Lumacaftor and Ivacaftor
Lumacaftor and ivacaftor (Orkambi) is a combination medicine used to treat cystic fibrosis in patients aged 1 year and older who carry two copies of a specific CFTR gene mutation called F508del.
🧬 Where this information comes from Click to expand
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 1 clinical label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
📖 Lumacaftor and Ivacaftor: Patient Information Guide
A plain-language walkthrough of Lumacaftor and Ivacaftor — what it treats, how it works, how it’s taken, side effects, warnings and interactions. Tap any section to expand it.
🎯What it's used for▾
Lumacaftor and ivacaftor, sold together as Orkambi, is a combination medicine approved specifically for cystic fibrosis (CF) with the F508del/F508del mutation.
- Orkambi is approved to treat cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene — meaning they carry two copies of this specific mutation, one inherited from each parent.
- If a patient's genetic type (genotype) is not already known, an FDA-cleared CF mutation test must be used to confirm the F508del mutation on both copies of the CFTR gene before starting Orkambi.
- Orkambi has not been proven safe or effective for patients with CF who have a different mutation type — it is only for F508del/F508del.
⚙️How it works▾
In CF caused by the F508del mutation, the CFTR protein — a channel in cell surfaces that controls the flow of salt (chloride) — is misfolded. Most of it gets destroyed before it ever reaches the cell surface, and the little that does arrive there barely opens. Lumacaftor corrects the folding problem so more CFTR protein can make it to the cell surface. Ivacaftor then acts as a potentiator — it helps the channel open more often once it's there, improving chloride transport. Together they increase both the amount and the function of working CFTR protein, addressing the root defect in this type of CF.
💊How it's taken▾
Orkambi is taken by mouth twice a day, roughly every 12 hours. It must be taken with fat-containing food — things like eggs, cheese, nuts, butter, or whole milk — because food dramatically increases how much medicine your body absorbs. The right dose depends on your age and weight; your prescriber will determine this for you. Do not change your dose on your own, and do not skip doses without talking to your care team.
- Orkambi is available as tablets and as an oral powder — the powder formulation is generally used for younger children who cannot swallow tablets.
- If you are starting Orkambi while already taking a strong CYP3A inhibitor (such as certain antifungals), your doctor may adjust the starting schedule for the first week.
🤒Side effects — in detail▾
The most common side effects reported with Orkambi include:
- Shortness of breath or unusual breathing (especially in the first days of treatment)
- Runny nose, nasal congestion, or upper respiratory infections
- Nausea
- Diarrhea
- Fatigue
- Rash
- Gas (flatulence)
- Elevated creatine phosphokinase (a muscle enzyme) on blood tests
More serious reactions — including liver problems, severe allergic reactions (anaphylaxis), increased pressure in the skull, and mood or mental health changes including suicidal thoughts — have been reported and require prompt medical attention. Tell your doctor right away if you notice yellowing of the skin or eyes, severe headaches, vision changes, or any new or worsening anxiety or depression.
⚠️Warnings & precautions▾
- Liver problems: Elevated liver enzymes are common, and serious liver failure has been reported in patients with pre-existing advanced liver disease (cirrhosis with portal hypertension). Liver blood tests are required before starting and regularly during treatment.
- Respiratory effects: Breathing symptoms like chest tightness and shortness of breath are more common when first starting Orkambi — especially in patients with advanced lung disease. This can be serious enough to require stopping the medicine.
- Intracranial hypertension (raised pressure in the skull): Reported after Orkambi came to market. Signs include unusual or severe headaches and visual disturbances. Stop the medicine and seek evaluation if these occur.
- Neuropsychiatric effects: Anxiety, depression, insomnia, and suicidal thoughts or behaviors have been reported in both adults and children — sometimes within the first three months. Monitor closely.
- Serious allergic reactions: Anaphylaxis and angioedema (severe swelling) have been reported. Seek emergency help immediately if these occur.
- Blood pressure: Elevated blood pressure has been observed more often in patients taking Orkambi compared to placebo. Blood pressure should be monitored during treatment.
- Eye changes (cataracts): Non-congenital lens opacities have been reported in pediatric patients. Baseline and follow-up eye exams are recommended for children.
🔀What it interacts with▾
Orkambi interacts with a wide range of medications because lumacaftor is a strong inducer of an important liver enzyme (CYP3A) that processes many drugs. Key interactions include:
- Hormonal contraceptives (pill, patch, ring, implant, injection) — Orkambi significantly reduces their effectiveness and can cause irregular menstrual bleeding; a non-hormonal backup method is required.
- Rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, St. John's Wort — these reduce Orkambi's effectiveness and should be avoided.
- Azole antifungals (itraconazole, ketoconazole, voriconazole, posaconazole) — Orkambi reduces their levels significantly; this combination is not recommended.
