AtriCure, Inc.’s Post

Exciting news from Geron Corporation. The FDA Oncologic Drugs Advisory Committee has voted 12 to 2 in favor of the clinical benefit/risk profile of imetelstat for treating transfusion-dependent anemia in adult patients with low-to-intermediate-1 risk Myelodysplastic Syndromes (LR-MDS) who have not responded to, or are ineligible for erythropoiesis-stimulating agents (ESAs). This marks a significant step forward in the treatment of hematologic malignancies. Read the full article here: https://bit.ly/3IEok6j #CancerResearch #MDSResearch #MDSTreatment #ClinicalTrials #ClinicalResearch

A year after initially rejecting it, the FDA has now granted approval for Ascendis Pharma’s once-daily injectable YORVIPATH for the treatment of hypoparathyroidism. Previously known as TransCon PTH, YORVIPATH (palopegteriparatide) has been authorized for use in adults with this condition. The FDA’s decision followed a review of clinical data from Ascendis’ New Drug Application, which included results from the global Phase II PaTH Forward and Phase III PaTHway trials. This approval comes at a critical time for hypoparathyroidism patients, as Takeda Pharmaceutical is set to withdraw the only current medication for this rare disorder by the end of the year. With the existing drug soon to be unavailable, there has been significant anticipation for an alternative treatment, making the FDA’s approval of YORVIPATH particularly timely, albeit later than expected. Ascendis Pharma will face competition from other companies such as Amolyt Pharma, Extend Biosciences, Inc., EnteraBio, MBX Biosciences, Inc., and others, who are also advancing their lead treatments. Stay informed about how YORVIPATH's approval might impact the treatment landscape for hypoparathyroidism @ https://lnkd.in/gjwidtkh

NRX-101 has been awarded Breakthrough Therapy Designation, Fast Track Designation, a Biomarker Letter of Support, and a Special Protocol Agreement by the FDA for treatment of suicidal bipolar depression. It is the only oral medication to have demonstrated reduced suicidal ideation in patients with bipolar depression, a lethal disease that claims the lives of one in five who live with it. "This is the first clinical trial, to the company's knowledge, conducted among patients with suicidal bipolar depression in the outpatient setting. Our previous trial measured the ability of NRX-101 to maintain the anti-depressant and anti-suicidal effect of ketamine administered in the hospital setting. These patients, whose clinical need is urgent and extraordinary have routinely been excluded from the clinical trials of all previously-known anti-depressant drugs. said Dr. Jonathan Javitt, Founder, Chairman and Chief Scientist of NRx Pharmaceuticals. Although there were patients whose depression worsened and required hospitalization (we don't yet know whether they were on NRX-101 or comparator), patient safety was maintained, and no trial participant suffered a serious unexpected adverse outcome. #depression #clinicaltrial #breakthroughdrug #innovation #fasttrackapproval #fda

Drug treatments for critically ill #COVID19 patients in #ECMO?? PK is affected! Ex vivo study on an adult #ECLS model aimed to measure extracorporeal circuit effects on concentrations of remdesivir, nafamostat, tocilizumab: 💉 relative to baseline, study drug recoveries in both controls & circuits at 6h significantly lower for remdesivir (32.2% - 12.4%), nafamostat (21.4% - 0%) Results confirm significant circuit drug loss for highly protein-bound & lipophilic drugs. Reduced concentrations could be a clinical concern, and alternative dosing strategies should be considered. Particularly, remdesivir and nafamostat may need dose adjustments. ASAIO Journal 🖇️ https://bit.ly/3x1BtnV

A Game-Changer for Neuromyelitis Optica Spectrum Disorder Treatment: FDA Approves Ultomiris The FDA has approved Ultomiris for the treatment of adults with neuromyelitis optica spectrum disorder. The approval was based on positive results from a Phase III trial, in which Ultomiris demonstrated a significant reduction in relapse risk. Marc Dunoyer, CEO of Alexion, highlighted the potential of Ultomiris to transform the treatment landscape for this condition. The approval of Ultomiris in the US follows previous approvals in Japan and the EU. NMOSD is a rare condition that primarily affects women. Ultomiris is a long-acting C5 inhibitor that has shown promising results in reducing relapses. Overall, the approval of Ultomiris offers hope for improved outcomes for patients with NMOSD. For more details please click the link! https://lnkd.in/dyeswHtE #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

STAT+: Pharmalittle: We’re reading about Zepbound trial results, an Alnylam heart drug, and more

NICE Approves Etrasimod for Ulcerative Colitis: A New Hope for Patients Aged 16 and Over The National Institute for Health and Care Excellence has endorsed the use of Etrasimod for ulcerative colitis patients who have not responded well to conventional treatments. Etrasimod, sold under the name Velsipity by Pfizer, has shown promising results in clinical trials and is a cost-effective option. The guidance includes a commercial arrangement with the NHS to offer discounts on the drug. NHS England and integrated care boards are required to ensure availability of Etrasimod within a specified timeframe. For more details please click the link! https://lnkd.in/d85SBxXY #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

STAT+: Pharmalittle: We’re reading about Zepbound trial results, an Alnylam heart drug, and more

AR882, a selective URAT1 inhibitor, for gout was given Fast Track designation by the FDA for the potential treatment of clinically visible tophi in patients with gout. In the US 13 mill have gout & 2 mill have visible tophi. AR882 was presented at EULAR24 https://buff.ly/3AqHvQ8 https://buff.ly/4cA4BRG

AR882, a selective URAT1 inhibitor, for gout was given Fast Track designation by the FDA for the potential treatment of clinically visible tophi in patients with gout. In the US 13 mill have gout & 2 mill have visible tophi. AR882 was presented at EULAR24 https://buff.ly/4dv4x7c https://buff.ly/4dB2cYA