Pseudoxanthoma Elasticum

- Positive interim results in infants and young children with ENPP1 Deficiency showed improvements from baseline in multiple measures of disease, including survival, heart function, and stabilization or reduction in ectopic calcification and hypophosphatemia, with no radiographic evidence of rickets – - Enrollment complete in ENERGY 3 pivotal trial in pediatric patients with ENPP1 Deficiency; on track to complete dosing in January 2026, with topline data to follow in early 2026 – - Regulatory guidance from FDA and EMA supports planned ASPIRE pivotal trial focused on addressing severe complications of ABCC6 Deficiency in children – Jan. 10, 2025 -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, today announced positive interim data from its ENERGY 1 trial and Expanded Access Program (EAP) evaluating INZ-701 in infants and young children with ENPP1 Deficiency, completion of enrollment in the ENERGY 3 pivotal trial in pediatric patients with ENPP1 Deficiency and regulatory guidance for the ASPIRE pivotal trial in children with ABCC6 Deficiency. “We believe these highly encouraging outcomes in infants and young children, combined with previously reported data from adult studies, provide strong support for the potential impact of INZ-701 on rickets, a key clinical endpoint in the ongoing pivotal ENERGY 3 trial, and underscore its potential to address the significant needs of pediatric patients,” said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma. Matt Winton, Ph.D., Senior Vice President and COO of Inozyme Pharma added, “Our team and global collaborators worked tirelessly to identify and diagnose these rare patients and initiate treatment as quickly as possible. Tragically, in some cases, we have been unable to begin treatment before the infant passed. This only deepens our commitment to the patient community and strengthens our resolve to address unmet needs across all populations as we advance INZ-701.” Interim data from the ENERGY 1 trial (three infants) and the EAP (two infants and one child -2.5 years old) evaluated patients with generalized arterial calcification of infancy (GACI), a severe manifestation of ENPP1 Deficiency. Patients were treated with INZ-701 for periods of three weeks to 22 months. The data presentation can be accessed here on Inozyme’s Investor Relations site. Key results include: Improved Survival: 80% of infants treated with INZ-701 survived beyond their first year, compared to a historical survival rate of approximately 50%. Reduction in Arterial Calcifications: Substantial reductions or stabilization of arterial calcifications were observed in all surviving patients, including complete resolution in some instances. There was no evidence of progression of arterial calcification in any patient. Improved Heart Function: Stabilization or improvement in left ventricular ejection fraction (LVEF) was noted in all surviving patients. Reduced Risk of Rickets: No radiographic evidence of rickets was observed in patients evaluated beyond one year of age and at-risk of rickets development (n=3), supported by stabilization or increases in serum phosphate levels. Favorable Safety Profile: INZ-701 was well-tolerated, with no serious treatment-related adverse events in infants and young children. Observed treatment-related events were limited to mild injection site reactions. Across studies to-date low, often transient, anti-drug antibody (ADA) levels were noted in some children and adults, with no impact on pharmacokinetics (PK) or pharmacodynamics (PD). In the ENERGY 1 trial and EAP, higher ADA levels in some infants significantly affected PK and PD. In infants with high ADA levels, data collected pre- and post-dosing demonstrated substantial transient increases in PPi and drug exposure following INZ-701 administration, consistent with the clinical effects observed. ADAs were not associated with adverse events in any patient. The Company today announced completion of enrollment in its ENERGY 3 pivotal trial of INZ-701 in patients with ENPP1 Deficiency aged >1 to With 25 patients enrolled, the trial’s 2:1 randomized design provides >90% power to detect meaningful differences in RGI-C between treatment and control groups. Strong patient interest and scheduled screenings may result in the enrollment of additional participants in January 2025. Inozyme anticipates completing the one-year dosing period for all patients by January 2026, with topline data expected in early 2026. Regulatory Progress for ASPIRE Pivotal Trial in Children with ABCC6 Deficiency: Preliminary Support from U.S. and EU Regulators Inozyme is advancing the development of INZ-701 in ABCC6 Deficiency. In April 2024, the Company reported topline data from an open-label, dose-escalation study in adults, along with findings from a natural history study documenting the significant disease burden in patients with the early-onset form of the disease, known as GACI Type 2 (GACI-2). The adult study demonstrated positive improvements in vascular and retinal pathology after 48 weeks of treatment with INZ-701, as well as normalization of PPi levels at the highest dose tested, supporting further development in additional age groups. The natural history study revealed a high disease burden characterized by childhood strokes, arteriopathy, cardiovascular complications, and early