Acute congestive heart failure

今日，CDE官网显示，由华夏生生药业（北京）有限公司申报的硝普钠注射液拟纳入优先审评，拟定适应症为：用于在高血压危象中立即降低成年人和儿童患者的血压。应同时服用长效降压药，以尽量减少硝普钠的治疗时间。也可用于可控降压，以减少手术中的出血。也适用于治疗急性充血性心力衰竭。 根据CDE公布的拟纳入优先审评理由显示，经加快上市申请专家论证会议定，本品是临床急需的短缺药品，目前尚无硝普钠注射液仿制药上市申请纳入优先审评，并请申请人尽快提交上市申请。 据公开资料显示，硝普钠注射液是一种血管扩张剂，2022年4月，普利制药的硝普钠注射液以化学药品新注册分类3类获批上市，视同通过一致性评价。至今，硝普钠注射液在国内仍是普利制药的独家品种。 普利制药的硝普钠注射液仿制药成功研发后，相继提交了国内外多个国家的注册申请，2020年8月获得美国食药监局（FDA）批准，2020年10月获得加拿大卫生部批准，今年2月，硝普钠注射液获得德国上市许可 。 据药融云数据库显示，硝普钠注射液2023年全国医院（全终端）销售额近5000万元，相较于2022年销售额大增326%。

The main function of Aldosterone is to promote the reabsorption of sodium ions and excretion of potassium in the epithelial cells of the renal distal convoluted tubule and collecting duct, i.e., sodium preservation and potassium excretion. While reabsorbing sodium, it also osmotically reabsorbs water, thereby maintaining extracellular fluid volume and blood volume. Aldosterone can also act on sweat gland, salivary gland ducts, and gastrointestinal epithelial cells, reducing sodium ion excretion and increasing potassium excretion in sweat, saliva, and feces through sodium preservation and potassium excretion. Aldosterone can enhance the sensitivity of vascular smooth muscle to vasoconstrictive substances, indirectly causing other diseases in the body. Excessive aldosterone secretion can lead to sodium and water retention, causing high blood sodium, low blood potassium, alkalosis, and even persistent hypertension; conversely, low aldosterone secretion can lead to excessive excretion of sodium and water, resulting in high blood sodium, low blood potassium, acidosis or hypotension. The renin-angiotensin-aldosterone system plays a key role in the onset and development of many diseases. Therefore, blocking or inhibiting any stage in this mechanism can achieve the purpose of treating diseases, making aldosterone antagonists a subject of great interest. Aldosterone Competitive LandscapeAccording to the data provided by Patsnap Synapse-Global Drug Intelligence Database: the following figure shows that as of 3 Sep 2023, there are a total of 4 Aldosterone drugs worldwide, from 5 organizations, covering 8 indications, and conducting 10 clinical trials. 👇Please click on the picture link below for free registration or login directly if you have freemium accounts, you can browse the latest research progress on drugs , indications, organizations, clinical trials, clinical results, and drug patents related to this target. The analysis of the target Aldosterone reveals a competitive landscape with multiple companies involved in its development. Pfizer Inc. is leading with an approved drug, while Mayo Clinic is actively conducting research in Phase 1/2 and Phase 1. Japan, the United States, and South Africa are the countries/locations showing progress in the development of Aldosterone-related treatments. The indications for Aldosterone-related drugs include Hyperaldosteronism, Edema, Cardiac, Acute congestive heart failure, and Hypertension. The presence of small molecule drugs, biological products, and an unknown drug type indicates diverse approaches to targeting Aldosterone.  Overall, the analysis suggests a promising future for the development of Aldosterone-targeted therapies, with potential advancements in addressing various medical needs associated with Aldosterone-related conditions. Key Drug: Potassium CanrenoatePotassium Canrenoate is a small molecule drug that targets aldosterone and is used in the treatment of cardiovascular diseases, endocrinology and metabolic diseases, and other related conditions. 👇Please click on the image below to directly access the latest data (R&D Status | Core Patent | Clinical Trial | Approval status in Global countries) of this drug. The drug is indicated for the treatment of acute congestive heart failure, edema (cardiac), hyperaldosteronism, and edema. Acute congestive heart failure refers to a sudden onset of heart failure symptoms, such as shortness of breath and fluid retention. Edema, both cardiac and non-cardiac, refers to the accumulation of fluid in the body's tissues, leading to swelling. Hyperaldosteronism is a condition characterized by excessive production of aldosterone, which can lead to high blood pressure and fluid retention. Potassium Canrenoate received its first global approval in September 1967. It was developed by G.D. Searle & Co., Inc., and its approval signifies its efficacy and safety in treating the mentioned indications.

