CPS1

DUBLIN, Aug. 23, 2023 /PRNewswire/ -- The "Global Urea Cycle Disorders Treatment Market 2017-2027" report has been added to ResearchAndMarkets.com's offering. The global urea cycle disorders treatment market is poised for impressive growth in the forecast period from 2023 to 2027. This upward trajectory is fueled by a combination of factors, including the increasing prevalence of urea cycle disorders and substantial research and development efforts. Various catalysts are propelling this market's expansion, including heightened investments in research and development, growing initiatives by key industry players, heightened awareness, improved healthcare services, and a surge in innovative diagnostic methods. Rising Prevalence of Urea Cycle Disorders A surge in the prevalence of urea cycle disorders is expected to drive global market growth. Reports from the Centers for Disease Control and Prevention (CDC) indicate a projected incidence of 1 in 8500 births for urea cycle disorders. However, the actual number may be higher due to undiagnosed cases. It is estimated that up to 20% of Sudden Infant Death Syndrome (SIDS) cases may be attributed to undiagnosed inborn metabolic errors such as urea cycle disorders. This underscores the urgency of addressing and treating these disorders, thus catalyzing growth in the urea cycle disorders treatment market. Initiatives and Awareness Fueling Growth Governments and private organizations worldwide have ramped up efforts to support market growth. These initiatives encompass substantial investments in research and development, increased awareness campaigns, extensive research endeavors, clinical trials, and streamlined regulatory approvals. For instance, the merger of Mead Johnson Nutrition Company (MJN) with Reckitt Benckiser Group plc (RB) in 2017 bolstered the dietary management of urea cycle disorders by integrating brands like Enfa and Nutramigen into RB's consumer health portfolio. Similarly, Horizon Pharma plc's UCD in Common initiative offers resources and support for individuals with urea cycle disorders and their families. Leading Players and Geographic Outlook Market players shaping the global urea cycle disorders treatment market landscape include: Bausch Health Companies Inc. Recordati Rare Diseases Eurocept Pharmaceuticals Holding (Lucane Pharma SA) Acer Therapeutics Ultragenyx Pharmaceutical Aeglea BioTherapeutics Arcturus Therapeutics, Inc. Orpharma Pty. Ltd. Selecta Biosciences, Inc Abbott Laboratories, Inc. In terms of geographic spread, the United States is anticipated to be a pivotal market due to its escalating cases of urea cycle disorders. Conclusion: Navigating the Path Forward As the global urea cycle disorders treatment market marches ahead, it is driven by an amalgamation of factors that recognize the critical importance of addressing these genetic disorders. The rising prevalence, backed by initiatives, investments, and awareness, underscores the market's growth potential. A meticulously segmented market landscape, comprising diverse enzyme deficiency types, treatment options, routes of administration, and distribution channels, reflects the multifaceted approach adopted to combat urea cycle disorders. Amid these dynamics, leading players are contributing their expertise, while geographic trends emphasize the significance of targeted interventions. As the market evolves, it holds the promise of transforming lives and healthcare outcomes on a global scale. Report Scope In this report, global urea cycle disorders treatment market has been segmented into the following categories, in addition to the industry trends, which have also been detailed below. Urea Cycle Disorders Treatment Market, By Enzyme Deficiency Type: Carbamyl Phosphate Synthetase (CPS1) N-Acetylglutamate Synthetase (NAGS) Ornithine Transcarbamylase (OTC Deficiency) Argininosuccinic Acid Synthetase (AS) Argininosuccinate Lyase (AL or ASA Lyase) Arginase (AG) Urea Cycle Disorders Treatment Market, By Treatment Type: Amino Acid Formulas Phenylbutyrate Sodium Benzoate Others Urea Cycle Disorders Treatment Market, By Route of Administration: Oral Intravenous Urea Cycle Disorders Treatment Market, By Distribution Channel: Hospital Pharmacies Retail Pharmacies Online Pharmacies Urea Cycle Disorders Treatment Market, By Region: North America Asia-Pacific Europe & CIS South America Middle East & Africa Key Topics Covered 1. Product Overview 2. Research Methodology 3. Impact of COVID-19 on Global Urea Cycle Disorders Treatment Market 4. Voice of Customer 5. Executive Summary 6. Global Urea Cycle Disorders Treatment Market Outlook 7. North America Urea Cycle Disorders Treatment Market Outlook 8. Europe Urea Cycle Disorders Treatment Market Outlook 9. Asia-Pacific Urea Cycle Disorders Treatment Market Outlook 10. South America Urea Cycle Disorders Treatment Market Outlook 11. Middle East and Africa Urea Cycle Disorders Treatment Market Outlook 12. Market Dynamics 13. Market Trends & Developments 14. Competitive Landscape 15. Strategic Recommendations For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. 