mortality. Further research has identified a substantial pediatric population with ABCC6 Deficiency, underscoring the significant unmet medical need in this group. The natural history study data, supplemented by literature reports, has informed the design of the Company’s planned randomized, controlled ASPIRE trial of INZ-701 in children with ABCC6 Deficiency. The proposed primary endpoint, comprising major adverse clinical events over a two-year treatment period, has been reviewed and received preliminary support from U.S. and EU regulators. The trial is expected to enroll approximately 70 patients from infancy up to The Company plans to continue regulatory engagement over the coming months to finalize the trial protocol. Pending ongoing regulatory review and the availability of financial resources, Inozyme aims to initiate the ASPIRE trial in early 2026. ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Children with this condition typically develop rickets, specifically autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia, or softened bones. ARHR2 and osteomalacia cause pain and difficulty with movement. Additionally, patients may experience hearing loss, calcification in arteries and joints, and heart problems. Biallelic ENPP1 Deficiency affects approximately 1 in 64,000 pregnancies worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ENPP1 Deficiency. ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. Pediatric patients who survive beyond the first year of life may develop neurological disease, including strokes, and cardiovascular diseases due to ongoing vascular calcification and stenosis. In older individuals, ABCC6 Deficiency manifests as pseudoxanthoma elasticum (PXE), characterized by abnormal mineralization in blood vessels and soft tissues, affecting the skin, visual function, and vascular system. Biallelic ABCC6 Deficiency is estimated to affect 1 in 25,000 to 1 in 50,000 individuals worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many people with just one copy of the mutated gene (monoallelic ABCC6 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ABCC6 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ABCC6 Deficiency. Inozyme Pharma is a pioneering clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. We are experts in the PPi-Adenosine Pathway, where the ENPP1 enzyme generates inorganic pyrophosphate (PPi), which regulates mineralization, and adenosine, which controls intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurological conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL). Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these molecules. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases. The content above comes from the network. if any infringement, please contact us to modify.

- Data underscore the urgent need for therapies that address the cardiovascular and musculoskeletal complications and long-term systemic effects of ENPP1 and ABCC6 Deficiencies in children - - Inozyme and GACI Global launched PROPEL Registry to further understanding of these rare, life-threatening diseases (NCT06302439) - BOSTON, Sept. 26, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, is presenting four posters at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting, including new data that demonstrate the progression and impact of rare bone and blood vessel diseases in children. These presentations include findings highlighting the significant medical burden associated with ENPP1 Deficiency and early-onset ABCC6 Deficiency, new data from a natural history study that provides critical insights into the progression of ENPP1 Deficiency, and the launch of a global registry to enhance our understanding of these diseases. “Given their high mortality rate, there is an urgent unmet need for patients affected by these rare bone and blood vessel diseases. For instance, more than half of all infants with ENPP1 Deficiency die within six months, and those that survive often face major and potentially fatal complications throughout their lives,” said Kurt Gunter, M.D., Chief Medical Officer of Inozyme Pharma. “These new data underscore the significant and multifaceted impact of ENPP1 and ABCC6 Deficiencies, further highlighting the significant need for therapies that address the immediate and long-term systemic complications associated with these conditions. At Inozyme, we are dedicated to unlocking the full potential of our lead candidate, INZ-701, a potentially transformative treatment for patients and their families facing these diseases.” “As the data from these retrospective analyses demonstrates, the impact of these life-threatening conditions is more significant in both the short and long-term than we had understood, particularly in terms of cardiovascular and musculoskeletal complications,” said Christine O’Brien, co-president of GACI Global. “We’re excited to work with Inozyme to launch the PROPEL Registry to prospectively collect clinical data and patient-reported outcomes that will provide a comprehensive understanding of the burden of illness and progressive nature of ENPP1 Deficiency and early-onset ABCC6 Deficiency.” Natural History Study of ENPP1 Deficiency ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Key findings of the retrospective analysis included: In the 84 patients with ENPP1 Deficiency, cardiovascular