- Certain antibiotics (clarithromycin, erythromycin, telithromycin) — Orkambi reduces their effectiveness; alternatives like azithromycin, ciprofloxacin, or levofloxacin are preferred.
- Immunosuppressants (cyclosporine, tacrolimus, sirolimus, everolimus) — avoid combining with Orkambi due to dramatically reduced drug levels.
- Certain antidepressants (citalopram, escitalopram, sertraline) — levels may be reduced; dose adjustment may be needed.
- Warfarin — Orkambi may alter warfarin levels; INR should be monitored.
- Digoxin — blood levels should be monitored and the dose adjusted as needed.
- Corticosteroids (prednisone, methylprednisolone) — Orkambi may reduce their effectiveness; higher doses may be needed.
- Ibuprofen — Orkambi may reduce its effectiveness; higher doses may be needed.
This is not a complete list of interactions. Always talk with your doctor or pharmacist before starting, stopping, or changing any medication.
📋Before taking it▾
- Advanced liver disease or cirrhosis: Use with extreme caution only if the benefit outweighs the risk; close monitoring is required. Serious liver failure, including deaths, has been reported in CF patients with cirrhosis and portal hypertension.
- Pregnancy: Human safety data are limited. Discuss the risks and benefits with your doctor before starting or continuing Orkambi during pregnancy.
- Breastfeeding: It is not known if Orkambi passes into human breast milk. Weigh the benefits of breastfeeding against potential risk to the infant and discuss with your doctor.
- Hormonal contraception: Do not rely on hormonal birth control while taking Orkambi — it will likely not be effective. Use a non-hormonal method.
- Children under 1 year: Safety and effectiveness have not been established.
- Children and teens: Periodic eye exams are important — lens opacities (cataracts) have been reported in pediatric patients taking Orkambi or ivacaftor.
- Older adults (65+): Clinical trial data in this age group are limited; use with the same monitoring as younger patients.
- Hepatic (liver) impairment: Patients with moderate or severe liver impairment may need a dosage adjustment; discuss this with your prescriber.
🚑If you take too much▾
There are no reported cases of overdose with Orkambi in patients. In a clinical study where healthy volunteers took higher-than-usual doses for 7 days, reported side effects included headache, elevated liver enzymes, and a generalized rash. There is no specific antidote for an Orkambi overdose. If an overdose is suspected, seek medical attention; treatment involves supportive care and monitoring of vital signs and clinical status.
📡Pharmacodynamics — effects in the body▾
One measurable sign that Orkambi is working is a reduction in sweat chloride — a standard test used in CF care. In clinical trials, patients treated with Orkambi showed meaningful and sustained reductions in sweat chloride levels, starting as early as the first 2 weeks of treatment. When Orkambi was stopped, sweat chloride levels returned toward baseline within 2 weeks, confirming the effect is directly tied to the medication being active in the body.
🔬Pharmacokinetics — how your body processes it▾
Both lumacaftor and ivacaftor are absorbed through the gut and reach steady (stable) blood levels after about 7 days of twice-daily dosing. Taking the medicine with fat-containing food roughly doubles lumacaftor absorption and triples ivacaftor absorption compared to taking it on an empty stomach — which is why food is required. Lumacaftor is cleared slowly (half-life around 26 hours) and is mostly eliminated unchanged in the stool. Ivacaftor is processed more quickly (half-life around 9 hours when taken with lumacaftor), primarily broken down by a liver enzyme called CYP3A, and eliminated mainly in the stool as well. Notably, lumacaftor strongly activates CYP3A, which means it speeds up the breakdown of ivacaftor — and many other medications — in the body.
📦How to store it▾
Store at 20°C - 25°C (68°F - 77°F); excursions permitted to 15°C - 30°C (59°F - 86°F) .
📄 Written from Lumacaftor and Ivacaftor’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Every point is grounded in that labeling — nothing is invented. How we use AI →
Supplements & herbs that interact with Lumacaftor and Ivacaftor
153 supplements and herbal products have documented interactions with Lumacaftor and Ivacaftor in the licensed clinical database we use. Most are manageable — but your pharmacist should know about everything you take, including vitamins and herbals.
- Major · 3
- Moderate · 110
- Minor · 40
Educational information from a licensed clinical database (Natural Medicines) — see our data sources & update cadence. Not medical advice: an interaction being documented does not mean it will happen to you; do not start or stop medications or supplements without professional guidance.
🩺 Pharmacist Counseling Corner
Quick, plain-English answers to the questions patients ask most about Lumacaftor and Ivacaftor — the kind of thing our pharmacy team would talk through with you at the counter.
