Somatropin(Genentech, Inc.)

DUBLIN, Jan. 15, 2024 /PRNewswire/ -- The "Chronic Disease Markets for Sustained Released Injectables" report has been added to ResearchAndMarkets.com's offering. The report provides a comprehensive evaluation and analysis of the technology, products and participants providing the driving force behind this evolving segment of the healthcare sector. The study is designed to provide drug company decision makers, drug delivery developers, device designers, healthcare marketers, and supply chain participants with a detailed understanding of the economics, technologies, disease segments, and commercial opportunities for chronic disease markets for sustained release injectables. Provider organization business managers, healthcare administrators and investors will also benefit from this study. Evolving Engineered Solutions Chronic conditions require drug administration over long periods of time, placing a greater emphasis on self-administration. With the shift away from caregiver interaction, drug safety and compliance/adherence become prominent concerns. These concerns, which are not new, are becoming magnified as the number of powerful new drugs reaching the market increases. For healthcare managers and public health officials, one way to address these issues is less frequent dosing. Using formulation technologies designed to modulate the effect of therapeutic substances, drug developers are creating formulations that exhibit extended-release profiles. These chemistries include polymers such as polyethylene glycol-complexed (PEGylated), which are used to product encapsulated and coated versions of new APIs. The goal is to address the requirement for patient-dependent, and therefore compliance-sensitive, drug treatment protocols such as multiple dosing through controlled release formulations that provide the desired therapeutic effect with dosing of once-a-day or less. What You Will Learn What are the marketed sustained release injectable drug products, what sustained release formulation technologies are used, and who markets them? What are the major factors driving sustained release injectable drug demand? What sustained release injectable drug candidates are in late-stage clinical development and what is their potential impact? How important are drug developer-formulation technologist relationships in the sustained release market segment, and what are the key alliances in the industry? What are the essential formulation factors, delivery device selection issues, related technology factors and market development issues for sustained release injectable drug products? In what therapeutic market segments do sustained release injectables compete? What is their market share today? What will it be in 2028? What is their expected growth rate? What are the significant economic, technology, and regulatory factors affecting the market for sustained release injectables? Key Topics Covered: Injectable Drug Market Dynamics The Trend toward Self-Administration Injectable Drug Formulation Technology Innovation in Injectable Device Designs Therapeutic Demand Drivers Market Factors Competitive Landscape Risk Factors Sustained Release - Branded Formulation Technologies Biodegradable Polymers Branded SR Formulation Technologies Physical Process Control Methods Sustained Release Injectables - Development Factors Formulation Factors Stability Lyophilization Packaging Administration Factors SR Injectables - Product Analysis & Market Sector Forecasts Addiction Naltrexone Diabetes Exenatide Infectious Disease Interferon Hormone Deficiency Estradiol Somatropin Testosterone Metabolic Diseases Acromegaly Neurology Oncology Leukemia Lymphoma Cytarabine Prostate Pain Management Reproductive Health Fertility Endometriosis Market Profiles Companies Mentioned Alkermes Amylin AntriaBio Ascendis Pharma AstraZeneca Aurobundo Avadel Biomarin Camurus Critical Pharmaceuticals DURECT Eli Lilly Enzon Ipsen Janssen NanOlogy Novartis OctoPlus Oakwood Laboratories/PharmaSophia Pacira PhaseBio Pharmaceuticals Recipharm AB Roche Sanofi Taiwan Liposome Company Xbrane Bipharma 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. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

BUCHAREST, Romania, Jan. 09, 2024 (GLOBE NEWSWIRE) -- Er-Kim, an international pharmaceutical company specializing in the commercialization of novel therapies, today announced it has entered into an exclusive distribution agreement with Danish Company Ascendis Pharma A/S (Nasdaq: ASND) for its endocrinology portfolio. Under the agreement, Er-Kim is appointed as the exclusive Ascendis A/S endocrinology distributor in Albania, Bosnia and Herzegovina, Bulgaria, Croatia, Cyprus, Czechia, Estonia, Greece, Hungary, Kosovo, Latvia, Lithuania, Malta, Montenegro, North Macedonia, Poland, Romania, Serbia, Slovakia, Slovenia, and Türkiye. Once-weekly SKYTROFA (lonapegsomatropin) is a human growth hormone (hGH) approved in the European Union for growth failure in children and adolescents aged from 3 to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]).2 YORVIPATH (palopegteriparatide) is a first-in-class parathyroid hormone (PTH) replacement therapy to treat chronic hypoparathyroidism, a rare and potentially serious condition where the body produces no or abnormally low levels of PTH. It is approved in the European Union for the treatment of adults with chronic hypoparathyroidism.3 A third product – TransCon CNP (navepegritide) – is in development by Ascendis Pharma for the treatment of achondroplasia (ACH), the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, following successful Phase 2 trial results.4 “We are immensely honored to be selected as Ascendis Pharma’s partner in this pivotal agreement for our company,” stated Mert Zorlular, CFO of Er-Kim. He added, “Leveraging our exceptional expertise in rare diseases and our preeminent status in endocrinology throughout the region, we eagerly anticipate collaborating with Ascendis and the healthcare community. Our goal is to swiftly establish and broaden patient access to these innovative treatments in our markets.” “Making a meaningful difference in the lives of patients with rare diseases is a key focus for Ascendis Pharma,” said Camilla Harder Hartvig, Ascendis Pharma’s