Lumacaftor API Manufacturers & Suppliers

1 verified results

Get full market intelligence report

All Lumacaftor data. Full access. Full negotiation power

€399,-

Commercial-scale Suppliers

Senova Technology Co., Ltd.

Producer

Produced in China

Employees: 25+

Audit Report:

Certifications: USDMF

ISO9001

CoA

All certificates

USDMF

ISO9001

CoA

Request a quote

Take control of your API sourcing

Submit a Special Inquiry and have Pharmaoffer activate verified suppliers.

Start a Special Inquiry

Get full market intelligence report

€399,-

All Lumacaftor data. Full access. Full negotiation power

When insight is your advantage

Full data, full access, full negotiation power

Total market transparency

Supplier trade data access

Buyer / supplier flow comparison

Trusted by 30,000+ registered pharma professionals:

Reach multinationals, SMEs, compounding pharmacies & more!

Lumacaftor | CAS No: 936727-05-8 | GMP-certified suppliers

A medication that supports treatment of cystic fibrosis in patients with confirmed F508del mutations when used in combination therapy to enhance clinical management.

Therapeutic categories

AminesCytochrome P-450 CYP2B6 InducersCytochrome P-450 CYP2B6 Inducers (strength unknown)Cytochrome P-450 CYP2C19 InducersCytochrome P-450 CYP2C19 Inducers (strength unknown)Cytochrome P-450 CYP2C8 Inducers

Generic name

Lumacaftor

Molecule type

small molecule

CAS number

936727-05-8

DrugBank ID

DB09280

Approval status

Approved drug

ATC code

R07AX30

Primary indications

When used in combination with the drug [lumacaftor] as the product Orkambi, ivacaftor is indicated for the management of CF in patients aged one year and older who are homozygous for the _F508del_ mutation in the CFTR gene
If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the _F508del_ mutation on both alleles of the CFTR gene

Product Snapshot

Lumacaftor is an oral small‑molecule API supplied in granule and tablet forms
It is used in combination therapy for cystic fibrosis in patients homozygous for the F508del CFTR mutation
It is approved in the US, Canada, and the EU

Clinical Overview

Lumacaftor (CAS 936727-05-8) is a CFTR corrector used in fixed-dose combination with ivacaftor as Orkambi for the treatment of cystic fibrosis in patients with confirmed homozygous F508del mutations. This mutation leads to impaired CFTR protein folding, trafficking, and stability, resulting in reduced chloride transport and the characteristic viscous secretions affecting pulmonary and gastrointestinal function.

Lumacaftor acts by improving the conformational stability of F508del-CFTR, increasing the processing and delivery of mature protein to the cell surface. In vitro data show increased CFTR quantity and chloride transport, although translation to clinical outcomes is variable. When co-administered with ivacaftor, the corrector and potentiator target distinct defects of the same protein.

Pharmacodynamic assessments in adults and children show consistent reductions in sweat chloride concentrations, indicating improved CFTR function. Reported mean reductions range from approximately 8 to 32 mmol/L across dose levels and age groups, with reversibility after treatment washout. Clinical trials in patients aged 12 years and older demonstrate modest absolute improvements in ppFEV1 of about 2.6 to 3.0 percentage points over 24 weeks. No direct correlation between sweat chloride reduction and lung function improvement has been established.

Lumacaftor is a strong inducer of CYP3A and also affects CYP2B6, CYP2C8, and CYP2C9, with both induction and inhibition reported. These properties contribute to significant drug interaction potential. Decreases in heart rate of up to 8 bpm have been observed, though no meaningful QTc effects were detected in thorough QT studies. Safety considerations include respiratory symptoms during initiation, hepatic laboratory abnormalities, and increased blood pressure noted in clinical use.

Orkambi is widely used in regions where CFTR modulator therapy is approved. For API procurement, suppliers should demonstrate control of stereochemistry, process impurities, and residual solvents, along with robust documentation of stability, GMP compliance, and consistency suitable for fixed-dose combination manufacturing.

