Desmopressin Acetate

Biden-Harris Administration funds first Defense Production Act Title III award to onshore manufacturing of ingredients for essential medicines ST. LOUIS--(BUSINESS WIRE)--The API Innovation Center (APIIC) today announced that it has been awarded funding by the Administration for Strategic Preparedness and Response’s (ASPR) Center for Industrial Base Management and Supply Chain (IBMSC) to lead the development and domestic production of three critical active pharmaceutical ingredients (APIs) used in the treatment of asthma, diabetes, and anxiety disorders. APIIC will leverage advanced manufacturing technologies and cutting-edge research to further national strength in pharmaceutical innovation and supply chain resilience. The BioMaP-Consortium is a multi-purpose acquisition vehicle comprised of industry partners across the drug and vaccine manufacturing supply chain supported by ASPR’s Biomedical Advanced Research and Development Authority (BARDA). IBMSC will provide $14 million in funding and APIIC will invest an additional $2.4 million into the development of these critical APIs. This funding marks a significant step forward in securing the U.S. pharmaceutical supply chain and addressing national health security risks. At least 83% of the nation’s top 100 generic medicines have no U.S.-based source for the small-molecule APIs found in today’s medication. This dependency on foreign manufacturers has left the U.S. pharmaceutical supply chain vulnerable to shortages and geopolitical conflicts while domestic facilities operate below capacity. The decrease in U.S.-based production for APIs and key starting materials (KSMs) used to make them is widely recognized as a major driver behind this significant national health security risk. While much work remains to improve the return on investment for developing medicines domestically, funding through the BioMaP-Consortium represents a key investment in the ongoing efforts to reshore critical medicines and address ever-increasing drug shortages. Initiative to Address Availability of Essential Medications This initiative will see APIIC lead the development and production of three critical APIs, each chosen for their vital role in U.S. health care, shortage history, as well as inclusion on essential medicines lists: Albuterol: Used to treat asthma and other respiratory conditions. Desmopressin Acetate: Used to treat diabetes insipidus & blood coagulation conditions. Lorazepam: Used to treat anxiety. “This funding represents both a financial commitment and a significant step towards securing America’s pharmaceutical resiliency and patient access to critical medicines. By focusing on developing these high-impact molecules, we are creating a private-public blueprint for driving self-reliance that is essential for national health security and can be replicated across the country,” said Tony Sardella, chair and founder of APIIC. Partnership and Impact This project harnesses the strengths of APIIC’s extensive partner network, including collaborations with leading universities, pharmaceutical manufacturers, and suppliers of KSMs. APIIC will unite the resources and advanced manufacturing technologies of Mallinckrodt Specialty Generics, Apertus Pharmaceuticals, MilliporeSigma, and the University of Missouri–St. Louis (UMSL) to enhance the quality, efficiency, and global competitiveness of U.S.-based pharmaceutical manufacturing. These strategic collaborations form a dynamic product team, capitalizing on each member’s specific expertise, with APIIC providing funding and support to harness their strengths and advanced manufacturing capabilities. “Mallinckrodt Specialty Generics is proud to partner with the API Innovation Center on this critical initiative to bolster the U.S. pharmaceutical supply chain. Our proven expertise in the development, manufacture, and commercialization of active pharmaceutical ingredients in the U.S. positions us extremely well to contribute to this effort. By collaborating with APIIC and its partners, we are both addressing the nation’s drug shortage challenges and strengthening the nation’s ability to manufacture essential medicines domestically,” said Stephen Welch, executive vice president & head of Mallinckrodt Specialty Generics. APIIC will collaborate closely with Apertus Pharmaceuticals on research and development work, leveraging advanced manufacturing technology to produce these critical molecules. “Collaborating with the API Innovation Center has enabled us to scale our cutting-edge research and play a key role in this national initiative to strengthen our pharmaceutical supply chain,” said David Gindelberger, chief executive officer of Apertus Pharmaceuticals. MilliporeSigma, a small and large molecule contract development and manufacturing organization (CDMO) with facilities in St. Louis, will also play a pivotal role. “We are excited to bring our 25 years of CDMO expertise in organic synthesis and bioconjugation of ADCs, producing Active Pharmaceutical Ingredients (API), excipients and