Neurocrine Biosciences’ Post

New Drug Approval Afimetoran BMS-986256, WHO 11516 cas 2171019-55-7 2-[4-[2-(7,8-dimethyl-[1,2,4]triazolo[1,5-a]pyridin-6-yl)-3-propan-2-yl-1H-indol-5-yl]piperidin-1-yl]acetamide C26H32N6O,444.583, phase 1 Afimetoran is an immunomodulator and an antagonist of toll-like receptors 7 and 8.1,2 It is also is under investigation in clinical trial NCT04269356 (Study to Assess the Way the Body Absorbs, Distributes, Breaks Down and Eliminates Radioactive BMS-986256 in Healthy Male Participants). Afimetoran

Since 1992, FDA has paved the way for life-saving medicines to reach patients swiftly through the Accelerated Approval pathway, granted the drug demonstrates clinical benefit in a Phase IV confirmatory trial. In our new white paper, "RWE as Confirmatory Evidence for Accelerated Approvals," we delve into the transformative role of #RWE in helping drugs move through this process, including how it can help tackle challenges such as addressing surrogate endpoint validity. Download the white paper here: https://hubs.li/Q02tDTxV0

The State of Innovation in Pain and Addiction by David Thomas, CFA and Chad Wessel https://lnkd.in/djwYTH5T. Key takeaways: 1. Only 0.7% of new pain drugs progress from Phase I clinical trials to FDA approval, compared to the average success rate of 6.5% for novel drugs across all disease areas 2. The number of active clinical drug programs for pain has plummeted from 220 to 124 since 2017, a 44% decrease 3. The report’s authors describe a disconnect of investment focus, where private investment is not matching the magnitude of the societal and economic costs associated with the pain and addiction crises. They found that only 1.3% of total therapeutic venture capital investment went to pain and addiction companies in 2021

Pediatric drug development in the US and EU is subject to the continual evolution in local, regional, and global regulatory requirements and guidelines. Kimberly Belsky, FRAPS, MS, Karl-Heinz Huemer, and Linda McBride share some of the key considerations in establishing a global pediatric drug development strategy, including changes in regulatory requirements and clinical study design, as well as some real-world examples in this article from the latest issue of RF Quarterly. RAPS members get exclusive access to this article: https://bit.ly/3Ttp44O

🚨 FDA Approval Alert! 🚨 The #FDA has officially approved Purdue Pharma L.P.'s #Zurnai, a #nalmefene hydrochloride auto-injector designed for emergency treatment of known or suspected opioid overdoses. 🔬 How nalmefene Works? The mechanism of action of nalmefene involves its role as an opioid receptor antagonist. It primarily blocks the mu-opioid receptors in the brain which are the primary sites where opioid drugs exert their effects. By binding to these receptors, nalmefene prevents opioids from activating them thereby reversing the effects of opioid overdose such as respiratory depression and sedation.  Additionally, nalmefene has a high affinity for kappa and delta-opioid receptors, contributing to its effectiveness and longer duration compared to other antagonists like #naloxone. Zurnai is intended for immediate administration as emergency therapy in settings where opioids may be present. But, it is not a substitute for emergency medical care ! While nalmefene shares similarities with naloxone especially in reversing opioid overdoses, it is not a direct replacement. Nalmefene has a longer duration of action making it potentially more effective for overdoses involving long acting opioids. However, naloxone remains the standard first-line treatment due to its widespread availability and established use in emergency settings. Nalmefene could serve as an alternative or complementary option especially in situations where longer opioid antagonism is necessary. Despite this, nalmefene has significant potential for integration into standard medical care. *The image below is for illustrative purposes only and does not represent the actual product.* #PurduePharma #FDAApproval #OpioidOverdose

READ NOW: NAVLIN Last Week in Review https://ow.ly/KW7150SsPgf This week from NAVLIN by EVERSANA's “Last Week in Review,” recapping the latest regulatory changes, pricing & reimbursement updates, drug approvals and more from across the world: Policy: Learn how new criteria could impact market access and treatments like Gilead’s Trodelvy. Pricing & Reimbursement Discover how Vertex’s long-term agreement with NHS England marks a new era for cystic fibrosis treatment availability. HTA: Learn how CSL Behring’s Hemgenix is set to transform the treatment landscape for adults with severe hemophilia B, following NICE’s final draft guidance. Other: Discover how the EMA and HMA are addressing shortages of key GLP-1 receptor agonists like Ozempic, Saxenda, Trulicity, and Victoza. And more... #NAVLIN #CysticFibrosis #HemophiliaBTreatment #MarketAccess #DrugEvaluation

The FDA has approved Zunveyl (benzgalantamine) delayed-release oral tablets for the treatment of mild to moderate dementia of the #Alzheimer type in adults. For all the latest drug news, check out our drug referential resource: https://ow.ly/yUrV50SPGAT. #DrugNews #Pharmacy #Medication

Good news 🩵to help people not getting worse 🩵💚🩵 Alzheimer’s patients and families live in a cloud of uncertainty. It’s about to get worse! Alzheimer's disease is a brain disorder that gets worse over time. It's characterized by changes in the brain that lead to deposits of certain proteins. Alzheimer's disease causes the brain to shrink and brain cells to eventually die. Alzheimer's disease is the most common cause of dementia — a gradual decline in memory, thinking, behavior and social skills. These changes affect a person's ability to function ! About 6.5 million people in the United States age 65 and older live with Alzheimer's disease. Among them, more than 70% are 75 years old and older. Of the about 55 million people worldwide with dementia, 60% to 70% are estimated to have Alzheimer's disease. The rate of progression for Alzheimer's disease varies widely. On average, people with Alzheimer's disease live between three and 11 years after diagnosis. But some live 20 years or more. The degree of impairment at diagnosis can affect life expectancy.

"Currently, the most common drugs used against COVID-19 in the U.S. are FDA-approved drugs such as Paxlovid and Veklury (Remdesivir). Despite these options, COVID-19 drugs are costly and thus not readily available for the average person seeking relief. However, a new clinical trial shows hope with the successful results of a new COVID-19 drug: Simnotrelvir." To learn more, read Maahik Trivedi's article "Simnotrelvir: New COVID-19 Pill With Significant Promise" here: https://lnkd.in/gQFVkX77

This is an important landmark! With better drugs and improved outcomes for many cancers, current requirement of overall survival as an endpoint for drug approval will mean long study follow-ups and high study cost, delay in approval and access to treatment. Having an early robust surrogate endpoint would be a great way to mitigate these issues. Kudos to Drs Brian Durie, Nikhil Munshi, Ken Anderson, Bruno Paiva and the International Myeloma Foundation for their leadership and persistence in driving this (it has taken about 8 years!) and of course the entire myeloma community for contributing data to support the case. This again highlights the power of a cohesive community of thought leaders, practiotioners and patients in moving the needle forward for myeloma treatment! #mmsm #cancertreatment