Identification & chemistry

Generic name	Lumacaftor
Molecule type	Small molecule
CAS	936727-05-8
UNII	EGP8L81APK
DrugBank ID	DB09280

Pharmacology

Summary	Lumacaftor/ivacaftor targets the defective CFTR chloride channel caused by the F508del mutation in cystic fibrosis. Lumacaftor acts as a corrector that stabilizes and improves trafficking of F508del-CFTR to the cell surface, while ivacaftor increases the channel’s open probability to enhance chloride transport. Together, the combination increases the quantity and function of CFTR protein at epithelial surfaces, leading to measurable pharmacodynamic changes such as reduced sweat chloride concentrations.
Mechanism of action	The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. The F508del mutation results in protein misfolding, causing a defect in cellular processing and trafficking that targets the protein for degradation and therefore reduces the quantity of CFTR at the cell surface. The small amount of F508del-CFTR that reaches the cell surface is less stable and has a low channel-open probability (defective gating activity) compared to wild-type CFTR protein. Lumacaftor improves the conformational stability of F508del-CFTR, resulting in increased processing and trafficking of mature protein to the cell surface. In vitro studies have demonstrated that lumacaftor acts directly on the CFTR protein in primary human bronchial epithelial cultures and other cell lines harboring the F508del-CFTR mutation to increase the quantity, stability, and function of F508del-CFTR at the cell surface, resulting in increased chloride ion transport. In vitro responses do not necessarily correspond to in vivo pharmacodynamic responses or clinical benefits.
Pharmacodynamics	Changes in sweat chloride in response to relevant doses of lumacaftor alone or in combination with ivacaftor were evaluated in a double-blind, placebo-controlled, Phase 2 clinical trial in patients with CF 18 years of age and older either homozygous or heterozygous for the F508del mutation. In that trial, 10 patients (homozygous for F508del) completed dosing with lumacaftor alone 400 mg q12h for 28 days followed by the addition of ivacaftor 250 mg q12h for an additional 28 days and 25 patients (homozygous or heterozygous for F508del) completed dosing with placebo. The treatment difference between lumacaftor 400 mg q12h alone and placebo evaluated as mean change in sweat chloride from baseline to Day 28 compared to placebo was -8.2 mmol/L (95% CI: -14, -2). The treatment difference between the combination of lumacaftor 400 mg/ivacaftor 250 mg q12h and placebo evaluated as mean change in sweat chloride from baseline to Day 56 compared to placebo was -11 mmol/L (95% CI: -18, -4). Changes in sweat chloride in response to lumacaftor/ivacaftor were also evaluated in a 24-week, open-label, clinical trial (Trial 3) in 58 patients with CF, aged 6 through 11 years (homozygous for F508del) who received lumacaftor 200 mg/ivacaftor 250 mg q12h for 24 weeks. Patients treated with lumacaftor/ivacaftor had a reduction in sweat chloride on Day 15 that was sustained through Week 24. The within-group LS mean absolute change from baseline in sweat chloride was -20.4 mmol/L on Day 15 and -24.8 mmol/L on Week 24. In addition, sweat chloride was also assessed after a 2-week washout period to evaluate the off-drug response. The within-group LS mean absolute change in sweat chloride from Week 24 to Week 26 following the 2-week washout period was 21.3 mmol/L. Changes in sweat chloride in response to lumacaftor/ivacaftor were also evaluated in a 24-week, open-label, clinical trial (Trial 6) in 60 patients with CF, aged 2 through 5 years (homozygous for F508del) who received either lumacaftor 100 mg/ivacaftor 125 mg every 12 hours or lumacaftor 150 mg/ivacaftor 188 mg every 12 hours for 24 weeks. Treatment with lumacaftor/ivacaftor demonstrated a reduction in sweat chloride at Week 4 that was sustained through Week 24. The mean absolute change from baseline in sweat chloride was –31.7 mmol/L (95% CI: -35.7, -27.6) at Week 24. In addition, sweat chloride was also assessed after a 2-week washout period to evaluate the off-drug response. The mean absolute change in sweat chloride from Week 24 to Week 26 following the 2-week washout period was an increase of 33.0 mmol/L (95% CI: 28.9, 37.1; P<0.0001). Changes in sweat chloride in response to lumacaftor/ivacaftor were evaluated in a 24-week, open-label, clinical trial (Trial 7) in 46 patients with CF, aged 1 through 2 years (homozygous for F508del) who received lumacaftor 75 mg/ivacaftor 94 mg (patient weighing 7 kg to <9 kg at screening), lumacaftor 100 mg/ivacaftor 125 mg (patient weighing 9 kg to <14 kg at screening), lumacaftor 150 mg/ivacaftor 188 mg (patient weighing ≥14 kg at screening), every 12 hours for 24 weeks. Treatment with lumacaftor/ivacaftor demonstrated a reduction in sweat chloride at Week 4 which was sustained through Week 24. The mean absolute change from baseline in sweat chloride at Week 24 was -29.1 mmol/L (95% CI: -34.8, -23.4). In addition, sweat chloride was also assessed after a 2-week washout period to evaluate the off-drug response. The mean absolute change in sweat chloride from Week 24 at Week 26 following the 2-week washout period was 27.3 mmol/L (95% CI: 22.3, 32.3). There was no direct correlation between the decrease in sweat chloride levels and an improvement in lung function (ppFEV1). The effect of multiple doses of lumacaftor 600 mg once daily/ivacaftor 250 mg q12h and lumacaftor 1000 mg once daily/ivacaftor 450 mg q12h on QTc interval was evaluated in a randomized, placebo- and active-controlled (400 mg moxifloxacin), parallel, thorough QT study in 168 healthy subjects. No meaningful changes in QTc interval were observed with either lumacaftor 600 mg once daily/ivacaftor 250 mg q12h and lumacaftor 1000 mg once daily/ivacaftor 450 mg q12h dose groups. A maximum decrease in mean heart rate of up to 8 beats per minute (bpm) from baseline was observed with lumacaftor/ivacaftor treatment. In Trials 1 and 2, a similar decrease in heart rate was observed in patients during the initiation of ORKAMBI (lumacaftor 400 mg/ivacaftor 250 mg q12h). Results from two randomized, double-blind, placebo-controlled, 24-week clinical trials of Orkambi (lumacaftor/ ) indicates that a lung function improvement, as demonstrated by a mean absolute change in ppFEV1 from baseline at Week 24 of 2.6 percentage points [95% CI (1.2, 4.0)] in Trial 1 (P=0.0003) and 3.0 percentage points [95% CI (1.6, 4.4)] in Trial 2.

