Orkambi Price, Side effects, Dosage for Cystic Fibrosis Treatment

Orkambi, breakthrough drugs for Cystic Fibrosis treatment was approved by FDA approved in 2015. Orkambi is orally-delivered medicine in the form of fully co-formulated tablets i.e. a combination therapy that contains two drugs lumacaftor and ivacaftor.

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) is a protein that is responsible for regulating the proper flow of important salts such as chloride and sodium (salt) in as well as out of cells lining the lungs and other organs. In patients, above mentioned protein is either defective or missing, that leads towards malfunctioning of CFTR protein, i.e. a poor flow of salt and water into and out of cells in a number of organs, including the lungs. This causes thick, sticky mucus to develop inside the body and very salty perspiration.

Orkambi (Lumacaftor-Ivacaftor) is an oral medication that is used to treat CF. It basically targets the underlying cause of the disease in people having two copies of most common CF gene mutation, inherited from each parent though parents doesn’t have the disease. This type of gene mutation causes not only abnormal flow of salts across the cells but it can also lead to complications such as infections, diabetes as well as life threatening lung damage.

Orkambi Price, Side effects, Dosage for Cystic Fibrosis Treatment

Orkambi Price, Side effects, Dosage for Cystic Fibrosis Treatment

Please find below the detailed description about Orkambi price, side effects, dosage recommended for Cystic Fibrosis treatment:

Orkambi (Lumacaftor-Ivacaftor) Side effects

Shortness of breath, upper respiratory tract infection, nausea, diarrhea, and rash are the most common side effects of the drug. In women, increased menstrual abnormalities such as increased bleeding can also occur. Sore throat and nasal congestion (nasopharyngitis), fatigue, runny nose, and flu symptoms are also expected as side effects of this drug.

The first and foremost precaution is that if you are allergic to ivacaftor or lumacaftor, you shouldn’t use Orkambi. In addition, there are some medicines that can interact with Orkambi, so they shouldn’t use at same time. If you are using following drugs, then you should consult your doctor and take advice before using Orkambi, rifabutin or rifampin, St. John’s wort, seizure medicine like carbamazepine, phenobarbital, phenytoin, medicine that are used to prevent organ transplant rejection like everolimus (Zortress), sirolimus, tacrolimus, a sedative – triazolam or oral midazolam e.t.c.

In case of having liver, kidney or lung disease or asthma, you should also tell your doctor before using Orkambi. In case of pregnancy or having a plan for it, consultancy is also important although an unborn baby is not expected to be harmed. Birth control precautions may no longer effective during treatment. Mothers should also avoid breastfeeding during treatment.

Orkambi Dosage in Adults

Recommended dose for Adults and pediatric patients with age 12 years or above is two tablets (each containing lumacaftor 200 mg/ivacaftor 125 mg), taken orally every 12 hours. Patients are recommended to take tablet with some fat containing products such as butter, whole milk dairy products like whole milk, yogurt or cheese, peanut butter as well as nuts. Patients having moderate or severe hepatic impairment are advised to reduce the dose.

Patients taking strong CYP3A inhibitors should also reduce Orkambi dose for the first week of treatment and afterwards they can take normal dose. If a patient misses a dose, then there can be two possibilities, patient should take the dose if he remembers the missed dose within 6 hours, but in case of more than six hours after the usual dosing time, the patient should skip that dose and resume the normal schedule for the following dose. In any case, double dose is not recommended.

Orkambi Cost/Price for Cystic Fibrosis treatment

According to some authentic sources, the company plans to charge about $259,000 per patient annually. Though this drug is not approved yet in other continents such as Europe but company will start shipping the drug to US care centers within days. In clinical trials, the improvement that Orkambi yielded was not as much as Kalydeco’s , and as a result it is widely expected that Vertex Orkambi Price will be little lower as compare to Vertex Kalydeco Price which was $312,000 annually.

VERTEX is often blamed for overcharging but the company spokesman justified the high Orkambi price by giving the following reasons. He said that small patient population and the clinical benefit of a drug combination that actually treats the underlying cause of the disease are the factors behind the higher price In addition, the time and cost of Vertex’s efforts to bring the medicine to market is also contributing in its price.

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