Prevail Therapeutics’ Post

Missed our keynote session “Can you overcome complexity and deliver value for rare disease patients in Europe?" Review the summary

Today, we announced the U.S. FDA has cleared our Investigational New Drug Application for CLN-978 in systemic lupus erythematosus (#SLE).  CLN-978 is the first development stage CD19 T cell engager to receive IND Clearance in #autoimmune diseases. CLN-978 is a novel bispecific T cell engager that targets CD19, offering a highly differentiated approach to deliver the potency of T cell redirecting therapy with off-the-shelf access and convenient dosing through subcutaneous administration.  We are advancing CLN-978 in our global Phase 1 clinical trial for patients living with SLE.   Learn more: https://bit.ly/3zX2F8P

Another important step completed - with some valuable experience of the new(ish) CTIS clinical trial approval system gained along the way!

What challenges do orphan medicines face when entering European markets? In our 4-part series, we explore the key roadblocks to market access for orphan medicines and rare disease treatments: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/e5whjpQQ 2️⃣ Clinical evidence development 👉https://lnkd.in/eW8tD5w9 3️⃣ Economic evidence development 👉https://lnkd.in/ex2fTzKu 4️⃣ Operational challenges 👉 https://lnkd.in/eg5kDAqF Planning to launch an orphan medicine in Europe in 2025? Our experts are here to guide you through the process. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

Supporting awareness is essential in all fields, but when #rarediseases are involved, it is even more important. The main players in the rare diseases field, i.e. patients, clinicians, caregivers, researchers, and the pharmaceutical industry must work together to make sure 'rare' does not mean 'invisible'. Today we are celebrating Global #NiemannPickAwareness Day and, as part of the rare disease community, AZAFAROS B.V. wants to use this opportunity to reinforce our commitment to the development of new treatments for these devastating diseases. #NNPDF, #NPC, #collaboration, #awareness, #rarediseases

I'm pleased to share that we received our 2nd FDA Breakthrough Therapy Designation (BTD) this year for one of our innovative, investigational biologic therapies. The designation, for the treatment of patients with moderate-to-severe Sjögren’s disease (SjD), represents an important milestone for this systemic autoantibody-driven disease that it is believed to impact some three million people in the U.S. where, unfortunately, there are no FDA-approved advanced therapies. Read more about this milestone here: https://bit.ly/4fHGiTM At Johnson & Johnson Innovative Medicine, we never rest in our commitment to change treatment paradigms and transform patients’ lives. #MyCompany #JNJImmunology #SjögrensDisease #Innovation #PatientDriven

#raredisease #drugdevelopment is really hard and inherently risky - and it can be tremendously rewarding! When else do you have a chance to bring the first treatment - and a glimmer of hope - to a community affected by really serious conditions, with incredible unmet medical need, and an almost unprecedented resolve to change the world. Rare disease drug development is a big priority for FDA. Great primer and summary of many of the efforts #CDER is undertaking to help spur rare disease drug development published this week: https://lnkd.in/eZRs3Dsv

Starts Today! LIVE CE series on T2 inflammation's implications for managed care.

Rare diseases, such as sickle cell disease and thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. Our new report provides recommendations for enhancing and promoting rare disease drug development in the U.S. and the European Union by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between the FDA and the European Medicines Agency. "While rare diseases by definition have small patient populations, together, they afflict hundreds of millions of people — meaning very large numbers of people across the world have no drug designed to treat their condition," said Jeffrey Kahn, chair of the committee that wrote the report. Learn more at https://ow.ly/6ME850Tm7wx. #RareDisease #RareDiseases #DrugDevelopment #DrugDiscovery

FDA Oncologic Drugs Advisory Committee (ODAC) meeting voted in favor of J&J's #Carvykti (11-0) and BMS & 2seventy bio's #Abecma (8-3)—recommending both CAR T therapies for the treatment of #RRMM patients. This is something to start a conversation about: https://lnkd.in/eY--MxYT