Neurotech International Ltd’s Post

In 2023, the FDA approved two new drugs for rare diseases: Trofinetide for Rett syndrome and Vamorolone for Duchenne Muscular Dystrophy. Both have shown promising results in phase 2 and 3 randomized clinical trials, offering hope for patients. Here is a brief summary on those two drugs: https://lnkd.in/es68S4a9 #rarediseaseresearch #drugdiscovery #drugdevelopment

Amazing news for the #Duchenne community! After a few decades of ongoing drug development efforts, the 9th drug has now been approved by either the FDA or the European Medicines Agency for patients living with Duchenne Muscular Dystrophy. The previous 8 drugs belonged to one of 2 classes of drugs, namely, the dystrophin restoration therapies that target specific mutation types or the corticosteroid class that reduces inflammation. The new drug, #Duvyzat (givinostat) is an HDAC inhibitor belonging to a different class. #Givinostat targets the pathogenesis of the disease that leads to muscle loss and can benefit patients regardless of their mutation. #drugapproval #italfarmaco #HDACi #duchennemusculardystrophy

In case you missed it, the European Commission granted orphan drug designation to our treatment for eosinophilic granulomatosis with polyangiitis (EGPA). Read the article from Pharmaceutical-Technology to learn more. #Pulmonology #RareDisease

GC Biopharma (GC녹십자) announced that GC1130A, a therapy for mucopolysaccharidosis type IIIA (MPS IIIA) developed with #NovelPharma, has received fast-track designation from the U.S. FDA. MPS IIIA, or Sanfilippo syndrome, is a rare, fatal genetic disorder in children. GC1130A uses high-concentration protein formulation technology for intracerebroventricular injection, previously used in the approved MPS II therapy, Hunterase. Recognized as an innovative treatment, GC1130A has also been designated an orphan drug and a rare pediatric disease drug by the FDA and EMA. The fast-track status will expedite its development, with clinical trials planned in Korea, the U.S., Japan, and other countries. #GC1130A #FastTrack #FDA #RareDisease #MPSIIIA #SanfilippoSyndrome #Biopharma #InnovativeTreatment #ClinicalTrials #OrphanDrug #RarePediatricDisease #SouthKorea #APAC #RASLifeScienceSolutions #MarketIntelligence #MarketResearch #MarketEntryStrategy #CompetitiveIntelligence #PartnerIdentification #StrategySupport For detailed news: https://lnkd.in/dzCvm_vm Follow our page for more industry updates: https://lnkd.in/de5zNWmK

Congratulations to Dr. Richard Novak, cofounder and CEO of Unravel Biosciences, and 2024 Termeer Scholar. Under his leadership, Unravel Biosciences has just received FDA Orphan Drug Designation for Vorinostat (RVL-001), targeting the rare neurogenetic disorder, Rett Syndrome. This milestone is a testament to the innovative spirit and relentless pursuit of patient-centric solutions that define our Fellows. Richard's work, from rapid drug prototyping to initiating global clinical trials, exemplifies the type of transformative leadership that the Termeer Foundation aims to foster. His efforts not only advance medical science but also embody our mission to empower healthcare innovators to bring new treatments to those in dire need. Join us in supporting leaders like Richard, who are not just creating change but are dedicated to making a meaningful difference in the lives of patients worldwide. #biotech #biotechnology #leadership #STEM #TermeerFoundation #Healthcareinnovation #raredisease #patientcare #lifescience #peopleempoweringcures #connectionsempoweringcures #leadershipmatters #healthcare #mentorship #science #mentor #leader #ceo #termeerfellows

Olezarsen receives Orphan Drug designation from U.S. FDA for familial chylomicronemia syndrome: The FDA has awarded Orphan Drug status to the investigational drug olezarsen for addressing familial chylomicronemia syndrome (FCS), a rare genetic disorder marked by significantly high triglyceride levels and recurring acute pancreatitis (AP). Orphan Drug designation is granted by the FDA to treatments aimed at addressing, preventing, or diagnosing rare diseases or conditions affecting fewer than 200,000 individuals in the United States at the time of designation. FCS impedes the body's ability to metabolize fats, leading to fasting triglyceride levels that can soar to 10 to 100 times above the normal range. Individuals with FCS face a heightened risk of acute pancreatitis (AP), characterized by pancreas inflammation, often requiring hospitalization and posing life-threatening complications. In severe instances, critical organs like the heart, lungs, kidneys, and pancreas may sustain damage.   Explore more about Olezarsen and FCS: #olezarsen #fcs #familialchylomicronemiasyndrome #drugtherapy #lifesciences

🤩 Kicking off 2024 with positive news in the world of Pharmacovigilance with the promising outcomes in a Kidney Disease Trial! 💊 The study’s findings revealed that half of the participants treated had a clinically meaningful reduction by 30% or more in albuminuria. 🌍 The results of this study will help to inform a Phase 3 clinical trial and will be evaluated in 11,000 patient participants worldwide. #pharmacovigilance #drugsafety #clinicaltrial #phase2 #research https://lnkd.in/eVVsbufN

The US Food and Drug Administration’s (FDA) accelerated approval pathway allows approval of investigational drugs treating unmet medical needs based on changes to surrogate measures considered “reasonably likely” to predict clinical benefit. Post approval clinical trials are then required to confirm whether these drugs offer clinical benefit. 👉 Per recent JAMA article on the clinical benefit of cancer drugs granted accelerated approval from 2013 to 2017, 41% (19/46) did not improve overall survival or quality of life in confirmatory trials after more than 5 years of follow-up.   👉 Patients should be clearly informed about the cancer drugs that use the accelerated approval pathway and do not end up showing benefits in patient-centered clinical outcomes.

Imugene Limited (ASX:IMU, OTC:IUGNF) has opened higher on being granted 'Fast Track' designation from the US Food and Drug Administration (FDA) for its MAST (metastatic advanced solid tumours) clinical program — evaluating the safety and efficacy of novel cancer-killing virus CF33-hNIS (VAXINIA).  The FDA’s Fast Track designation is awarded to help important new therapies reach patients earlier. It is designed to facilitate the development and expedite the review of drug candidates to treat serious conditions and fill an unmet medical need.  More at #Proactive #ProactiveInvestors #ASX #OTC #IMU #IUGNF http://ow.ly/Z3R81053UUk

💥 Hot Special Issue of EM journal in 2023! 💡 Topic: Beyond Weed: Clinical Applications of #Cannabis and #Cannabinoids (https://lnkd.in/enX3Q7t4) Four Hot Articles with more than 10,000 reads: ✅ A U.S. Food and Drug Administration perspective on cannabis research and drug development 🔗 https://lnkd.in/gY-hpJWp ✅ A large-scale survey of cannabis use for sleep: preferred products and perceived effects in comparison to over-the-counter and prescription sleep aids 🔗 https://lnkd.in/exTEbXge ✅ Cannabis-based medicinal products (CBMPs) for the treatment of Long COVID symptoms: current and potential applications 🔗 https://lnkd.in/gZDEqJGT ✅ Cannabis use in cancer patients: acute and sustained associations with pain, cognition, and quality of life 🔗 https://lnkd.in/gr_KQhMe ⭕ Follow me for more special issue information.