Zevra Therapeutics’ Post

BREAKING NEWS We are delighted to announce that the Pharmaceutical Benefits Advisory Committee (PBAC) has today recommended Trikafta® for children aged 2 to 5 years who have at least one F508del mutation in the CFTR gene. This is a positive step in the right direction and builds on the two previous decisions to recommend and list Trikafta for those 12 and above in April 2022, and more recently 6 to 11-year-olds in May 2023, both of which now have access to Trikafta through the Pharmaceutical Benefits Scheme (PBS). There are approximately 300 young children in Australia that stand to benefit from this decision. The recommendation now heads to the Federal Government for review and a decision as to when it will be added to the PBS. We know that early access is vital; reducing infections, hospitalisations and increasing quality of life; and we urge all stakeholders to expedite this important listing on the PBS. We appreciate that this recommendation was reached promptly and thank all who advocated for this important change. 🎉 #CFTR #Trikafta #PBACDecision #CysticFibrosis

The role of companion diagnostics in the development of targeted therapeutics in hereditary diseases CDx have enormous potential to advance treatment in other areas, such as hereditary diseases. Germline testing offers a compelling way forward to identify the patients with a hereditary disease who may benefit from new compounds. This can lead to a significant improvement in treatment outcomes. Join our expert speakers from QIAGEN, Neuron23 and Helix and discover how CDx can de-risk your clinical development programs in hereditary diseases. https://lnkd.in/dvdmyxSg #drugdiscovery #drugdevelopment #pharma #biotech #personalizedmedicine #medicine

Longitude portfolio company Inozyme Pharma, which focuses on developing novel therapeutics for rare diseases, recently shared that the U.S. FDA has granted Fast Track designation to INZ-701 for the treatment of ABCC6 Deficiency. INZ-701 is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. In pre-clinical studies, the experimental therapy has shown potential to prevent pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in devastating genetic disorders such as ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. Learn more about INZ-701 and this major milestone: https://lnkd.in/er5Csmbf #biopharma #rarediseases

FDA recently approved the 50th biosimilar drug product, a major milestone in reducing healthcare cost and expanding patient access to life-changing biotheraputics. A biosimilar is a biological drug product (biologic) that is highly similar to, and has no clinically meaningful differences from, an existing FDA-approved biologic (also called the reference product). Biosimilars are now approved for 15 different reference products, and treat illnesses like rheumatoid arthritis, inflammatory bowel disease, some cancers, psoriasis, diabetes, macular degeneration, osteoporosis, and more. Here is a list of FDA-approved biosimilars: https://lnkd.in/ewspn2B3

For #RareDiseaseDay2024, here's a round-up of the latest breakthroughs in the treatment of rare disease from Drug Discovery World. #rarediseaseday #raredisease #drugdiscovery https://lnkd.in/exgtVJFz

🙌 Raising awareness of DPD / DYPD deficiency Fluorouracil (5-FU) and its prodrug Capecitabine/Xeloda are chemotherapy agents with a narrow therapeutic index. Approximately 5-7% of patients carry genetic DPYD variants that cause DPD deficiency and reduce the metabolic elimination of 5-FU. In the US, the death rate is estimated to be between 700 and 1,400 annually. You can significantly reduce the risk of severe toxicity by undergoing a pharmacogenomic test for DPYD. Currently, testing for DPYD is not a standard of care in the US. However, with collaborative efforts, we can make it happen. 🙏 Joanne McIntyre lost a loved one due to DPD deficiency. Let's work together to prevent such tragedies. #pharmacogenomics #DPYD #DPD https://test4dpd.org

#ACROHighlights [Featured Product] 🔬 Overcoming Autoimmune Diseases with Cytokine-Targeted Therapeutics Cytokines are key regulators of immune responses and inflammation. In autoimmune diseases (AIDs), their dysregulation plays a significant role in disease progression. By understanding cytokines, we can develop targeted therapies to improve patient outcomes in AIDs. ACROBiosystems provides comprehensive cytokine solutions to accelerate your autoimmune disease drug development. Discover more: https://smpl.is/97eif #AutoimmuneDisease #Cytokines #TargetedTherapies #DrugDevelopment #Biotechnology #Healthcare #ACROBiosystems

Principle Diagnostics has the solution for frequent falls in older adults. Contact us today to learn more. #prinicplediagnostics #elderlycare #longtermcare #pharmacogenomics #genetics #pgx #genetictesting #medicationmanagement #pharmd #pharmacy #pharmacist #prescriptionmedication #medicationmetabolization #assistedliving #medicationsolutions #precisionmedicine

Massive news in the #genomics theme! Last Friday, the FDA approved the ground-breaking gene-editing therapy, Casgevy, developed by CRISPR Therapeutics and Vertex Pharmaceuticals. This therapy marks a significant advancement as the first gene-editing treatment in the US for severe Sickle Cell Disease (SCD), impacting around 100,000 people in the US and 20 million worldwide. This approval sets the stage for potential advancements in addressing other rare blood diseases, with a PDUFA meeting on March 30, 2024, discussing Casgevy's approval for beta-thalassemia (TDT). A promising step forward in the pursuit of curing genetic disorders. Considering the recent challenges faced by genomics stocks over the past few years, is it now opportune for investors to contemplate allocation? https://lnkd.in/eZCEaXJ3 ARK Investment Management LLC Rize ETF #HealthcareInnovation #GeneEditing #FDAApproval

The incredible weight loss transformations induced by obesity drugs targeting GLP-1 have amplified interest in this pathway to target metabolic diseases. We offer a variety of humanized mouse models to evaluate preclinical efficacy and toxicity of novel therapeutic candidates targeting GLP1R, PCSK9, and other regulators implicated in metabolic diseases such as obesity and hyperlipidemia. Check out our B-hGLP1R mice (https://lnkd.in/eTcsmYay), B-hGLP1R/hGCGR mice (https://lnkd.in/eF2WrC_7) and B-hPCSK9 mice (https://lnkd.in/e3VDXpU5). #metabolicdisease #Obesity #glp1forweightloss #gpcr #GLP1 #PCSK9 #glp1medication #glp1weightloss #MouseModel #pharmacology