MND Scotland’s Post

The US FDA Accepts Incyte’s BLA for Axatilimab with Priority Review to Treat Chronic Graft-Versus-Host Disease #incyte #axatilimab #chronicgraftversushostdisease #regulatory #bla #acceptance #usfda #priorityreview #phase2 #agave #trials #safety #efficacy #tolerability #agave201 #ash23 #pediatricpatients #systemictherapy

EMA Recommends Agilus for Malignant Hyperthermia: A Leap Forward in Rare Disease Treatment The European Medicines Agency (EMA) has recommended the approval of two new medications for rare conditions. Agilus is a new treatment for malignant hyperthermia, a life-threatening disorder characterized by muscle hyperstimulation and an inability to relax. It incorporates the trusted muscle relaxant dantrolene sodium and targets the ryanodine receptor 1 to alleviate symptoms. Agilus offers improvements in its formulation, making it easier to use and safer for patients. Neoatricon is a new treatment for hypotension in children, utilizing dopamine hydrochloride to enhance blood pressure. Both medications represent advancements in therapeutic development and patient care, and their approval signifies progress in addressing unmet medical needs. For more details please click the link! https://lnkd.in/dudphE6j #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

JP Alliance Board Members had a fun time stuffing gift bags for our sold out Save-a-Life Golf Tournament this past weekend! With the tournament only ten days away, I thought I would share some positive trends* surrounding the opioid epidemic: - Over the last 13 years, the number of opioid prescriptions written by doctors and other health care professionals has declined by nearly 50%. -Queries of state prescription drug monitoring programs, known as PDMPs, reached more than 1.3 billion in 2022, a 197% increase from 2018. -There has been a 200% increase in naloxone dispensed since 2018. Check out our website to learn more about who we are, what we do, and how you can help! https://lnkd.in/gSf6vt9x * Overdose Epidemic Report – 2023, American Medical Association

Transformative treatment for ADPKD patients - first in class VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants. ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease. The majority of ADPKD cases are caused by variants in the PKD1 gene, which encodes the polycystin 1 (PC1) protein. These inherited variants lead to a loss of PC1 function that results in cyst growth. VX-407 is a first-in-class small molecule corrector that is designed to target the underlying cause of ADPKD in a subset of patients with PKD1 variants, estimated at ~25,000 (or ~10%) of the overall ~250,000 ADPKD patient population, by restoring function to the variant PC1 protein. Vertex plans to initiate a Phase 1 clinical trials

Good policy can be a driver of drug discovery and treatments. It’s a point I emphasized during opening remarks at STAT in DC: A Policy Prescription, and a strong reason why Congress needs to pass the ORPHAN Cures Act and restore needed incentives for the research and development of rare disease treatments. My Alexion colleague, SVP for US Business Scott Weintraub, further underscored the importance of the legislation during his fireside chat with STAT Editor Rick Berke. Interested in learning more about the ORPHAN Cures Act? Check out the clip below and visit the Save Rare Treatments website. https://lnkd.in/e2TarKah #STATinDC #raredisease 

Did you get your fill of #pharmacynews this week? Take the #RxRoundup quiz of the week and test your knowledge about the latest stories published by the Drug Topics team from February 25 to March 1. #Pharmacy #QuizOfTheWeek https://lnkd.in/eZ5hesjz

My step-daughter has a rare disease called Ataxia Telangiectasia, which occurs in an estimated 1 out of every 100,000 people worldwide. At #Calyx, we’re proud of the work we do to support the worldwide pharmaceutical industry as they strive to develop cures for the millions of patients afflicted with #RareDisease, today and every day. https://lnkd.in/e43xBv5T #RareDiseaseDay #ATSociety #ActionForAT #AtaxiaTelangiectasia

🎙️The staggering potential for hepatitis C elimination: A call for political will ahead of World #Hepatitis Day (28 July)🎙️ "The ability to eliminate a viral disease is pretty staggering. It's not something that has been done many times in our history," says Dr Joss O’Loan, General Practitioner and Co-Founder of the Kombi Clinic. "We just really need the political desire to achieve the 2030 goal." Despite making early headway towards the 2030 elimination goal, recent progress has stalled in Australia. "We've reached a stage where the prospect of elimination is real," highlights Dr Paul Slade, Country Medical Director at Gilead Sciences. "The challenge for us now is that we're in a steady state." "The time for talk is over, the time for action is now if we are to achieve the elimination goals by 2030," Dr O’Loan emphasises. Link to full podcast: https://lnkd.in/gqxbW8Y4 #WorldHepatitisDay #gilead #hepc #hepatitisc #pharmaceuticals #medicine #pharma #primarycare #generalpractice The Royal Australian College of General Practitioners (RACGP)

Learn more about the latest developments in kidney disease treatment and FDA approvals. #PharmaNews #FDAApproval

A Groundbreaking Milestone in Pustular Psoriasis Treatment with Expanded Approval in China and the US SPEVIGO has received expanded approval for the treatment of generalized pustular psoriasis (GPP) in pediatric and adult patients in both China and the United States. This makes it the first targeted therapy approved to address acute and chronic treatment for GPP. SPEVIGO is a humanized selective antibody that specifically targets the interleukin-36 receptor (IL-36R) to interrupt the inflammatory cascade responsible for GPP. Clinical trial data showed that SPEVIGO significantly reduced GPP flares compared to placebo and had a favorable safety profile. This approval marks a milestone in GPP treatment and offers hope to patients. For more details please click the link! https://lnkd.in/gjxp7X5V #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical