HI-Bio: A Biogen Company’s Post

Yesterday was #RareDiseaseDay. The annual observance highlights how far the #biopharma industry has come and serves as a wake-up call for how far it must still go to meet the needs of #raredisease patients. Despite recent investment and breakthroughs for #rarediseases, experts told BioSpace that helping a much larger patient population will require cross-sector support and desperately needed funding. We looked at some of the investments to date, as well as the biopharma industry’s promising pipelines and what is required to bring more groundbreaking therapies to patients with unmet medical needs. #Patientadvocacy groups have been critical to the development of the first marketed drugs for certain rare diseases, including progeria and Friedreich's ataxia. We spoke with leaders of these groups formed by families coping with rare diseases, providing case studies on their partnerships with biopharma companies. https://lnkd.in/g7R6hU23 #RareDiseaseDay2024 #genetherapy #genetherapies #duchenne #duchennemusculardystrophy #biopharmaceuticals

Yesterday, the FDA announced plans to launch a Rare Disease Innovation Hub that will utilise new and collaborative approaches to expedite the development and approval of safe and effective drugs and biologics that treat rare diseases. One out of every 10 people in the US are affected by a rare disease and around half of these people are children. Many rare conditions are life-threatening, and most do not have approved treatments. The new Hub will work across rare diseases with a focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. It will serve as a single point of connection and engagement with the rare disease community; enhance agency collaboration to address common scientific, clinical and policy issues related to rare disease product development; and advance regulatory science. Having supported organisations across the globe with the development of medicines for rare diseases, Boyds warmly welcomes this promising initiative which ultimately aims to improve outcomes for people living with rare diseases by providing new therapeutic options. Find out more: https://lnkd.in/egJKYwvs #rarediseases #fda #innovationhub #medicine

For #RareDiseaseDay2024, here's a round-up of the latest breakthroughs in the treatment of rare disease from Drug Discovery World. #rarediseaseday #raredisease #drugdiscovery https://lnkd.in/exgtVJFz

🇺🇸The FDA is launching a Rare Disease Innovation Hub to enhance the development and approval of treatments for rare diseases. Connecting with patient communities, fostering intercenter collaboration, and advancing regulatory science will be the focus. This initiative co-led by the directors of CDER and CBER is expected to significantly improve outcomes for patients with rare diseases by streamlining the development of new therapies and leveraging cross-agency expertise and existing programs. 🇬🇧 The Medicines and Healthcare products Regulatory Agency (MHRA) has initiatives similar to the FDA's Rare Disease Innovation Hub. One notable initiative is the Rare Therapies Launch Pad (RTLP) announced in the UK’s 2023 Autumn Statement. This program aims to develop a new pathway for the development and access to novel medicines, particularly targeting children with rare and life-threatening conditions. It involves collaboration among Genomics England, the MHRA, Oxford-Harrington Rare Disease Centre, the Association of the British Pharmaceutical Industry (ABPI), and Mila’s Miracle Foundation. Additionally, the UK government has established the Rare Diseases Action Plan, which aligns with the UK Rare Diseases Framework. This plan focuses on improving diagnosis, increasing awareness among healthcare professionals, coordinating care, and enhancing access to specialist treatments. It includes actions like enhancing genomic testing communication, implementing networked models of care, and addressing health inequalities for those with rare conditions. However, the continuity of the UK's Rare Disease Action Plan is a topic of concern with the new Labour government which has not specifically mentioned rare diseases in their manifesto but have committed to advancing health innovation, such as genomics, which is crucial for rare disease diagnostics and treatments.