Executive Vice President and Chief Commercial Officer. “We are therefore very pleased to partner with Er-Kim, a company that shares this commitment, to broaden and accelerate access to our innovative Endocrinology Rare Disease treatment options across Central and Eastern Europe and Turkey.” About Er-Kim Established in 1981, Er-Kim Pharmaceuticals stands at the forefront of biopharmaceutical innovation, partnering with over 40 global leaders to revolutionize patient care in key international markets. Our pioneering business models, tailored for sustainability and flexibility, have positioned us as a full-service solution, extending our reach to over 500 million patients through our fully-owned affiliates. With a dedicated team of over 250 professionals worldwide and revenues exceeding EUR 100M, Er-Kim is not just a partner but a trailblazer in healthcare, continually setting new standards in commercialization and patient access. For more information, please visit . About Ascendis Pharma Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Please visit to learn more. About SKYTROFA (lonapegsomatropin) SKYTROFA™ (lonapegsomatropin, developed as TransCon™ hGH) is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous GH.5 TransCon hGH is approved and marketed as SKYTROFA (lonapegsomatropin-tcgd) in the United States1 and as SKYTROFA (lonapegsomatropin) in the European Union2 as a once-weekly treatment for children and adolescents with GHD. SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve patients and patients switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.1,2 The dose may be adjusted based on the child’s weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).2 TransCon hGH was studied in over 300 children with GHD across the Phase 3 program, which consisted of the Height Trial5 (for treatment-naïve patients), the Flight Trial6 (for treatment-experienced patients), and the Enlighten Trial7 (a long-term extension trial). Patients who completed the Height or Flight Trials were able to continue in Enlighten, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing global Phase 3 Foresight Trial of TransCon hGH in adults with GHD.8 About Paediatric Growth Hormone Deficiency Paediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough GH. Children with GHD are not only characterised by short stature; they also may experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH to improve growth and overall endocrine health. About YORVIPATH (palopegteriparatide) YORVIPATH™ (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug with sustained release of active PTH approved by the European Union as a PTH replacement therapy for the treatment of adults with chronic hypoparathyroidism. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism.3 TransCon PTH met all primary and key secondary endpoints in the Phase 3 Pathway Trial, demonstrating a response rate of 78.7% compared to 4.8% for control (p-value <0.0001) for the primary composite endpoint, and statistically significant improvements compared to control on all key secondary endpoints, which included measures evaluating patient-reported disease symptoms and impacts.9 About Hypoparathyroidism Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium/phosphate balance in the body, acting directly on bone and kidneys and indirectly on intestines. Hypoparathyroidism is considered chronic if it persists >6 months following surgery. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment.9 About TransCon CNP (navepegritide) TransCon™ CNP (navepegritide) is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels, via a single, weekly subcutaneous dose, for the treatment of children with ACH.4 The Phase 2 Accomplish Trial, a randomised, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in prepubertal children with ACH aged 2 to 10 years old, met its primary objectives, and demonstrated that TransCon CNP at 100 µg/kg/week was superior to placebo for the primary efficacy endpoint of annualised growth velocity (AGV) at 52 weeks4. All 57 randomised children completed the blinded portion of Accomplish and are currently continuing in the open label extension at the 100 μg/kg/week dose4. Ascendis Pharma recently confirmed that these 57 clinical trial patients have all completed one year of treatment with TransCon CNP at 100 µg/kg/week, and announced that TransCon CNP is the first investigational product to demonstrate improvements in health-related quality of life and disease impacts in children with ACH.10 About Achondroplasia Achondroplasia is the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, caused by a genetic mutation in the fibroblast growth factor receptor 3 (FGFR3). This leads to an imbalance between the stimulatory and inhibitory signaling pathways involved in regulating bone growth. People living with ACH may experience serious complications and comorbidities due to inhibited skeletal development. Complications may include sleep apnoea and respiratory problems, chronic back and leg pain from lower spine impingement, and sudden infant death from compression of the brain stem. Chronic ear infections due to eustachian tube problems can lead to hearing loss and speech delay. Children with ACH may also experience social and emotional challenges. References SKYTROFA (lonapegsomatropin-tcgd) US Prescribing Information. SKYTROFA (lonapegsomatropin) EU Summary of Product Characteristics. YORVIPATH (palopegteriparatide) EU Summary of Product Characteristics. Savarirayan, R, Hoemschemeyer DG, Ljungberg M, et al., Lancet. 2023;65:1–10. Thornton PS, Maniatis AK, Aghajanova E, et al., J Clin Endocrinol Metab. 2021;106(11):3184–3195. Maniatis AK, Nadgir U, Saenger P, et al., Horm Res Paediatr. 2022;95(3):233–243. Maniatis AK, Casella SJ, Nadgir UM, et al. J Clin Endocrinol Metab. 2022;107(7): e2680–e2689. Foresight Trial (NCT05171855), . Khan AA, Rubin MR, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14–25. Ascendis Pharma A/S, Significant Health and Quality of Life Improvements Achieved in Children with Achondroplasia Treated for One Year with TransCon™ CNP (Navepegritide) at 100 µg/kg/week, Press Release, 20 December 2023, . Media contact: Rosalia Scampoli Marketcom PR Tel: (914) 815-1465 rscampoli@marketcompr.com