Hypoprothrombinemias

NEW YORK--( BUSINESS WIRE )--Pathway to Cures (P2C) announces two new members to its board of directors, Sharon Richardson, PhD , a Director on the National Bleeding Disorders Foundation Board, and Philip M. Gattone, M.Ed. , President and CEO of the National Bleeding Disorders Foundation. Pathway to Cures is the venture philanthropy fund for the National Bleeding Disorders Foundation (NBDF) focused on investing in emerging biotech companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community. Inheritable blood and bleeding disorders include rare and ultra-rare blood disorders, hemophilia A and B, von Willebrand disease, Sickle Cell disease, anemia, clotting disorders, and other hematological disorders, affecting more than 20 million people worldwide. Sharon Richardson, PhD, is a result-driven strategist with 28 years of experience in the pharmaceutical industry as a medical affairs leader committed to creating healthier communities through scientific exchange, patient advocacy, and community engagement. “P2C offers a unique opportunity for the National Bleeding Disorders Foundation to leverage their 75 years of serving people living with blood and bleeding disorders,” said Sharon Richardson. “It is a privilege to lend my time and assistance to advance the science and treatment options available to our community and to work alongside this talented and dedicated group of staff and volunteers.” “Sharon has led a life of selfless service to others and has used her strong leadership skills to make a difference for our community.” said Phil Gattone “We are thrilled to have Sharon join the Pathway to Cures Board and look forward to working with her to make a positive impact in more lives.” Philip M. Gattone, M.Ed., joined the National Bleeding Disorders Foundation as president and chief executive officer earlier this year. Gattone is a seasoned leader dedicated to amplifying the impact of nonprofit organizations through his distinctive blend of leadership and strategic insight. He has served as a senior executive in local, regional, national, and global nonprofit organizations and healthcare companies. “We’re honored and delighted to have Sharon and Phil supporting our efforts for the inheritable blood and bleeding disorders community!” said Teri Willey, Managing Director of the Venture Philanthropy Fund “Their knowledge and experience will enhance our ability to ensure there are more therapies and cures available for our community.” About Pathway to Cures Pathway to Cures (P2C) is the venture philanthropy fund of the National Bleeding Disorders Foundation created to accelerate the development of cures across all inheritable blood and bleeding disorders. In collaboration with other organizations, P2C invests in innovative therapies and technologies, leveraging the deep resources and scientific community relationships of the National Bleeding Disorders Foundation. By reinvesting proceeds from investments back into the Fund, P2C will amplify the investment impact, support promising biotech companies, and build a portfolio of investments that further the mission of the National Bleeding Disorders Foundation. For more information, visit www.pathwaytocures.org or contact Renee Peck at rpeck@pathwaytocures.org . About National Bleeding Disorders Foundation The National Bleeding Disorders Foundation (NBDF) is dedicated to finding cures for inheritable blood and bleeding disorders and addressing and preventing these disorders' complications through research, education, and advocacy, enabling people and families to thrive. Today, NBDF serves people across the United States with all bleeding disorders, including hemophilia, von Willebrand disease, rare factor deficiencies, and platelet disorders. The foundation also supports a network of 50 chapters across the country. To learn more, visit www.bleeding.org and follow NBDF across social media @nbd_foundation.

