dihydrochloride used for?▼
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 (HPA) due to tetrahydrobiopterin-(BH4-) responsive Phenylketonuria (PKU). Sapropterin dihydrochloride powder for oral solution is to be used in conjunction with a Phe-restricted diet. Sapropterin dihydrochloride powder for oral solution is a phenylalanine hydroxylase activator 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).
What are the side effects of Sapropterin Dihydrochloride?▼
6 ADVERSE REACTIONS Most common adverse reactions (≥4%) are: headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, and nasal congestion. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Annora Pharma Private Limited at 1-866-495-1995, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to the rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. PKU Clinical Studies The safety of sapropterin dihydrochloride was evaluated in 7 clinical studies in patients with PKU (aged 1 month to 50 years) [see Clinical Studies (14) ].
Can I take Sapropterin Dihydrochloride during pregnancy?▼
8.1 Pregnancy Risk Summary Available data from pregnancy safety studies, pharmacovigilance, and published case reports with sapropterin dihydrochloride use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes (see Data) . Uncontrolled blood phenylalanine concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects (see Clinical Considerations) .
What are the important warnings for Sapropterin Dihydrochloride?▼
5 WARNINGS AND PRECAUTIONS • Hypersensitivity Reactions Including Anaphylaxis : Sapropterin dihydrochloride is not recommended in patients with a history of anaphylaxis to sapropterin dihydrochloride; discontinue treatment in patients who experience anaphylaxis and Initiate appropriate medical treatment. Continue dietary Phe restrictions. ( 5.1 ) • Upper Gastrointestinal Mucosal Inflammation : Monitor patients for signs and symptoms of these conditions including esophagitis and gastritis. ( 5.2 ) • Hypophenylalaninemia : Pediatric patients younger than 7 years treated with sapropterin dihydrochloride doses of 20 mg/kg per day are at increased risk for low levels of blood Phe compared with patients 7 years and older. 4 CONTRAINDICATIONS None. None. ( 4 )
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Medical Disclaimer
This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.