bioSeedin’s Post

Recent update of the European Medicines Agency's Agency's critical medicines list emphasises the importance of plasma-derived therapies for EU healthcare systems and patients. This makes plasma donation and collection even more crucial for the well-being of European patients. How can industry gain visibility into the medicine supply chain and avoid future crises? Governmental experts will dive into these topics at the #IPPC2024 next month: 👉 Cemre Balaban, Junior Trade Policy Analyst, Trade Policy Division, Trade and Agriculture Directorate - OECD - OCDE 👉 Klaus Kruttwig Medicines and Medical Devices Shortages Specialist - European Medicines Agency 👉 Stelios Kympouropoulos, Member of European Parliament, European Parliament 👉 Domenico Di Giorgio, Head of Inspection and Certification Department and of the Pharmaceutical Crime Counteracting -Office - AIFA Agenzia Italiana del farmaco   In the coming months, as part of the Critical Medicines Alliance, PPTA looks forward to collaborate and share our expertise, scientific evidence, and experience to strengthen the frameworks of sustainable and resilient supply chains for plasma-derived medicines. #IPPC2024 https://lnkd.in/gRma-WAB

Island Pharmaceuticals is pleased to release the September 2024 Quarterly Activity Report.   Island had an active first quarter of this financial year, achieving milestones including signing a binding Letter of Intent for the potential acquisition of antiviral molecule galidesivir, the appointment of Chris Ntoumenopoulos to the Island board as a Non-Executive Director, and clinical update of the Phase 2a/b PROTECT challenge study in dengue disease. These events are followed by significant post quarter activities, including the commencement of the ISLA-101 Phase 2a/b clinical trial and an A$3.5m Placement being secured to support Island’s clinical programs. Data readout of the Phase 2a cohort can be expected by the end of this calendar year.   Commenting on this quarter, Island CEO Dr David Foster said: “This quarter marked a breakthrough for Island. After submitting our amended protocol to the US FDA for ISLA-101, we were able to commence a Phase 2a/b trial, incorporating both prophylactic and therapeutic arms across two cohorts. We were delighted to welcome Chris Ntoumenopoulos to the Board this quarter. In his short time with the Company, Chris has proven to be a valuable asset, bringing fresh insights and a proactive approach that have positively influenced our direction and decision-making.”   Access the full Activities Report via this link: https://shorturl.at/lbFL5   #mosquitobornedisease #antiviraltherapeutics #ILA #biotech

📢 * JUST ANNOUNCED * Agios Pharmaceuticals has announced the successful completion of #patientenrollment in its Phase 3 RISE UP #clinicalstudy, evaluating the investigational oral medication #mitapivat for its effectiveness and safety in treating #sicklecell disease in individuals aged 16 and older. The Phase 3 RISE UP study trial has enrolled over 200 #patients in total. The trial's primary endpoints include haemoglobin response and the annualized rate of sickle cell pain crises, which are significant indicators of the disease's impact on patient #QualityOfLife. The company's Chief Medical Officer, Dr. Sarah Gheuens, expressed gratitude to patients, investigators, and advocates for their role in achieving full enrollment. The company anticipates reporting the topline results from this comprehensive 52-week study in late 2025. It is also noteworthy that mitapivat has shown promising results in two Phase III studies for transfusion-dependent #thalassemia (#TDT) and non-transfusion-dependent thalassemia (#NTDT), thus Agios is actively planning to submit a #MarketingApplication for mitapivat targeting alpha- or beta-thalassaemia patients to regulatory authorities in the #US and #EU by the end of 2024. https://lnkd.in/dxqGdxXp...

Attralus Receives European Medicines Agency Committee for Orphan Medicinal Products (COMP) Positive Opinions for AT-02 for the Treatment of Both ATTR and AL Amyloidosis ✳ Read the press release - https://lnkd.in/eXuMjqUd.   ✳ AT-02 has been granted COMP positive opinions for orphan medicinal product designations in the EU for both ATTR and AL amyloidosis. ✳ AT-02, the company’s lead pan-amyloid removal therapeutic candidate, is currently being evaluated in a Phase 1 three-part trial and a Phase 2 open label extension trial in ATTR and AL amyloidosis patients to support its use as an immunotherapy in patients with systemic amyloidosis. #amyloidosis #rarediseases

Island Pharmaceuticals is pleased to report highly positive pharmacokinetic (PK) data for ISLA-101 from its 24-subject Single Ascending Dose clinical study. The new data shows that the required levels of ISLA-101 concentration in the blood were observed after only a single dose. PK data is critical for determining how the drug acts in the body and for establishing appropriate dosing regimens for Island's planned Phase 2a clinical trial. CEO Dr David Foster said, “This new pharmacokinetic data reinforces our strong confidence in ISLA-101. The data has shown us that even following a single dose, we are achieving blood concentrations that have previously been shown to be effective against dengue fever infections. This is exactly the outcome we hoped for - it provides a critical datapoint to underpin our dosing regimen as we prepare for the Phase 2 clinical study, which will include dosing for multiple days.” For further information, refer to the full ASX Announcement here: https://shorturl.at/nqyO8 #ILA #DengueFever #MosquitoBorneViruses

