Blueprint Medicines’ Post

Very tough week for the Parkinson's community with back-to-back failures of Roche's prasinezumab and UCB's minzasolmin, both targeting alpha-synuclein proteins: https://lnkd.in/dHfwzyjg A lot of data remains to be unpacked, but prasinezumab now qualifies as one of the most heavily studied Parkinson's drugs ever. Over the past 11 years, it has been tested in >900 patients, including some who have been on it for >4 years in an open-label extension. In 2025, we’ll join the rest of the Parkinson's community in reflecting on what these results teach us about alpha-synuclein’s role in Parkinson’s disease—and, more broadly, how to improve the process of drug development for Parkinson’s going forward. One big missing piece is the lived experience of patients who participated, especially those in the 4+ year open-label extension. I hope their stories emerge in the coming months. 🧠

In 2024, the U.S. Food and Drug Administration approved groundbreaking treatments addressing high unmet needs across multiple therapeutic areas—including several for liver disease. In an article from Citeline, ZS Principal Emily Mandell discusses the challenges companies face in ensuring these innovations effectively reach patients. Her insights shed light on market readiness, streamlining care and bringing these breakthroughs to those who need them most. Read the full article here: https://bit.ly/3Cvo8a7

In 2024, the U.S. Food and Drug Administration approved groundbreaking treatments addressing high unmet needs across multiple therapeutic areas—including several for liver disease. In an article from Citeline, ZS Principal Emily Mandell discusses the challenges companies face in ensuring these innovations effectively reach patients. Her insights shed light on market readiness, streamlining care and bringing these breakthroughs to those who need them most. Read the full article here: https://bit.ly/3Cvo8a7

In 2024, the U.S. Food and Drug Administration approved groundbreaking treatments addressing high unmet needs across multiple therapeutic areas—including several for liver disease. In an article from Citeline, ZS Principal Emily Mandell discusses the challenges companies face in ensuring these innovations effectively reach patients. Her insights shed light on market readiness, streamlining care and bringing these breakthroughs to those who need them most. Read the full article here: https://bit.ly/3Cvo8a7

In 2024, the U.S. Food and Drug Administration approved groundbreaking treatments addressing high unmet needs across multiple therapeutic areas—including several for liver disease. In an article from Citeline, ZS Principal Emily Mandell discusses the challenges companies face in ensuring these innovations effectively reach patients. Her insights shed light on market readiness, streamlining care and bringing these breakthroughs to those who need them most. Read the full article here: https://bit.ly/3Cvo8a7

With over 7,000 rare diseases affecting approximately 300 million people globally, the collective burden of these conditions is profound. When it comes to rare disease clinical development, Perspectives covers a wide range of topics. Here are 2024’s top posts: Orphan Drug Designation for Rare Diseases https://hubs.ly/Q030pVy00 Understanding Market Exclusivity for Orphan Drug Products https://hubs.ly/Q030pVJ70 Quantitative Strategies for Rare Disease Clinical Trials https://hubs.ly/Q030pVzL0 Sample Size Re-Estimation for Rare Disease Clinical Trials https://hubs.ly/Q030pVPL0 Thank you to Natalia Mühlemann, Boaz Adler, Valeria Mazzanti, and the Strategic Consulting team for sharing your insights!

New results from SQUEEZE and REMEDY - Center for Treatment of Rheumatic and Musculoskeletal Diseases show that adalimumab levels above 4.0 mg/L are associated with remission throughout the first year of adalimumab therapy in RA patients. https://lnkd.in/dFdA5ab5 Using this target level, we are currently running the #RA-DRUM trial to assess the role of proactive therapeutic drug monitoring of adalimumab. Espen A. Haavardsholm Johanna Elin Gehin Daniel Aletaha Oslo universitetssykehus

An interesting perspective from a leader in the biotech space on the FDA's accelerated approval pathway for medicines with potentially minor benefits for the most severe diseases. Despite controversies, this pathway has driven important momentum. Learn more in this BioSpace opinion piece: https://lnkd.in/etDX-_Rv

Unfortunately, even with fee reductions and other incentives, such as additional patent protection, it often doesn't make commercial sense to develop a drug for a rare disease.     As a result, around 95% of rare conditions lack a regulatory-approved treatment*.   This needs to change, argues our CEO, Simon Estcourt. In this article for RARE Revolution Magazine, he discusses the current considerations for conducting clinical trials for rare diseases, approaches for overcoming challenges and how he thinks the rare disease landscape will evolve in the coming years. You can read Simon’s thoughts on page 60… *The Lancet Group #RareDiseases #CRO #ClinicalResearch #MedicalResearch #ClinicalTrials

There's still time to register for this expert webinar! » https://bit.ly/3wEFJta The world of drug development is evolving with the increased acceptance of real-world evidence (RWE). Disease registries, one of the most clinically rich sources of real-world data (RWD), are at the forefront of this change. These registries are purpose-built for research, allowing the collection of fit-for-purpose data, especially in rare diseases. Register for this expert webinar to gain insights into how RWE and RWD from registries enhance clinical development, approval and post-marketing analyses, particularly for rare diseases.