Gordian Biotechnology’s Post

Will History Repeat Itself ?! 2023 was a landmark year for Alopecia Areata in that the 1st FDA and Health Canada approved targeted treatment is now available with Ritlecitinib. 20 Years ago in 2003 (Yes, that long!), the 1st Biologic was approved by FDA for Psoriasis, and what an amazing 20 years it’s been for Psoriasis & Psoriatic Arthritis Treatment, with at least 12 Biologics and 4 oral molecules significantly helping and indeed life changing for Psoriasis & Psoriatic Arthritis Patients, and wait, there is more coming, as no 1 drug works for all. Can we look forward to same with Alopecia Areata? Imagine when a severe Alopecia Areata patient comes for treatment, we have 12 or 16 approved agents to choose from?! Hopefully History repeats itself and may be even better from all the lessons we have learned in new drug development! National Alopecia Areata Foundation Canadian Alopecia Areata Foundation National Psoriasis Foundation Canadian Psoriasis Network

Choosing predictive and clinically relevant endpoints is a key aspect of drug development in the regulatory space. By assessing orphan drug development for sickle cell disease (SCD) over the past two decades, we observed that failures in clinical development were mainly found in phase 3 due to lack of efficacy on vaso-occlusive crises as the primary study endpoint. This was likely related to variable definitions and heterogeneity of pain scoring and treatment. If you are interested in improving the debate on how fostering pharmaceutical R&D on SCD (and other rare diseases), you may wish to read our last work on this. https://lnkd.in/difgpBep #sicklecell #orphandrugs

𝐂𝐞𝐥𝐥𝐭𝐫𝐢𝐨𝐧 𝐔𝐒𝐀 𝐒𝐮𝐛𝐦𝐢𝐭𝐬 𝐁𝐋𝐀 𝐟𝐨𝐫 𝐂𝐓-𝐏𝟒𝟕: 𝐀 𝐍𝐞𝐰 𝐄𝐫𝐚 𝐢𝐧 𝐑𝐡𝐞𝐮𝐦𝐚𝐭𝐨𝐢𝐝 𝐀𝐫𝐭𝐡𝐫𝐢𝐭𝐢𝐬 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 📄 Celltrion USA Celltrion USA has submitted the Biologics License Application (BLA) for CT-P47, a biosimilar candidate of ACTEMRA® (tocilizumab), to the U.S. FDA. 💊 CT-P47, containing tocilizumab, aims to provide a more accessible treatment option for rheumatoid arthritis. The submission is based on positive Phase III trial results, evaluating efficacy, safety, and more. 👥 Thomas Nusbickel Tom Nusbickel, Chief Commercial Officer at Celltrion USA, emphasizes the importance of this step in offering patients a diverse product lineup in the autoimmune disease market. 📆 Stay tuned as Celltrion actively cooperates with the FDA for a potential new treatment option for rheumatoid arthritis. #HealthcareInnovation #Biotech #FDASubmission #RheumatoidArthritis #BioPharma #MedicalAdvancements #CTP47 #ACTEMRA #CelltrionUSA #ClinicalTrials

AMX0035 Shows Promise for Treating Wolfram Syndrome 🌟🧬 Amylyx Pharmaceuticals' AMX0035 demonstrates potential in treating Wolfram syndrome, a rare neurodegenerative disease. Initial clinical data reveal improvements in patient outcomes, marking a significant step forward in addressing this debilitating condition. Continued research aims to validate these findings and expand therapeutic options for Wolfram syndrome patients. To read the full news, visit 👉 https://lnkd.in/dfKyj8uD Stay updated with the latest in pharmaceutical advancements at https://lnkd.in/dKhZfHxi #PharmaNow #pharmanews #healthcareinnovation #globalpharma #wolframsyndrome #rarediseases #clinicaltrials #AmylyxPharmaceuticals