未经授权，不得转载维生素K1混合胶束包封率测定方法的适用性研究Applicability Study of Determination Methods for Encapsulation Efficiency of Vitamin K1-loaded Mixed Micelles郭丽珊1，李晰月1,2，姚成丽1，奚 泉1*(1. 中国医药工业研究总院上海医药工业研究院，上海 201203；2. 中国药科大学，江苏南京 211198)摘要：维生素K1(VK1) 混合胶束(VK1-MM) 注射液中游离的VK1 会导致安全性风险，包封率可评价混合胶束中游离VK1 的量以及胶束的载药性能。该研究比较了萃取法、离心法和过滤法用于测量VK1-MM 包封率的适用性，并讨论了不同方法中影响包封率测定准确性的主要因素。结果表明，萃取法中使用的萃取剂会影响VK1-MM 的结构；游离VK1 与水的密度差异较小，导致离心法不能彻底分离游离VK1 和VK1-MM ；过滤法选用合适的滤膜孔径及材质时，可有效分离游离VK1 和VK1-MM，且方法回收率为100.40％ (RSD 0.80％，n=3)，适用于测定VK1-MM 的包封率。该研究对不同包封率测定方法的原理及待测样品特性的适用性分析结果为其他胶束制剂建立科学、合理、适用的包封率评价方法提供了参考。关键词：维生素K1 ；混合胶束；包封率；适用性以下为文章节选维生素K1(vitamin K1，VK1) 为脂溶性维生素，是肝脏中凝血因子Ⅱ、Ⅶ、Ⅸ和Ⅹ合成凝血酶原所必须的辅助因子[1]，用于治疗低凝血酶原血症、维生素K 缺乏症，以及预防和治疗新生儿出血性疾病等[2]，临床应用时有口服、肌内注射、皮下注射及静脉注射等给药途径。其中，注射给药的剂型不可或缺，在紧急情况，如术中华法林抗凝引起颅内出血时，需要尽快静脉注射给药[2]，新生儿也需要肌内注射给药以预防晚期低凝血酶原血症[3]。20 世纪60 年代，VK1 静脉注射剂以聚氧乙烯蓖麻油作乳化剂，以增加VK1 的溶解度，但成人静脉注射时可能会发生严重的过敏反应[3]。因此，VK1 注射给药的安全性受到广泛关注。胆盐和磷脂组成的混合胶束(mixed micelles，MM) 作为新型载药体系，生物安全性较高，能提高难溶性药物的溶解度[4]。MM 可代替聚氧乙烯蓖麻油、聚山梨酯80 及丙二醇等增溶剂[3,5]，用于递送VK1 时可增加VK1 溶解度，同时显著降低产生过敏反应的风险。然而，有研究[2]报道临床应用VK1混合胶束溶液(vitamin K1 loaded mixed micelles solution，VK1-MMs) 仍存在不良反应，可能是由注射液中的游离VK1 引发。例如，VK1 可通过一氧化氮途径激活血管扩张剂的释放，并阻断主要离子电流，对心脏动作电位产生直接影响，静脉注射时可导致血流动力学变化，引起心血管衰竭等不良反应[6]。因此，VK1-MM 的包封率是影响生物安全性的重要评价指标。有研究[7]采用高速离心法测定VK1 纳米脂质载体的包封率，另有研究[8] 采用萃取法测定VK1固体脂质纳米粒的包封率，但尚未见适用于VK1-MM 的包封率测定方法和方法准确性的研究报道。因此，本研究比较了萃取法、离心法和过滤法检测VK1-MM 包封率的适用性，并探究了影响包封率检测准确性的主要因素，以期建立可准确检测VK1-MM 包封率的方法、为辅助评估其安全性提供数据参考。↑长按二维码阅读全文作者简介：郭丽珊(1997—)，女，硕士研究生，专业方向：复杂注射剂研究。通信作者：奚 泉(1979—)，男，研究员，博士生导师，从事口服缓控释制剂、吸入制剂和复杂注射剂的研究。E-mail：xqsipi@163.com文章详情欢迎登录《中国医药工业杂志》官网查看。年度发布2023年《全球药研新动态》《中国医院市场用药格局》（2023年Q2版）2023年《数图药讯》权威发布2022年度中国医药工业百强榜单解读中国仿制药发展报告（2022版）《2023年专利到期药品概述》医药行业专项报告中国医药中间体和原料药行业调研报告中国非甾体抗炎类原料药市场调研报告中国祛痰类原料药市场调研报告中国钙拮抗剂类原料药市场调研报告中国血管紧张素Ⅱ受体拮抗剂类原料药市场调研报告中国口服血糖调节类原料药市场调研报告中国中枢兴奋类原料药市场调研报告中国抗痛风类原料药市场调研报告中国脑血管病类原料药市场调研报告END如需获取更多数据洞察信息或公众号内容合作，请联系医药地理小助手微信号：pharmadl001

Cortrophin Gel is the only FDA-approved purified corticotropin (ACTH) indicated for the treatment of appropriate patients with acute gouty arthritis flares New 1-mL size furthers ANI’s commitment to providing ACTH options for appropriate acute gouty arthritis flare patients and their healthcare providers; available through distributors to help enable in-office administration and through specialty pharmacies Cortrophin Gel will now be available in two sizes, including the 5-mL vial size launched in January 2022 BAUDETTE, Minn., Oct. 02, 2023 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced U.S. Food and Drug Administration (FDA) approval and commercial availability of a 1-mL vial (80 USP units/1 mL) of Purified Cortrophin® Gel (repository corticotropin injection USP) (Cortrophin Gel). The 1-mL vial is now available via Cortrophin Gel’s existing specialty pharmacy network and will also be available for ordering via national specialty distributors. Cortrophin Gel is already available in a 5-mL vial. Cortrophin Gel is indicated for short-term administration as an adjunctive therapy during an acute episode or exacerbation in acute gouty arthritis. Cortrophin Gel is contraindicated for intravenous administration. For additional important safety information, please see below. ANI reintroduced Cortrophin Gel to the market in January 2022. At that time, Cortrophin Gel became the first newly commercialized drug indicated for appropriate patients with acute gouty arthritis flares in 12 years. This smaller 1-mL product configuration was developed with the goal of helping enable rapid time to therapy, at point-of-care, for appropriate patients with acute gouty arthritis flares. "More than 9 million people in the US are affected by gout, and each year a portion of these patients experience acute gouty arthritis flares as a symptom of their underlying disease. For some of these patients, Cortrophin Gel may be an appropriate additional treatment option for their flares. The approval of the 1-mL vial for Cortrophin Gel is another step forward in our mission to increase access for appropriate acute gouty arthritis flare patients in need. Scaling up our Rare Disease business is ANI’s largest growth driver and key to achieving our goal of building a high-growth biopharma company,” stated Nikhil Lalwani, President and Chief Executive Officer of ANI. “With this approval, we look forward to continuing the Cortrophin Gel launch momentum we have seen to date,” Lalwani continued. “The commercial launch of the 1-mL vial will be supported by ANI’s existing Cortrophin Gel field sales force, which is already engaging rheumatologists and nephrologists who treat patients with acute gouty arthritis