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Anivive's antifungal vaccine afforded protection in 100% of vaccinated dogs First vaccine with demonstrated efficacy against a systemic fungal disease Due to climate change, more than 30 million dogs are now at risk LOS ANGELES, June 17, 2021 /PRNewswire/ -- Anivive, a pet health technology company transforming the therapeutic discovery and development process, today announced positive results from a study on its groundbreaking fungal vaccine for Valley Fever in dogs. The primary endpoint of the study was met, with the vaccine affording protection in 100% of vaccinated dogs, preventing clinical disease. In this randomized, blinded, placebo-controlled study, Anivive's vaccine was administered to dogs who were then challenged with a disease-causing strain of Coccidioides, the fungus that causes the systemic fungal disease, Valley Fever. The dogs also received a booster one month later. When compared to unvaccinated controls, with a mean disease score of 123.7, the three dose groups vaccinated with avirulent Coccidioides spores (Δcps1), had mean composite disease scores of 9.5, 10.7, and 11.7, respectively. These study results indicate that vaccinated dogs did not develop clinical signs of Valley Fever. Vaccinated dogs in all groups had their fungal burdens, radiographic abnormalities, gross and histopathological lung lesions greatly reduced in comparison to unvaccinated controls (p=0.0002). "This study provides important data that validates our model and demonstrates the ability of the vaccine to afford protection against exposure to an extremely virulent fungal pathogen," said Anivive Chief Medical Officer David Bruyette, DVM, DACVIM. "To date, no vaccine exists to protect against systemic fungal disease, so this is an important step forward in our progress to develop a vaccine for Valley Fever and other fungal diseases in animals and humans." The Anivive team is using breakthrough gene deletion technology to remove CPS1, a gene key to Coccidioides' ability to produce disease, to create a modified fungal organism for the vaccine. Clinical studies are proving that the modified organism, while unable to spread, provokes a strong immune reaction in mice and dogs. The vaccine is designed to be given as injection, the same way dogs receive other vaccines, with two injections the first year and a single booster annually. Valley Fever is caused by inhaling Coccidioides, found most commonly in the soil of the Western and Southwestern United States. Symptoms in humans resemble those of the flu, but dogs are more likely to develop more severe disseminated forms of the disease. More than 30 million dogs live in or travel to parts of the country where Valley Fever is prevalent, highlighting the importance of developing a vaccine to prevent disease. While antifungal therapies exist, they most commonly require costly life-long treatments with harsh side effects. Anivive's work with Dr. John Galgiani and his research team at the Valley Fever Center for Excellence at the University of Arizona College of Medicine was featured in a recent Scientific American story. Anivive's technology is also pushing the envelope on over eight therapeutics in their pipeline. To learn more, visit: . Disclaimer: The results of the efficacy study for the therapeutic claim stated in this press release have not been reviewed by the USDA Center for Veterinary Biologics. About Anivive Anivive is reinventing the drug development process in pet pharma. Our proprietary software accelerates the delivery of new, affordable treatments for pets. Backed by over $45 million in seed funding, Anivive has gained conditional approval for LAVERDIA™-CA1 (verdinexor), the first oral tablet to treat canine lymphoma and created a specialty sales force dedicated to supporting veterinarians. Anivive's pipeline includes 8+ first-in-class pharmaceuticals, biologics, and vaccines. For more information on how we address unmet needs for pets, visit anivive.com. View original content to download multimedia: SOURCE Anivive Lifesciences Inc.

LEBANON, N.J., Jan. 26, 2021 /PRNewswire/ -- Recordati Rare Diseases Inc. today announced the U.S. Food and Drug Administration (FDA) has approved a new indication for CARBAGLU® (carglumic acid) tablets 200mg as adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA) in pediatric and adult patients. CARBAGLU is the first and only FDA approved medication for the treatment of acute hyperammonemia due to PA and MMA. CARBAGLU was initially approved by the FDA for N-acetylglutamate synthase (NAGS) deficiency, another rare metabolic disorder, as adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to NAGS deficiency, and maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency. PA and MMA are rare inherited metabolic disorders that result in the dysfunction of a specific step of amino acid catabolism, or breaking down of certain fatty acids, due to deficient enzyme activity. As a result, toxic metabolites accumulate, which can cause hyperammonemia, a potentially life-threatening condition. CARBAGLU acts as a replacement for N-acetylglutamate (NAG) in NAGS deficiency, PA, and MMA patients by activating carbamoyl phosphate synthetase (CPS 1), improves or restores the function of the urea cycle, and facilitates ammonia detoxification and urea production. "There are few approved drugs that treat hyperammonemia, and none that are indicated for the treatment of acute hyperammonemia in PA and MMA patients," said Mendel Tuchman, MD, Medical Geneticist and Professor Emeritus of Pediatrics at The George Washington University School of Medicine and Health Science. "Carbaglu has the potential to impact these patients by reducing high plasma ammonia levels during critical situations." FDA approval of the new indication was supported by a randomized, double-blind, placebo-controlled, multicenter clinical trial comparing the effectiveness of CARBAGLU to placebo in the treatment of hyperammonemic episodes in patients with PA or MMA. The