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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.
It is recognized, however, that there is a learning curve across the industry and FDA that is inherent to the development of such novel technologies. To prevent this learning curve from becoming a roadblock on the path to regulatory approval, the FDA has developed several collaborative programs to support advanced manufacturing.
The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene therapy applications it later brings to the regulator.
FDAs withdrawal authority when a confirmatory trial is not conducted with due diligence was expanded to include that FDA could specify the conditions for a postapproval study. Considering FDAs new authority to specify the conditions for such confirmatory trials (e.g.,
The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.
Unlearn’s early collaboration with regulators has helped it navigate this space effectively. The company worked closely with agencies including the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) from the outset, aligning its methodology with evolving guidance.
FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.
Farquhar This is the first in a series of blog posts on tips for successfully handling an FDA inspection. Using publicly available examples, these lessons will illustrate potential pitfalls and strategies for interacting with FDA during and after an inspection. FD&C Act 501(j).
On April 10, 2025, the US FDA announced that it has a long-term plan to eliminate conventional animal testing in drug development, starting with monoclonal antibodies (mAbs).[ 2] An overview of the 3Rs The FDA and other global regulatory health authorities have long embraced the 3Rs of animal research (replace, reduce, and refine).
For example, transcriptomic processes are showing the potential to identify and track failures in gene expression and gene regulation of amyloid and tau-related biomarkers, understood as precursors to the onset of Alzheimers disease (AD). References FDA-NIH Biomarker Working Group.
Enhanced Regulatory Compliance : Integrated CDMOs can provide comprehensive supervision over all research, development, and manufacturing initiatives, ensuring compliance with strict regulatory guidelines set by entities like the FDA and EMA.
Koblitz Integral to the careful balance Congress struck when passing the Hatch-Waxman Amendments, the patent term extension (PTE) is intended to restore patent life that was consumed during regulatory review of an FDA-regulated product. FDA has already been there , of course, with respect to 180-day exclusivity.)
But growing ethical scrutiny, supply shortages, cost burdens, scientific innovation and regulatory shifts like the US Food and Drug Administration (FDA)’s new alternative methods roadmap are bringing the continued reliance on live NHPs into question, and opening the door to next-generation solutions that could eventually replace them altogether.
Collaboration with Regulatory Agencies : Collaboration with regulatory agencies such as the FDA and EMA is crucial in navigating the regulatory landscape. This involves working closely with regulators to ensure that the analytical characterization and clinical trial design meet the necessary standards for approval.
Drug/Biologic/Device combination products are subject to the requirements of all three FDA centers: CDER, Center for Biologics Evaluation and Research (CBER), and CDRH. Developers must ensure that their CMC documentation is comprehensive, covering both the APIs and the device.
Beyond bioanalysis, understanding the mechanism of action is equally important, as different classes of oligonucleotidessuch as antisense oligonucleotides (ASOs)interact with mRNA in distinct ways to regulate gene expression. Regulatory Considerations for Oligonucleotide Drug Development and Safety In 2024, the U.S.
Regulators may reasonably ask is a drug crystallized in orbit the same as if made on Earth? Initial discussions between FDA, NASA, and industry have begun, but a solidified regulatory framework is years away. Regulatory : there is no regulatory framework for products made in space.
Claud The ongoing DOGE-led reductions to the federal workforce and recent sweeping policy changes have spawned many questions for compliance officers and quality managers in FDA-regulated companies. How will the cuts at FDA impact inspections and enforcement? Clarity of purpose is a good place to start.
Fast Track and Orphan Drug Designations from FDA The U.S. Food and Drug Administration (FDA) recognized the significance of rilzabrutinib’s potential by granting Fast Track designation in May 2025 and Orphan Drug designation in April 2025 for its development in IgG4-related disease.
Food and Drug Administration (FDA). He most recently served for over six years in FDA’s Office of Chief Counsel, first as an Assistant and then as an Associate Chief Counsel. He also provides strategic litigation support and proactive counsel on agency engagement.
These standards are widely recognized by regulatory agencies such as the FDA and EMA for use in analytical method development, validation, and quality control. For manufacturers, researchers, and regulators alike, this alliance represents more than a logistical convenience—it’s a blueprint for the next era of therapeutic innovation.
With global regulators including the FDA, EMA, and MHRA signaling alignment and implementation in 2025 [FDA, 2025; EMA, 2025; MHRA, 2025], sponsors must act now to align their strategies with the new expectations. FDA Statement on Adoption of ICH E6(R3). Learn more References: ICH Harmonised Guideline. link] MHRA UK.
However, many other types of toxicity remain challenging: We have the FDA DILIRank, and now the DICTrank list, which is the cardiotox equivalent. ” Hosseini-Gerami noted the value of established resources for well-understood toxicities: “We have the FDA DILIRank , and now the DICTrank list , which is the cardiotox equivalent.
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.
Members of the PI3K family regulate cellular processes such as cell growth and proliferation, survival, remodelling, and intracellular transport of organelles. [15] 3] [6] [20] [21] The drug application was granted priority review and breakthrough therapy designations by the FDA. [3] Food and Drug Administration (FDA).
s specific mutation, creating a mouse model of the disease, determining the optimal base editor, performing extensive safety analyses, working with Danaher to manufacture the therapeutic, conducting toxicity studies, and securing FDA approval for the trial. This unprecedented feat required diagnosing K.J.’s
Let’s dive in… Wave 2: B7-H3 Optimistically named B7-homologue-3, B7-H3, a distant relative of the immune regulatory proteins B7-1 and B7-2, has recently moved into the spotlight - not as an immune regulator but as a target of advanced therapeutic modalities including bispecific antibodies, antibody drug conjugates (ADC) and CAR-T cells.
Interestingly, the US Food and Drug Administration (FDA) is starting to enforce diversity policy in clinical trial recruitment regulations. Cancer doesn’t discriminate between men and women and it’s important to ensure clinical trials are balanced. In conclusion, I will continue to champion women in STEM careers.
In the decades since the FDA first introduced Good Manufacturing Practice (GMP) regulations, the drug and device industry has undergone significant transformation. Additionally, as regulators worldwide adopt more rigorous data integrity and compliance requirements, scrutiny of quality systems has intensified.
In regulated domains, every step must be documented, auditable and reproducible to meet stringent regulatory requirements. Current generative AI models often fall short of these expectations, posing challenges in gaining trust from regulators who demand a high bar for reliability and accountability.
Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). Importantly, the independent expert panel’s recommendations will be reviewed by the FDA before implementation. The FDA agrees with this cautious approach. ELEVIDYS is currently the only U.S.
The FDA, 2024 FDA guidance M12 Drug Interaction Studies , pg. The potential for an investigational drug to cause pharmacokinetic interactions both as an object (effect of other drugs on the investigational drug) and as a precipitant (effect of the investigational drug on concomitant drugs) should be evaluated.
Drug development is a complex and highly regulated process. 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. Regulatory agencies, such as the U.S.
Following clear processes, in accordance with GLP and current FDA/EMA guidelines—and supported by our unique array of platforms and large list of validated assays —our team ensures the most effective methods for your individual programs. We have state-of-the-art, purpose-built laboratories at our locations in the U.S.
FDA Approves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. The FDA approval of Dupixent for bullous pemphigoid represents a significant advancement in dermatological and immunological care.
Food and Drug Administration (FDA) granted approval to the tablet formulation of BRUKINSA for all five of its approved indications. Regulatory Developments The CHMP’s favorable opinion in Europe follows a similar regulatory milestone in the United States. Earlier this month, the U.S.
Marijuana down-rescheduled to schedule III would still be regulated for legitimate medical, scientific and industrial use. Requirements will vary depending on the registered business activity. Schedules of Controlled Substances, 44,621. Marijuana would also remain subject to applicable provisions of the Food, Drug and Cosmetics Act.
The February 2024 FDA draft guidance on DMCs highlights the necessity of incorporating diverse, global expertise into these critical oversight functions. A globally diverse DMC ensures the trial adheres to local regulations while maintaining high ethical standards.
FDA is in the pipeline, and subsequent filings in other regions including the European Union, Japan, and Latin America are expected to follow. We look forward to working closely with regulators, clinicians, and patients to ensure pimicotinib becomes available as swiftly and broadly as possible.” A New Drug Application (NDA) to the U.S.
Main Goals Community Resources Conversations on Cancer A public panel discussion series Discuss cancer-related social issues with inclusive and diverse panel members from the US and within the FDA. Provides the Oncology Dosing Toolkit. first or second line setting), considering clinical, scientific, regulatory, and operational factors.
Greater Engagement with Regulatory Authorities Regulatory agencies, including EMA and FDA, are focused on building strong relationships with patient communities and organizations. For example, Voice of the Patient and Patient-Focused Drug Development meetings are focused on one disease or a group of similar diseases.
The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. Notably, this has led to the development of new medicines to treat genetic diseases — Casgevy was the first of these to gain FDA approval and is used to treat two blood disorders, called sickle cell disease and beta thalassemia.
Their results underscore the importance of early detection and treatment, such as with vorasidenib , which targets IDH and was recently approved by the FDA for low-grade gliomas. said study senior author Brad Bernstein , an institute member at Broad and director of the Gene Regulation Observatory at Broad. “We Nature Cancer.
The Foundational Pillars ACTO believes in implementing four foundational pillars that will ensure responsible AI solutions that address pharmas highly regulated parameters, while providing value. a walled garden of sorts), ensures data sovereignty and compliance with regional regulations.
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