Otto-von-Guericke-Universität Magdeburg

SAN RAFAEL, Calif., Nov. 16, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive and consistent results from multiple real-world evidence studies of VOXZOGO® (vosoritide) in children with achondroplasia. These results, as well as data from the investigational research program for VOXZOGO in hypochondroplasia, were presented at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Liverpool, England, Nov. 16-18, 2024. "The extensive clinical data supporting VOXZOGO in children with achondroplasia are well-known, and at ESPE, we shared further results in children who greatly benefited from the medicine in a real-world setting," said Klaus Mohnike, M.D., Ph.D., pediatric endocrinologist at Children's Hospital, Otto-von-Guericke-University in Magdeburg, Germany. "Furthermore, the breadth of clinical and real-world evidence underscores why we continue to advocate for treatment with VOXZOGO as early as possible." Real-world longitudinal data from the achondroplasia module of the European CrescNet registry involving 452 children at 30 centers across eight countries showed outcomes of treatment with VOXZOGO were consistent with previously reported clinical trials. The median age at time of enrollment was 6.12 years. Among 143 participants treated with VOXZOGO for 12 months, the average height increase was 6.36 centimeters (cm), with a height Z-score improvement of 0.7 compared to an achondroplasia reference population. For 73 participants treated for 24 months, the average height increase was 11.86 cm, with a height Z-score improvement of 1.15 compared to the same reference population. In addition, in a real-world study in France that included 62 children, results from 17 children above the age of 5 (for whom data is available for 18 months after initial treatment) showed continued efficacy of VOXZOGO. The children who received VOXZOGO experienced an 8.76 cm increase in height, on average, and experienced a mean 0.56 improvement in Z-score compared to an untreated natural history population with achondroplasia and a 0.44 improvement in Z-score compared to a general U.S. population, indicating progress in height compared to the untreated population. The average annualized growth velocity was 5.85 cm/year, representing a substantial improvement in growth-related development over time. There were no discontinuations, and longer-term safety and effectiveness will continue to be monitored. "These real-world data further reinforce the value of VOXZOGO as the first and only approved treatment for children, including infants, with achondroplasia," said Greg Friberg, M.D., executive vice president and chief research & development officer at BioMarin. "We have now collected more than 6,000 patient-years of safety data through our industry-leading CANOPY clinical program, providing the scientific basis for the rapid development of VOXZOGO in new indications such as hypochondroplasia, where we hope to replicate our success in achondroplasia." Key presentations for VOXZOGO in achondroplasia and hypochondroplasia at ESPE, including two oral presentations and four poster presentations, are listed below, with all times in Greenwich Mean Time (GMT): Oral Presentations Phase 2 Trial of Vosoritide Use in Patients with Hypochondroplasia: Pharmacokinetic/Pharmacodynamic Analysis from 12 Month Data Abstract #FC5.5 Saturday, Nov. 16 at 3:35 p.m. Expansion of the CrescNet Registry Achondroplasia Module: Real-World Demographic Data and Outcomes After Up to 2 Years of Vosoritide Treatment Abstract #FC2.6 Saturday, Nov. 16 at 3:45 p.m. Poster Presentations Real-World Effectiveness of Vosoritide in Children with Achondroplasia: Results from 18 Months Follow-Up in France Abstract #P2-56 Patient-Centered Data Collection Provides Comprehensive Insights into Healthcare Resource Use in Achondroplasia: Data From the Pilot Phase of the VIrtual STudy in Achondroplasia (VISTA) Abstract #P1-118 Design and Objectives of Study 111-902: a Multicenter, Prospective and Retrospective Observational Study of Children with Hypochondroplasia Abstract #P3-164 Comparison of the Diagnostic Yield of Whole Exome Sequencing (WES) and Targeted Panel Sequencing for Children with Idiopathic Short Stature (ISS) Abstract #P1-61 About the CANOPY Clinical Program The CANOPY clinical program was designed to evaluate the potential of VOXZOGO (vosoritide) in children with various genetic skeletal conditions, including achondroplasia, hypochondroplasia, Noonan syndrome, SHOX deficiency, Turner syndrome and idiopathic short stature, with the goal of addressing the unmet needs of and expanding treatment options for children and families impacted by these conditions. Studies underway as part of the CANOPY program beyond achondroplasia include: CANOPY HCH-OS, a multinational observational study in children with hypochondroplasia. CANOPY HCH-3, a Phase 3 randomized, placebo-controlled, double-blind multicenter study in children with hypochondroplasia. CANOPY ISS-OS, a multinational observational study in children with idiopathic short stature. CANOPY ISS-2, a Phase 2 randomized, controlled, multicenter study in children with idiopathic short stature. CANOPY NS, TS, SHOX-D-2, a Phase 2 study in multiple genetic skeletal conditions including Noonan syndrome, Turner syndrome and SHOX deficiency. About VOXZOGO In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history. To date, approximately 3,800 infants and children with achondroplasia around the world have received VOXZOGO. Patient Support Accessing VOXZOGO To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected]. For more information about VOXZOGO, please visit . For additional information regarding this product, please contact BioMarin Medical Information at [email protected]. About Achondroplasia Achondroplasia is a rare genetic skeletal condition caused by a variation in the FGFR3 gene. It is characterized by disproportionate short stature and a potentially high burden of complications related to impaired endochondral bone growth. Approximately 80% of children with achondroplasia are born to parents of average stature as a result of a spontaneous variation in the FGFR3 gene. The worldwide incidence of achondroplasia is around one in 25,000 live births. VOXZOGO U.S. Important Safety Information What is VOXZOGO used for? VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO? VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO? The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken? VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO? Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Please see additional safety information in the full Prescribing Information and Patient Information. About BioMarin BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin pursues treatments that offer new possibilities for patients and families around the world navigating rare or difficult-to-treat genetic conditions. To learn more, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data presented at European Society for Paediatric Endocrinology (ESPE) 2024, including the oral and poster presentations; VOXZOGO's efficacy, safety and impact on children with achondroplasia, including the potential benefits of early treatment with VOXZOGO; the potential benefits of VOXZOGO for children with hypochondroplasia; the development of BioMarin's VOXZOGO program generally, including BioMarin's ability to rapidly develop of VOXZOGO in new indications such as hypochondroplasia; and BioMarin's CANOPY clinical program, including BioMarin's plans and expectations for clinical trials for various genetic skeletal conditions, including achondroplasia, hypochondroplasia, Noonan syndrome, SHOX deficiency, Turner syndrome and idiopathic short stature. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned pre-clinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the Food and Drug Administration, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, BioMarin RareConnections® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc. SOURCE BioMarin Pharmaceutical Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Dedicated memory tests on smartphones enable the detection of 'mild cognitive impairment', a condition that may indicate Alzheimer's disease, with high accuracy. Dedicated memory tests on smartphones enable the detection of "mild cognitive impairment," a condition that may indicate Alzheimer's disease, with high accuracy. Researchers from DZNE, the Otto-von-Guericke University Magdeburg and the University of Wisconsin-Madison in the United States who collaborated with the Magdeburg-based company "neotiv" report these findings in the scientific journal npj Digital Medicine. Their study is based on data from 199 older adults. The results underline the potential of mobile apps for Alzheimer's disease research, clinical trials and routine medical care. The app that has been evaluated is now being offered to medical doctors to support the early detection of memory problems. Memory problems are a key symptom of Alzheimer's disease. Not surprisingly, their severity and progression play a central role in the diagnosis of Alzheimer's disease and also in Alzheimer's research. In current clinical practice, memory assessment is performed under the guidance of a medical professional. The individuals being tested have to complete standardized tasks in writing or in conversation: for example, remembering and repeating words, spontaneously naming as many terms as possible on a certain topic or drawing geometric figures according to instructions. All these tests necessarily require professional supervision, otherwise the results are not conclusive. Thus, these tests cannot be completed alone, for example at home. Prof. Emrah Düzel, a senior neuroscientist at DZNE's Magdeburg site and at University Magdeburg as well as entrepreneur in medical technology, advocates a new approach: "It has advantages if you can carry out such tests on your own and only have to visit the doctor's office to evaluate the results. Just as we know it from a long-term ECG, for example. Unsupervised testing would help to detect clinically relevant memory impairment at an earlier stage and track disease progression more closely than is currently possible. In view of recent developments in Alzheimer's therapy and new treatment options, early diagnosis is becoming increasingly important." Comparison between remote at-home and supervised in-clinic testing In addition to his involvement in dementia research, Düzel is also "Chief Medical Officer" of "neotiv," a Magdeburg-based start-up with which the DZNE has been cooperating for several years. The company has developed an app with which memory tests can be carried out autonomously with no need for professional supervision. The software runs on smartphones and tablets, and has been scientifically validated; it is used in Alzheimer's disease research and is now also offered as a digital tool for medical doctors to support the detection of mild cognitive impairment (MCI). Although MCI has little impact on the affected individuals daily living, they have nevertheless an increased risk of developing Alzheimer's dementia within a few years. Dr. David Berron, research group leader at DZNE and also co-founder of neotiv explains: "As part of the validation process, we applied these novel remote and unsupervised assessments as well as an established in-clinic neuropsychological test battery. We found that the novel method is comparable to in-clinic