Targets

Target	Organism	Actions
Cystic fibrosis transmembrane conductance regulator	Humans	modulator

ADME / PK

Absorption	When a single dose of lumacaftor/ivacaftor was administered with fat-containing foods, lumacaftor exposure was approximately 2 times higher than when taken in a fasting state. Following multiple oral dose administrations of lumacaftor in combination with ivacaftor, the exposure of lumacaftor generally increased proportionally to doses over the range of 200 mg every 24 hours to 400 mg every 12 hours. The median (range) t<sub>max</sub> of lumacaftor is approximately 4.0 hours (2.0; 9.0) in the fed state.
Half-life	The half-life of lumacaftor is approximately 26 hours in patients with cystic fibrosis.
Protein binding	Lumacaftor is extensively protein bound in the plasma (99%), and binds primarily to albumin.
Metabolism	Lumacaftor is mostly excreted unchanged in the feces and is not extensively metabolized. When metabolism does occur, oxidation and glucuronidation are the main processes involved.
Route of elimination	Following oral administration of lumacaftor, the majority of lumacaftor (51%) is excreted unchanged in the feces. There was minimal elimination of lumacaftor and its metabolites in urine (only 8.6% of total radioactivity was recovered in the urine with 0.18% as the unchanged parent drug).
Volume of distribution	Following oral administration of 200 mg of lumacaftor every 24 hours to cystic fibrosis patients in a fed state for 28 days, the mean (+/-SD) for apparent volumes of distribution was 86.0 (69.8) L.
Clearance	The typical apparent clearance, CL/F (CV), of lumacaftor was estimated to be 2.38 L/hr.

