FDA’s Post

Today we announced a significant step in our clinical development of SRP-001 by enrolling the first subjects in the multiple ascending dose portion of our ongoing Phase 1 trial. SRP-001 is a first-in-class, non-opioid investigational drug that avoids liver toxicity linked to acetaminophen, positioning itself to meet the urgent need for non-toxic and non-addictive solutions for acute and chronic pain. For more details, read the full release https://lnkd.in/e-tPD8ms #gamechanger #LifeScience #biotech #acetaminophen #medicationsafety #healthcare #clinicalresearch #innovation #Innovation #DrugDevelopment

Several new FDA guidances released this month including: "Rare Diseases: Considerations for the Development of Drugs and Biological Products" which provides comprehensive guidelines for the development of drugs for rare diseases. It covers various aspects including natural history studies, nonclinical studies, clinical development considerations (effectiveness, safety, pharmacology, biomarker use), pharmaceutical quality considerations, and interactions with the FDA. Key differences from the previous guidance include: -Enhanced focus on natural history studies to understand rare diseases better. -Detailed guidance on nonclinical studies tailored to rare diseases. -Specific considerations for clinical development, emphasizing the uniqueness of rare diseases. -Expanded discussion on patient participation, pediatric considerations, and expedited programs. -Emphasis on early and continuous interactions with the FDA for successful drug development.

FDA has published final guidance on “Rare Diseases: Considerations for the Development of Drugs and Biological Products” -- not only an early Christmas gift for #raredisase patients and first-time and long-term sponsors, but also the latest in the Agency’s many steps to facilitate development of new rare disease treatments. Just ten days ago, #FDA announced its new Genetic Metabolic Diseases Advisory Committee (GeMDAC) to advise CDER on “[g]enetic metabolic diseases include very #rarediseases that individually affect a limited number of patients.” The final guidance – ·     provides clear, overarching advice on key nonclinical and clinical trial design considerations, use of natural history studies, and a nod to use of decentralized trials; ·     hints it “may exercise some flexibility on the type and extent of manufacturing information… expected at the time of submission and approval… on a case-by-case basis”; ·     most notably “encourages involvement of patients, their caregivers, and advocates in rare disease drug development”; and ·     underscores the availability of heightened engagement with CDER review divisions through the new INitial Targeted Engagement for Regulatory Advice on CBER/CDER ProducTs (INTERACT, possibly the most tortured acronym I’ve seen 😇) meetings, Critical Path Innovation Meetings (CPIM) and Accelerating Rare disease Cures (ARC) program.   FDA has underscored the importance of partnering with rare disease patients and trusted partners like #NORD to find treatments for the ~95% of the >7,000 known rare diseases lacking an approved therapy. The recent milestone approvals against #sicklecelldisease underscore Commissioner Robert Califf’s call to capitalize on the enormous promise of cell and gene therapies, ASOs, and other breakthrough advances against rare diseases. May 2024 be a truly landmark year for rare disease #innovation! #kendallsquarepolicy 

We understand the pivotal role high-quality biospecimens play in advancing clinical studies from exploratory R&D to achieving regulatory compliance. Our extensive inventory of ethically approved biospecimens spans diverse biomarkers, supporting your research needs. Whether you are conducting Self-Test Studies, utilising Remnant Samples, or planning Prospective Collections, we have you covered. Why PeploBio? ✅ Ethically Approved Biospecimens ✅ Diverse Biomarker Coverage including rare disease types. ✅ Seamless Support for Self-Test Studies, Remnant Samples, and Prospective Collections For a comprehensive look at our inventory, leave a comment with your e-mail address and our Commercial Manager, Jack Purvis will get in touch with you. #IVDs #Biobank #IVDR #TGA #FDA #Biotechnology #IVDmanufacturer #pharmaceuticalindustry #Regulatory #Clinicalresearch #Clinicaltrials #Infectiousdiseases #SampleProcurement #ClinicalStudies #Biospecimens #PeploBio