-- LGMD is a Rare Form of Muscular Dystrophy with No Approved Therapy -- Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101)* for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is a novel investigational recombinant adeno-associated virus (AAV) based gene therapy currently being developed as a one-time intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I/R9), a disease subtype affecting 4.5 people per million worldwide, including more than 5,000 people in the EU and US. The EC decision, dated February 15, 2023, follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) and was received through AskBio's EU-based subsidiary BrainVectis. AB-1003 is being investigated in the US in a Phase 1/2 multicenter study that will evaluate the safety, tolerability and efficacy of a single IV infusion of gene therapy in adult subjects with genotypically confirmed LGMD2I/R9. "The EC orphan drug designation for AB-1003 is an important recognition of the unmet medical need in LGMD, which has no approved therapy," said Sheila Mikhail, Co-Founder & CEO, AskBio. "The burden of this rare form of muscular dystrophy on patients and their families is significant, and this decision supports our efforts to potentially bring a new therapeutic option to people in the EU living with the 2I/R9 type of this devastating disease." The EC grants orphan drug designation for medicinal products intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000 in the EU, provided there is no other satisfactory treatment option or the medicine can be of significant benefit to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees. In addition, if the medicine is approved for marketing, this designation will provide 10 years of marketing exclusivity. About Limb-Girdle Muscular Dystrophy (LGMD) and Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) Limb-girdle muscular dystrophy (LGMD) is a term for a group of diseases that cause progressive weakness and wasting of the muscles in the arms and legs.1 The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs.1 The severity, age of onset, and features of LGMD vary among the many subtypes of the condition and are often inconsistent, even within the same family.1 Signs and symptoms may first appear at any age and generally worsen with time, although in some cases they remain mild.1 Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD and is caused by mutations in the FKRP gene.2 In LGMD2I/R9, signs and symptoms often develop in late childhood and may include difficulty running and walking.2 The symptoms gradually worsen over time toward significant disability, and affected people generally rely on a wheelchair for mobility approximately 23-26 years after onset.2 Currently, there is no treatment that modifies the disease progression, and management is based on the signs and symptoms present in each individual.2 Visit the National Institutes of Health Medline Plus website to learn more about LGMD and LGMD2I/R9. About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease, and congestive heart failure. AskBio's gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. An early innovator in the gene therapy field, with over 800 employees in five countries, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at   or follow us on LinkedIn. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability, and quality throughout the world. In fiscal 2021, the Group employed around 100,000 people and had sales of 44.1 billion euros. R&D expenses before special items amounted to 5.3 billion euros. For more information, visit . * European Commission orphan drug designation is for "adeno-associated viral vector serotype 9 expressing fukutin related protein." 1. National Institutes of Health – Medline Plus: Limb-Girdle Muscular Dystrophy – Description. Accessed February 17, 2021. Limb-girdle muscular dystrophy: MedlinePlus Genetics 2. National Institutes of Health — National Center of Advancing Translational Sciences: Genetic and Rare Diseases Information Center. Limb-Girdle Muscular Dystrophy Type 2I – Summary. Accessed February 17, 2021. Limb-girdle muscular dystrophy type 2I | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program (nih.gov) 内容来源于网络，如有侵权，请联系删除。