Formulation & handling

Low aqueous solubility and high lipophilicity support oral solid formulations using solubility‑enhancing approaches such as dispersions or lipid‑based systems.
Oral absorption is food‑dependent, with higher exposure when administered with dietary fat, which should be considered in clinical and formulation planning.
Chemically stable small‑molecule solid; standard handling for hydrophobic APIs applies, including control of particle size to support consistent dissolution.

Regulatory status

Lifecycle	Most U.S. patents protecting the API are scheduled to expire between late 2026 and mid‑2027, indicating that the product is approaching a late‑lifecycle stage in its primary market. With current availability in the US, Canada, and the EU, market conditions are likely to shift as patent expiries enable potential generic entry.

Markets	US, Canada, EU

Supply Chain

Supply chain summary	Lumacaftor is supplied by a single originator company, with branded Orkambi products marketed in the United States, Canada, and the European Union, indicating established global distribution. Patent protections in the United States extend through late 2026 to mid‑2027. These expiration dates suggest that generic development may emerge as exclusivities conclude.

Safety

Toxicity	In animal reproduction studies, oral administration of lumacaftor to pregnant rats and rabbits during organogenesis demonstrated no teratogenicity or adverse effects on fetal development at doses that produced maternal exposures up to approximately 8 (rats) and 5 (rabbits) times the exposure at the maximum recommended human dose (MRHD). No adverse developmental effects were observed after oral administration of lumacaftor to pregnant rats from organogenesis through lactation at doses that produced maternal exposures approximately 8 and 5 times the exposures at the MRHD, respectively. There are no animal reproduction studies with concomitant administration of lumacaftor and ivacaftor. There have been no reports of overdose with ORKAMBI. The highest repeated dose was lumacaftor 1000 mg once daily/ivacaftor 450 mg q12h administered to 49 healthy subjects for 7 days in a trial evaluating the effect of ORKAMBI on electrocardiograms (ECGs). Adverse events reported at an increased incidence of ≥5% compared to the lumacaftor 600 mg/ivacaftor 250 mg dosing period and placebo included: headache (29%), transaminase increase (18%), and generalized rash (10%). No specific antidote is available for overdose with ORKAMBI. Treatment of overdose consists of general supportive measures including monitoring of vital signs and observation of the clinical status of the patient. A two-year study in Sprague-Dawley rats and a 26-week study in transgenic Tg.rasH2 mice were conducted to assess the carcinogenic potential of lumacaftor. No evidence of tumorigenicity was observed in rats at lumacaftor oral doses up to 1000 mg/kg/day (approximately 5 and 13 times the MRHD on a lumacaftor AUC basis in males and females, respectively). No evidence of tumorigenicity was observed in Tg.rasH2 mice at lumacaftor oral doses up to 1500 and 2000 mg/kg/day in female and male mice, respectively. Lumacaftor was negative for genotoxicity in the following assays: Ames test for bacterial gene mutation, in vitro chromosomal aberration assay in Chinese hamster ovary cells, and in vivo mouse micronucleus test. Lumacaftor had no effects on fertility and reproductive performance indices in male and female rats at an oral dose of 1000 mg/kg/day (approximately 3 and 8 times, respectively, the MRHD on a lumacaftor AUC basis).