flares. Importantly, we are enhancing Cortrophin in Your Corner™, our reimbursement and access hub, to include support for this new 1-mL configuration of Cortrophin Gel.” “Gout is the most common type of inflammatory arthritis,” said Mary Pao, MD, PhD, Chief Medical Officer of ANI. “Acute gouty arthritis flare patients who are in need of an additional flare treatment options, beyond currently recommended first line therapies, have a need for alternatives. The development of the 1-mL vial represents ANI’s commitment to this patient population.” Cortrophin Gel is an adrenocorticotropic hormone (ACTH), also known as purified corticotropin, and it is approved for the treatment of certain chronic autoimmune disorders, including acute exacerbations of multiple sclerosis (MS) and rheumatoid arthritis (RA), in addition to excess urinary protein due to nephrotic syndrome. Cortrophin Gel is the only FDA-approved ACTH therapy indicated for the treatment of appropriate patients with acute gouty arthritis flares. For a full list of indications, please see below. Indications Cortrophin Gel is a prescription medicine that is injected subcutaneously or intramuscularly. It is indicated for: Short-term administration as an adjunctive therapy during an acute episode or exacerbation in: acute gouty arthritisrheumatoid arthritis, including juvenile rheumatoid arthritis; psoriatic arthritis; and ankylosing spondylitis. Exacerbations or as maintenance therapy in select cases of systemic lupus erythematosus and systemic dermatomyositis (polymyositis).Severe erythema multiforme (Stevens-Johnson syndrome) and severe psoriasis.Atopic dermatitis and serum sickness.Severe acute and chronic allergic and inflammatory conditions affecting the eye and its adnexa, such as allergic conjunctivitis, keratitis, iritis and iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, and anterior segment inflammation.Symptomatic sarcoidosis.Inducing a diuresis or remission of proteinuria due to nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus.Acute exacerbations of multiple sclerosis. Important Safety Information Contraindications Cortrophin Gel is contraindicated for intravenous administration.Cortrophin Gel is contraindicated in patients who have any of the following conditions: scleroderma; osteoporosis; systemic fungal infections; ocular herpes simplex; recent surgery; history of or the presence of a peptic ulcer; congestive heart failure; hypertension; primary adrenocortical insufficiency; adrenocortical hyperfunction; or sensitivity to proteins derived from porcine sources. Warnings and Precautions Infections: Corticotropin therapy may increase susceptibility to infections and may mask the symptoms of infections.Adrenal insufficiency: Prolonged corticotropin therapy can increase the potential for adrenal insufficiency after withdrawal of the medication. Adrenal insufficiency may be minimized by gradually reducing the corticotropin dosage. Hormone therapy should be reinstituted if stressful situations arise during discontinuation.Elevated blood pressure, salt and water retention, and hypokalemia: Corticotropin can cause elevation of blood pressure, salt and water retention, and increased excretion of potassium or calcium.Masking symptoms of other diseases: Corticotropin may only suppress signs and symptoms of chronic disease without altering the natural course of disease.Psychiatric reactions: Psychic derangements may appear when corticotropin is used, ranging from euphoria, insomnia, mood swings, personality changes, and depression to psychosis. Existing conditions may be aggravated.Ophthalmic reactions: Prolonged use of corticotropin may produce posterior subcapsular cataracts and glaucoma with possible damage to the optic nerves.Immunogenicity potential: Prolonged administration of Cortrophin Gel may increase the risk of hypersensitivity reactions. Neutralizing antibodies with chronic administration may lead to loss of endogenous ACTH and Cortrophin Gel activity.Vaccination: Patients should not be vaccinated against smallpox while on corticotropin therapy. Other immunizations should be undertaken with caution due to possible neurologic complications and lack of antibody response.Use in patients with hypothyroidism and cirrhosis: There is an enhanced effect in patients with hypothyroidism and in those with cirrhosis.Use in patients with latent tuberculosis or tuberculin reactivity: Closely observe for reactivation of the disease.Comorbid diseases: Corticotropin should be used with caution in patients with diabetes, abscess, pyogenic infections, diverticulitis, renal