Orkambi
Cystic Fibrosis
Treatment
2 FDA approvals
0 Active Studies for Orkambi
Treatment for
Cystic Fibrosis
What is Orkambi
Lumacaftor
The Generic name of this drug
Treatment Summary
Lumacaftor is a drug used to treat Cystic Fibrosis (CF), a genetic disorder that causes thick and sticky mucus in the lungs, pancreas, and other organs. Lumacaftor is often used in combination with the drug ivacaftor (brand name Kalydeco) and sold as the fixed dose combination product Orkambi, which is approved to treat CF in patients age 6 and older. Lumacaftor helps stabilize the CFTR protein, which is affected by CF, allowing for increased production of ion channels and increased fluid and ion transport across cell membranes. Orkambi has
Orkambi
is the brand name
Orkambi Overview & Background
Brand Name
Generic Name
First FDA Approval
How many FDA approvals?
Orkambi
Lumacaftor
2015
5
Approved as Treatment by the FDA
Lumacaftor, otherwise known as Orkambi, is approved by the FDA for 2 uses such as Cystic Fibrosis (CF) and Cystic Fibrosis .
Cystic Fibrosis (CF)
Used to treat Cystic Fibrosis (CF) in combination with Ivacaftor
Cystic Fibrosis
Used to treat Cystic Fibrosis (CF) in combination with Ivacaftor
Effectiveness
How Orkambi Affects Patients
Studies have shown that Orkambi (lumacaftor/[DB08820]) helps improve lung function, reduces the risk of breathing problems, lowers sweat chloride, increases weight gain, and makes CF symptoms and quality of life better. It has not been found to cause any significant increase in the QTc interval, which is a measure of electrical activity in the heart.
How Orkambi works in the body
Lumacaftor helps the body process a mutated protein that causes Cystic Fibrosis. It keeps the protein from becoming misshapen and getting destroyed in the body. This helps the body process it correctly, so it can do its job of regulating the flow of salt in and out of cells.
When to interrupt dosage
The advised measure of Orkambi is contingent upon the determined disorder. The amount of dosage fluctuates as per the delivery methodology (e.g. Granule - Oral or Granule) noted in the table beneath.
Condition
Dosage
Administration
Cystic Fibrosis
200.0 mg, , 100.0 mg, 150.0 mg
Tablet, film coated - Oral, Tablet, film coated, Oral, , Tablet, Tablet - Oral, Granule - Oral, Granule
Warnings
There are 20 known major drug interactions with Orkambi.
Common Orkambi Drug Interactions
Drug Name
Risk Level
Description
1,2-Benzodiazepine
Major
The metabolism of 1,2-Benzodiazepine can be increased when combined with Lumacaftor.
3,5-diiodothyropropionic acid
Major
The metabolism of 3,5-diiodothyropropionic acid can be increased when combined with Lumacaftor.
5-androstenedione
Major
The metabolism of 5-androstenedione can be increased when combined with Lumacaftor.
6-O-benzylguanine
Major
The metabolism of 6-O-benzylguanine can be increased when combined with Lumacaftor.
7-ethyl-10-hydroxycamptothecin
Major
The metabolism of 7-ethyl-10-hydroxycamptothecin can be increased when combined with Lumacaftor.
Orkambi Toxicity & Overdose Risk
Common side effects of Orkambi include shortness of breath, sore throat, nausea, diarrhea, upper respiratory infection, fatigue, abnormal breathing, increased levels of an enzyme in the blood, rash, gas, runny nose, and the flu.
Orkambi Novel Uses: Which Conditions Have a Clinical Trial Featuring Orkambi?
One active trial is examining the effectiveness of Orkambi in relieving symptoms associated with Cystic Fibrosis (CF).
Condition
Clinical Trials
Trial Phases
Cystic Fibrosis
0 Actively Recruiting
Orkambi Reviews: What are patients saying about Orkambi?
2.3
Patient Review
10/16/2017
Orkambi for Cystic Fibrosis with Homozygous F508del Genetic Mutation in CFTR Gene
Patient Q&A Section about orkambi
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What are the side effects of Orkambi?
"The following are some of the common side effects that may occur when taking this medication: breathing problems such as shortness of breath and chest tightness; nausea; diarrhea; fatigue; increase in a certain blood enzyme called creatine phosphokinase; rash; gas; and the common cold, including sore throat, stuffy or runny nose."
Answered by AI
How effective is Orkambi?
"Orkambi, a lumacaftor/ivacaftor combination, can improve overall health in adolescents and adults with cystic fibrosis. However, the study found that its use can be risky for those who do not tolerate the therapy."
Answered by AI
What does the drug Orkambi do?
"These cells are also responsible for sweeping bacteria and other particles out of the lungs.
Orkambi is a medicine used to treat cystic fibrosis in patients aged 2 years and above. Cystic fibrosis is an inherited disease that has severe effects on the lungs and the digestive system. It affects the cells that produce mucus and digestive juices. These cells are also responsible for sweeping bacteria and other particles out of the lungs."
Answered by AI
What is the difference between Orkambi and Trikafta?
"Trikafta is a three-drug combination of ivacaftor, tezacaftor and elexacaftor (previously coded as VX-445). Symdeko is a two-drug combination of ivacaftor and tezacaftor, while Orkambi is a combination of ivacaftor with lumacaftor."
Answered by AI
How much does Orkambi cost?
"The list prices for Orkambi and Symdeko are around $272,000 and $292,000 respectively. Analysts expect that the early approval of Trikafta will lead to an acceleration of its launch date, with the drug expected to make $630 million in 2020."
Answered by AI
What is Orkambi used for?
"ORKAMBI is a prescription medicine that is used to treat cystic fibrosis in patients that are 1 year old or older and have two copies of the F508del mutation in their CFTR gene."
Answered by AI