Erythromycin Gluceptate

STOCKHOLM, Sept. 19, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) ("Calliditas"), today announced upcoming data presentations from the NeflgArd Phase 3 Study at the 17th International Symposium on IgA Nephropathy (IIgANN). Investigators will be presenting additional analyses of the Phase 3 NefIgArd study's 2-year data with Nefecon (TARPEYO® (budesonide) delayed-release capsules/Kinpeygo®) for the treatment of Primary IgA nephropathy (IgAN), along with the biomarker data from the NeflgArd study. IIgANN will be held virtually and in person in Tokyo, Japan, September 25-27, 2023. Poster presentation details are below and will be available on the Presentation and Publication page on the Calliditas' corporate website following the meeting. Oral Presentation Details: Title: Analysis of the NefIgArd Part A study population confirms Nefecon suppresses circulating levels of BAFF, APRIL, and soluble BCMA in IgA nephropathy Presenter: Nadia Nawaz, University of Leicester Date and Time: Thursday, September 28, 14:35 - 15:35 p.m. JST Title: Nefecon treatment response in Asian and White patient populations with immunoglobulin A nephropathy: A 2-year analysis of the phase III NefIgArd trial Presenter: Jonathan Barratt, M.B.Ch.B., Ph.D., University of Leicester Date and Time: Saturday, September 30, 10:55 - 11:55 a.m. JST Title: Hematuria reduction in patients with IgAN following Nefecon treatment: A secondary analysis of the full 2-year NefIgArd Phase III trial results Presenter: Richard Lafayette, M.D., F.A.C.P., Stanford Healthcare Date and Time: Saturday, September 30, 10:55 - 11:55 a.m. JST Poster Presentation Details: Title: Long-term renal benefit over 2 years with Nefecon verified: The NefIgArd Phase III full trial results Authors: Richard Lafayette, Jens Kristensen, Andrew Stone, Jürgen Floege, Vladimir Tesar, Hernán Trimarchi, Hong Zhang, Necmi Eren, Alexander Paliege, Heather N. Reich, Brad H. Rovin, and Jonathan Barratt Date and Time: Friday, September 29, 11:20 a.m. - 12:35 p.m. JST Title: Durable proteinuria reduction over 2 years with Nefecon treatment: A secondary analysis of the full NefIgArd Phase III trial results Authors: Richard Lafayette, Jens Kristensen, Andrew Stone, Jürgen Floege, Vladimir Tesar, Hernán Trimarchi, Hong Zhang, Necmi Eren, Alexander Paliege, Heather N. Reich, Brad H. Rovin, and Jonathan Barratt Date and Time: Friday, September 29, 11:20 a.m. - 12:35 p.m. JST Title: Analysis of the NefIgArd Part A study population confirms Nefecon suppresses circulating levels of IgA-containing immune complexes in IgA nephropathy Authors: Vicky Cotton, Nadia Nawaz, Karen Molyneux, and Jonathan Barratt Date and Time: Friday, September 29, 11:20 a.m. - 12:35 p.m. JST Title: Analysis of the NefIgArd Part A study population confirms that Nefecon modulates circulating levels of the chemokines CXCL5, CCL11, and CCL13 in IgA nephropathy Authors: Roisin Thomas, Nadia Nawaz, Karen Molyneux, and Jonathan Barratt Date and Time: Friday, September 29, 11:20 a.m. - 12:35 p.m. JST For more information, visit the IIgANN website here. Indication TARPEYO® (budesonide) delayed release capsules is a corticosteroid indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g. This indication is approved under accelerated approval based on a reduction in proteinuria. It has not been established whether TARPEYO slows kidney function decline in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory clinical trial. Important Safety Information Contraindications: TARPEYO is contraindicated in patients with hypersensitivity to budesonide or any of the ingredients of TARPEYO. Serious hypersensitivity reactions, including anaphylaxis, have occurred with other budesonide formulations. Warnings and Precautions Hypercorticism and adrenal axis suppression: When corticosteroids are used chronically, systemic effects such as hypercorticism and adrenal suppression may occur. Corticosteroids can reduce the response of the hypothalamus-pituitary-adrenal (HPA) axis to stress. In situations where patients are subject to surgery or other stress situations, supplementation with a systemic corticosteroid is recommended. When discontinuing therapy [see Dosing and Administration] or switching between corticosteroids, monitor for signs of adrenal axis suppression. Patients with moderate to severe hepatic impairment (Child-Pugh Class B and C, respectively) could be at an increased risk of hypercorticism and adrenal axis suppression due to an increased systemic exposure to oral budesonide. Avoid use in patients with severe hepatic impairment (Child-Pugh Class C). Monitor for increased signs and/or symptoms of hypercorticism in patients with moderate hepatic impairment (Child-Pugh Class B). Risks of immunosuppression: Patients who are on drugs that suppress the immune system are more susceptible to infection than healthy individuals. Chicken pox and measles, for example, can have a more serious or even fatal course in susceptible patients or patients on immunosuppressive doses of corticosteroids. Avoid corticosteroid therapy in patients with active or quiescent tuberculosis infection; untreated fungal, bacterial, systemic