Cystic Fibrosis (CF)
1 FDA approval
0 Active Studies for Orkambi

What is Orkambi

LumacaftorThe Generic name of this drug
Treatment SummaryLumacaftor 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
Orkambiis the brand name
image of different drug pills on a surface
Orkambi Overview & Background
Brand Name
Generic Name
First FDA Approval
How many FDA approvals?

Approved as Treatment by the FDA

Lumacaftor, otherwise known as Orkambi, is approved by the FDA for 1 uses such as Cystic Fibrosis (CF) .
Cystic Fibrosis (CF)
Used to treat Cystic Fibrosis (CF) in combination with Ivacaftor


How Orkambi Affects PatientsStudies 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 bodyLumacaftor 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.
Cystic Fibrosis (CF)
, 150.0 mg, 100.0 mg, 75.0 mg, 200.0 mg
Granule - Oral, Granule, Tablet - Oral, Tablet, film coated, Oral, , Tablet, film coated - Oral, Tablet


Orkambi Contraindications
Risk Level
Severe Hypersensitivity Reactions
Do Not Combine
Lumacaftor may interact with Pulse Frequency
There are 20 known major drug interactions with Orkambi.
Common Orkambi Drug Interactions
Drug Name
Risk Level
The metabolism of 1,2-Benzodiazepine can be increased when combined with Lumacaftor.
3,5-diiodothyropropionic acid
The metabolism of 3,5-diiodothyropropionic acid can be increased when combined with Lumacaftor.
The metabolism of 5-androstenedione can be increased when combined with Lumacaftor.
The metabolism of 6-O-benzylguanine can be increased when combined with Lumacaftor.
The metabolism of 7-ethyl-10-hydroxycamptothecin can be increased when combined with Lumacaftor.
Orkambi Toxicity & Overdose RiskCommon 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.
image of a doctor in a lab doing drug, clinical research

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).
Clinical Trials
Trial Phases
Cystic Fibrosis (CF)
0 Actively Recruiting

Orkambi Reviews: What are patients saying about Orkambi?

2.3Patient Review
Orkambi for Cystic Fibrosis with Homozygous F508del Genetic Mutation in CFTR Gene
My 14 year old daughter's PFTs have decreased since she started taking this medication two years ago. Additionally, she has been having vision problems, brain fog, and joint pain. We're attributing all of these issues to the medicine and will be stopping it as soon as we get approval from her doctor.
image of drug pills surrounding a glass of water symbolizing drug consumption

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