efficacy evaluation, based on 90 hyperammonemic episodes occurring in 24 patients, showed that patients receiving CARBAGLU demonstrated a quicker reduction of ammonia compared to patients receiving placebo. The primary endpoint was the time from the first dose to the earlier of blood ammonia level below 50 micromol/L or hospital discharge. Throughout the first three days of treatment, a higher proportion of CARBAGLU-treated episodes reached the primary endpoint compared to placebo-treated episodes. In the clinical trial, at least 1 adverse reaction was reported in 42.2% of the 90 hyperammonemic episodes that occurred. The most common adverse events were (≥5%) neutropenia, anemia, vomiting, electrolyte imbalance, decreased appetite, hypoglycemia, lethargy/stupor, encephalopathy and pancreatitis/lipase increased. "People living with these conditions face potentially serious complications from hyperammonemia that, without treatment, can lead to coma or even death," said Andrea Recordati, CEO of Recordati S.p.A.. "We are pleased to receive this new indication for Carbaglu which will enable Recordati to address an unmet medical need in these patients." About Propionic Acidemia (PA) and Methylmalonic Acidemia (MMA) Propionic acidemia (PA) and methylmalonic acidemia (MMA) are rare inherited metabolic disorders. PA results from a deficiency of the enzyme propionyl-CoA carboxylase. Most PA patients present with symptoms in the first few days of life. MMA results from different types of enzyme deficiencies or defects. The most common cause is a deficiency of the enzyme methylmalonyl-CoA mutase. In both disorders, complete lack of enzyme or very limited enzyme causes more severe symptoms. Hyperammonemia is one of the most severe and life-threatening events that can occur in patients with PA or MMA. Hyperammonemia is a medical emergency that if left untreated, can progress to irreversible brain damage, coma, or death. Please click here for Full Prescribing Information and Instructions for Use. Important Safety Information Contraindications: None. NAGS deficiency: Most common adverse reactions (≥13%) are: vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache. PA and MMA: Most common adverse reactions (≥5%) are: neutropenia, anemia, vomiting, electrolyte imbalance, decreased appetite, hypoglycemia, lethargy/stupor, encephalopathy and pancreatitis/lipase increased. To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1-800-FDA-1088 or If CARBAGLU is administered during pregnancy to women with NAGS deficiency, health care providers should report CARBAGLU exposure to the pregnancy pharmacovigilance program by calling 1-888-575-8344. About Recordati Rare Diseases Inc. Recordati Rare Diseases Inc. is a biopharmaceutical company committed to providing often-overlooked orphan therapies to the underserved rare disease communities of the United States. Recordati Rare Diseases is part of the Recordati Group, a public international pharmaceutical company committed to the research and development of new specialties with a focus on treatments for rare diseases. Recordati Rare Diseases' mission is to reduce the impact of extremely rare and devastating diseases by providing urgently needed therapies. We work side-by-side with rare disease communities to increase awareness, improve diagnosis and expand availability of treatments for people with rare diseases. The company's U.S. corporate headquarters is located in Lebanon, NJ, with global headquarter offices located in Milan, Italy. For a full list of products please click here: . For additional information, please visit our website: . Recordati, established in 1926, is an international pharmaceutical group, listed on the Italian Stock Exchange (Reuters RECI.MI, Bloomberg REC IM, ISIN IT 0003828271), with a total staff of more than 4,300, dedicated to the research, development, manufacturing and marketing of pharmaceuticals. Headquartered in Milan, Italy, Recordati has operations throughout the whole of Europe, including Russia, Turkey, North Africa, the United States of America, Canada, Mexico, some South American countries, Japan and Australia. An efficient field force of medical representatives promotes a wide range of innovative pharmaceuticals, both proprietary and under license, in several therapeutic areas including a specialized business dedicated to treatments for rare diseases. Recordati is a partner of choice for new product licenses for its territories. Recordati is committed to the research and development of new specialties with a focus on treatments for rare diseases. Consolidated revenue for 2019 was € 1,481.8 million, operating income was € 465.3 million and net income was € 368.9 million. Forward Looking Statement Statements contained in this release, other than historical facts, are "forward-looking statements" (as such term is defined in the Private Securities Litigation Reform Act of 1995). These statements are based on currently available information, on current best estimates, and on assumptions believed to be reasonable. This information, these estimates and assumptions may prove to be incomplete or erroneous, and involve numerous risks and uncertainties, beyond the Company's control. Hence, actual results may differ materially from those expressed or implied by such forward-looking statements. All mentions and descriptions of Recordati products are intended solely as information on the general nature of the company's activities and are not intended to indicate the advisability of administering any product in any particular instance.