Canary Regulatory Affairs

FDA Novel Drug Approvals 2024 #medicine That is a wrap for 2024! And another year of relentless drug discovery! There you have it, drug-hunting aficionados, the complete list of 2024 novel FDA-approved drugs, 50 NMEs so far, down from 2023 and up from 2022 (55 & 38 approvals, respectively). Notable first-in-class approvals 💊 Revumenib, a menin inhibitor that disrupts the interaction between menin and MLL proteins, halting cancer cell growth. It is indicated for relapsed or refractory acute leukemia with KMT2A translocation, providing hope to patients with resistant disease. 💉 Zanidatamab-hrii, a bispecific antibody targeting two epitopes of the HER2 receptor, enhancing anti-tumor activity. It is approved for unresectable or metastatic HER2-positive biliary tract cancer, addressing a rare and aggressive condition with limited treatment options. 💊 Acoramidis stabilizes the transthyretin (TTR) protein to prevent misfolding and amyloid plaque deposition. It is used for cardiomyopathy associated with transthyretin-mediated amyloidosis (ATTR-CM), improving outcomes for patients with heart failure due to amyloid deposits. 💉 Landiolol, an ultra-short-acting beta-1 adrenergic receptor antagonist offering rapid and safe heart rate control for supraventricular tachycardia (SVT), with a reduced risk of hypotension—a significant advancement for arrhythmia management. 💊 Crinecerfont provides a novel approach for classic congenital adrenal hyperplasia (CAH), a hormonal disorder. Acting as a corticotropin-releasing factor type 1 receptor antagonist, it reduces androgen production and restores hormonal balance. About 40% of the approved drugs target cancer, including lung cancer, leukemia, and gastric adenocarcinoma. Cardiovascular therapies account for 15%, focusing on conditions like transthyretin-mediated amyloidosis. Neurological disorders account for 10%, addressing diseases such as congenital adrenal hyperplasia. Another 10% tackle infectious diseases with new antibiotics for resistant infections. The remaining 25% address diverse conditions, including metabolic and genetic disorders. My drug of the year – what is yours? Cobenfy, a groundbreaking schizophrenia treatment, combines xanomeline, a muscarinic receptor agonist, with trospium chloride, a peripheral antagonist, to uniquely target central nervous system receptors while reducing side effects. Schizophrenia, which affects 24 million globally, has seen limited innovation for decades. By targeting muscarinic rather than dopamine pathways, Cobenfy offers improved efficacy and fewer side effects, such as weight gain and movement disorders. As a novel class of medication, it provides a vital option for treatment-resistant patients, solidifying its status as my drug of the year. Follow Chris De Savi or ring the bell 🔔 icon to be notified of all his posts. #pharmaceuticals #healthcare

🌟What an enriching and inspiring week spent connecting with industry leaders and innovators at #AusBioInvest, at an #OrdMinnett presentation on philanthropy, and #AusBiotech! At #AusBioInvest, I had the privilege to hear insights from early-stage, innovative companies across the #Pharma💊,#Biotech🧬and #MedTech 💉sectors. Special thanks to Rhenu Bhuller Buller for curating such a valuable program for investors💲 in the sector. A heartfelt thank you to Julian W. Reeves CFA Reeves at #OrdMinnett for the opportunity to listen to Maureen Wheeler, Co-founder of #LonelyPlanet🪐, and her unique perspective on the power of giving. #AusBiotech was the week's highlight. Huge thanks to Aditya Yadav, PhD and Chris Ozga for all your support in preparing for and attending a productive meeting💼schedule. And to Jenny Petering and #FB Rice, thank you for hosting a pre-dinner event which always facilitates stenghtening connections. Thank you also to all the companies we met—too many to list—for their time, for sharing their background and offerings and exploring potential collaborations. Finally, I would like to acknowledge Rebekah Cassidy, Rosanne Hyland, Tanya Daw, the entire #AusBiotech team, and the volunteers whose dedication ensured a smooth, engaging experience for all. Looking forward to seeing how these connections evolve.🍀

The Canary Regulatory Affairs team is excited to attend #AusBio24. If you'll be there, we'd love to connect. Reach out to us via #AusPartnering to schedule a meeting. Whether you need support with product development, manufacturing, non-clinical and clinical studies design, or with your regulatory strategy, we’re here to help. Specializing in #biotechnology, #pharmaceuticals, and #medicaldevices, we provide tailored scientific and #regulatory guidance. With extensive experience working with global authorities, we streamline the approval process from early discussions to final review. Let’s connect at #AusBio24 and explore how we can support your success!

#MentalHealthMatters #FDAApproval #SchizophreniaTreatment #PharmaInnovation #BMS #MentalHealthBreakthrough #CanaryRegulatoryAffairs #RegulatorAffairs #FDA 

This morning Aditya Yadav, PhD and I have attended Lecture Four of the Second Series on #mRNA technology organised by the #BiomelbourneNetwork focusing on the regulation of the mRNA platform. The session was adeptly facilitated by Amanda Caples, Victoria's Chief Scientist. Carolyn Tucek-Szabo Ph.D. gave a clear historical summary of the platform's development and described how a working group of leading Australian scientists, including herself, Prof John Skerritt, Brett Sutton AO and Terry Nolan proposed a novel approach to regulating mRNA products. By leveraging similarities in the manufacturing process, analysis and preclinical and clinical development of different products, bridging studies can minimise the regulatory burden without compromising safety and efficacy. The proposed approach aligns with initiatives currently under development by the #FDA, #EMA and the #WHO. Prof Skerritt elaborated on the key considerations driving regulatory discussions, which must also account for advancements towards individualised neoantigen therapies in oncology and mRNA therapeutics in rare diseases, where patient populations are inherently limited. It was exciting to learn about these recent and complex developments in regulatory science and reassuring to hear that scientifically driven regulatory flexibility is being exercised.

What a week! ⭐ It was lovely to catch up with Leab Sek of Moderna and Kim Steel of SAPRO Consulting Pty Limited at the annual BioMelbourne Network lunch 🍽. I then had a very informative week in Adelaide attending #AusMedTech and #AusMedtechInvest 💲 with Chris Ozga. Thank you, Chris for sharing your expertise in #MedicalDevices. Thank you to all those we met (too many to name, but you know who you are 😊) for sharing your time and insights. I look forward to the opportunity for further collaborations. It was great to hear an update on the #Vaxxas story from David Hoey and to learn about the potential of the #TekCyte technology presented by Antonio (Tony) Simula amongst many other notable presentations💡. The always impressive Sarah Meibusch facilitated several panel discussion making them lively and engaging 🎤 and Rosanne Hyland masterfully directed the proceedings with grace and professionalism 👩🏫 . A special thank you to #PharmOut and Trevor Schoerie for the opportunity to present at the #PharmaandDevicesForum2024 to wrap up the week. It was so nice to reconnect with old colleagues Wayne Ingram and Tanja Varglien and introduce them to new ones. Thank you to Carl Bufe for the introduction and to Aditya Yadav, PhD for representing Canary Regulatory Affairs at the event and for the support. 🙏 It was fascinating to hear the #Polyactiva development story masterfully presented by David Valade. Congratulations to the entire team.