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Produced in:
Established in: 2013
MOQ: 1 kg
Employees: 25+
ISO9001 certified
Drug R&D center (in Hangzhou)
From lab scale (mg/g) to commercial scale (kgs/tons)
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Looking for Lumacaftor API 936727-05-8?
- Description:
- Here you will find a list of producers, manufacturers and distributors of Lumacaftor. You can filter on certificates such as GMP, FDA, CEP, Written Confirmation and more. Send inquiries for free and get in direct contact with the supplier of your choice.
- API | Excipient name:
- Lumacaftor
- Synonyms:
- lumacaftor
- Cas Number:
- 936727-05-8
- DrugBank number:
- DB09280
- Unique Ingredient Identifier:
- EGP8L81APK
About Lumacaftor
You can find a short description about Lumacaftor here. Lumacaftor is a drug used in combination with Ivacaftor as the fixed dose combination product Orkambi for the management of Cystic Fibrosis (CF) in patients aged 6 years and older. Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, a transmembrane ion channel involved in the transport of chloride and sodium ions across cell membranes of the lungs, pancreas, and other organs. Mutations in the CFTR gene result in altered production, misfolding, or function of the CFTR protein and consequently abnormal fluid and ion transport across cell membranes As a result, CF patients produce thick, sticky mucus that clogs the ducts of organs where it is produced making patients more susceptible to infections, lung damage, pancreatic insufficiency, and malnutrition.
Lumacaftor improves CF symptoms and underlying disease pathology by aiding the conformational stability of F508del-mutated CFTR proteins, preventing misfolding and resulting in increased processing and trafficking of mature protein to the cell surface. Results from clinical trials indicated that treatment with Orkambi (lumacaftor/ivacaftor) results in improved lung function, reduced chance of experiencing a pulmonary exacerbation, increased weight gain, and improvements in CF symptoms.
Check who is selling Lumacaftor in the overview above.
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 manufacturers | traders | suppliers
We have 1 companies offering Lumacaftor produced in 1 different countries.
Get in contact with the supplier of your choice:
- Senova Technology Co., Ltd. from China, product country of origin China
Let the supplier know whether you are looking for a product with a specific monograph such as EP (Ph. Eur.), USP, JP, BP or another quality. Or, whether you are looking for hydrochloride (HCl), anhydricum, base, micronisatum or a specific purity.
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