Information about Orfadin Suspension (Nitisinone)
Orfadin Suspension (Nitisinone) is an oral liquid form of the medication Nitisinone, which is used primarily in the treatment of hereditary tyrosinemia type 1 (HT-1). HT-1 is a rare and potentially life-threatening metabolic disorder caused by a deficiency of the enzyme fumarylacetoacetate hydrolase (FAH). The suspension formulation of Orfadin is especially helpful for patients who have difficulty swallowing capsules, such as young children or those with certain medical conditions.
Product Highlights
- Orfadin Suspension is used to treat HT-1, a genetic disorder where the body cannot break down the amino acid tyrosine due to a deficiency in the FAH enzyme.
- This leads to the accumulation of toxic metabolites, resulting in damage to the liver, kidneys, and nervous system.
- Orfadin works by inhibiting the enzyme HPPD, thereby preventing the buildup of these toxic metabolites.
Key Ingredient
Key Benefits of Orfadin Suspension (Nitisinone)
- By inhibiting HPPD, Orfadin reduces the production of harmful byproducts like succinylacetone that accumulate in HT-1, helping to prevent damage to vital organs.
- Orfadin helps to protect the liver and kidneys from the severe damage that untreated HT-1 can cause, reducing the risk of liver failure, kidney dysfunction, and other complications.
- By controlling the toxic byproducts of tyrosine metabolism, Orfadin can help to prevent neurological symptoms such as developmental delays, movement disorders, and intellectual impairment associated with HT-1.
- The oral suspension formulation makes it easier for patients, particularly children or those with difficulty swallowing pills, to take the medication.
Direction of Use
- Orfadin Suspension is taken by mouth, with the dosage typically based on the patient’s body weight.
- The usual starting dose is 1 mg per kg of body weight, taken once daily. The dose may be adjusted by the healthcare provider based on the patient’s response and clinical status.
- The suspension should be measured with an accurate device (e.g., a syringe or dosing cup) to ensure the correct dose is given.
- Orfadin Suspension can be taken with or without food, but it is important to follow any specific instructions provided by the healthcare provider.
- Patients will typically require regular blood tests to monitor tyrosine levels, liver function, and other parameters to ensure the dose is correct and well-tolerated.
Safety Concerns
- Prolonged use of Orfadin can cause eye-related issues, such as changes in the cornea or other ocular complications. Regular eye exams are recommended for patients on long-term therapy.
- Monitoring tyrosine levels is crucial during treatment, as excessive tyrosine can lead to adverse effects. The dose may need to be adjusted to maintain a safe balance.
- Orfadin can affect liver function, so liver enzymes and function should be monitored regularly during treatment.
- Some patients may experience side effects such as nausea, vomiting, diarrhea, skin rash, or irritability. Serious side effects are rare but can include eye-related issues, liver dysfunction, and changes in blood counts.
- Orfadin may interact with other medications, so it is important to inform the healthcare provider of all other drugs being taken, including over-the-counter medications and supplements.
Avoid Orfadin Suspension (Nitisinone) If
- Do not use Orfadin if you have a known allergy or hypersensitivity to nitisinone or any of the other ingredients in the formulation.
- Patients with existing serious eye conditions (e.g., significant corneal disorders) should avoid Orfadin due to the potential for eye-related side effects.
- While Orfadin is used to treat HT-1-related liver problems, caution is needed in individuals with other forms of severe liver disease that are not associated with HT-1.
- The safety of Orfadin during pregnancy and breastfeeding has not been well-established. Pregnant or breastfeeding women should only use the medication if the benefits outweigh the potential risks, and this should be discussed with their healthcare provider.
This product requires special packaging to maintain its integrity during the shipping process. DO NOT USE THIS MEDICATION if the attached temperature indicator shows that the medication was exposed to temperatures below 2 degrees or above 8 degrees Celsius, and contact the pharmacy immediately.
