Where is orkambi available?

The CF drug Orkambi is now available in Austria, Germany, Ireland, Italy, the Netherlands, Sweden and the U.S. Read the full statement from Vertex below: Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI® (lumacaftor/ivacaftor)

How old do you have to be to take Orkambi? What is the drug for? ORKAMBI is a treatment for cystic fibrosis (CF) in patients 12 years and older with two copies of a specific gene mutation called F508del. Having two copies of this mutation (one inherited from each parent) is the leading cause of CF.

Who is the manufacturer of Orkambi in the UK? Orkambi is a drug manufactured by Vertex Pharmaceuticals, and treats the F508del mutation, which around 50 per cent of sufferers in the UK have.

How much does Orkambi cost for cystic fibrosis? Orkambi (ivacaftor/lumacaftor) is a member of the CFTR combinations drug class and is commonly used for Cystic Fibrosis. The cost for Orkambi oral granule for reconstitution (125 mg-100 mg) is around $21,849 for a supply of 56 granule for reconstitution, depending on the pharmacy you visit.

Who are the patients in the Orkambi clinical trial? The efficacy of ORKAMBI in patients with cystic fibrosis (CF) who are homozygous for the F508del mutation in the CFTR gene was evaluated in two randomized, double‑blind, placebo‑controlled, 24‑week clinical trials (Trials 1 and 2) in 1108 clinically stable patients with CF.

How often should I give my Child Orkambi?

How often should I give my Child Orkambi? Take one sachet of ORKAMBI every 12 hours. Giving ORKAMBI to your child: Hold sachet of granules with cut line on top. Shake sachet gently to settle contents. Tear or cut sachet open along cut line. Mix the entire content of a sachet with 5 mL of age-appropriate soft food or liquid.

When to take Orkambi and what foods to eat? Orkambi is usually taken every 12 hours. Follow all directions on your prescription label and read all medication guides or instruction sheets. Take Orkambi with food that contains fat, such as butter, peanut butter, eggs, avocados, nuts, whole milk, cheese, yogurt, or cheese pizza. Follow your doctor’s dosing instructions very carefully.

When to see a doctor for Orkambi blood work? Ask your doctor or pharmacist if you are not sure about this list of medicines. Your doctor will do some blood tests to check your liver prior to and while you are taking ORKAMBI, particularly during the first year (every 3 months), and once a year after that.

Can you take Orkambi if you have cystic fibrosis? Orkambi is for use only in patients with a specific gene mutation related to cystic fibrosis. Before you take Orkambi, you may need a medical test to make sure you have this gene mutation.