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𝐀𝐫𝐫𝐨𝐰𝐡𝐞𝐚𝐝 𝐏𝐡𝐚𝐫𝐦𝐚𝐜𝐞𝐮𝐭𝐢𝐜𝐚𝐥𝐬 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐏𝐡𝐚𝐬𝐞 𝟑 𝐑𝐞𝐬𝐮𝐥𝐭𝐬 𝐟𝐨𝐫 𝐅𝐚𝐦𝐢𝐥𝐢𝐚𝐥 𝐂𝐡𝐲𝐥𝐨𝐦𝐢𝐜𝐫𝐨𝐧𝐞𝐦𝐢𝐚 𝐒𝐲𝐧𝐝𝐫𝐨𝐦𝐞 Arrowhead Pharmaceuticals (NASDAQ: ARWR) Arrowhead Pharmaceuticals announced positive results from the Phase 3 PALISADE study on investigational plozasiran for familial chylomicronemia syndrome (FCS), a severe genetic disease with no approved treatments in the U.S. The study successfully met its primary and key secondary endpoints, including significant reductions in triglycerides (TGs), apolipoprotein C-III (APOC3), and the risk of acute pancreatitis (AP). These results were presented at the ESC Congress 2024 and published in The New England Journal of Medicine. Arrowhead plans to file a New Drug Application with the FDA by the end of 2024 and seek regulatory approval with global authorities. #Pharma #Biotech #GeneticDisorders #RareDisease #FCS #Triglycerides #ClinicalResearch #FDA #DrugDevelopment #ESC2024

Biosimilar Competition Does Not Reduce Patient Out-of-Pocket Costs for Biologic Drugs P.S. If you like this post please 1) Like it (so I know it's useful) and 2) Repost and share it (so others can see it too)

Thoughts on this? >> Novartis to seek approvals in two rare kidney diseases after positive Phase 3 trials >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #productmarketing #biotech #healthcare

#GeneTherapy promises huge benefits - but when is a treatment too expensive? Orchard Therapeutics’ Lenmeldy (also known as Libmeldy) has made headlines since its approval by the FDA by becoming the world’s most costly treatment, with a US wholesale price of $4.25 million for a single course. Results suggest it has the potential to be life-changing for the tiny minority of children (diagnosis prevalence is around 0.0001%) who suffer from metachromatic leukodystrophy (MLD). But to date, it has only generated sales of $12.7 million across Europe… Is there a place for treatments that are so prohibitively expensive? Is there a market? #Pharmaceuticals #LifeSciences

#Pharmacokinetics101: Distribution - How Drugs Spread Throughout the Body In our ongoing series on pharmacokinetics, today we’re diving into drug distribution. This is the process by which a drug spreads from the bloodstream to various tissues in the body. What is Drug Distribution? After a drug is administered, it doesn’t stay put. It travels, or ‘distributes’ itself throughout the body. This distribution is a crucial step in determining how a drug will affect us and how long its effects will last. Factors Influencing Drug Distribution Blood flow to tissues: Organs with a rich blood supply like the heart, liver, and kidneys receive the drug more quickly. Drug solubility: Drugs that are water-soluble tend to stay within the bloodstream, while fat-soluble drugs can penetrate cell membranes and distribute throughout body tissues. Protein binding: Many drugs bind to proteins in the blood. Only the unbound portion of the drug can distribute to tissues. The Role of the Blood-Brain Barrier The blood-brain barrier (BBB) is a unique hurdle in drug distribution. It’s a protective shield that prevents potentially harmful substances, including many drugs, from entering the brain. Only drugs that are small, lipophilic (fat-soluble), and not bound to plasma proteins can cross the BBB. Why Understanding Drug Distribution Matters Understanding drug distribution is crucial for predicting a drug’s effects and potential side effects. It helps us answer questions like: How quickly will the drug take effect? How long will the effects last? Which tissues will the drug affect? In conclusion, drug distribution is a complex but vital aspect of pharmacokinetics. It’s a key player in determining the journey of a drug in our body and its ultimate effects on us. Stay tuned for our next post in this series where we’ll explore the next phase of pharmacokinetics: Metabolism. #patelkwan #pharmacokinetics #drugdevelopment #clincalpharmacology #DMPK

Study: No Differences Between Biosimilar TNFα-inhibitor, Reference Products for Long-Term Survival: Investigators compared the reference products of adalimumab (Humira; AbbVie) and etanercept (Enbrel; Amgen) with biosimilar products. #finance #pharmacy #lifesciences

Thoughts on this? >> Expanded US FDA approval of Elevidys in DMD - The Pharma Letter >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #competitivemarketing #pharma #biotech #healthcare #pharmaceutical

🚀 Looking for answers? TransHeps has the tools to diagnose iDILI with precision! At TransHeps, we’re driving innovation in drug safety with our MetaHeps® assay, supporting clinical trials with precise, patient-specific diagnostics. Check out our website and let’s connect if you’re a clinical trial expert looking to enhance drug development outcomes! #TransHeps #ClinicalTrials #Innovation #DrugSafety

Biosimilar Competition Does Not Reduce Patient Out-of-Pocket Costs for Biologic Drugs P.S. If you like this post please 1) Like it (so I know it's useful) and 2) Repost and share it (so others can see it too)

09 Adverse Drug Reactions - Leading Causes - Factors that lead to ADRs - Pharmacological Factors - Patient-Related Factors - Genetic Factors Factors that lead to ADRs - Drug Interactions - Medication Errors - Social Factors How to get started with UGenome's Personalized Medication Service? 1. Order $399 test kit 2. Swab to collect sample 3. Return sample free of charge 4. Receive PGx report in 3-4 weeks https://lnkd.in/gZhay7Rn Sources - Marsh (2023). Adverse Drug Reactions. Merck Manual - Alomar (2014). Factors affecting the development of adverse drug reactions. Saudi pharmaceutical journal. - Woo et al. (2020). Common causes and characteristics of adverse drug reactions in older adults: BMC Pharmacology and Toxicology, - Siddiqui et al. (2022). Pharmacogenomics of Adverse Drug Reactions. Frontiers in Genetics.. #ugenome, #pharmacogenetics, #genomicmedicine, #personalizedmedicine, #precisionmedicine, #health, #medicationmanagement