Sapropterin Dihydrochloride
Sapropterin Dihydrochloride Prescribing Information
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.
Take sapropterin dihydrochloride powder for oral solution orally with a meal, preferably at the same time each day
Sapropterin dihydrochloride powder for oral solution should be dissolved in 120 to 240 mL of water or apple juice and taken orally within 30 minutes of dissolution. Sapropterin dihydrochloride powder for oral solution may also be stirred in a small amount of soft foods such as apple sauce or pudding. Empty the contents of the packet(s) in water, apple juice, or a small amount of soft foods and mix thoroughly. The powder should dissolve completely.
For infants weighing 10 kg or less, sapropterin dihydrochloride powder for oral solution can be dissolved in as little as 5 mL of water or apple juice and a portion of this solution corresponding to a 10 mg/kg dose may be administered orally via an oral dosing syringe. Table 1 provides dosing information for infants at the recommended starting dose of 10 mg/kg per day. Refer to Table 2 for dosing information at 20 mg/kg per day if dosage adjustment is needed.
Patient Weight (kg) | Starting Dose: 10 mg/kg per day* | |||
Dose (mg) | Sapropterin Dihydrochloride Powder for Oral Solution 100 mg Packets Dissolved† | Dilution Volume (mL)‡ | Administered Dose volume (mL)§ | |
1 | 10 | 1 | 10 | 1 |
2 | 20 | 1 | 10 | 2 |
3 | 30 | 1 | 10 | 3 |
4 | 40 | 1 | 10 | 4 |
5 | 50 | 1 | 10 | 5 |
6 | 60 | 1 | 5 | 3 |
7 | 70 | 1 | 5 | 3.5 |
8 | 80 | 1 | 5 | 4 |
9 | 90 | 1 | 5 | 4.5 |
10 | 100 | 1 | 5 | 5 |
*Starting dose for infants is 10 mg/kg per day. Dosing information for 20 mg/kg per day is provided in Table 2.
†Powder for oral solution provided in single use packets containing 100 mg sapropterin dihydrochloride per packet
‡Volume of water or apple juice to dissolve Sapropterin Dihydrochloride Powder for Oral Solution.
§Discard remainder of mixture after volume to be administered is drawn.
Patient Weight (kg) | 20 mg/kg per day | |||
Dose (mg) | Sapropterin Dihydrochloride Powder for Oral Solution 100 mg Packets* Dissolved | Dilution Volume (mL)† | Administered Dose volume (mL)§ | |
1 | 20 | 1 | 5 | 1 |
2 | 40 | 1 | 5 | 2 |
3 | 60 | 1 | 5 | 3 |
4 | 80 | 1 | 5 | 4 |
5 | 100 | 1 | 5 | 5 |
6 | 120 | 2 | 5 | 3 |
7 | 140 | 2 | 5 | 3.5 |
8 | 160 | 2 | 5 | 4 |
9 | 180 | 2 | 5 | 4.5 |
10 | 200 | 2 | 5 | 5 |
*Powder for oral solution provided in single use packets containing 100 mg sapropterin dihydrochloride per packet
†Volume of water or apple juice to dissolve Sapropterin Dihydrochloride Powder for Oral Solution.
§Discard remainder of mixture after volume to be administered is drawn.
Sapropterin dihydrochloride powder for oral solution is available as a unit dose packet containing 500 mg of sapropterin dihydrochloride (equivalent to 384 mg of sapropterin base). The powder is off-white to lightly yellow in color.
None.
Some patients with PKU do not show biochemical response (reduction in blood Phe) with treatment with sapropterin dihydrochloride. In two clinical trials at a sapropterin dihydrochloride dose of 20 mg/kg per day, 56% to 75% of pediatric PKU patients showed a biochemical response to sapropterin dihydrochloride, and in one clinical trial at a dose of 10 mg/kg per day, 20% of adult and pediatric PKU patients showed a biochemical response to sapropterin dihydrochloride
Biochemical response to sapropterin dihydrochloride treatment cannot generally be pre-determined by laboratory testing (e.g., molecular testing), and should be determined through a therapeutic trial (evaluation) of sapropterin dihydrochloride response
The following adverse reactions have been reported during post-approval use of sapropterin dihydrochloride. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.