Toxicity

In animal reproduction studies, oral administration of lumacaftor to pregnant rats and rabbits during organogenesis demonstrated no teratogenicity or adverse effects on fetal development at doses that produced maternal exposures up to approximately 8 (rats) and 5 (rabbits) times the exposure at the maximum recommended human dose (MRHD). No adverse developmental effects were observed after oral administration of lumacaftor to pregnant rats from organogenesis through lactation at doses that produced maternal exposures approximately 8 and 5 times the exposures at the MRHD, respectively. There are no animal reproduction studies with concomitant administration of lumacaftor and ivacaftor. There have been no reports of overdose with ORKAMBI. The highest repeated dose was lumacaftor 1000 mg once daily/ivacaftor 450 mg q12h administered to 49 healthy subjects for 7 days in a trial evaluating the effect of ORKAMBI on electrocardiograms (ECGs). Adverse events reported at an increased incidence of ≥5% compared to the lumacaftor 600 mg/ivacaftor 250 mg dosing period and placebo included: headache (29%), transaminase increase (18%), and generalized rash (10%). No specific antidote is available for overdose with ORKAMBI. Treatment of overdose consists of general supportive measures including monitoring of vital signs and observation of the clinical status of the patient. A two-year study in Sprague-Dawley rats and a 26-week study in transgenic Tg.rasH2 mice were conducted to assess the carcinogenic potential of lumacaftor. No evidence of tumorigenicity was observed in rats at lumacaftor oral doses up to 1000 mg/kg/day (approximately 5 and 13 times the MRHD on a lumacaftor AUC basis in males and females, respectively). No evidence of tumorigenicity was observed in Tg.rasH2 mice at lumacaftor oral doses up to 1500 and 2000 mg/kg/day in female and male mice, respectively. Lumacaftor was negative for genotoxicity in the following assays: Ames test for bacterial gene mutation, in vitro chromosomal aberration assay in Chinese hamster ovary cells, and in vivo mouse micronucleus test. Lumacaftor had no effects on fertility and reproductive performance indices in male and female rats at an oral dose of 1000 mg/kg/day (approximately 3 and 8 times, respectively, the MRHD on a lumacaftor AUC basis).

High Level Warnings:

High‑dose exposure in humans was associated with increased incidence of headache, elevated transaminases, and rash
No specific antidote is defined, and management relies on general supportive measures
Long‑term studies in rats and Tg

Lumacaftor is a type of CFTR regulator

A CFTR regulator is a pharmaceutical Active Pharmaceutical Ingredient (API) that belongs to the subcategory of drugs targeting the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. This category of drugs plays a crucial role in the treatment of cystic fibrosis, a genetic disorder that affects the lungs and other organs.

CFTR regulators work by modulating the activity of the CFTR protein, which is responsible for controlling the movement of ions across cell membranes. In individuals with cystic fibrosis, mutations in the CFTR gene result in the production of faulty CFTR proteins that impair the normal functioning of the lungs and other affected organs.

The goal of CFTR regulators is to restore or enhance the function of the CFTR protein, ultimately improving the flow of chloride ions and fluids across cell membranes. By doing so, these drugs help to alleviate the symptoms of cystic fibrosis and improve the overall quality of life for affected individuals.

CFTR regulators can act through various mechanisms, such as potentiating the opening of CFTR channels or correcting the folding and trafficking of CFTR proteins. Some examples of CFTR regulators include correctors, potentiators, and modulators, each targeting specific defects in the CFTR protein.

These pharmaceutical APIs are developed through rigorous research, clinical trials, and regulatory approval processes to ensure their safety and efficacy. CFTR regulators represent a significant advancement in the treatment of cystic fibrosis, offering hope and improved therapeutic options for individuals living with this challenging condition.

Lumacaftor (CFTR regulator), classified under Enzyme Replacements/modifiers

Enzyme replacements/modifiers are a crucial category of pharmaceutical active pharmaceutical ingredients (APIs) utilized in the treatment of various enzyme-related disorders. Enzymes play a vital role in the normal functioning of the body by catalyzing specific biochemical reactions. However, in certain medical conditions, the body may lack or produce dysfunctional enzymes, leading to serious health complications.

Enzyme replacement therapy (ERT) involves administering exogenous enzymes to compensate for the enzyme deficiency in patients. These enzymes are typically derived from natural sources or produced using recombinant DNA technology. By introducing these enzymes into the body, they can effectively substitute the missing or defective enzymes, thereby restoring normal metabolic processes.

On the other hand, enzyme modifiers are API substances that regulate the activity of specific enzymes within the body. These modifiers can either enhance or inhibit the enzyme's function, depending on the therapeutic objective. By modulating enzyme activity, these APIs can restore the balance of enzymatic reactions, leading to improved physiological outcomes.

Enzyme replacements/modifiers have shown remarkable success in treating various genetic disorders, such as Gaucher disease, Fabry disease, and lysosomal storage disorders. Additionally, they have demonstrated potential in managing enzyme deficiencies associated with rare diseases and certain types of cancer.

