PharmaVoice’s Post

On the 12th of April, the US Food and Drug Administration (FDA)’s Oncology Drugs Advisory Committee (ODAC) voted 12 – 0 to approve minimal residual disease (MRD) as an accepted endpoint for accelerated approval of new myeloma drugs. MRD can supply reliable information on the efficacy of a drug before currently accepted survival indicators. This decision can potentially help patients in the US get faster temporary access to newly developed treatments. As MRD still has limitations, final regulatory approvals will not be based on MRD alone. MPE prepared a Q&A on this topic with details on the main scientific concepts and their limitations, data submitted to the FDA and potential implications of this decision for European patients. Please see more information here: https://lnkd.in/dEMCeqKC

A very important decision from FDA on the use of minimal residual disease in accelerated approvals for new myeloma drugs! If you are interested in this and would like to know more, MPE prepared a Q&A on this topic (details below).

Amylyx Pharmaceuticals is expanding its portfolio with the #acquisition of avexitide, a Phase III ready GLP-1 receptor antagonist, for $35 million. Avexitide targets hyperinsulinemic hypoglycemia in post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). Key Insights - ▶ Unique Mechanism: Unlike #GLP1 agonists, avexitide is an antagonist, designed to stabilize glucose levels by reducing excessive #insulinsecretion. ▶ FDA Designations: Avexitide has Breakthrough Therapy, Rare Pediatric Disease, and #OrphanDrug Designations, indicating strong potential. ▶ Phase III Trials: Amylyx plans to initiate Phase III trials for PBH in early 2025 and engage with experts on HI following promising Phase II results. Questions to Consider - ▶ How will avexitide’s unique mechanism impact treatment for PBH and HI? ▶ What strategies will Amylyx employ to successfully bring avexitide to market? ▶ How can Amylyx’s commitment to rare disease treatments shape future developments in the field? #PharmaInnovation #RareDiseases #AmylyxPharmaceuticals #DrugDevelopment

🌟 Exciting News from the FDA! 🌟 🔍 The FDA’s Center for Drug Evaluation and Research (CDER) has just released its "New Drug Therapy Approvals 2023" report. This comprehensive document, released on Jan 16, 2024, shines a light on the groundbreaking achievements in drug approvals over the past year. 💡 Key Highlights: • CDER approved a remarkable total of 55 novel drugs in 2023. • These drugs target a wide array of conditions, ranging from COVID-19 and RSV to neurological conditions, anemia, and type 2 diabetes. • 🚀 A significant milestone: Over half of these drugs were approved under the orphan drug designation program, focusing on rare diseases. • 🎗️ This includes treatments for conditions like Friedreich’s ataxia, rare cancers such as mantle cell lymphoma, and more. • 🆕 2023 also saw the approval of five biosimilars, bringing new options to the market. 📈 Efficiency and Impact: • CDER’s report also highlights their efficient review process, with 89% of drug approvals meeting the Prescription Drug User Fee Act goal dates. • 🥇 84% of these approvals were achieved on the “first cycle,” and 35 of the 55 novel drugs were approved ahead of other countries. • 🌍 36 drugs benefited from the FDA’s expedited programs. 🔬 A Collaborative Effort: • CDER Director Patrizia Cavazzoni emphasizes the collaborative nature of the approval process, involving expertise from various sectors. • She lauds the dedication of the teams involved, whose efforts significantly impact patient health and well-being nationwide. 👏 As we celebrate these advancements, let's acknowledge the hard work and dedication of everyone at the FDA and the broader medical community. These approvals are not just numbers; they represent hope, innovation, and a brighter future for patients around the world. #FDA #DrugApproval #HealthcareInnovation #Pharmaceuticals #MedicalResearch #PublicHealth

2024-04-01 US FDA grants fast track status to LISCure’s LB-P8 drug for PSC LISCure plans to conduct a Phase II study of investigational drug LB-P8 across multiple sites in the US and Europe. The US Food and Drug Administration (FDA) has granted fast track designation to LISCure Biosciences’ investigational drug LB-P8, for the treatment of primary sclerosing cholangitis (PSC). With the designation, the company will fast track the development of the drug to fulfill the unmet medical needs of people affected by PSC. LB-P8 is designed to treat inflammation and fibrosis as well as to modulate bile acids. LISCure said the LB-P8 live biotherapeutic product (LBP) is the only one of its kind reported to be in clinical development for PSC, a rare and chronic liver disease with no approved treatment options. The drug previously received orphan drug designation for PSC in 2022. https://lnkd.in/g5VjhV-G

Madrigal Pharmaceuticals’ resmetirom, soon available as Rezdiffra, has achieved the milestone of becoming the primary therapy for NASH to receive FDA clearance, after a protracted journey through drug development. Rezdiffra marked a significant milestone as the first medication to achieve both NASH resolution and improvement in fibrosis during a phase III trial. While Rezdiffra benefits from being the first to enter the NASH treatment market, other treatments have shown encouraging early clinical results, aiming to capture a significant portion of the NASH treatment market. To know more about the emerging treatment options in the NASH market, visit: https://lnkd.in/gzYBPRM7 #NASH #NASHtherapies #therapies #madrigalpharmaceuticals #resmetirom

News from First Report Managed Care from Asembia AXS24: The specialty pharmacy pipeline was the subject of one of many informative sessions at the annual Asembia Summit this year. Key insights were shared about current drugs in the pipeline according to their drug designations, the phase of each drug’s development, and projected first launch dates after receiving FDA approval. Access the latest insights from Asembia's AXS24 Summit! #FirstReportManagedCare #FRMC #asembia2024 #axs24 #raredrugs #specialtydrugs #pharmaceuticalpipeline

Thinking about investing in that new drug because the FDA granted accelerated approval? ... "Of the 46 cancer drugs that the U.S. Food and Drug Administration (FDA) granted accelerated approval between 2013-2017, 63% were converted to regular approval even though only 43% demonstrated a clinical benefit in confirmatory trials after more than five years of follow-up, according to a study presented at the American Association for Cancer Research (AACR) Annual Meeting 2024, held April 5-10 ... The study was simultaneously published in JAMA." #FDA #EMA #21USC #21CFR #GMP #cGMP #pharma #pharmainvesting #investing #drugs #drugtreatments #clinicaltrial #clinicalresearch #NDA #ANDA #IND #BLA #approval #acceleratedapproval (https://lnkd.in/gi5wYzWX)

𝐅𝐃𝐀 𝐆𝐫𝐚𝐧𝐭𝐬 𝐏𝐫𝐢𝐨𝐫𝐢𝐭𝐲 𝐑𝐞𝐯𝐢𝐞𝐰 𝐭𝐨 𝐈𝐨𝐧𝐢𝐬' 𝐍𝐞𝐰 𝐃𝐫𝐮𝐠 𝐀𝐩𝐩𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧 𝐟𝐨𝐫 𝐎𝐥𝐞𝐳𝐚𝐫𝐬𝐞𝐧 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) Ionis Pharmaceuticals, Inc. announced that the U.S. FDA has accepted for Priority Review the New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for the treatment of adults with familial chylomicronemia syndrome (FCS). The FDA set an action date of December 19, 2024, and indicated no advisory committee meeting is currently planned for olezarsen. "FCS is a debilitating, rare, genetic disease with no approved treatment options in the U.S.," said Brett Monia, Ph.D. Brett P. Monia, PhD, CEO of Ionis. "The Priority Review underscores the urgent need for a medicine to lower triglyceride levels and reduce life-threatening acute pancreatitis events. We look forward to working with the FDA to bring this potentially breakthrough medicine to patients before the end of 2024." Priority Review is for medicines that may provide significant improvements in treating serious diseases, with FDA action expected within six months. The NDA was based on positive results from Balance, a global, multicenter, Phase 3 study, presented at the 2024 ACC Annual Meeting and published in The New England Journal of Medicine. The FDA granted olezarsen Fast Track designation for FCS in January 2023, and Orphan Drug and Breakthrough Therapy designations in February 2024. Ionis plans additional regulatory filings in the European Union this year. Ionis is also evaluating olezarsen for severe hypertriglyceridemia (sHTG) in three Phase 3 trials – CORE, CORE2, and ESSENCE – all of which completed enrollment in the first half of 2024. Data are expected in the second half of 2025. #IonisPharmaceuticals #Olezarsen #FCS #RareDisease #FDAApproval #OrphanDrug #BreakthroughTherapy #FastTrack

Small molecule drugs were 70% of the drugs selected for price setting in the #inflationreductionact in 2026 and are likely to make up 93% of the drugs selected for price setting in 2027, and 87% in 2028. Small molecule oncology therapies are estimated to be the predominant therapeutic class affected by price setting, in the next three years, followed by small molecule therapies for respiratory conditions and for diabetes. If CMS sets the price at the #inflationreductionact ceiling (conservatively estimated) the expected revenue from those small molecule drugs will be reduced by 28% over 18 years and by 11%-15% for large molecule drugs. Less revenue means less investment in clinical studies and less information about how those medicines can improve human health, analysis with Rajini Yamuna Jayasuriya and Yamini Jena of Charles River Associates