Tiemonium Methylsulfate

Fragile X Syndrome typically affects males more severely compared to females and is a significant cause of inherited intellectual disability and autism. Credit: bangoland via Shutterstock. Spinogenix has received approval from the US Food and Drug Administration (FDA) for a Phase II clinical trial of SPG601 for Fragile X Syndrome (FXS), the same week that the European Commission gave orphan drug status to Shionogi’s zatolmilast (BPN14770). FXS is a rare genetic condition that causes a range of developmental problems, including learning disabilities and cognitive impairment. The disorder typically affects males more severely than females and is a significant cause of inherited intellectual disability and autism. These issues arise from the silencing of the Fmr1 gene, leading to impaired BK channel function, which is essential for neuron communication. Spinogenix’s SPG601 is a novel small molecule BK channel activator that works by binding to BK channels and increasing their activation to restore synaptic function. The Phase IIa trial will assess the neurophysiological and clinical impacts of SPG601, a once-a-day pill, in adult men diagnosed with FXS. This trial aims to determine how effective the drug is in altering disease markers and alleviating symptoms compared to a placebo. CEO of Spinogenix Stella Sarraf emphasised the gap in effective and patient-friendly solutions for neurodevelopmental conditions such as FXS: “Like many other conditions, loss of synaptic function remains a key driver of disease, and for that reason, we are excited to launch our first US trial to address this unmet need in FXS.” This development follows the 11 April announcement that the European Commission has granted orphan medicinal product designation for Shionogi’s FXS drug, zatolmilast, a cAMP-specific 3′,5’ cyclic phosphodiesterase 4D inhibitor. Zatolmilast is developed by Tetra Therapeutics, a Shionogi Group company. Shionogi acquired Tetra in 2020 as part of its efforts to develop medications for unmet medical needs. Zatolmilast received orphan drug designation for FXS in the US in September 2023. See Also: FDA grants orphan drug status to NovelMed’s PNH treatment Norgine seeks approval for high-risk neuroblastoma treatment In a Phase II clinical trial assessing zatolmilast, cognitive assessments using the NIH Toolbox revealed significant benefits in oral reading recognition, picture vocabulary, and cognition crystallised composite score (NCT03569631), in males aged 18 to 45 years. The drug is forecast to generate $64m in sales in 2030, according to GlobalData. GlobalData is the parent company of Pharmaceutical Technology. There are currently no approved treatments for FXS, and prescribed treatment consists exclusively of off-label drugs that target individual symptoms of the disease, such as SSRIs for depressive symptoms and anxiety, and stimulants that include methylphenidate for hyperactivity, inattention, and impulsivity. According to a report on the GlobalData Pharma Intelligence Center, the FXS market across two major markets (US and Germany) was valued at $21.9m in 2020 and is projected to grow at a compound annual growth rate of 7.6% reaching $45.8m by 2030.

AMSTERDAM, April 15, 2024 /PRNewswire/ -- Norgine B.V. today announced its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology. Norgine and USWM, LLC (dba US WorldMeds), a Kentucky-based specialty pharmaceutical company, have an exclusive licensing agreement by which Norgine will register and commercialise eflornithine, also referred to as DFMO, in Europe, Australia and New Zealand. On 13 December 2023, the US Food and Drug Administration (FDA) approved eflornithine as the first oral maintenance therapy for high-risk neuroblastoma (HRNB), indicated to reduce the risk of relapse in adult and paediatric patients who have received certain prior therapies.1 The approval decision was based on findings from a trial comparing outcomes from Study 3b (NCT02395666; investigational arm)2,4 and Study ANBL0032 (NCT00026312; clinical-trial-derived external control arm)3,4, where use of eflornithine resulted in improved event-free survival and overall survival when compared to outcomes for patients with high-risk neuroblastoma treated with the standard of care (SoC) without the drug.1 Dr David Gillen, Chief Medical Officer at Norgine, added, "These submissions via Project Orbis represent an important first step in the regulatory process for eflornithine and re-emphasise Norgine's passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need." Follow @norgine Notes to Editors: Project Orbis Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health). HRNB background Children diagnosed with HRNB undergo an intense SoC regimen that still leaves them vulnerable to relapse and death, a risk that is particularly acute during the first two years.5 Approximately 30% of patients who attain remission following upfront therapy will relapse, resulting in a poor prognosis and low likelihood of long-term survival (e.g. estimates as low as 15% of patients will live for five years after relapsing).6 Avoiding relapse is key to long-term survival, yet outside of the United States there are no approved therapies for sustaining remission following SoC treatment. The data with eflornithine demonstrate using it as maintenance therapy extends remission and reduces risk of relapse in patients with HRNB. About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world. NORGINE and the sail logo are trademarks of the Norgine group of companies. References FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma. FDA. News release. 13 December 2023. Available from: FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA Clinical Trial NCT02395666; Preventative trial of difluoromethylornithine (DFMO) in high risk patients with neuroblastoma that is in remission. Available from: Study Details | Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission | ClinicalTrials.gov. Clinical Trial NCT00026312; Isotretinoin with or without dinutuximab, aldesleukin, and sargramostim following stem cell transplant in treating patients with neuroblastoma. Available from: Study Details | Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma | ClinicalTrials.gov Oesterheld J, Ferguson W, Kraveka JM, Bergendahl G, Clinch T, Lorenzi E et al. Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score-Matched Survival Outcome Comparisons. 2024; 42 (1): 90-102. Basta NO, Halliday GC, Makin G, Birch J, Feltbower R, Bown N, et al. Factors associated with recurrence and survival length following relapse in patients with neuroblastoma. Br J Cancer. 2016;115:1048-57. London WB, Bagatell R, Weigel BJ, Fox E, Guo D, Van Ryn C, et al. Historical time to disease progression and progression-free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children's Oncology Group early-phase trials. Cancer. 2017;123:4914-23. Logo -