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Seladelpar
Seladelpar, sold under the brand name Livdelzi, is a medication used for the treatment of primary biliary cholangitis. It is used as the lysine dihydrate salt. It is a peroxisome proliferator-activated receptor delta (PPARδ) receptor agonist. The compound was licensed from Janssen Pharmaceutica NV. Seladelpar was approved for medical use in the United States in August 2024. __TOC__ Medical uses Seladelpar is indicated for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid in adults who have an inadequate response to ursodeoxycholic acid, or as monotherapy in people unable to tolerate ursodeoxycholic acid. Clinically, seladelpar reduces pruritus (itching) and interleukin-31 in people with primary biliary cholangitis. Society and culture Legal status Seladelpar was approved for medical use in the United States in August 2024. The FDA granted the application breakthrough therapy designation. In December 2024, the Committee for Medici ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Primary Biliary Cholangitis
Primary biliary cholangitis (PBC), previously known as primary biliary cirrhosis, is an autoimmune disease of the liver. It results from a slow, progressive destruction of the small bile ducts of the liver, causing bile and other toxins to build up in the liver, a condition called cholestasis. Further slow damage to the liver tissue can lead to scarring, fibrosis, and eventually cirrhosis. Common symptoms are tiredness, itching, and in more advanced cases, jaundice. In early cases, the only changes may be those seen in blood tests. PBC is a relatively rare disease, affecting up to one in 3,000–4,000 people. As with many other autoimmune diseases, it is much more common in women, with a sex ratio of at least 9:1 female to male. The reasons for this disparity are unclear, but may involve the expression of sex hormones such as estrogen, which impact immune system response. The condition has been recognised since at least 1851, and was named "primary biliary cirrhosis" in 1 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
By Mouth
Oral administration is a route of administration whereby a substance is taken through the Human mouth, mouth, swallowed, and then processed via the digestive system. This is a common route of administration for many medications. Oral administration can be easier and less painful than other routes of administration, such as Injection (medicine), injection. However, the onset of action is relatively low, and the effectiveness is reduced if it is not absorbed properly in the digestive system, or if it is broken down by digestive enzymes before it can reach the bloodstream. Some medications may cause gastrointestinal side effects, such as nausea or vomiting, when taken orally. Oral administration can also only be applied to conscious patients, and patients able to swallow. Terminology ''Per os'' (; ''P.O.'') is an adverbial phrase meaning literally from Latin "through the mouth" or "by mouth". The expression is used in medicine to describe a treatment that is taken orally (but not ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Breakthrough Therapy
Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "breakthrough therapy" designation is not intended to imply that a drug is actually a "breakthrough" or that there is high-quality evidence of treatment efficacy for a particular condition; rather, it allows the FDA to grant priority review to drug candidates if preliminary clinical trials indicate that the therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases. The FDA has other mechanisms for expediting the review and approval process for promising drugs, including fast track designation, accelerated approval, and priority review. Requirements A breakthrough therapy designation can be assigned to a drug if "it is a drug which is intended alone or in combination wit ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
PPAR Agonists
In the field of molecular biology, the peroxisome proliferator–activated receptors (PPARs) are a group of nuclear receptor proteins that function as transcription factors regulating gene expression. PPARs play essential roles in regulating cellular differentiation, development, and metabolism (carbohydrate, lipid, protein), and tumorigenesis Nomenclature and tissue distribution Three types of PPARs have been identified: alpha, gamma, and delta (beta): * α (alpha) - expressed in liver, kidney, heart, muscle, adipose tissue, and others * β/δ (beta/delta) - expressed in many tissues, especially in brain, adipose tissue, and skin * γ (gamma) - although transcribed by the same gene, this PPAR, by way of alternative splicing, is expressed in three forms: ** γ1 - expressed in virtually all tissues, including heart, muscle, colon, kidney, pancreas, and spleen ** γ2 - expressed mainly in adipose tissue; it is 30 amino acids longer than γ1 ** γ3 - expressed in macrophag ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drugs
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Acetic Acids
Acetic acid , systematically named ethanoic acid , is an acidic, colourless liquid and organic compound with the chemical formula (also written as , , or ). Vinegar is at least 4% acetic acid by volume, making acetic acid the main component of vinegar apart from water. Historically, vinegar was produced from the third century BC and was likely the first acid to be produced in large quantities. Acetic acid is the second simplest carboxylic acid (after formic acid). It is an important chemical reagent and industrial chemical across various fields, used primarily in the production of cellulose acetate for photographic film, polyvinyl acetate for wood glue, and synthetic fibres and fabrics. In households, diluted acetic acid is often used in descaling agents. In the food industry, acetic acid is controlled by the food additive code E260 as an acidity regulator and as a condiment. In biochemistry, the acetyl group, derived from acetic acid, is fundamental to all forms of life ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Medicine
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
European Medicines Agency
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or European Medicines Evaluation Agency (EMEA).Set up by EC Regulation No. 2309/93 as the European Agency for the Evaluation of Medicinal Products, and renamed by EC Regulation No. 726/2004 to the European Medicines Agency, it had the acronym EMEA until December 2009. The European Medicines Agency does not call itself EMA either – it has no official acronym but may reconsider if EMA becomes commonly accepted (secommunication on new visual identity an). The EMA was set up in 1995, with funding from the European Union and the pharmaceutical industry, as well as indirect subsidy from member states, its stated intention to harmonise (but not replace) the work of existing national medicine regulatory bodies. The hope was that this plan would ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Committee For Medicinal Products For Human Use
The Committee for Medicinal Products for Human Use (CHMP), formerly known as the Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regarding medicinal product Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy ( pharmacotherapy) is an important part of the ...s for human use. See also * Committee for Medicinal Products for Veterinary Use References External links Committee for Medicinal Products for Human Use (CHMP) Health and the European Union {{eu-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Interleukin-31
Interleukin-31 (IL-31) is a protein that in humans is encoded by the ''IL31'' gene that resides on chromosome 12. IL-31 is an inflammatory cytokine that helps trigger cell-mediated immunity against pathogens. It has also been identified as a major player in a number of chronic inflammatory diseases, including atopic dermatitis. IL-31 is produced by a variety of cells, namely type 2 helper ( TH2) T-cells. IL-31 sends signals through a receptor complex made of IL-31RA and oncostatin M receptor β (OSMRβ) expressed in immune and epithelial cells. These signals activate three pathways: ERK1/2 MAP kinase, PI3K/AKT, and JAK1/ 2 signaling pathways. Structure IL-31 is a cytokine with an anti-parallel four-helix bundle structure in the gp130/IL-6 cytokine family. This family includes IL-6, IL-11, IL-27, leukemia inhibitory factor ( LIF), oncostatin M (OSM), ciliary neurotrophic factor (CNTF), cardiotrophin-1 (CT-1), cardiotrophin-like cytokine (CLC), and neuropoietin (NP). T ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
