Cogstate’s Post

Transformative treatment for ADPKD patients - first in class VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants. ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease. The majority of ADPKD cases are caused by variants in the PKD1 gene, which encodes the polycystin 1 (PC1) protein. These inherited variants lead to a loss of PC1 function that results in cyst growth. VX-407 is a first-in-class small molecule corrector that is designed to target the underlying cause of ADPKD in a subset of patients with PKD1 variants, estimated at ~25,000 (or ~10%) of the overall ~250,000 ADPKD patient population, by restoring function to the variant PC1 protein. Vertex plans to initiate a Phase 1 clinical trials

In 2023, the FDA's Center for Drug Evaluation and Research (CDER) played a pivotal role in bringing groundbreaking drug therapies to patients and consumers. From the approval of the first oral antiviral pill for COVID-19 to advancements in combating opioid overdose, CDER's efforts are transforming patient care. But that's not all! CDER's 2023 approvals cover a wide range of diseases and conditions, offering hope to patients with rare diseases and enhancing treatment options across the board. Curious to learn more about CDER's impactful work? Click the link to read the full information. #HealthcareInnovation #FDAApprovals #Raredisease #PatientCare #InviMedsHealth #ReadMore

There are fewer than 150 individuals living with #BarthSyndrome in the United States - and this #ultrarare disease is devastating. Fewer than 15% of affected individuals survive their fifth birthday, and there’s currently no FDA-approved treatment on the market.   But Stealth BioTherapeutics is a company of firsts when it comes to Barth Syndrome. They were the first to introduce the disease to the FDA; the first to conduct a clinical trial; the first to show potential improvements in how affected individuals feel and function; and the first to submit a new drug application. They recently resubmitted that application with additional supportive data, and hope FDA will, for the first time, review the application to evaluate a first potential therapy for this devastating disease.   Stealth’s story of innovation and frustration is all too common in #rare and #ultrarare drug development, where inconsistency in FDA review can compound challenges and risks already inherent to rare disease drug development.   When it comes to #BarthSyndrome and countless other rare diseases, time is of the essence. The FDA must ensure appropriate application of regulatory flexibility to all #ultrarare disease drug reviews to ensure patients have timely access to therapies.   #OneRareVoice #LifeScience #Healthcare #RareDiseaseWeek

The significance of Regulatory CMC is magnified in the context of Orphan Drug Status due to the urgent need for therapies in rare disease populations. These patients frequently have limited or no treatment options, underscoring the necessity for rapid development and approval of new drugs. The FDA's focused efforts to address this need are evident, with over half of the Center for Drug Evaluation and Research's (CDER) novel drug approvals in 2023 targeting rare diseases. This emphasis highlights the critical role of Regulatory CMC in ensuring the quality, safety, and efficacy of these urgently needed treatments but also underscores the importance of streamlined and efficient regulatory pathways to bring these therapies to patients swiftly. The detailed outcomes and impact of these approvals on rare disease treatment landscapes can be further explored in the specified sections of the recent FDA report, providing valuable insights into the advancements in this vital area of drug development. FDA Enkrisi #FDA #CMC

Today, on May 1st, the beginning of #PWSAwarenessMonth, we're thrilled to announce the release of the Voice of the Patient Report. This timely filing marks a significant moment for the Prader-Willi syndrome community! The report gives crucial insights that amplify the perspective of individuals and families affected by PWS and sheds light on aspects of PWS that may not be fully captured in drug trials alone. Integrating the patient's voice throughout the drug approval process for PWS treatments is essential for the U.S. Food and Drug Administration's (FDA) comprehensive understanding and impactful decision-making. The Voice of the Patient Report was created following last year's PWS Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting and is compiled of testimonials shared during the EL-PFDD meeting, as well as those submitted virtually. PWSA | USA, FPWR, and IPWSO extend heartfelt gratitude to the entire PWS community for contributing to the success of this project. To truly amplify the patient's voice, our entire community must commit to ongoing advocacy efforts. This entails incorporating patient perspectives into all facets of processes and systems, ensuring genuine and enduring change. It's not solely about isolated advocacy initiatives—it's about steadfast dedication to embracing and elevating the voices of those directly affected by Prader-Willi syndrome. You can read the Voice of the Patient Report at https://lnkd.in/gx_Nv-qX.

Explore the FDA's guidance on rare disease drug development! This insightful blog breaks down the key insights recommendations and strategies to advance treatments. Read more: https://lnkd.in/ggHpCvJd #FDAguidance #RareDisease #DrugDevelopment #HealthcareNews

In July, the #FDA approved Eli Lilly’s Kisunla, the newest treatment for Alzheimer’s disease after its approval of Eisai and Biogen’s Leqembi last year. Kisunla does not permanently cure Alzheimer’s or stop its progression, instead, it slows down the process. In a study of more than 1,700 participants, Kisunla slowed symptoms by 35% compared to the control group. Hence, Kisunla would only benefit patients in the early stages of Alzheimer's and has limited results with later-stage patients. Commercial insurance’s coverage of the drug varies on a case-by-case basis, yet Medicare covers the cost of Kisunla as long as the patient’s symptoms and side effects are recorded in an approved database. With more than 140 drugs in trial for Alzheimer’s disease as of 2022, many more new drugs are to arrive on the market. Written by: Ethan Sun Sources: Wall Street Journal

In this week's edition of "IntellaTurn's Weekly Scoop," we're taking a look at recent and upcoming drug approval decisions. Headlines include: Notable approvals | 5 upcoming FDA decisions | Around the industry | Sickle cell disease treatment slow to catch on | and more. Read it all at https://lnkd.in/edesyfTQ.

The FDA is adding a Boxed Warning for Amgen's Prolia (denosumab) subcutaneous injection regarding the risk of severe hypocalcemia in patients with advanced chronic kidney disease. Further information can be found at https://ow.ly/yvOh50QtbYX. #DrugNews #Medication #Pharmacy #ChronicKidneyDisease