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Clinicaltrials are expensive, slow and often limited by outdated design constraints. They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data. Accelerating timelines and de-risking development decisions across the pipeline.”
Advanced strategies and tools are being implemented to establish the safety and efficacy of new therapeutic modalities, with the development of new biomarkers becoming one of the most relevant approaches for enhancing the precision and utility of early-stage studies.
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDAClinicalTrials Deals Drug Pricing Gene Therapy An article from Dive Brief Actithera draws new investors to radiopharma drug pitch The four-year-old biotech raised about $75 million in a Series A round that involved nine venture capital firms.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinicaltrials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
FDA Orphan Drug Designation for Antibody-Mediated Rejection in Solid Organ Transplantation The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.
Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This trial will build on previous positive data from the earlier Phase 1/2 HALOS study and is designed to rigorously assess the potential of ION582 as a disease-modifying therapy. “We
Safety Profile Consistent with Previous Studies : The safety profile of rilzabrutinib was well-understood from previous trials, with no new safety signals. We look forward to further evaluating rilzabrutinib in subsequent phases of its clinicaldevelopment.” Fast Track and Orphan Drug Designations from FDA The U.S.
Researchers must characterize the anti-drug-antibody (ADA) response in preclinical and clinical studies and report any ADA-positive samples as a risk-based approach. Regulatory Considerations for Oligonucleotide Drug Development and Safety In 2024, the U.S.
Two large and randomized clinicaltrials, IMpower-133 and CASPIAN, showing statistically significant improvements in outcomes when chemotherapy was combined with atezolizumab or durvalumab, respectively, in the first-line treatment of ES-SCLC. This Phase 3 trial compared tarlatamab with chemotherapy as second-line treatment.
However, the FDA’s recent decision to remove the Risk Evaluation and Mitigation Strategy (REMS) requirements for certain approved Chimeric Antigen Receptor T-cell (CAR T) therapies could dramatically shift these numbers, making life-saving treatments more accessible to those in need.
Regulatory bodies such as the FDA oversee clinicaltrials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.
Rilzabrutinib: A Potentially Transformative Therapy in ITP Among the most anticipated presentations is new data from the LUNA 3 phase 3 clinicaltrial evaluating rilzabrutinib, an investigational, reversible, oral BTK inhibitor, in adults with immune thrombocytopenia (ITP). In the U.S.,
Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other global counterparts, set rigorous standards to ensure that drugs are safe, effective, and high-quality.
5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] 5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] Jump up to: a b c d e f g h i j “FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome” U.S.
Balancing generative AI opportunities and risks Generative AI offers unparalleled opportunities for transforming various aspects of health care and clinical research. In clinicaltrials, generative AI accelerates trial designs through protocol optimization and enrollment forecasting, leading to faster and more effective studies.
FDA Grants Priority Review to Merck’s WINREVAIR™ Based on Landmark ZENITH Trial Showing Dramatic Reduction in Morbidity and Mortality in PAH Patients Merck (NYSE: MRK), operating as MSD outside the U.S. label of WINREVAIR based on compelling new evidence from the Phase 3 ZENITH trial. risk score of 9 or greater.
Published June 11, 2025 Ben Fidler Senior Editor post share post print email license The FDA on June 11, 2025 approved Nuvation's Ibtrozi for ROS1-positive non-small cell lung cancer. Among those who had gotten another such therapy, the rates were 52% and 62% in those trials. You can unsubscribe at anytime.
Reporting in Nature Reviews Drug Discovery, @AsherMullard noted that Merus must provide the FDA with confirmatory evidence of benefit in both NSCLC and pancreatic adenocarcinoma in order to achieve full approval and that analysts predict annual sales of around $180 million by 2030 per consensus forecast via Cortellis.
Groundbreaking Data from the Phase III ARANOTE Trial The CHMP’s recommendation is founded on data from the pivotal Phase III ARANOTE clinicaltrial, which demonstrated that darolutamide, when used in combination with ADT, significantly reduced the risk of radiological progression or death in patients with mHSPC.
ClinicalTrials: Pritelivir is currently in phase II clinicaltrials, with ongoing research into its effectiveness and safety. Pritelivir (development codes AIC316 or BAY 57-1293 ) is a direct-acting antiviral drug in development for the treatment of herpes simplex virus infections (HSV). Johnston, C.
FDA Approves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S.
Grifols’ BT524 Fibrinogen Therapy Shows Promise in Phase 3 Trial Published in The Lancet’s eClinicalMedicine Grifols , a globally recognized healthcare company and a leader in the production of plasma-derived therapies, has marked a significant advancement in the treatment of acquired fibrinogen deficiency (AFD).