complications and arterial calcifications developed early in life, predominately within the first few months.A striking 88% of patients experienced ectopic calcifications, with 81% showing arterial calcification and 66.7% showing aortic calcification. Cardiovascular complications, including hypertension, heart valve defects and heart failure, were noted in 76% of patients.Among the 84-patient cohort, 51 were diagnosed with Generalized Arterial Calcification of Infancy (GACI) alone and only 19 survived beyond infancy; 22 were diagnosed with GACI, survived beyond infancy and progressed to Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2); and 11 patients presented initially with ARHR2.Musculoskeletal complications, including phosphate-wasting rickets, bowed extremities, and pain, were documented in all patients diagnosed with ARHR2, with 45% of ENPP1 Deficiency survivors showing symptoms by age 3 and 75% by age 11.The data highlight that 95% of surviving ENPP1 Deficiency patients are expected to experience cardiovascular, musculoskeletal, and other organ complications, underscoring the lifelong burden of the disease. Title: Lifetime Accumulation of Calcification and Clinical Complications in Patients with ENPP1 Deficiency (GACI and ARHR2)Format: Poster PresentationAuthor/Presenter: Carlos Ferreira, M.D., of the National Institutes of Health (NIH)Session Dates and Times: Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET Early Career Spotlight: Friday, September 27, 2024, 4:54 p.m. ET Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Retrospective Observational Study of ENPP1 Deficiency and Early-onset ABCC6 Deficiency Like ENPP1 Deficiency, ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. This study described the clinical events and interventions in patients with ENPP1 Deficiency (n=14) and early-onset ABCC6 Deficiency (n=9).ENPP1 Deficiency was associated with a high burden of cardiovascular complications in infancy, with additional frequent medical interventions including orthopedic surgery (64%) and hearing aids (43%).Early-onset ABCC6 Deficiency patients showed significant cardiovascular complications, and 44% experienced stroke. Title: The Medical Burden of ENPP1 Deficiency and Early-Onset ABCC6 Deficiency from a Retrospective Observational StudyFormat: Poster PresentationAuthor/Presenter: David R. Weber, M.D., MSCE, Attending Physician and Medical Director of the Center for Bone Health at Children’s Hospital of PhiladelphiaSession Dates and Times: Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Additional poster presentations include: Title: PROPEL: Prospective Observational Registry for ENPP1 Deficiency and Infantile-Onset ABCC6 DeficiencyFormat: Poster PresentationAuthor/Presenter: Lothar Seefried, M.D., of the Orthopedic Department of the University Hospital at the Julius-Maximilians University Würzburg, GermanySession Dates and Times: Energy Metabolism in Skeletal Development and Disease: Thursday, September 26, 2024, 8:30 a.m. – 5:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Title: Therapeutic Application of ENPP1-Fc Prevent Muscle Calcification Following Cardiotoxin-induced-muscle Damage in Abcc6 -/- miceFormat: Poster PresentationAuthor/Presenter: Kevin O’Brien, Principal Scientist, Inozyme PharmaSession Date and Time: Late Breaking Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET About ENPP1 Deficiency ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Children with this condition typically develop rickets, specifically autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia, or softened bones. ARHR2 and osteomalacia cause pain and difficulty with movement. Additionally, patients may experience hearing loss, calcification in arteries and joints, and heart problems. Biallelic ENPP1 Deficiency affects approximately 1 in 64,000 pregnancies worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ENPP1 Deficiency. About ABCC6 Deficiency ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. Pediatric patients who survive beyond the first year of life may develop neurological disease, including strokes, and cardiovascular diseases due to ongoing vascular calcification and stenosis. In older individuals, ABCC6 Deficiency manifests as pseudoxanthoma elasticum (PXE), characterized by abnormal mineralization in blood vessels and soft tissues, affecting the skin, visual function, and vascular system. Biallelic ABCC6 Deficiency is estimated to affect 1 in 25,000 to 1 in 50,000 individuals worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many people with just one copy of the mutated gene (monoallelic ABCC6 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ABCC6 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ABCC6 Deficiency. About Inozyme PharmaInozyme Pharma is a pioneering clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. We are experts in the PPi-Adenosine Pathway, where the ENPP1 enzyme generates inorganic pyrophosphate (PPi), which regulates mineralization, and adenosine, which controls intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurological conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL). Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these molecules. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases. For more information, please visit https://www.inozyme.com/ or follow Inozyme on LinkedIn, X, and Facebook. Cautionary Note Regarding Forward-Looking StatementsStatements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential benefits of INZ-701. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to conduct its ongoing clinical trials of INZ-701 for ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis; enroll patients in ongoing and planned trials; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain, and protect intellectual property rights related to its product candidates; manage expenses; comply with covenants under its outstanding loan agreement; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. Contacts Investors:Inozyme PharmaStefan Riley, Senior Director of IR and Corporate Communications(857) 330-8871Stefan.riley@inozyme.com Media:Biongage CommunicationsTodd Cooper(617) 840-1637Todd@biongage.com

All html pdf doc xls ppt match recent 10 20 30 none small medium large Home About Chugai News Chugai Files in Japan for Additional Indication of Vabysmo for Angioid Streaks, a Leading Cause of Vision Loss The filing is based on the results from a Japanese phase III study for angioid streaks associated with neovascularization, a rare disease with a poor prognosis The application will be reviewed under priority review in Japan If approved, Vabysmo will be the first product for angioid streaks in Japan TOKYO, September 6, 2024 -- Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) announced today that it filed for an additional indication with the Ministry of Health, Labour and Welfare (MHLW) for anti-VEGF/anti-Ang-2 bispecific antibody Vabysmo® Intravitreal Injection 120 mg/mL [generic name: faricimab (genetical recombination)] (Hereafter Vabysmo), for the treatment of angioid streaks associated with neovascularization. For this indication, Vabysmo received orphan drug designation from the MHLW, and the applications will be reviewed under priority review. “Angioid streaks is a rare disease with no approved products in Japan. We will continue to work for approval as soon as possible so that Vabysmo, which demonstrated vision improvement in a Japanese phase III study, can contribute to the treatment of patients as a new option for angioid streaks,” said Chugai’s President and CEO, Dr. Osamu Okuda. This application is based on the results of a Japanese phase III study, the NIHONBASHI study for angioid streaks associated with neovascularization. [Reference] ・NIHONBASHI study Positive Phase III Results Showed Vabysmo Improved Vision for the First Time in Japanese in Angioid Streaks (Press release by Chugai issued on April 15, 2024) https://www.chugai-pharm.co.jp/english/news/detail/20240415113000_1064.html About angioid streaks Angioid streaks is a disease characterized by cracks in parts of the retina, causing-discoloration (pigmented streak) of the fundus. The disease is often asymptomatic, and when choroidal neovascularization extends to the macula region of the fundus, it causes symptoms such as decreased or distorted vision. People with neovascularization have a poor prognosis, but conventional treatments such as surgery and laser are not sufficiently effective, and new treatment options are needed. The number of patients with angioid streaks in Japan is unknown, but approximately 300 patients have pseudoxanthoma elasticum (one of the designated intractable diseases), which is known to be associated with angioid streaks in approximately half of patients.1,2 About NIHONBASHI study NIHONBASHI study is a multicenter, single-arm phase III study in Japan evaluating the efficacy and safety of Vabysmo for the people with angioid streaks associated with neovascularization. 24 people were enrolled into this study. The primary endpoint is the change in best-corrected visual acuity (BCVA) score (the best distance vision a person can achieve – including with correction such as glasses – when reading letters on an eye chart) from baseline at 12 weeks. Secondary endpoints include the mean change in BCVA score at 52 weeks, change in central subfield thickness from baseline over time, and safety. About Vabysmo Vabysmo is the first bispecific antibody approved for the eye. 3,4 It targets and inhibits two signaling pathways linked to a number of vision-threatening retinal conditions by neutralizing angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). Ang-2 and VEGF-A contribute to vision loss by destabilizing blood vessels, causing new leaky blood vessels to form and increasing inflammation. By blocking pathways involving Ang-2 and VEGFA, Vabysmo is designed to stabilize blood vessels. 4,5 Vabysmo is approved in nearly 100 countries around the world, including the United States (US), Japan, the United Kingdom and the European Union, for people living with neovascular or ‘wet’ age-related macular degeneration and diabetic macular edema, and in several countries, including the US, EU and Japan, for the treatment of macular edema following retinal vein occlusion. Review by other health authorities is ongoing. 3,6 Trademarks used or mentioned in this release are protected by law. Sources Chatziralli I, Saitakis G, Dimitriou E, Chatzirallis A, Stoungioti S, Theodossiadis G, et al. ANGIOID STREAKS: A Comprehensive Review From Pathophysiology to Treatment. Retina. 