Orfadin helps prevent the buildup of specific metabolic compounds.
Orfadin (previously known as NTBC) used in conjunction with dietary restriction of tyrosine and phenylalanine was approved by the FDA in 2002 for the treatment of patients with HT-1.
Before the drug Orfadin (nitisinone) was available, the only treatment for tyrosinemia was diet and ultimately a liver transplant. A liver transplant is a major operation, and people who have one usually need to take medicines (known as immunosuppressants) for the rest of their lives.
Orfadin can help block the breakdown of tyrosine before it can be changed into harmful substances. Remember that staying on a diet that limits tyrosine and phenylalanine is required when taking Orfadin. Your doctor will prescribe you with a special low-protein diet. Please contact your doctor with any questions regarding your dietary needs.
Orfadin is prescribed for daily administration. Orfadin can be administered as capsules or as a 4 mg/mL Oral Suspension; the dose and form of Orfadin will be decided by your child's doctor. If you have questions about Orfadin capsules or the new Oral Suspension, please speak with your doctor or contact an Orfadin4U specialist at 1-877-473-3179 (between 7 AM - 6 PM CST).
For patients who prefer capsules, Orfadin is available in 2 mg/kg, 5 mg/kg, 10 mg/kg and 20 mg/kg capsules. Our 20 mg capsules may help reduce the number of pills you (or your child) take at morning and even doses. For young children, capsules may be opened and the contents suspended in a small amount of water, formula or applesauce immediately before use. Orfadin capsules must be kept refrigerated at 36-46°F (2-8°C).
Orfadin Oral Suspension was specially developed for infants and toddlers or those who may have difficulty swallowing capsules. Strawberry-flavored and available as a 4-mg/mL liquid, the Oral Suspension is administered in the child's mouth without dilution using a dosing oral syringe. The Oral Suspension may be stored for up to 60 days at room temperature (not any higher than 77°F (25°C)). After 60 days at room temperature, any unused Orfadin needs to be discarded, even if you had put it in the refrigerator for a short time. For the longest shelf life, keep Orfadin in the refrigerator all the time at 36-46°F (2-8°C). For additional information on how to properly dispense and store Oral Suspension please see the "Instructions for Use" section within the full Prescribing Information
The amount of medication needed will change as your child grows, so don't be surprised when the dose alters. There are side effects associated with Orfadin. It is important to talk to your doctor about any adverse events your child experiences.
Important safety information related to Orfadin.
- Treatment with Orfadin should be initiated by a physician experienced in the treatment of HT-1. Orfadin should be taken at least one hour before, or two hours after a meal.
- For young children, capsules may be opened and the contents suspended in a small amount of water, formula or apple sauce immediately before use.
- Patients and their parents or caregivers should be advised of the need to maintain dietary restriction of tyrosine and phenylalanine when taking Orfadin.