insufficiency, and myasthenia gravis.Growth and development: Carefully observe growth and development of infants and children on prolonged corticotropin therapy.Acute gouty arthritis: Treatment of acute gouty arthritis should be limited to a few days. Conventional concomitant therapy should be administered during corticotropin treatment and for several days after it is stopped.Drug interactions: Aspirin should be used cautiously with corticotropin in hypoprothrombinemia.Pregnancy: Since fetal abnormalities have been observed in animals, Cortrophin Gel should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Adverse Reactions Adverse reactions for Cortrophin Gel include fluid or sodium retention; muscle weakness; osteoporosis; peptic ulcer with possible perforation and hemorrhage; injection site reactions; impaired wound healing; hypertension; convulsions; headache; development of Cushingoid state; suppression of growth in children; and weight gain. These are not all the adverse reactions reported with Cortrophin Gel. Please click here for full prescribing information for Cortrophin Gel. About ANI ANI Pharmaceuticals, Inc. (Nasdaq: ANIP) is a diversified bio-pharmaceutical company serving patients in need by developing, manufacturing, and marketing high quality branded and generic prescription pharmaceutical products, including for diseases with high unmet medical need. Our team is focused on delivering sustainable growth by scaling up our Rare Disease business through successful launch of our lead asset, Purified Cortrophin® Gel, strengthening our generics business with enhanced development capability, innovation in established brands and leveraging our North American manufacturing capabilities. For more information, please visit our website www.anipharmaceuticals.com. Forward-Looking Statements To the extent any statements made in this release deal with information that is not historical, these are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, those relating to the commercialization and potential sales of the product and any additional product launches from the Company’s generic pipeline, other statements that are not historical in nature, particularly those that utilize terminology such as “anticipates,” “will,” “expects,” “plans,” “potential,” “future,” “believes,” “intends,” “continue,” other words of similar meaning, derivations of such words and the use of future dates. These forward-looking statements include statements regarding the commercial launch of Cortrophin Gel and the potential benefit of Cortrophin Gel to patients as a treatment option. Risks and uncertainties that may cause the Company’s actual results to be materially different than those expressed in or implied by such forward-looking statements include, but are not limited to, the costs involved in commercializing Cortrophin Gel, the ability to maintain regulatory approval of the product and maintain sufficiency of the product, the ability to obtain reimbursement from third-party payors for this product, evolving government legislation, the opinions and views of key opinion leaders and physicians who treat patients with chronic diseases and who may prescribe Cortrophin Gel, manufacturing difficulties or delays, ANI’s ability to generate projected net product revenue and gain market share on the timeline expected, actions taken by competitors in response to a new market entrant, ANI’s reliance on third parties over which it may not always have full control, costs and regulatory requirements relating to contract manufacturing arrangements, increased competition and strategies employed by competitors, uncertainties regarding the COVID-19 pandemic, market trends for our products, regulatory environment and changes, and other risks and uncertainties. More detailed information on these and additional factors that could affect the Company’s actual results are described in the Company’s filings with the Securities and Exchange Commission (“SEC”), including its most recent annual report on Form 10-K and quarterly reports on Form 10-Q, as well as other filings with the SEC. All forward-looking statements in this news release speak only as of the date of this news release and are based on the Company’s current beliefs, assumptions, and expectations. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. Contacts: Investor:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Media:Faith Pomeroy-WardT: 817-807-8044E: faith.pomeroyward@anipharmaceuticals.com Source: ANI Pharmaceuticals, Inc. A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/2efb6d4a-2b60-468e-93f0-ab6ceeb02958