viral, or parasitic infections; or ocular herpes simplex. Avoid exposure to active, easily transmitted infections (eg., chicken pox, measles). Corticosteroid therapy may decrease the immune response to some vaccines. Other corticosteroid effects: TARPEYO is a systemically available corticosteroid and is expected to cause related adverse reactions. Monitor patients with hypertension, prediabetes, diabetes mellitus, osteoporosis, peptic ulcer, glaucoma, cataracts, a family history of diabetes or glaucoma, or with any other condition in which corticosteroids may have unwanted effects. Adverse reactions: In clinical studies, the most common adverse reactions with TARPEYO (occurring in ≥5% of TARPEYO patients and ≥2% higher than placebo) were hypertension (16%), peripheral edema (14%), muscle spasms (13%), acne (11%), dermatitis (7%), weight increase (7%), dyspnea (6%), face edema (6%), dyspepsia (5%), fatigue (5%), and hirsutism (5%). Drug interactions: Budesonide is a substrate for CYP3A4. Avoid use with potent CYP3A4 inhibitors, such as ketoconazole, itraconazole, ritonavir, indinavir, saquinavir, erythromycin, and cyclosporine. Avoid ingestion of grapefruit juice with TARPEYO. Intake of grapefruit juice, which inhibits CYP3A4 activity, can increase the systemic exposure to budesonide. Use in specific populations Pregnancy: The available data from published case series, epidemiological studies, and reviews with oral budesonide use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with IgAN. Infants exposed to in utero corticosteroids, including budesonide, are at risk for hypoadrenalism. Please see Full Prescribing Information. About TARPEYO1 Calliditas has introduced TARPEYO, the first FDA-approved therapy for the treatment of the autoimmune renal disease primary IgA Nephropathy, or IgAN, to reduce proteinuria in adults with primary IgAN who are at risk of rapid disease progression, generally a UPCR≥1.5g/g. This indication is approved under accelerated approval based on a reduction in proteinuria. It has not been established whether TARPEYO slows kidney function decline in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. TARPEYO is an oral, delayed release formulation of budesonide, a corticosteroid with potent glucocorticoid activity and weak mineralocorticoid activity that undergoes substantial first pass metabolism. TARPEYO is as a 4 mg delayed release capsule and is enteric coated and designed to remain intact until it reaches the ileum. Each capsule contains coated beads of budesonide that target mucosal B-cells present in the ileum, including the Peyer's patches, which are responsible for the production of galactose-deficient IgA1 antibodies (Gd-Ag1) causing IgA nephropathy. It is unclear to what extent TARPEYO's efficacy is mediated via local effects in the ileum vs systemic effects. About the NeflgArd Study The global clinical trial NefIgArd is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of TARPEYO 16 mg once daily vs placebo in adult patients with primary IgAN (N=364), as an addition to optimized RAS inhibitor therapy. Part A of the study included a 9-month blinded treatment period and a 3-month follow-up period. The primary endpoint was UPCR, and eGFR was a secondary endpoint. Part B included a 12-month observational period off drug and assessed eGFR over the entire 2-year period for patients who were treated with the TARPEYO or placebo regimen in Part A. The full NefIgArd trial met its primary endpoint. Topline data from the full NefIgArd study were reported on March 12, 2023. About Primary Immunoglobulin A Nephropathy Primary immunoglobulin A nephropathy (IgA nephropathy or IgAN or Berger's Disease) is a rare, progressive, chronic autoimmune disease that attacks the kidneys and occurs when galactose-deficient IgA1 is recognized by autoantibodies, creating IgA1 immune complexes that become deposited in the glomerular mesangium of the kidney.2,3 This deposition in the kidney can lead to progressive kidney damage and potentially a clinical course resulting in end- stage renal disease. IgAN most often develops between late teens and late 30s.3,4 About Calliditas Calliditas Therapeutics is a biopharma company headquartered in Stockholm, Sweden, focused on identifying, developing, and commercializing novel treatments in orphan indications, with an initial focus on renal and hepatic diseases with significant unmet medical needs. Calliditas is listed on Nasdaq Stockholm (ticker: CALTX) and the Nasdaq Global Select Market (ticker: CALT). Visit Calliditas.com for further information. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding Calliditas' strategy, business plans, regulatory submissions, and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties, and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, any related to Calliditas' business, operations, continued FDA approval for TARPEYO, the potential to expand TARPEYO's FDA approval to the entire Phase 3 study population, the potential to achieve full approval of Kinpeygo from the EC and MHRA, market acceptance of TARPEYO, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Calliditas' reports filed with the Securities and Exchange Commission. Calliditas cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Calliditas disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent Calliditas' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. For further information, please contact: Åsa Hillsten, Head of IR & Sustainability, Calliditas Tel.: +46 76 403 35 43, email: [email protected] The information was sent for publication, through the agency of the contact person set out above, on September 19, 2023, at 4.00 p.m. CET. 1 TARPEYO® (budesonide) [prescribing information]. Stockholm, SE: Calliditas Therapeutics AB; 2021 2 Barratt, J., & Feehally, J. (2005). IgA nephropathy. J Am Soc Nephrol, 16(7), 2088-2097. 3 Barratt, J., Rovin, B. H., Cattran, D., et al. (2020). Why Target the Gut to Treat IgA Nephropathy? Kidney Int Rep, 5(10), 1620-1624. 4 Jarrick, S., Lundberg, S., Welander, A., et al. (2019). Mortality in IgA Nephropathy: A Nationwide Population-Based Cohort Study. J Am Soc Nephrol, 30(5), 866-876. The following files are available for download: SOURCE Calliditas Therapeutics

-If approved, more than 1,200 children would be newly eligible for a medicine that could treat the underlying cause of their disease- BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “KAFTRIO has demonstrated unprecedented clinical benefit for eligible people living with CF,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex. “Treating the underlying cause of CF as early as possible in life has the potential to slow disease progression, which is why we are pleased the CHMP is supportive of expanding the indication for KAFTRIO to patients as young as 2 years.” In the European Union, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is already approved for the treatment of people with CF ages 6 years and older who have at least one copy of the F508del mutation in the CFTR gene. About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s. About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in A Combination Regimen With Ivacaftor In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is approved in the European Union for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one copy of the F508del mutation in the CFTR gene. For complete product information, please see the Summary of Product Characteristics that can be found on . U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) INDICATIONS AND USAGE TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 2 years of age. IMPORTANT SAFETY INFORMATION Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: are allergic to TRIKAFTA or any ingredients in TRIKAFTA, have kidney problems, have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk. Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin. Patients should avoid food or drink that contains grapefruit while taking TRIKAFTA. TRIKAFTA can cause serious side effects, including: Liver damage and worsening of liver function in patients with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in patients without liver disease. High liver enzymes in the blood, which is a common side effect in patients treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patient’s doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine. Serious allergic reactions have happened to patients who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include: rash or hives; tightness of the chest or throat or difficulty breathing; swelling of the face, lips and/or tongue; difficulty swallowing; and light-headedness or dizziness. Abnormality of the eye lens (cataract) has been noted in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts. The most common side effects of TRIKAFTA include headache, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, flu (influenza), inflamed sinuses, and increase in blood bilirubin. Patients should tell their doctor if they have any side effect that bothers them or that does not go away. These are not all the possible side effects of TRIKAFTA. For more information, patients should ask their doctor or pharmacist. Please click here to see the full U.S. Prescribing Information for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor). About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency. Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by Nia Tatsis, Ph.D., in this press release and statements regarding our expectations for regulatory approval and a label extension for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, the estimated number of children eligible for a medicine that can treat the underlying cause of their disease for the first time and our beliefs regarding the benefits of our medicines. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company’s development programs may not support a label extension for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, the European Commission may not approve the company’s applications for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor on a timely basis or at all, and other risks listed under the heading “Risk Factors” in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at vrtx.com and . You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN)