Pharma Now - Empowering Pharma Leadership’s Post

Regulatory Milestone: minimal residual disease (MRD) has been recognized by the FDA's advisory committee as an 'approvable' endpoint for accelerated approval in Multiple Myeloma. Similar to how PFS has become standard as a surrogate for OS in solid tumors, it is nice to see MRD as an acceptable target to help get breakthrough therapies to patients faster. https://lnkd.in/gDc4cn4E

𝐑𝐞𝐠𝐞𝐧𝐞𝐫𝐨𝐧 𝐚𝐧𝐝 𝐒𝐚𝐧𝐨𝐟𝐢 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞 𝐅𝐃𝐀 𝐀𝐩𝐩𝐫𝐨𝐯𝐚𝐥 𝐨𝐟 𝐊𝐞𝐯𝐳𝐚𝐫𝐚® 𝐟𝐨𝐫 𝐏𝐨𝐥𝐲𝐚𝐫𝐭𝐢𝐜𝐮𝐥𝐚𝐫 𝐉𝐮𝐯𝐞𝐧𝐢𝐥𝐞 𝐈𝐝𝐢𝐨𝐩𝐚𝐭𝐡𝐢𝐜 𝐀𝐫𝐭𝐡𝐫𝐢𝐭𝐢𝐬 (𝐩𝐉𝐈𝐀) Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) Regeneron Pharmaceuticals, Inc. and Sanofi Sanofi have announced the FDA approval of Kevzara® (sarilumab) for treating patients weighing 63 kg or greater with active polyarticular juvenile idiopathic arthritis (pJIA), a form of arthritis that affects multiple joints simultaneously. "Polyarticular juvenile idiopathic arthritis can be a painful and disruptive disease for children," said George D. Yancopoulos, M.D.george yancopoulos, Ph.D., President and Chief Scientific Officer at Regeneron. "Kevzara provides a new FDA-approved treatment option to help these vulnerable patients." The approval is based on well-controlled studies and pharmacokinetic data from adults with rheumatoid arthritis, as well as a study in pediatric patients with pJIA. pJIA symptoms include joint pain, stiffness, and swelling, which can lead to permanent joint damage and delayed growth without adequate treatment. No new adverse reactions were identified in the pJIA population compared to the rheumatoid arthritis population. Common adverse reactions included nasopharyngitis, neutropenia, and upper respiratory tract infections. "This approval brings a new treatment option with an established safety profile to pediatric patients with pJIA," said Brian Foard Brian Foard, Executive Vice President, Head, Specialty Care at Sanofi. "We are committed to supporting younger patients with this chronic condition." Sanofi and Regeneron offer support services through KevzaraConnect®, helping eligible patients access the medicine and receive needed support. #Regeneron #Sanofi #Kevzara #pJIA #FDAApproval #ArthritisTreatment #Biopharma #HealthcareInnovation #PediatricCare #Rheumatology #PharmaNews

🌟 Essential Reminders for Oncology Clinical Pharmacists 🌟 In antiemetic protocols, understanding key drug-drug interactions with NK-1 inhibitors like aprepitant is crucial. Here are four vital points to keep in mind: 1️⃣ Dexamethasone Adjustment: - Apprepitant may elevate Dexamethasone AUC. - Consider a 40% dosage reduction on day 2 or 3 if administered orally. No adjustment needed for IV administration due to first-pass metabolism. 2️⃣ Oral Contraceptives Alert: - Effectiveness of oral contraceptives may be diminished. - Recommend alternative birth control methods for women of childbearing age during aprepitant use. 3️⃣ Warfarin Interaction: - Potential decrease in INR (clinically significant). - Advise INR check 7-10 days post-completion of a 3-day aprepitant course. 4️⃣ Caution in Lymphoma Patients: - Especially in R-CHOP protocol recipients. - Studies indicate increased neuropathy risk with aprepitant in R-CHOP patients, as aprepitant acts as a CYP3A4 inhibitor. Stay informed, stay vigilant. Your expertise ensures optimal patient care! 💊👩⚕️ #ClinicalPharmacy #OncologyCare #DrugInteractions #PharmacyAlerts #NadaGinawi

