Idorsia Ltd
🇨🇭Switzerland
FDA Approves First TAVR Therapy for Asymptomatic Severe Aortic Stenosis Patients
• Edwards Lifesciences' SAPIEN 3 platform has received FDA approval for treating asymptomatic patients with severe aortic stenosis, marking the first such approval for transcatheter aortic valve replacement in this patient population.
• The groundbreaking EARLY TAVR trial demonstrated superior outcomes with TAVR compared to watchful waiting, with 26.8% versus 45.3% of patients experiencing death, stroke, or unplanned cardiovascular hospitalization over a median 3.8-year follow-up.
• This approval challenges the current "watchful waiting" approach to asymptomatic severe aortic stenosis, potentially transforming treatment pathways as symptoms can develop suddenly and unpredictably in these patients.
Aprocitentan Shows Significant Blood Pressure and Proteinuria Reduction in Black Patients with Resistant Hypertension
• Aprocitentan, a dual endothelin receptor antagonist, demonstrated clinically meaningful and sustained blood pressure reductions in Black patients with resistant hypertension when added to existing antihypertensive regimens.
• The drug significantly reduced proteinuria by approximately 65% at the 25mg dose in patients with micro- or macroalbuminuria, addressing a critical need in a population disproportionately affected by hypertension-related complications.
• Published in Hypertension, the PRECISION study subgroup analysis highlights aprocitentan as the first approved treatment targeting the endothelin system, potentially preventing thousands of cardiovascular events in Black patients who often struggle to achieve blood pressure control.
FDA Removes REMS Requirement for TRYVIO, Simplifying Access to Novel Hypertension Treatment
• The US FDA has eliminated the Risk Evaluation and Mitigation Strategy (REMS) requirement for TRYVIO (aprocitentan), determining that standard labeling is sufficient to communicate safety information.
• TRYVIO, a dual endothelin receptor antagonist, is the first new antihypertensive medication with a novel mechanism of action in over 40 years, capable of reducing systolic blood pressure by more than 15 mmHg.
• The removal of REMS requirements simplifies prescribing for healthcare providers and improves access for patients with difficult-to-control hypertension, including those with chronic renal failure.
Hypersomnia Pipeline Shows Promise with 10+ Emerging Therapies in Development
• DelveInsight's latest report reveals 8+ companies developing 10+ therapies for hypersomnia, with Avadel's Lumryz, Zevra's KP1077, and Alkermes' ALKS 2680 among the key candidates advancing through clinical trials.
• Recent clinical milestones include Axsome's AXS-12 meeting its Phase III primary endpoint with 72-82% reduction in cataplexy attacks, and Takeda's TAK-861 showing promise as the first treatment targeting the underlying pathophysiology of narcolepsy type 1.
• The hypersomnia market is driven by increased awareness, unmet medical needs, and advances in neuroscience, though challenges remain including lack of biomarkers, high trial costs, and regulatory hurdles.
Owkin Launches ATLANTIS: AI-Powered Patient Data Mining Initiative Across 20 Global Healthcare Centers
• Owkin has initiated ATLANTIS, a comprehensive patient data mining program partnering with 20 leading healthcare institutions across seven countries, set to conclude by May 2025.
• The project will analyze data across 11 therapeutic areas, including seven oncology programs, three immunology and inflammation conditions, and Alzheimer's disease, utilizing Owkin's AI platform Owkin K.
• This initiative aims to break down data silos by integrating diverse medical data sources, including health records, imaging, and diagnostic results, to accelerate drug discovery and development.
Nxera Pharma Partners with Holling Bio-Pharma to Launch Daridorexant for Insomnia in Taiwan
• Nxera Pharma has established a strategic partnership with Holling Bio-Pharma, Taiwan's largest pharmaceutical distributor, to commercialize daridorexant for insomnia treatment in the Taiwanese market.
• The agreement addresses a significant medical need in Taiwan, where sleep disorders affect 12-20% of the adult population, approximately 4-5 million people as of 2021.