Developing orphan drugs for rare diseases? At Quest Diagnostics, we understand the challenges of developing treatments for rare diseases. Traditional diagnostic methods have limitations, hindering patient selection and drug development timelines. Read "Navigating the Complexities of Rare Disease – A Quest Diagnostics Perspective" by Juha Koskenvuo to gain valuable insights for Pharma & researchers: https://lnkd.in/ePGwesT9

FDA Rare Disease Innovation Hub FDA take another step to expedite development and approval of treatments for rare diseases. The Food and Drug Administration (FDA) announced the creation of a Rare Disease Innovation Hub to “expedite development and approval of safe and effective drugs and biologics” for rare diseases. What it will do, according to the FDA: ·   “Serve as a single point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations.” ·   “Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development.” ·   “Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.” https://lnkd.in/d7fqn6tB

Vertex Pharmaceuticals to Acquire Alpine Immune Sciences for $4.9 Billion US-based Vertex Pharmaceuticals has agreed to pay approximately $4.9 billion in cash to acquire Alpine Immune Sciences, Inc., a compatriot #biotechnology company focused on discovering and developing innovative, protein-based #immunotherapies. The transaction was unanimously approved by the boards of directors of both companies and is expected to close later this quarter. Alpine’s lead molecule, povetacicept (ALPN-303), is a dual antagonist of BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand). Through Phase 2 development, povetacicept has shown potential best-in-class efficacy in IgA nephropathy (IgAN), the companies said in a joint statement. IgAN is a serious, progressive, autoimmune disease of the kidney that can lead to end-stage-renal disease. So far, there are no approved therapies that target the underlying cause of IgAN. Povetacicept is on track to enter Phase 3 clinical development in the second half of 2024. Read more on #CMI online: https://lnkd.in/ebDN63QP

In our latest article published in PLOS Neglected Tropical Diseases, we reviewed the snakebite envenoming (SBE) therapeutics pipeline and antivenom market. Here’s what we found: 💉 We identified 127 products, which may seem sizeable, but this does not translate into a robust antivenom market. Some products have efficacy and safety issues, are expensive to produce, and many exist in a loosely regulated environment. 💊 We also found a diverse pipeline of 196 candidates, but progress is slow moving. Further investment and more diverse actors are needed to push candidates through clinical development and scale up 🕒 Antivenoms save lives and will continue to play a key role in the treatment of SBE, but our data validates a shift towards a dual R&D agenda: focusing both on improving existing antivenoms in the short to medium-term, and on developing more cost-effective and safer, next-generation SBE therapeutics in the longer term. 🤝 Achieving the short- and long-term priorities will require more coordination and consensus. Established governance structures could help funders and researchers to align on an R&D agenda, prioritise a portfolio of work, and better understand the community’s capacities and needs. 📣 We’re seeing unprecedented levels of R&D funding and exciting scientific developments but we will soon be faced with a stark funding landscape. Advocacy and evidence will play a key role in engaging funders to ensure SBE stays on the agenda, and innovations can reach those who need them most. Read the article here: https://lnkd.in/gB2keeUR Thanks to the co-authors, José María Gutiérrez, Cecilie Knudsen, Abdulrazaq Habib, Maya Goldstein, Andrew Tuttle; and the Advisory Committee, Ian Cameron, Nicholas Casewell, Andreas Laustsen-Kiel, Matthew Lewin, Devin Sok and Julien Potet Funding: Wellcome Trust

The DOJ's recent ruling on sponsored genetic testing has major implications for rare disease pharma. What does it mean for patient identification and research efforts? Our new blog post from General Counsel and Chief Privacy Officer Hilary Weckstein breaks down: - Key takeaways from the DOJ's decision - Why real-world data (RWD) is now essential for rare disease companies - How harmonized lab data can revolutionize HCP and patient identification Read the full post here: https://bit.ly/3Yr2yvT Stay ahead of the curve. Learn how RWD and lab data can drive your success in this evolving regulatory landscape. #raredisease #pharma #biotech #RWD #healthdata

Chronic #HepatitisDelta is a devastating viral disease with no approved treatments in the United States. I'm energized to see today's preliminary results from Vir Biotechnology, Inc.'s Phase 2 SOLSTICE clinical trial, evaluating mono- and combination therapies in the treatment of #HepatitisDelta. It's days like today that make me even more excited to come to work tomorrow. #PatientsAreWaiting https://lnkd.in/gHHhgdVS