Ginsenoside Rg5 as an anticancer drug: a comprehensive review on mechanisms, structure–activity relationship, and prospects for clinical advancement.  Our latest  article published in “Pharmacological Report”   is now available online @ https://lnkd.in/ebyfAura  

Gwen Eak and Kelli Phillips, experts in bioanalysis, outline the evolution of biosimilar drug development regulations in the last two decades, giving background on harmonization efforts in the U.S. and EU and key milestones, as well as delving into key areas specific to bioanalysis in biosimilar programs, including: • Method development and validation • Immunogenicity assays • PK considerations • Comparability assessment Read more » https://bit.ly/46IyU6V

"This exploration has revealed that major phytocannabinoids, including cannabidiol (CBD), cannabigerol (CBG), tetrahydrocannabinol (THC), and their derivatives interact with a wide array of receptors, ion channels, and enzymes. Recent studies have illuminated the complex pharmacology of cannabinoids, demonstrating their actions on receptors such as transient receptor potential (TRP) channels, peroxisome proliferator-activated receptors (PPARs), and serotonin receptors, to name a few. This intricate web of interactions offers new insights into the therapeutic potential of cannabinoids, extending well beyond their psychoactive properties." Mahalo to Mike for this paper; I learned something while working! Please use my Aloha Discount Code DAVID20 and save 20% at EndoCare #HappyHealthy #Receptors https://lnkd.in/gHdGsKZb

First Patient Treated in Phase 2 Study of Synthetic THC Prodrug for IOP Lowering: Skye Bioscience, Inc, initiated its phase 2 clinical trial evaluating SBI-100 for IOP. https://lnkd.in/ei2HgrxT #glaucoma

Genentech, a member of the Roche Group announced that the U.S. Food and Drug Administration (FDA) has approved Vabysmo (faricimab-svoa) for the treatment of macular edema following retinal vein occlusion (RVO). RVO is the third indication for Vabysmo, in addition to wet, or neovascular, age-related macular degeneration (AMD) and diabetic macular edema (DME). Read more news on FDA-approved medical devices: https://lnkd.in/gxGkVumk #Genentech #ai #roche #smarteeva #fda #medicaldevice #postmarketsurveillance #ai

Exciting news from FDA today on the future of Platform Clinical trials in its newly released guidance. In 2022 FDA released guidances “Master Protocols: Efficient Clinical Trial Design Strategies To Expedite Development of Oncology Drugs and Biologics” (March 2022) and “Studying Multiple Versions of Cellular or Gene Therapy Product in Early-Phase Clinical Trials (November 2022)” that set the stage for “Master Protocols” studying more than one indication or more than one drug simultaneously, applicable to certain specific product subsets. For Master Protocols these guidances formalized “Basket” trials involves unification of multiple diseases into a single 'basket', and “Umbrella” trials combining evaluation of multiple targeted therapies in one clinical trial, often cross referenced in multiple INDs. The newly released guidance is much more broad, applying to all drugs and biologics, and introduces several new concepts: ➡️ The new “Platform” trial: or trial s designed to evaluate multiple medical products for a disease or condition in an ongoing manner, with medical products entering or leaving the platform. ➡️ Ways to meet with FDA to discuss master protocols, or REQUEST FOR MEETING-MASTER PROTOCOL (Meeting Type B) ➡️ Specific information on how to cross-reference Master Protocol data in other INDs and approval packages Master protocols, historically seen as a risky option outside of limited oncology settings, have the potential to accelerate drug development— while making it more efficient and less costly. It looks like Master Protocols are breaking new ground across FDA regulated drugs, beyong just oncology. Draft Guidance: https://lnkd.in/e97JfggM FDA Podcast Recap: https://lnkd.in/e-v4tiGS FDA Info Sheet: https://lnkd.in/eGPaPW6u #fda #drugdevelopment #regulatoryaffairs #clinicaltrials #clinicalresearch #clinicaltrial #nursesonlinkedin #healthcare #vaccinedevelopment #genetherapy #celltherapy