Why does my doctor want me to eat something fatty before taking Orkambi? ▾
I use the birth control pill. Do I need to change anything while taking Orkambi? ▾
What should I watch for in the first few weeks of starting Orkambi? ▾
My child is taking Orkambi — do they need any special monitoring? ▾
Will Orkambi interact with my other CF medications? ▾
Is Orkambi a cure for cystic fibrosis? ▾
Is Lumacaftor and Ivacaftor available over the counter? ▾
When was Lumacaftor and Ivacaftor first available? ▾
Who makes Lumacaftor and Ivacaftor? ▾
💬 Answers drafted from Lumacaftor and Ivacaftor’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Grounded in that labeling — nothing is made up. How we use AI →
🧰 Keep exploring
💊 How Lumacaftor and Ivacaftor comes
Lumacaftor and Ivacaftor is available in 2 forms. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Oral powder
How this form is used
- Orkambi oral powder is used to treat cystic fibrosis in patients with two copies of the F508del mutation in the CFTR gene
- Orkambi oral powder is the appropriate formulation for younger pediatric patients who cannot swallow tablets
- Orkambi oral powder is taken by mouth twice daily (every 12 hours)
- Orkambi oral powder must be taken with fat-containing food to maximize absorption
- Follow your prescriber's exact instructions for how to prepare and give Orkambi oral powder based on your child's age and weight
Tablet
How this form is used
- Orkambi tablets are used to treat cystic fibrosis in patients with two copies of the F508del mutation in the CFTR gene
- Orkambi tablets are approved for use in patients aged 2 years and older (based on the age-appropriate dosing regimen)
- Orkambi tablets are taken by mouth twice daily (every 12 hours)
- Orkambi tablets must be taken with fat-containing food to maximize absorption
- Swallow Orkambi tablets whole; follow your prescriber's exact instructions for your age group and weight
📊 Lumacaftor and Ivacaftor across the U.S. market
How Lumacaftor and Ivacaftor appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
FDA-listed product records by dose form (tablet variants merged) — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
Share of FDA-listed product records by manufacturer — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
📈 How widely Lumacaftor and Ivacaftor is used
A general measure of how commonly Lumacaftor and Ivacaftor is prescribed, based on Medicaid — one of the largest public drug programs. The figure below is prescriptions filled in Q1–Q4 2025, summed across every form and brand of the drug.
Think of this as a proxy for how widely Lumacaftor and Ivacaftor is used, drawn from freely available public data. Medicaid is just one program — these numbers don’t include Medicare, other government coverage, or commercial and cash-pay prescriptions, so real-world use is higher than what’s shown here.
How commonly is Lumacaftor and Ivacaftor prescribed? Of the 1,594 medications we measure, Lumacaftor and Ivacaftor ranks #1,420 by Medicaid prescriptions — more than 11% of them.
Utilization by Lumacaftor and Ivacaftor product
Pick a product to see its own Medicaid numbers. Each chip is one clinical product — a specific strength & dosage form as defined by RxNorm — with brand-name and generic versions combined.
ivacaftor 125 MG / lumacaftor 100 MG Oral Granules Most dispensed
Summed across the 1 of 1 listed NDC products with Medicaid activity — every brand and generic of this exact strength & form. RxNorm 2053509
ivacaftor 94 MG / lumacaftor 75 MG Oral Granules
Summed across the 1 of 1 listed NDC products with Medicaid activity — every brand and generic of this exact strength & form. RxNorm 2611458
ivacaftor 125 MG / lumacaftor 100 MG Oral Tablet
Summed across the 1 of 1 listed NDC products with Medicaid activity — every brand and generic of this exact strength & form. RxNorm 1812468
Shares are of the Medicaid prescriptions shown here. Product grouping follows RxNorm (the National Library of Medicine’s drug terminology), so “one product” means one strength & dosage form regardless of manufacturer. Dollar figures are gross Medicaid reimbursement before mandatory rebates — rebates (often large, especially for brand-name drugs) are confidential, so actual net cost to Medicaid is lower than shown.
Source: Medicaid State Drug Utilization Data (CMS), aggregated by generic ingredient — summed across every NDC (all brands, strengths, salt forms and dosage forms) of Lumacaftor and Ivacaftor, and across all states and both fee-for-service and managed-care claims. A general popularity signal; it excludes Medicare, commercial insurance and cash prescriptions, so it is not total U.S. use. Based on the 3 of 3 listed Lumacaftor and Ivacaftor NDCs with Medicaid activity.
