Claritas Rx’s Post

argenx is working toward a speedy FDA decision on using its drug Vyvgart to treat chronic inflammatory demyelinating polyneuropathy (CIDP) by using a priority review voucher. The company is working to bring a treatment to CIDP patients as quickly as they can following a trial that indicated a 61% reduction in risk of relapse over placebo. A subcutaneous version of Vyvgart was approved earlier this year, which positions it as a flexible, game-changing treatment for this set of autoimmune diseases. By making it easier for patients to receive the drug, Argenx should be able to help a broader population. https://hubs.la/Q027nljQ0

Missed our keynote session “Can you overcome complexity and deliver value for rare disease patients in Europe?" Review the summary

Biosimilars Real World Evidence (RWE) plays a key role in addressing important questions on Biosimilars long term effectiveness, safety and immunogenicity while also providing additional confidence on the outcomes of switching patients from the reference biological to the Biosimilars.

Today I was able to present on one of my favorite topics: celiac disease! As someone who suffers from celiac disease, this project gave me a new hope for the future. So many clinical trials right now are looking promising for a new treatment that does not involve adherence to a gluten free diet. These trials will change lives and I hope they succeed! In this presentation, I focused mainly on the KAN-101 drug trial. I also briefly discussed TPM502, DONQ52, and TAK-101. I am thrilled about all of these trials and can’t wait to see how things play out. More information can be found on clinicaltrials.gov

Drug repurposing has often been suggested as a logical solution for targeting rare disease therapies. This strategy is a particularly attractive method to develop therapies in a more efficient and cost-effective way. The International Rare Diseases Research Consortium (IRDiRC) has developed a new guidebook to invigorate drug repurposing for rare diseases. #drugdiscovery #repurposing #drugdevelopment

“In 2022, two landmark studies showed that EBV is the leading cause of MS, with prior EBV infection increasing susceptible individuals’ risk of developing MS 32-fold.”   In part 8 of Pharma Intelligence, now Citeline ‘s Scrip Asks series, CMO Manher (AJ) Joshi provided his thoughts on what’s in store for R&D progress in 2023, including the possible therapeutic implications of targeting EBV-infected immune cells in #MS. Read more from AJ and other industry leaders here: http://bit.ly/3FCOR30  

This TNF-blocking biologic prescription drug helps treat a wide variety of autoimmune diseases with very few adverse reactions. Because of this, it has quickly become the most profitable prescription drug in the world. Autoimmune diseases cause the immune system to attack its own healthy cells. Humira helps block that process from occurring, making it a vital tool against rheumatoid and psoriatic arthritis, Crohn’s disease, ulcerative colitis, and other autoimmune diseases. In 2020, Humira’s global revenue reached almost $20 billion, with nearly that entire amount coming from the U.S. Stay tuned for more updates and photos from this exciting venture on our LinkedIn page! #export #Innovation #dailyupdate #pharmaceuticalindustry 

Etrasimod is a new generation of oral S1P receptor modulator, which can specifically bind to S1P receptors 1, 4, and 5. It is being studied for a range of immune inflammatory diseases, including ulcerative colitis, Crohn's disease, atopic dermatitis, eosinophilic esophagitis and alopecia areata. At present, the highest stage of research and development of the drug is to apply for marketing, which is used to treat ulcerative colitis and is expected to be approved by FDA in this month. Chemenu has been working to develop more compounds for drug discovery. Here come the related building blocks we can provide: https://lnkd.in/gckhBZS9 Etrasimod.html 👇 #Chemenu #Etrasimod #S1Preceptor #immuneinflammatorydiseases #ulcerativecolitis #Crohn'sdisease #atopicdermatitis #eosinophilicesophagitis #alopeciaareata #FDA #buildingblocks

One Life, One Liver Under the theme, “One Life, One Liver,” this year's World Hepatitis Day will highlight the importance of the liver for a healthy life, and the need to scale up viral hepatitis prevention, testing and treatment to optimize liver health, prevent liver disease and achieve the 2030 hepatitis elimination goals.   At AXIS Clinicals, we are committed to support the Clinical Development related to Hepatitis treatment. Our collective efforts with Pharma and Biotech companies will ensure a real impact in creating a healthier future.   #OneLifeOneLiver #axisclinicals #Clinicaltrials #Clinicalstudies #worldhepatitisday #AXISClinicals #AXISINDIA #AXISUSA #AXISMEXICO #ClinicalTrials #Bioequivalence #Biostudies #CDM #Clinicaldatamanagement #Clinicalresearch #505B2 #VCStudies #PhaseI #PhaseII #PhaseIII #PhaseTrials #PhaseItoIV #FirstInHuman #Regulatory #clinicaldevelopment #AXISHyderabad #AXISAhmedabad #CRO #PatientPK #ClinicalEndPoint #Clinicaltrials 

C-Path's Rare Disease Clinical Outcome Assessment Consortium today announced the launch of the Rare Disease Clinical Outcome Assessment Resource. The Rare Disease COA Resource provides information on published COAs that have the potential to be used to support efficacy endpoints in treatment trials for rare diseases. The Rare Disease COA Resource seeks to accelerate drug development by freely providing information on published COAs that may have applicability for use in drug development programs across multiple rare diseases. Full details: https://lnkd.in/dvFWu_MS To learn more, visit https://lnkd.in/d8GrwibV  To access the Resource, visit https://rdcoas.c-path.org/ Lindsey Murray Barbara Brandt #CPath #FDA #drugdevopment #raredisease #datasharing #collaboration #clinicaloutcomes #collaboration #globalhealth