2019;39(1):1-11. Japan Intractable Diseases Information Center. Pseudoxanthoma elasticum (designated intractable disease 166) [Internet; cited September 2024]. Available from: https://www.nanbyou.or.jp/. (Japanese only) United States Food and Drug Administration (U.S. FDA). Highlights of prescribing information, Vabysmo. 2022 [Internet; cited September 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761235s000lbl.pdf. Heier JS, et al. Efficacy, durability, and safety of intravitreal faricimab up to every 16 weeks for nAMD (TENAYA and LUCERNE): two randomised, double-masked, Phase III, non-inferiority trials. The Lancet. 2022; 399:729-40. Wykoff C, et al. Efficacy, durability, and safety of intravitreal faricimab with extended dosing up to every 16 weeks in patients with DME (YOSEMITE and RHINE): two randomised, double-masked, Phase III trials. The Lancet. 2022; 399:741-755. Roche data on file. [PDF 252KB] Contact: For Media Chugai Pharmaceutical Co., Ltd. Media Relations Group, Corporate Communications Dept., Hideki Sato Tel: +81-3-3273-0881 E-mail: pr@chugai-pharm.co.jp For Investors Chugai Pharmaceutical Co., Ltd. Investor Relations Group, Corporate Communications Dept., Takayuki Sakurai Tel: +81-3-3273-0554 E-mail: ir@chugai-pharm.co.jp Back Home About Chugai News Chugai Files in Japan for Additional Indication of Vabysmo for Angioid Streaks, a Leading Cause of Vision Loss About Chugai Message from the CEO Chugai’s Five Strengths Company Outline Our Vision Our Strategy History of Chugai Pharmaceutical Chugai Group R&D Digital Transformation "CHUGAI DIGITAL" Videos & Advertisements Press Room News Media Conference Partnering To our suppliers Investor Relations Management Policy Financial Results Reports & Downloads Shareholder Information Corporate Governance ESG at Chugai Main Products / Development Pipeline See how Chugai compares to other companies Strategic Alliance with Roche Chugai’s Five Strengths Events & Presentations Contacts & Information FAQs Recruitment Chugai Recruiting Site Sustainability Basic Policy (Creation of Shared Value) Sustainability and Business Strategy Sustainable Patient-Centric Healthcare Global Health Human Resources and Diversity & Inclusion Ethics and Compliance Supply Chain Management Human Rights Social Contribution Global Environment Health and Productivity Management Governance External Evaluations Activity Reports Data Chugai's Transparency Guidelines You are now moving to an external website. Access the link Our use of cookiesWe use cookies to improve your experience on our website (www.chugai-pharm.co.jp), to provide social media features and to analyze our traffic. By clicking “Accept Cookies”, you consent to the use of all cookies. For more information, configuration and to withdraw your consent, please click “Cookie Settings”. You can manage the settings at any time from the “Cookie Policy” link at the bottom of each page.Cookie Policy Privacy Preference CenterWhen you visit any website, it may store or retrieve information on your browser, mostly in the form of cookies. This information might be about you, your preferences or your device and is mostly used to make the site work as you expect it to. The information does not usually directly identify you, but it can give you a more personalized web experience. Because we respect your right to privacy, you can choose not to allow some types of cookies. Click on the different category headings to find out more and change our default settings. However, blocking some types of cookies may impact your experience of the site and the services we are able to offer. More information Manage Consent PreferencesStrictly Necessary CookiesAlways ActiveThese cookies are necessary for the website to function and cannot be switched off in our systems. They are usually only set in response to actions made by you which amount to a request for services, such as setting your privacy preferences, logging in or filling in forms. You can set your browser to block or alert you about these cookies, but some parts of the site will not then work. These cookies do not store any personally identifiable information. Performance Cookies Performance Cookies These cookies allow us to count visits and traffic sources so we can measure and improve the performance of our site. They help us to know which pages are the most and least popular and see how visitors move around the site. All information these cookies collect is aggregated and therefore anonymous. If you do not allow these cookies we will not know when you have visited our site, and will not be able to monitor its performance. Functional Cookies Functional Cookies These cookies enable the website to provide enhanced functionality and personalisation. They may be set by us or by third party providers whose services we have added to our pages. If you do not allow these cookies then some or all of these services may not function properly. Targeting Cookies Targeting Cookies These cookies may be set through our site by our advertising partners. They may be used by those companies to build a profile of your interests and show you relevant adverts on other sites. They do not store directly personal information, but are based on uniquely identifying your browser and internet device. If you do not allow these cookies, you will experience less targeted advertising. Cookie List checkbox label label Consent Leg.Interest checkbox label label checkbox label label checkbox label label