critical raw materials, to this partnership. This initiative will not only mitigate risks associated with supply disruptions but can also enhance the U.S. bioeconomy,” said Mark Cooley, site director of one of the St. Louis MilliporeSigma facilities involved in the consortium. “Partnering with the API Innovation Center on this important initiative allows us to leverage our strengths in research, workforce development, and community engagement to support a more secure and self-reliant pharmaceutical supply chain. UMSL is proud to play a key role in advancing these critical efforts, which will have lasting impacts on both our region’s economy and the nation’s health security,” said Dr. Chris Spilling, vice chancellor for research and economic and community development at the University of Missouri-St. Louis. A Roadmap for a Resilient Supply Chain in Missouri APIIC, together with the State of Missouri and the Missouri Technology Corporation, has been developing a state-supported model in the St. Louis region and a strategic framework for creating a resilient national pharmaceutical manufacturing ecosystem. This model is designed to be a replicable blueprint that compliments Missouri’s strengths in infrastructure and workforce development to restore U.S. leadership in pharmaceutical production. To date, the State of Missouri has committed approximately $18 million to API reshoring efforts, demonstrating to other states how to support the “Missouri Model” and take the lead in addressing national supply chain challenges. “Missouri is leading in restoring America’s pharmaceutical manufacturing strength. By focusing on developing active pharmaceutical ingredients right here in Missouri, we are tackling a pressing national health security issue—our over-reliance on foreign suppliers,” said Gov. Mike Parson. “Recognizing our strengths, particularly in pharmaceutical manufacturing, we collaborated with the General Assembly to invest in bringing critical medicine production back to the U.S. The state’s investment, now bolstered by federal support, is a clear indicator that what we are doing in Missouri is working. We are not just talking about solutions. We’re delivering results that are necessary steps toward safeguarding the health and security of our nation.” As it looks to the future, APIIC is committed to driving the U.S. towards pharmaceutical self-reliance, ensuring every hospital, pharmacy, and patient has access to domestically produced critical medication to support national health security. This project has been funded in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response (ASPR); Office of Industrial Base Management and Supply Chain (IBMSC), under OT number #75A50123D00003. About the API Innovation Center: The API Innovation Center (APIIC) is a 501(c)(3) nonprofit public benefit organization headquartered in St. Louis, Missouri, dedicated to driving health security and economic growth for the nation and its citizens through U.S.-based production of critical medicines. By investing in and collaborating along the supply chain, APIIC’s model contracts with leading U.S.-based specialists within their pharmaceutical ecosystem to develop and manufacture high-quality domestically produced Active Pharmaceutical Ingredients. APIIC is also committed to expanding access to critical medications through strategic partnerships to commercialize market-competitive supplies of finished drug products and build resiliency in the supply chain. Their mission is to ensure every hospital, pharmacy, and patient has access to domestically produced critical medication to fortify national health security. APIIC is supported by the state of Missouri through the Advanced Manufacturing Resiliency Grant awarded by the Missouri Technology Corporation. To learn more, visit: apicenter.org . About Apertus Pharmaceuticals: Apertus Pharmaceuticals is a contract development and manufacturing organization (CDMO) that develops and manufactures device, OTC and prescription pharmaceutical products. Services include product de-formulation, formulation development, process development, and manufacturing (clinical batch to intermediate scale), including product serialization. Apertus performs an extensive range of analytical method development and validation as well as provides stability programs for all of the medications it produces. Apertus currently manufactures solid oral dose, suppository, liquid and cream products. Apertus is cGMP compliant and maintains Drug Enforcement Administration research and analytical registrations for Schedules II-V. To learn more about Apertus Pharmaceuticals, visit: apertuspharma.com . About Mallinckrodt Specialty Generics: Mallinckrodt’s Specialty Generics business segment is a 150+ year-old business that manufactures approximately 50 FDA-approved active pharmaceutical ingredients and approximately 100 FDA-approved finished dose pharmaceutical products across our network of U.S.