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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 clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
In just two years, CTMC has advanced eight therapies into clinical trials, harnessing genetic engineering to enhance T-cell effectiveness in the fight against cancer. Over the years, Ive had the privilege of working on more than 25 different products, Bock shared.
A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms.
2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1] 26 March 2025.
Biosimilar clinical trials and study designs’ Considerations The rising prevalence and large market share of biological products emphasize their importance as treatment options for several cancers and autoimmune diseases.
High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. The development of Taxol involved multiple patents, including those for the isolation method, synthetic production, and various formulations.
Despite their exciting potential, the smooth operation of cell therapy development trials requires extraordinary orchestration, perfectly aligning the product and patient journeys. While clinical supply is essential to any successful trial, autologous cell therapy trials occupy the far end of the spectrum regarding risk tolerance.
The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. These findings underscore the potential of mRNA to redefine cancer treatment by offering precise, patient-specific therapies.
Most of these conditions are genetic in origin and the majority have no effective treatment. Symptoms were reversed in mouse models and a clinical trial is planned for later this year. That includes countless rare peripheral diseases, including many for which there are currently no treatments.
Advancing reduction of drug use as an endpoint in addiction treatmenttrials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&
Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first. Kidneys perform essential functions such as managing blood filtration, the balance of water and electrolytes, blood pressure, and hormone production.
Though they do positively impact patients’ quality of life and hemodynamic parameters, these treatments — prostacyclin analogs and receptor agonists, phosphodiesterase 5 inhibitors, endothelin-receptor antagonists, and cGMP activators — have shown limited beneficial effects on survival and disease progression.
Identifying branded drugs with a low likelihood of generic entry has become a crucial strategy for companies looking to expand their product portfolio through in-licensing. Branded drugs, developed through extensive research and clinical trials, often enjoy patent protection for a limited period.
Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. This limited pool poses significant barriers to conducting statistically significant studies and validating the efficacy and safety of new treatments.
LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. As a single-arm trial (i.e., Join us as we break down what went wrong this time. What Happened?
These designs facilitate streamlined trial logistics and centralized governance and create higher-quality data. Master protocols allow a trial to perform multiple tests on diverse patient populations or diseases under a unified design. But how do they work, and when are they appropriate? What Are Master Protocols?
Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). How does the DCI mechanism compare to the design of other drugs for cancer treatment?
Authors: Matt Cooper, PhD, Executive Director, Therapeutic Strategy Lead, Oncology; Megan Morrison, Vice President, Asia Pacific Strategy Lead Adaptive trial designs have become essential in oncology, offering a flexible and efficient approach for conducting clinical trials.
Regulatory bodies such as the FDA oversee clinical trials 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.
These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.
Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in Clinical Trials ” that revises the 2006 guidance “Establishment and Operation of Clinical Trial Data Monitoring Committees” and, when final, will replace the 2006 guidance.
Cost and complexity go hand-in-hand The rising costs and growing complexity in clinical trials are deeply linked, with patient recruitment, extended timelines and meeting regulatory demands emerging as some of the key drivers. Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024.
AI has the potential to revolutionize drug discovery, clinical trials, and personalized medicine. However, before integrating an AI product into a life sciences workflow , it is crucial to evaluate its effectiveness and reliability rigorously. Improve clinical trial predictions? Or perhaps optimize patient diagnosis?
Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. There are so many patients suffering from diseases that have no treatments. We are committed to supporting reimbursement and exploring ways to increase patient access to treatments. “We
In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches. There has also been an increase in government and regulatory support for CGT trials, in conjunction with an increase in investments for these products to get to market.
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Both Durveqtix and Adzynma were major advancements this year, as well as the EMA granting PRIME designation for Ultragenyx’s Angelman syndrome, Sangamo’s Fabry disease, and Spur’s Gaucher disease products. DA: Several things! These innovations show that the FDA are caring more about leveraging collective lessons learned in drug approvals.
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 clinical trials.
As the clinical trial landscape evolves, drug developers are faced with novel challenges and changes in study recruitment, trial size and structure, and more. Decentralized Trials Digital and decentralized clinical trials (DCTs) continue to expand and have become a standard solution for drug developers.
Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations.
AI also has the potential to incorporate real-world data (RWD) obtained from electronic health records (EHRs), medical claims or other data sources to inform the design and optimization strategy of clinical trials.
Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.
Clinical trial data management is critical to pharmaceutical research, yet it remains a significant challenge for many organizations. Outdated systems: Many organizations rely on legacy data management tools that fail to meet the demands of modern clinical trials.
2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment. Both trials demonstrated a statistically significant superior reduction in pain with Journavx compared to placebo. Suzetrigine is taken by mouth. [1]
Less clear is whether we can productively and safely inhibit TGF- activity at all given the toxicity issues associated with TGF- inhibition. No chemotherapy regimen is used in the trial. Note one important difference in the trials is the inclusion of HPV-positive patients, which the Bicara trial will exclude.
However, accelerated approval comes with a condition: sponsors must conduct confirmatory trials to verify the drug’s anticipated clinical benefits using robust outcome measures. Concerns have arisen over delays—sometimes spanning over 7–8 years—that may expose patients to risks before confirmatory trials are completed.
When health research is published, we tend to focus our minds on the findings and consider the implications for future treatments. Yet, what about those who took part in the trial? In recent years there has been an increasing call to let trial participants know the results of trials in which they have taken part.
Author: Lona Sheeran, SVP, Clinical Operations, Early Phase At this year’s Clinical Trials Nexus, I had the privilege of representing Worldwide Clinical Trials as the sole CRO on a panel discussion: “Reversing the Conversation: What the Clinical Trial Industry Really Wants from its Service Providers.”
Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.
Cancer Treatments, Clinical Trials, and the Motivation to Innovate nbartlett Tue, 06/27/2023 - 20:58 Getting to the Heart of Science with Michelle Newby At the heart of science are stories—stories of courage, challenges, successes, lessons, and people. Q: What was your cancer diagnosis and was treatment options were you given?
Biological products have unlocked the potential for the management of several diseases such as cancers and autoimmune diseases for which treatment with small molecule, chemically synthesized drug molecules remain suboptimal. […]. The post Biosimilar Clinical Trials and US FDA Guidance appeared first on ProRelix Research.
The Rise of Biosimilars Biosimilars are biological products that are highly similar to reference biologics in terms of structure, function, and efficacy. This growth is driven by the increasing demand for affordable biologic treatments and the loss of patent exclusivity for several blockbuster biologics.
This has a positive impact on the treatment of patients when reduction of heart rate without decrease in arterial blood pressure is desired. [9] Treatment of phenol 143 with bromo epoxide 144 in the present of K2CO3 afforded ether 145 in 76% yield. 9] It is used as landiolol hydrochloride.
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