The development and production of enzyme replacements/modifiers involve rigorous research, formulation optimization, and adherence to stringent quality control measures. Pharmaceutical companies invest substantial resources in developing these APIs to ensure their safety, efficacy, and compliance with regulatory standards.

Overall, enzyme replacements/modifiers represent a vital therapeutic category in modern medicine, offering hope and improved quality of life for patients with enzyme-related disorders.

Lumacaftor API manufacturers & distributors

Compare qualified Lumacaftor API suppliers worldwide. We currently have 1 companies offering Lumacaftor API, with manufacturing taking place in 1 different countries. Use the table below to review supplier type, countries of origin, certifications, product portfolio and GMP audit availability.

Supplier	Type	Country	Product origin	Certifications	Portfolio
Senova Technology Co., Lt...	Producer	China	China	CoA, ISO9001, USDMF	157 products

When sending a request, specify which Lumacaftor API quality you need: for example EP (Ph. Eur.), USP, JP, BP, or another pharmacopoeial standard, as well as the required grade (base, salt, micronised, specific purity, etc.).

Use the list above to find high-quality Lumacaftor API suppliers. For example, you can select GMP, FDA or ISO certified suppliers. Visit our help page to learn more about sourcing APIs via Pharmaoffer.

Frequently asked questions about Lumacaftor API

Sourcing

What matters most when sourcing GMP-grade Lumacaftor?

Key considerations include confirming GMP compliance and alignment with US, Canadian, and EU regulatory standards. Because Lumacaftor is supplied by a single originator company, verifying legitimate supply chain provenance is essential. Patent protections in the United States through late 2026 to mid‑2027 should also be reviewed to ensure appropriate use and timing for any development activities.

Which documents are typically required when sourcing Lumacaftor API?

Request the core API documentation set: CoA (1 company), USDMF (1 company), ISO9001 (1 company). Confirm versions and validity dates match the destination market to avoid delays in qualification.

Which manufacturers are known to produce Lumacaftor API?

Known or reported manufacturers for Lumacaftor: Senova Technology Co., Ltd.. Evaluate their GMP history, scale, and regional coverage before requesting dossiers or allocating demand.

How can I request quotes for Lumacaftor API from GMP suppliers?

Submit quote requests through the supplier listings with your specs and required documents (specifications, target volume, delivery timeline, and destination). Providing consistent details upfront speeds comparable offers and clarifies technical feasibility.

Is a GMP audit report available for Lumacaftor manufacturers?

Audit reports may be requested for Lumacaftor: 0 GMP audit reports available. Confirm the scope and recency of any audit before relying on it for qualification decisions.

How many suppliers offer Lumacaftor API on Pharmaoffer?

Reported supplier count for Lumacaftor: 1 verified suppliers. Filter listings by certifications, regions, and delivery options to match your qualification plan.

Which countries are known to manufacture Lumacaftor API?

Production countries reported for Lumacaftor: China (1 producer). Knowing the manufacturing geography helps anticipate logistics lead times and import compliance needs.

Which certifications do suppliers of Lumacaftor usually hold?

Common certifications for Lumacaftor suppliers: CoA (1 company), USDMF (1 company), ISO9001 (1 company). Always verify issuing authorities and expiry dates when reviewing audit packages.

Technical

What is Lumacaftor (CAS 936727-05-8) used for?

Lumacaftor is used as a CFTR corrector in combination with ivacaftor to treat cystic fibrosis in patients who have confirmed homozygous F508del CFTR mutations. It improves the folding, processing, and stability of the F508del-CFTR protein, increasing its delivery to the cell surface and enhancing chloride transport. This combination therapy addresses complementary defects in CFTR function associated with the mutation.

Which therapeutic class does Lumacaftor fall into?

Lumacaftor belongs to the following therapeutic categories: Amines, Cytochrome P-450 CYP2B6 Inducers, Cytochrome P-450 CYP2B6 Inducers (strength unknown), Cytochrome P-450 CYP2C19 Inducers, Cytochrome P-450 CYP2C19 Inducers (strength unknown). This positioning helps teams compare alternative APIs, anticipate pharmacology expectations, and align early research priorities.

