"The cystic fibrosis (CF) drug Orkambi (Vertex Pharmaceuticals) has received a green light from the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP).
Orkambi is a fixed-dose oral combination "...
Orfadin Consumer (continued)
This medication may rarely cause a low number of blood cells such as white cells and platelets. This effect can decrease your body's ability to fight an infection or cause easy bruising/bleeding. Tell your doctor immediately if you develop any of the following unlikely symptoms: signs of infection (such as fever, chills, persistent sore throat), easy bruising/bleeding.
Your condition and this medication can cause liver problems. Tell the doctor immediately if any of these symptoms of liver problems occur: severe stomach/abdominal pain, persistent nausea or vomiting, yellowing eyes/skin, dark urine.
Your condition and this medication can cause high levels of tyrosine in the blood. Therefore, it is very important to follow the low protein/low tyrosine diet recommended by your doctor. See also Precautions section. Too much tyrosine may cause skin, eye, or brain problems. Tell the doctor immediately if any of these symptoms occur: eye redness/itching/discharge, vision changes, eye pain, eye sensitivity (especially to light), dry/itchy skin, sores on palms of hands/soles of feet, developmental delays (such as holding head up, rolling over, crawling).
A very serious allergic reaction to this drug is rare. However, seek immediate medical attention if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.
This is not a complete list of possible side effects. If you notice other effects not listed above, contact the doctor or pharmacist.
In the US -
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
In Canada - Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.
Read the Orfadin (nitisinone) Side Effects Center for a complete guide to possible side effects
PRECAUTIONS: Before giving nitisinone, tell the doctor or pharmacist if your child is allergic to it; or if your child has any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.
Before using this medication, tell the doctor or pharmacist your child's medical history, especially of: eye problems (such as cataracts, corneal ulcers).
Patients taking this medication are at risk for developing high levels of tyrosine in the blood, possibly leading to eye, skin, and nervous system problems. (See Side effects section.) To decrease the risk of high tyrosine levels, patients should follow a diet low in tyrosine and phenylalanine. Avoid foods and beverages that contain aspartame or are high in tyrosine or phenylalanine. Foods high in tyrosine include soy products, turkey, fish, avocados, bananas, yogurt, and lima beans. Foods high in phenylalanine include milk, chicken, eggs, cheese, peanut butter. (See also Drug Interactions section.)
During pregnancy, this medication should be used only when clearly needed. Consult your doctor before breast-feeding.
It is not known whether this drug passes into breast milk. If you are taking this medication and wish to breast-feed, consult your doctor first.
Additional Orfadin Information
Report Problems to the Food and Drug Administration
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit the FDA MedWatch website or call 1-800-FDA-1088.
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