If you are attending #ASCPT2024, stop by my poster PII-207 - PIPERACILLIN PHARMACOKINETICS AND TARGET ATTAINMENT IN PEDIATRIC LIVER TRANSPLANT RECIPIENTS

Our team at GeneDx has worked SO hard to launch this exciting new program for patients, clinicians, and biopharma partners, and I'm honored to be involved in an initiative that increases access to Whole Exome Sequencing (#WES) for pediatric #epilepsy patients. I'd love to chat if you want more information! #raredisease #drugdiscovery #exome #genetictesting

Time is brain. Time is muscle. The list goes on. The goal of this #epilepsy Patient Access Program is to get the right test to the right patients at the right time. #GeneDx #DEE #GeneticTesting #WES #Exome #RareDisease #DrugDevelopment #ChildNeurologyFoundation #Dravet #LennoxGastaut

With only 6 products evaluated and 1 interventional procedure guidance, June was the slowest month for NICE in terms of the published guidelines. Hematology and Oncology were the main disease areas impacted by the published guidance.  Last month NICE recommended 4 pharmaceutical treatments for reimbursement across three therapeutic areas: Orthopaedics, Cardiovascular and Haematology.  Two products (Baricitinib and Nivolumab) had their appraisal terminated due to non submission of the data by the company. Both products were seeking indications in young patients.   In Summary: 📆 In June 2024 NICE published 7 set of guidance. One being an interventional procedure guidance, and the other six correspond to single technology assessment. 📈 NICE published across 5 different therapy areas: Cardiovascular, Haematology, Immunology, Oncology and Orthopaedics. 🌐 4 treatments were recommended, three of them with patient access schemes (simple discounts) #NICE #pharma #HTA #haematology #immunology #psychiatry #ophthalmology #metabolism #oncology #infectiousdisease #respirology 

The wide range of topics in the edition includes: A special feature on haematology. ✔️ New therapies for chronic lymphocytic leukemia ✔️ Research by Greek doctors at the European Hematology Congress ✔️ Hematological malignancies in pregnancy ✔️ Large inequalities in access to new haematological therapies at international level 🛈 Still: National Cancer Action Plan Steps and gaps for planning in Greece Innovation Fund for new medicines What the University of West Attica's proposal includes 8th Health Innovation Conference Innovation in Health: Catalyst for improving the NHS Patients Forum 2024 Patient-centred evidence in health decision-making The pharmaceutical industry in Europe in numbers How key market figures evolved in 2024 👉 Find out about FULL PACK or DIGI PACK subscriptions: https://lnkd.in/d6B2457a #health #pharma #hematology #cancer #healthcare #patients #corporate #magazine

The wide range of topics in the edition includes:   A special feature on haematology.  ✔️ New therapies for chronic lymphocytic leukemia  ✔️ Research by Greek doctors at the European Hematology Congress  ✔️ Hematological malignancies in pregnancy  ✔️ Large inequalities in access to new haematological therapies at international level    🛈 Still:   National Cancer Action Plan  Steps and gaps for planning in Greece    Innovation Fund for new medicines  What the University of West Attica's proposal includes    8th Health Innovation Conference  Innovation in Health: Catalyst for improving the NHS    Patients Forum 2024  Patient-centred evidence in health decision-making    The pharmaceutical industry in Europe in numbers  How key market figures evolved in 2024      👉 Find out about FULL PACK or DIGI PACK subscriptions: https://lnkd.in/d6B2457a   Virus.com.gr #health #pharma #hematology #cancer #healthcare #patients #corporate #magazine

This "Hot Topic" presentation sheds light on the pediatric cytogenetic testing at Mayo Clinic Laboratories. Learn how these tests impact pediatric patients with hematologic malignancies. Listen now. https://bit.ly/3PoN1HQ