assessments and detects mild cognitive impairment, also known as MCI, with high accuracy. This technology has enormous potential to provide clinicians with information that they cannot obtain during a patient vist to the clinic." These findings have now been published in the scientific journal npj Digital Medicine. Participants from Germany and the USA A total of 199 women and men over the age of 60 participated in the current study. They were located either in Germany or the USA and were each involved in one of two long-term observational studies, both of which address Alzheimer's -- the most common dementia: DZNE's DELCODE study (Longitudinal Cognitive Impairment and Dementia Study) and the WRAP (Wisconsin Registry for Alzheimer's Prevention) study of the University of Wisconsin-Madison. The study sample reflected varying cognitive conditions as they occur in a real world situation: It included individuals who were cognitively healthy, patients with MCI and others with subjectively perceived but not measurable memory problems. The diagnosis was based on established assessments that included e. g. memory and language tasks. In addition, all participants completed multiple memory assessments with the neotiv app over a period of at least six weeks, using their own smartphones or tablets -- and wherever it was convenient for them. "We found that a majority of our WRAP participants were able to complete the unsupervised digital tasks remotely and they were satisfied with the tasks and the digital platform," says Lindsay Clark, PhD, neuropsychologist and lead investigator of the Assessing Memory with Mobile Devices study at the University of Wisconsin-Madison. Remembering images and detecting differences "Assessments with the neotiv app are interactive and comprise three types of memory tasks. These address different areas of the brain that can be affected by Alzheimer's disease in different disease stages. Many years of research have gone into this," Düzel explains. Essentially, these tests involve remembering images or recognizing differences between images that are presented by the app. Using a specially developed score, the German-US research team was able to compare the results of the app with the findings of the established in-clinic assessments. "Our study shows that memory complaints can be meaningfully assessed using this digital, remote and unsupervised approach," says Düzel. "If the results from the digital assessment indicate that there is memory impairment typical of MCI, this paves the way for further clinical examinations. If test results indicate that memory is within the age-specific normal range, individuals can be given an all-clear signal for the time being. And for Alzheimer's disease research, this approach provides a digital cognitive assessment tool that can be used in clinical studies -- as is already being done in Germany, the USA, Sweden and other countries." Outlook Further studies are in preparation or already underway. The novel memory assessment is to be tested on even larger study groups, and the researchers also intend to investigate whether it can be used to track the progression of Alzheimer's disease over a longer period of time. Berron: "Information about how quickly memory declines over time is important for medical doctors and patients. It is also important for clinical trials as new treatments aim to slow the rate of cognitive decline." The cognitive neuroscientist describes the challenges: "To advance such self-tests, a patient's clinical data must be linked to self-tests outside the clinic, in the real-world. This is no easy task, but as our current study shows, we are making progress as a field."

FRIDAY, Jan. 26, 2024 -- For patients with low-risk Staphylococcus aureus ( S. aureus ) bloodstream infection, early switch to oral antimicrobial therapy is noninferior to intravenous standard therapy, according to a study published online Jan. 17 in The Lancet Infectious Diseases . Achim J. Kaasch, M.D., from Otto von Guericke University Magdeburg in Germany, and colleagues conducted an international, open-label, randomized, noninferiority trial conducted in 31 tertiary care hospitals involving patients with low-risk S. aureus bloodstream infection. After five to seven days of intravenous antimicrobial therapy, participants were randomly assigned to oral antimicrobial therapy or to continue intravenous standard therapy (108 and 105, respectively). The composite primary end point was the occurrence of any complication related to S. aureus bloodstream infection within 90 days. The researchers found that the primary end point was met by 13 and 12 percent of participants in the oral switch and intravenous groups, with a treatment difference of 0.7 percentage points (95 percent confidence interval, −7.8 to 9.1; P = 0.013, which met noninferiority). Within the safety population, 34 and 26 percent of participants in the oral switch and intravenous groups, respectively, had at least one serious adverse event. "This study supports an early switch to oral antimicrobial therapy in patients with low-risk S. aureus bloodstream infection provided a rigorous clinical assessment and close monitoring for complications are done," the authors write. Several authors disclosed ties to the pharmaceutical industry. Abstract/Full Text (subscription or payment may be required) Editorial (subscription or payment may be required) Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions. Posted January 2024 More news resources FDA Medwatch Drug Alerts Daily MedNews News for Health Professionals New Drug Approvals New Drug Applications Drug Shortages Clinical Trial Results Generic Drug Approvals Subscribe to our newsletter Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.