🔍 Compare Lumacaftor and Ivacaftor products
A representative set of FDA-listed product records for Lumacaftor and Ivacaftor — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Oral powder | ||||
| LUMACAFTOR 75 mg/1; IVACAFTOR 94 mg | Oral | Vertex Pharmaceuticals Incorporated | NDA | View NDC → |
| LUMACAFTOR 100 mg/1; IVACAFTOR 125 mg | Oral | Vertex Pharmaceuticals Incorporated | NDA | View NDC → |
| LUMACAFTOR 150 mg/1; IVACAFTOR 188 mg | Oral | Vertex Pharmaceuticals Incorporated | NDA | View NDC → |
| 94; 75 mg/1; mg | Oral | Vertex Pharmaceuticals Incorporated × 2 listings | NDA | View NDC → |
| 125; 100 mg/1; mg | Oral | Vertex Pharmaceuticals Incorporated × 2 listings | NDA | View NDC → |
| 188; 150 mg/1; mg | Oral | Vertex Pharmaceuticals Incorporated × 2 listings | NDA | View NDC → |
| 💊Tablet | ||||
| IVACAFTOR 125 mg/1; LUMACAFTOR 100 mg | — | Aesica Queenborough Limited | DRUG FOR FURTHER PROCESSING | View NDC → |
| LUMACAFTOR 200 mg/1; IVACAFTOR 125 mg | — | Aesica Queenborough Limited | DRUG FOR FURTHER PROCESSING | View NDC → |
| LUMACAFTOR 100 mg/1; IVACAFTOR 125 mg | Oral | Vertex Pharmaceuticals Incorporated | NDA | View NDC → |
| LUMACAFTOR 200 mg/1; IVACAFTOR 125 mg | Oral | Vertex Pharmaceuticals Incorporated | NDA | View NDC → |
| 125; 100 mg/1; mg | Oral | Vertex Pharmaceuticals Incorporated × 2 listings | NDA | View NDC → |
| 125; 200 mg/1; mg | Oral | Vertex Pharmaceuticals Incorporated × 2 listings | NDA | View NDC → |
Showing 17 of 17 products — FDA National Drug Code Directory. See every product, package size and price: browse all 17 Lumacaftor and Ivacaftor NDCs →
📈 What people report — real-world data (FDA FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Lumacaftor and Ivacaftor — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Lumacaftor and Ivacaftor in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
🏛️ The road to your pharmacy
How Lumacaftor and Ivacaftor went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
🚨 Recall history
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
Source: openFDA drug enforcement (recall) reports.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one exact multiple-ingredient concept (MIN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). A product with a different ingredient combination belongs on a different page.
RxCUI 1655927 2 dose forms · 5 strengths
-
Dose form (SCDF) Oral Granules RxCUI 2053508In current FDA listingsClinical drug / strength (SCD) ivacaftor 125 MG / lumacaftor 100 MGRxCUI 2053509ivacaftor 188 MG / lumacaftor 150 MGRxCUI 2053517ivacaftor 94 MG / lumacaftor 75 MGRxCUI 2611458 -
Dose form (SCDF) Oral Tablet RxCUI 1655926In current FDA listingsClinical drug / strength (SCD) ivacaftor 125 MG / lumacaftor 100 MGRxCUI 1812468ivacaftor 125 MG / lumacaftor 200 MGRxCUI 1655928
Look up RxCUI 1655927 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
📦 Supply & shortage status
Whether Lumacaftor and Ivacaftor is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
🏷️ Sold under these brand names
🏭 Manufacturers & labelers
RxNorm drug class
Lumacaftor and Ivacaftor belongs to the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator class.
Classified by RxNorm RxClass — the U.S. National Library of Medicine's standardized drug-classification service (Established Pharmacologic Class from the FDA, the ATC drug family from the WHO, and mechanism of action). Reference only, not medical advice.
🔬 Where this comes from
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How we combine label and product data
HelloPharmacist combines public FDA and NLM records; we don’t author the underlying clinical facts. We identified 1 FDA-filed SPL label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. The representative label is the starting point for the displayed reference monograph; the 12 largest stored in-scope SPL records, which may include that representative, are compared section by section, and the longest available version of each section is retained. Forms, routes, brands, labelers, product-listed strengths, and product-record counts are aggregated separately from ingredient-matched FDA NDC Directory records; package records listed above are associated with the linked SPL; and the normalized concept hierarchy comes from NLM RxNorm.
For canonical ingredient pages, bounded product-attributed indication and administration excerpts may be added to the AI evidence, and the generator is instructed to preserve available product, brand, route, and form qualifiers instead of treating one label as universal. Where a section is identified as AI-written, its plain-language text is generated from bounded label evidence. New draft drug pages remain in the admin review queue before first publication. Vertex Pharmaceuticals Incorporated is the representative DailyMed label linked for verification and dates; it is not the sole source used for the page.
This is general education, not medical advice — always consult your doctor or pharmacist about your medications and any medical condition. For the exact wording of the linked representative label, view that label on DailyMed; some displayed composite sections may come from other in-scope SPL labels. Learn how we use AI and our data sources & update cadence.