We have seen how quickly vaccines and treatments can be developed when innovation is prioritised and stakeholders work together against the backdrop of imminent societal needs. Converting these challenges into opportunities, we must embrace new clinicaldevelopment strategies that promote agility, efficiency and effectiveness.
The drug’s clearance came after an unusual clinicaldevelopment journey and, in the ensuing months, has swelled Soleno’s market value past $4 billion amid optimism about the drug’s sales prospects. You can unsubscribe at anytime. Condulis, for instance, predicts Vykat could reach $2.5 billion in worldwide yearly sales at its peak.
This expansion is creating opportunities for clinicaltrials related to a range of new therapy areas and their subpopulations. The SELECT trial set out to understand whether the drug has similar effect on patients without diabetes.
Dive Insight: Lexeo is developing treatments for heart conditions, such as cardiomyopathy associated with Friedreich’s ataxia and plakophilin-2 arrhythmia. You can unsubscribe at anytime. Both programs are in early human studies. Informa PLCs registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales.
Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. Section #1: FDA Interactions Given the wide range of sponsors (i.e., Tobolowsky & Charles G. Raver & James E.
Food and Drug Administration (FDA) within 30 days, providing a structured timeline for progressing with clinicaltrials. Early engagement with the FDA can help set expectations for future regulatory steps and ensure that non-clinical and clinicaltrial data meet global regulatory requirements.
Real-world data paired with machine learning is a game changer in drug development RWD is becoming increasingly important in influencing the drug development landscape, particularly when used to develop the natural history and patient journey through disease. AI has the potential to disrupt the entire clinicaltrial process.
Patients are the backbone of clinicaltrials, playing an essential role in the drug development process. However, patients also play a vital role in engaging directly with the FDA. It also means sponsors are less likely to waste time and resources developing programs that don’t work for patients.”
Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in ClinicalTrials ” that revises the 2006 guidance “Establishment and Operation of ClinicalTrial Data Monitoring Committees” and, when final, will replace the 2006 guidance.
The trial, which will explore the pharmacokinetics and safety of ATX01 in healthy volunteers, is due to start in January 2021. AlgoTherapeutix recently raised a 12M€ Series A that will fund the Phase 1 and 2 clinicaldevelopment of ATX01. More information at www.algotx.com. View source version on businesswire.com: [link].
Speakers discussed investigator, regulatory (FDA), industry, and patient perspectives during the special symposium “Challenging the Status Quo of Early Phase ClinicalTrial Design: Project Optimus.” To address this challenge, the FDA Oncology Center of Excellence initiated Project Optimus.
The FDA defines a serious condition as “a disease or condition associated with morbidity that has a substantial impact on day-to-day functioning.” Concerns have arisen over delays—sometimes spanning over 7–8 years—that may expose patients to risks before confirmatory trials are completed.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Please select at least one newsletter. Registered in England and Wales. TechTarget, Inc.s
Two of the three planned late-stage efficacy trials for NVX-CoV2373 sponsored by Novavax are fully enrolled, and more than 20,000 participants have been dosed to-date. In alignment with Novavax’ commitment to transparency, Phase 3 clinicaltrial protocols are posted to the company’s website at Novavax.com/resources upon finalization.
Insights in FIH studies provide valuable information on potential dosing, efficacy, and safety, and serve as a good guide for the regulatory requirements needed throughout clinicaldevelopment. jpg Tags ClinicalTrials Weight 1
The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & ClinicalDevelopment , the team discussed why they choose this type of gene therapy to treat HIV. Air Force photo by Kemberly Groue.
This process can be daunting, but understanding how to manage feedback effectively is crucial for developing and ultimately gaining approval for new therapies, especially in oncology clinicaltrials. An illustrative example of harmonization between agencies exists via the European Medicines Agency (EMA) and U.S.
Data monitoring committees (DMCs) review ongoing clinicaltrial data to make recommendations regarding trial conduct based on risk-benefit. DMCs are an essential component to ensuring the integrity and safety of clinicaltrials. New draft guidance published by the U.S.
The FDA guidance, Evaluating Drug Effects on the Ability to Operate a Motor Vehicle , indicates that testing in early-phase clinicaldevelopment should emphasize sensitivity over specificity in CNS effects. Tags ClinicalTrials Weight 12 View the Driving Simulation Fact Sheet for more information.
Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA.
Food and Drug Administration (FDA) has approved an updated label for ADUHELM (aducanumab-avwa) injection 100 mg/mL solution. The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinicaltrials, as seen below ( italics to note updated language).
Read the Guide FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies One frustrating aspect of traditional drug development, especially for rare disease communities, is the tempo of regulatory decisions on potential drugs.
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