-based manufacturing sites. To learn more about Mallinckrodt Specialty Generics, visit . About the Life Science business of Merck KGaA, Darmstadt, Germany The Life Science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has more than 27,000 employees and more than 55 total manufacturing and testing sites worldwide, with a portfolio of more than 300,000 products focused on scientific discovery, biomanufacturing and testing services. Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and electronics. Follow MilliporeSigma on Twitter @MilliporeSigma, on Facebook @MilliporeSigma and on LinkedIn . About the University of Missouri–St. Louis: A Tier 1 national university and Carnegie R2 classification—Doctoral University with High Research Activity— public research university, UMSL, and its four-campus University of Missouri System, provides strong research programs in chemical organic synthesis and advanced pharmaceutical manufacturing methods, such as continuous flow technologies, along with strong industry partnerships. To learn more about the University of Missouri–St. Louis, visit: umsl.edu . Contacts Kaylee Schreiner kaylee.schreiner@fleishman.com

Octapharma to present new clinical and scientific findings for wilate® and Nuwiq® at the upcoming ISTH 2024 Congress New data and clinical study developments to be showcased in seven oral presentations, two posters and two Supported Symposia LACHEN, Switzerland, June 10, 2024 /PRNewswire/ -- The most recent developments from Octapharma's Haematology portfolio will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) on June 22–26, 2024, in Bangkok, Thailand. The data will be featured in seven oral presentations, two poster presentations and two Supported Symposia during this key international meeting. Octapharma is a proud Silver Supporter of the ISTH 2024 Congress. Octapharma will also present the offer in modern solutions for acute bleeding and coagulation management in critical and intensive care. Bleeding disorders, such as haemophilia A and von Willebrand disease (VWD), carry the risk of recurrent and prolonged bleeds, which can not only be life-threatening but also take a heavy toll on patients' quality of life and mental health. The presentations at ISTH 2024 reflect Octapharma's long-term commitment to improving patient care and addressing unmet clinical needs. "Octapharma is excited to present our latest findings and study results from across our haematology and critical care portfolios at ISTH 2024." said Olaf Walter, Board Member at Octapharma. "Bleeding and coagulation disorder patients continue to face many challenges in their day-to-day care and bleeding management. Here at Octapharma we remain dedicated to frontline research towards improving patients' lives." Oral Presentations Although prophylaxis is the standard of care for severe haemophilia A, it remains underutilised in VWD. WIL-31, the largest study in VWD prophylaxis, demonstrated the safety and efficacy of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (wilate®) in patients with VWD. The lead investigator, Dr Robert F. Sidonio Jr., will present sub-analyses of data from the study which explore differences in bleed sites across VWD types and age groups. Prophylaxis was well-tolerated across all age groups in the 22 VWD type 3 patients who were included in the analysis population. MOTIVATE is an ongoing, international, investigator-initiated study supported through funding from Octapharma AG, evaluating the management of haemophilia A patients with inhibitors. Dr Carmen Escuriola-Ettingshausen, one of the study's coordinating investigators, will present an interim analysis of the study. Haemophilic arthropathy is a serious complication of haemophilia A and early diagnosis is critical to minimise its long-term impact. A proof-of-concept study has identified a signature of several circulating small non-coding microRNAs, which may be used as biomarkers to assist in the diagnosis of haemophilic arthropathy. Future work will investigate microRNAs associated with earlier stages of the condition in a sub-study of the PROVE clinical trial. Altered microvascular endothelial functionality has been observed in haemophilia A patients. Indeed, impaired tubulogenesis, migration and permeability of endothelial cells from haemophilia A patients has been observed, compared to those from healthy patients. New data will be presented showing that Nuwiq® can bind to endothelial cell surfaces, improving their functionality. FVIII physically interacts with platelets following vascular injury to ensure efficient clot formation. Different FVIII modifications may impact platelet binding and downstream signalling. Data will be presented showing that Nuwiq® exhibits significantly higher binding to platelets compared to comparator products, and that these differences in platelet binding may impact the efficacy of different rFVIII products in the treatment of haemophilia A. The oral presentations are scheduled at the following times: Sunday June 23 OC 27.5: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet clotting functionality. Presenting author: Viola Vogel. 