Seeking Participants for a New CMT Clinical Trial (for the US only):  A Chance to Help Advance Treatment! The sponsor of the study is NMD Pharma A/S. If you’re living with Charcot-Marie-Tooth (CMT) disease, specifically CMT1 or CMT2 subtypes, you may be eligible to participate in an exciting new clinical trial.  NMD Pharma, a biotechnology company, has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with the SYNAPSE-CMT Phase 2 trial. This trial will evaluate the safety, tolerability, and potential effectiveness of an investigational medication, NMD670, which could improve muscle strength and endurance in individuals with CMT. What’s the Goal of the Study? NMD670 may enhance communication between nerves and muscles where transmission is impaired, a common issue for people living with CMT. The hope is that this treatment will improve your strength, endurance, and overall daily functioning. By joining the study, you’ll play a critical role in determining whether this therapy could help others with CMT in the future. Learn more or to participate:  https://lnkd.in/eYSKTrKa https://lnkd.in/eHsEZ6MP #NMDpharma #cmtawareness #hnf4cmt #clinicaltrials #cmtresearch #charcotmarietooth #cmtneuropathy #cmtgene #charcotmarietoothdisorder #fda

Join us later this week in Florence at the Advanced Technologies & Treatments for Diabetes (ATTD) session to discuss the potential, concerns and what to expect from AI Driven Clinical Decision Support Systems for the management of #diabetes European Association for the Study of Diabetes e.V. (EASD) INTERNATIONAL SOCIETY FOR PEDIATRIC AND ADOLESCENT DIABETES (ISPAD) International Diabetes Federation Europe EFPIA - European Federation of Pharmaceutical Industries and Associations MedTech Europe PCDE JDRF International Société Francophone du Diabète (SFD) Tadej Battelino Stefano Del Prato Lutz, Prof. Dr. Heinemann Mia Bajramagic

📉 Impact ⤵ Ascendis Pharma’s long-awaited FDA approval of #Yorvipath brings much-needed relief to patients with #hypoparathyroidism, offering a new treatment option as Takeda’s Natpara is set to be discontinued. This approval positions Ascendis at the forefront of hypoparathyroidism care, but reimbursement challenges may influence its adoption in the U.S. 💡 Insight ⤵ Yorvipath’s approval highlights the importance of innovative drug development for rare diseases. With its TransCon technology, Ascendis is addressing critical gaps in treatment by providing a therapy that not only regulates hormone levels but also improves patient quality of life. However, securing reimbursement and navigating the transition from existing therapies will be key to its success. ❓ Questions to Think About ⤵ How will Ascendis overcome potential reimbursement hurdles for Yorvipath in the U.S.? What impact will this new therapy have on the broader landscape of hypoparathyroidism treatment? How might this approval influence future developments in rare disease therapies? #PharmaNews #RareDisease #Hypoparathyroidism #AscendisPharma #FDAApproval #BiotechInnovation #Endocrinology #HealthcareInnovation #PatientCare

🚀 Exciting News for Advanced Therapy Medicinal Products (ATMPs) Enthusiasts! On 30 December 2008, the Advanced Therapy Medicinal Products Regulation (EC No 1394/2007) came into effect, marking the beginning of a new era for ATMPs. Since then, these therapies have grown significantly, offering revolutionary treatments for unmet medical needs. To celebrate 15 years of progress, the European Medicines Agency (EMA) is hosting a symposium on 10 October 2024, at their headquarters in Amsterdam (also available remotely). 💡 This event will highlight: How the Committee for Advanced Therapies (CAT) has shaped the regulatory framework for ATMPs in Europe. The approval of 27 groundbreaking gene- and cell/tissue-based medicines that are now offering transformative treatment options for conditions previously considered untreatable. These therapies address critical unmet medical needs, providing hope for patients with rare and serious diseases. 👥 Who should attend? CAT members EMA committees & regulatory authority members Industry & academia representatives Healthcare professionals & patients Policymakers Anyone interested in the future of ATMPs 🗓️ Important Dates: In-person registration deadline: 24 September 2024 Remote attendance registration deadline: 1 October 2024 Register your interest and join the conversation on the future of ATMPs! 🎯 #ATMP #RegulatoryAffairs #InnovativeTherapies #EMA #Healthcare #AdvancedTherapies

Henlius and Organon have announced that the European Medicines Agency (EMA) has validated the marketing authorization applications (MAAs) for HLX14, an investigational biosimilar for Prolia and Xgeva (denosumab). This biosimilar aims to provide an alternative treatment for osteoporosis in postmenopausal women at high risk for fractures and for preventing skeletal-related events in patients with bone metastases. #femtech #womenshealth #womenshealthinnovation #health #healthcare #healthtech #pharma #biotech #regulatory #commercialization #bonehealth #osteopenia #osteoporosis #menopause