Yet Another Drug Approval in the field of rare liver disease~ Primary Billiary Cholangitis (PBC) 10th June 2024, marks the milestone day for Ipsen & GenFit, receiving FDA approval for the drug "Elafabrinor- Iqirvo". The results from the pivotal Phase 3 ELATIVE trial, proved Iqirvo to be effective for the second-line treatment of PBC. It is an oral, once daily PPAR agonist, inhibiting bile acid synthesis through activation of PPAR-alpha and PPAR-delta. The drug recieved Breakthrough Designation in 2019 by U.S. FDA. The approval sets these 2 French companies to directly compete with Intercept Pharmaceuticals' Ocaliva. Ocaliva has been available on the market for eight years. Eyes are set as, another drug expected to be approved in the indication in August—CymaBay Therapeutics’ seladelpar. #rareliverdiseases #FDA #drugapprovals #druglaunch #marketintelligence

https://lnkd.in/eEaTidDA Vertex Pharmaceuticals on Tuesday reported positive study results for its closely watched non-opioid painkiller. The drug lowered the moderate-to-severe acute pain reported by study volunteers, a sign it could be the first in a new class of painkiller to be approved for use. About 80 million Americans are prescribed a medicine for moderate-to-severe acute pain each year, according to Vertex. They can choose from a variety of treatments. Many of them take opioids because they work so well. Vertex’s drug, code-named VX-548, belongs to a new class of medicines that targets molecular mechanisms involved in feeling pain. Researchers have pursued this so-called #NaV class for years but experienced repeated setbacks. NaV refers to the channels through which sodium flows in and out of cells. VX-548 blocks the flow of sodium through a channel called NaV1.8, which plays a role in transmitting pain signals to the brain. VX-548 could open a new, potentially multibillion-dollar franchise in pain for the biotech company, Evercore ISI analyst Liisa Bayko said. “It’s a totally new class of drugs that have never made it this far,” she said in an interview before the company released the trial results. #NaV #nonopiod #painmedicine

Teva Pharmaceuticals and Alvotech have introduced SIMLANDI® (adalimumab-ryvk) injection in the U.S., an interchangeable biosimilar to Humira®. This new treatment option addresses various autoimmune conditions, such as rheumatoid arthritis, Crohn's disease, and juvenile idiopathic arthritis. SIMLANDI® offers a cost-effective alternative to Humira®, maintaining similar efficacy and safety while potentially reducing patient and healthcare system costs. Read the full article at https://lnkd.in/eXgAnP8y

$LLY - Eli Lilly wins US approval for drug to slow Alzheimer’s disease -Lilly's drug, branded as Kisunla™ (donanemab-azbt), is approved by the FDA. -Kisunla to cost $12,522 for 6 months course -Kisunla to cost $695.65 per vial Per Eli Lilly Press Release: -Kisunla slowed cognitive and functional decline by up to 35% compared to placebo at 18 months in its pivotal Phase 3 study and reduced participants' risk of progressing to the next clinical stage of disease by up to 39%  -Kisunla is the first and only amyloid plaque-targeting therapy that used a limited-duration treatment regimen based on amyloid plaque removal; nearly half of study participants completed their course of treatment with Kisunla in 12 months -Once-monthly infusions of 30 minutes reduced amyloid plaques on average by 84% compared to the start of the study

#ACR23: Post Hoc Analysis Reveals #CertolizumabPegol's Better Efficacy in #RheumatoidArthritis vs #Adalimumab with High Rheumatoid Factor Levels https://lnkd.in/gETjEQ-c #ACR2023 #pharma #pharmaceutical #Science #news #media #digitalhealth #patientcare #BREAKING #healthcare

#ACR23: Post Hoc Analysis Reveals #CertolizumabPegol's Better Efficacy in #RheumatoidArthritis vs #Adalimumab with High Rheumatoid Factor Levels https://lnkd.in/g-Ga-jh7 #ACR2023 #pharma #pharmaceutical #Science #news #media #digitalhealth #patientcare #BREAKING #healthcare