15:45–16:00 ICT, Room 210 A-D Monday June 24 OC 35.1: Factor VIII regulates extracellular matrix proteins to stimulate angiogenesis and vessel stability. Presenting author: Alessia Cucci. 9:30–9:45 ICT, Room 110 A-C OC 36.4: miRNA expression profiles as a potential biomarker of joint and bone health in haemophilia A. Presenting author: Yesim Dargaud. 10:15–10:30 ICT, Room 111 A-C OC 36.5: Factor VIII is a regulator of angiogenesis and a promoter of endothelial barrier stability. Presenting author: Antonia Follenzi. 10:30–10:45 ICT, Room 111 A-C Tuesday June 25 OC 63.4: Bleeding Sites in von Willebrand Disease Patients on Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 15:30–15:45 ICT, Ballroom B3 OC 61.4: Immune tolerance induction (ITI) strategies in haemophilia A patients with inhibitors – Interim analysis from the MOTIVATE study. Presenting author: Carmen Escuriola-Ettingshausen. 15:45–16:00 ICT, Ballroom B1 Wednesday June 26 OC 73.5: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 11:15–11:30 ICT, Ballroom B4 Poster Presentations Poster presentations are scheduled at the following times: Sunday June 23 PB 0251 - Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States. Presenting author: Craig M. Kessler. 13:45–14:45 ICT, Exhibition Hall Tuesday June 25 PB 1102 - Covalently linked complexes of coagulation factor VIII and von Willebrand factor fragment display increased stability and half-life in animal models and retain their function. Presenting author: Barbara Solecka-Witulska. 13:45–14:45 ICT, Exhibition Hall Supported Symposia Two Supported Symposia during the Congress will share new clinical and scientific data, providing updates on ongoing and new studies in the fields of haemophilia A and VWD. Learn About Tomorrow's Advances Today – Breakthrough in All-Round Bleed Protection Sunday June 23, 12:15–13:30 ICT, Room 208 Chair: Guy Young, Children's Hospital Los Angeles and Keck School of Medicine, Los Angeles, California, USA Octapharma's Symposium on haemophilia A this year is dedicated to cutting-edge research committed to bleed protection. Speakers will present the first results from the MOTIVATE study interim analysis and introduce the PROVE study investigating bone and joint health. This symposium will also explore the critical role of FVIII binding to platelets and endothelial cells in haemophilia A. Next Level Insights on Prophylaxis in von Willebrand Disease – Are You Addressing Your Patient's Needs? Monday June 24, 12:15–13:30 ICT, Room 210 Chair: Craig Kessler, Georgetown University Medical Center, Washington DC, USA This symposium will explore the critical role of prophylaxis in severe VWD. Topics presented will include the importance of prophylaxis and how to identify patients who may benefit from prophylactic treatment. Key lessons from the WIL-31 VWD prophylaxis study will be shared as well as the important role for prophylaxis in the management of heavy menstrual bleeding in women with VWD. Optimising care during pregnancy will also be discussed and the VIP and EMPOWER studies will be introduced. Both symposia will be open to congress participants in Bangkok and will be live streamed for participants not directly attending the Congress. An opportunity to ask questions will be available at both symposia. Larisa Belyanskaya, SVP and Head of IBU Haematology at Octapharma commented: "We are excited to present new insights from the WIL-31 data, and will take a deep dive into the importance of prophylaxis for specific patient groups, as well as discussing important clinical and scientific questions with the goal of improving the quality of life for people with von Willebrand disease and haemophilia A." About Nuwiq® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein1. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor1. Nuwiq® treatment has been assessed in nine1 2 3 completed clinical trials which included 201 previously treated patients (190 individuals)1 and 108 previously untreated patients2 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations4. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups4. About wilate® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production1. No albumin is added as a stabiliser5. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma1. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma1. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres6. wilate® is available in 500 IU and 1000 IU presentations. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)6. About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 195 plasma donation centers across Europe and the US. "Octapharma press releases are specifically for health specialist/medical media and are not for consumer press." [1] Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. [2]Liesner RJ et al. Thromb Haemost 2021; 121:1400–8. [3] Octapharma AG, data on file. [4] Nuwiq® Summary of Product Characteristics [5] Stadler M et al. Biologicals 2006; 34:281-8. [6] wilate® Summary of Product Characteristics. Logo: SOURCE Octapharma AG