What conditions is Lumacaftor mainly prescribed for?

The primary indications for Lumacaftor: When used in combination with the drug [Lumacaftor] as the product Orkambi, ivacaftor is indicated for the management of CF in patients aged one year and older who are homozygous for the _F508del_ mutation in the CFTR gene, If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the _F508del_ mutation on both alleles of the CFTR gene. These use cases frame the target patient populations and help prioritize formulation and safety evaluations.

How does Lumacaftor work?

The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. The F508del mutation results in protein misfolding, causing a defect in cellular processing and trafficking that targets the protein for degradation and therefore reduces the quantity of CFTR at the cell surface. The small amount of F508del-CFTR that reaches the cell surface is less stable and has a low channel-open probability (defective gating activity) compared to wild-type CFTR protein. Lumacaftor improves the conformational stability of F508del-CFTR, resulting in increased processing and trafficking of mature protein to the cell surface. In vitro studies have demonstrated that Lumacaftor acts directly on the CFTR protein in primary human bronchial epithelial cultures and other cell lines harboring the F508del-CFTR mutation to increase the quantity, stability, and function of F508del-CFTR at the cell surface, resulting in increased chloride ion transport. In vitro responses do not necessarily correspond to in vivo pharmacodynamic responses or clinical benefits.

What should someone know about the safety or toxicity profile of Lumacaftor?

Lumacaftor safety concerns include headache, elevated transaminases, and rash at higher exposures, as well as respiratory symptoms during treatment initiation. It is a strong inducer of CYP3A and affects other CYP enzymes, creating potential for clinically relevant drug interactions. Hepatic laboratory abnormalities and increases in blood pressure have been observed in clinical use. No specific antidote is available, and management of overdose relies on supportive care.

What are important formulation and handling considerations for Lumacaftor as an API?

Important considerations include its low aqueous solubility and high lipophilicity, which support the use of solubility‑enhancing approaches such as solid dispersions or lipid‑based systems for oral formulations. Because Lumacaftor absorption increases with dietary fat, food effects should be considered during formulation development and clinical administration planning. As a chemically stable, hydrophobic solid, standard precautions apply, including control of particle size to ensure consistent dissolution and performance.

Is Lumacaftor a small molecule?

Lumacaftor is classified as a small molecule. That classification shapes process design, impurity profiling, and analytical control strategies.

Are there special stability concerns for oral Lumacaftor?

Lumacaftor is a chemically stable, highly lipophilic solid, so routine handling for hydrophobic APIs applies. Its low aqueous solubility may require solubility‑enhancing formulation approaches to maintain consistent dissolution. Particle‑size control is important to support uniform performance, but no unusual instability issues are indicated.

Regulatory

Where is Lumacaftor approved or in use globally?

Lumacaftor is reported as approved in the following major regions: US, Canada, EU. Understanding geographic coverage informs regulatory filings, supply planning, and risk assessments before escalating procurement.

What’s the regulatory and patent landscape for Lumacaftor right now?

Lumacaftor is authorized for use in the United States, Canada, and the European Union. Patent protections and exclusivity periods follow the standard frameworks in these regions, with rights administered according to each jurisdiction’s intellectual property system.

Pharmaoffer

How does Pharmaoffer’s Smart Sourcing Service help with Lumacaftor procurement?

Pharmaoffer's Smart Sourcing Service coordinates compliant suppliers, documentation, and competitive quotes for Lumacaftor. It centralizes outreach, follow-ups, and document validation to shorten procurement timelines.

Is Lumacaftor included in the PRO Data Insights coverage?

PRO Data Insights coverage for Lumacaftor: 48 verified transactions across 24 suppliers and 16 buyers worldwide. Use the dataset to benchmark suppliers and monitor regulatory activity where available.

Where can I access the API market report for Lumacaftor?

Market report availability for Lumacaftor: . The report highlights demand trends, pricing drivers, and supplier landscape insights for procurement planning.