• Holling Bio-Pharma brings nearly five decades of experience in drug distribution and extensive expertise in CNS products, positioning the partnership for successful market penetration.
Statin Therapy Shows Significant Cardiovascular Risk Reduction in Elderly Patients Over 75
• New research demonstrates that statin therapy effectively reduces 5-year cardiovascular disease risk by up to 12.5% in adults aged over 85, without increasing adverse event risks.
• The study highlights statins' multiple beneficial mechanisms, including lipid-lowering, anti-inflammatory, and antioxidative properties, which help combat vascular inflammation and atherosclerosis in older adults.
• Clinical data shows varying effectiveness across age groups, with risk reduction ranging from 1.2% to 5% in patients aged 75-84, and 4.44% to 12.5% in those over 85.
Santhera's Agamree Receives NICE Recommendation and China NMPA Approval for Duchenne Muscular Dystrophy Treatment
• The National Institute for Health and Care Excellence (NICE) has recommended Agamree (vamorolone) for treating Duchenne muscular dystrophy (DMD) in patients aged four and older in England, Wales, and Northern Ireland.
• China's National Medical Products Administration (NMPA) has approved Agamree for DMD, marking it as the first approved treatment for the condition in China.
• Agamree demonstrated clinically significant safety benefits, including better bone metabolism and growth, compared to standard corticosteroids in clinical trials.
• Santhera is collaborating with NHS and Sperogenix Therapeutics to ensure rapid patient access to Agamree in the UK and China, respectively.
Kronos Bio Halts Istisociclib Development, Considers Strategic Alternatives
• Kronos Bio discontinued istisociclib (KB-0742) development after safety concerns arose in a Phase I/II trial for platinum-resistant high-grade serous ovarian cancer.
• The trial revealed neurological adverse events in five of seven patients, leading to treatment discontinuation or dosage reduction in some participants.
• Facing financial pressures, Kronos Bio is exploring strategic alternatives, including acquisition or merger, to maximize stockholder value and preserve cash.
• The company is also seeking partnerships for preclinical assets KB-9558 and KB-7898, while focusing on IND application for multiple myeloma and autoimmune disorders.
Seltorexant Shows Promise as First-in-Class Treatment for Depression with Insomnia in Phase III Trial
• Phase III trial demonstrates seltorexant's efficacy as an add-on therapy, showing significant improvement in depression scores with a -2.6 point difference on the MADRS scale compared to placebo.
• As the first selective orexin-2 receptor antagonist for depression, seltorexant significantly improved both depression and sleep disturbance symptoms in patients with inadequate response to conventional antidepressants.
• The drug demonstrated a favorable safety profile with fewer adverse events than placebo (36% vs 40.3%), positioning it as a potential first-in-class treatment for concurrent depression and insomnia.
Nxera Pharma Partners with Shionogi for Quviviq Distribution in Japan Following Approval for Insomnia
• Nxera Pharma has entered a commercial partnership with Shionogi for the distribution and sales of Quviviq in Japan, succeeding a prior agreement with Mochida Pharmaceutical.
• Quviviq, a dual orexin receptor antagonist, received approval in Japan for treating adult insomnia, marking a significant advancement in addressing sleep disorders.
• Under the agreement, Nxera will supply the drug, while Shionogi will manage distribution and sales, aiming to address the substantial public health issue of insomnia in Japan.
Viatris Expands Pipeline with Idorsia Collaboration, Focuses on Cardiovascular and Autoimmune Therapies
• Viatris collaborates with Idorsia Ltd., acquiring rights to selatogrel for acute myocardial infarction and cenerimod for systemic lupus erythematosus.
• Selatogrel is a self-administered medicine for heart attack patients, complementing Viatris' cardiovascular expertise.
• Cenerimod, a novel immunology asset, targets systemic lupus erythematosus and may expand to other autoimmune indications.
• Viatris' Q4 2023 earnings missed estimates, but key brands like Yupelri and Lipitor showed growth, and new generics contributed $107 million in revenue.
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