Edmonton Global’s Post

Mpox, previously known as monkeypox, presents Denmark with a unique opportunity to showcase its pharmaceutical prowess on an international stage. As the world grapples with emerging infectious diseases, Denmark's robust biopharmaceutical sector has positioned the nation as a leading player in the development and distribution of vaccines and treatments. The Danish government and its innovative companies can leverage the Mpox situation to not only enhance their global reputation but also reinforce their commitment to public health. By actively participating in international efforts to control and combat Mpox, Denmark can further cement its status as a frontrunner in the global health arena, highlighting the importance of swift and effective responses to health crises while fostering collaborations that can lead to groundbreaking advancements in medicine. This scenario exemplifies how Denmark's strategic investments in research and development can translate into real-world impact, demonstrating its capabilities to address global health challenges while capitalizing on its pharmaceutical strength.

Draft guidance to industry - Rare Diseases: Considerations for the Development of Drugs and Biological Products Key points include considerations for natural history studies, flexibility for nonclinical and CMC, detailed aspects of clinical design, involvement of patients, caregivers, and advocates in rare disease drug development, and a list of expedited and accelerated approaches for orphan drug development. 

The availability of innovative medicines for rare and orphan diseases in #Mexico is influenced by many factors. These include the relative low prevalence of these diseases in Mexico, the roll-out strategy of developers and the types of regulatory pathways that are available to facilitate the development and authorisation of these important medicines.   Our latest R&D Briefing “Challenges and Opportunities for Orphan Medicines’ Availability in Mexico” looks at this in more detail. #RegulatoryScience #Latam #raredisease #orphandrugs #RegulatoryAffairs

Atticus Medical and Medicines Development for Global Health announce that clearance has been received from the U.S. Food and Drug Administration (FDA) for the Investigational New Drug (IND) application for moxidectin for scabies. Moxidectin is in Phase 2 clinical trials as a potential single dose treatment for this debilitating disease and this clearance allows sites in the USA to be included in the study.   Clearing the IND hurdle is a significant milestone in the development of moxidectin to address neglected diseases. Scabies is a contagious skin infestation that has often been neglected by the global research community. It is caused by the Sarcoptes scabiei mite, and affects hundreds of millions of people worldwide, predominantly but not exclusively in resource-limited settings.   The IND is supported by data from a recently completed scabies clinical trial conducted in Australia and Europe, and data reviewed by the FDA as part of their 2018 marketing authorisation of moxidectin for the treatment river blindness (onchocerciasis), in adults and adolescents 12 years and older.   The clinical trial cleared under this IND pushes the program further into establishing the role of moxidectin as a potential oral treatment option for people suffering from scabies. For more information about Atticus Medical, please visit www.atticusmed.com. For more information about scabies, please visit https://lnkd.in/gr5Gg-nm. #moxidectin #scabies #investors #clinicaltrials #globalhealth

#liverdisease US approves drug for MASH liver disease The US has approved the first #drug to treat an obesity-linked liver disease that affects an estimated 5% of the world's adults. Resmetirom, to be marketed as Rezdiffra, treats metabolic dysfunction-associated steatohepatitis (MASH) — formerly known as non-alcoholic #steatohepatitis (NASH). After many earlier drug failures, resmetirom is the first to reduce scar tissue known as fibrosis in the liver. But researchers caution that evidence for long-term benefits is still needed. “Only time will tell,” says gastroenterologist Maya Balakrishnan. “In the end, what matters is: does this drug improve survival?” Nature | 4 min read A ‘hurrah moment’: go deeper into the development and approval of resmetirom in Nature Reviews Drug Discovery (10 min read)

Interesting to read the results of this new study on the #efficacy and #safety of drugs used in #compassionate use for #rarediseases, and the timing between compassionate use initiation and marketing approval of investigational drugs. Orphanet Journal of Rare Diseases (2023) 18:368, ‘Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022’ (Zhang B. et. al.)   Compassionate use of an investigational drug can be a significant treatment option for rare disease patients, and the study further reveals a critical challenge: The median time from compassionate use initiation to formal approval of the investigational drug 790 days in this study.   The authors urge action to abbreviate the time from compassionate use to market approval to #expedite #access to the wider rare disease population! What is Early Access?  #EarlyAccess also known as #ExpandedAccess, #CompassionateUse, or #NamedPatientSupply is a pathway for a patient with a life-threatening illness or severely debilitating disease or condition to gain access to an investigational medical product prior to approval by a regulatory authority. Learn more here: https://lnkd.in/eCURs-gM   Inceptua Group - #helpingpharmahelpingpatients

📢 The FDA has given its green light to the first interchangeable biosimilar to Soliris (eculizumab)—Bkemv (eculizumab-aeeb)—for the treatment of two rare diseases: Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS). ✔ 💉 Bkemv can be confidently prescribed, knowing that the FDA has confirmed it to be highly similar to Soliris with no clinically meaningful differences. Subject to state pharmacy laws, it may be substituted at the pharmacy level without consulting the prescriber. Bkemv holds the same expectations for safety and efficacy as Soliris and is only available via a restricted program to ensure the benefits outweigh the risks. 👀 👉 This marks the 53rd approved biosimilar in the US, a testament to the FDA's commitment to expanding patient access to affordable treatment options and supporting a competitive biologics marketplace. Let’s celebrate this major milestone that brings hope to those battling rare diseases.🎯 👏 Congratulations to Amgen Inc. on this remarkable achievement!🚀 For more information on this update, check out the link - https://lnkd.in/g9wyXrxg #FDA #biosimilar #rarediseases #treatment #accessibility #affordability #healthcare

Accenture's Rajanikanth (Raj) Bandaru penned a detailed summary of FDA's recent Draft Guidance on Rare Diseases: Considerations For The Development Of Drugs And Biological Products. He breaks down considerations for natural history studies, non-clinical and clinical studies, quality and #cmc, and more for #orphandrug development. #raredisease #regulatorycompliance #regulatory #cellandgenetherapy https://lnkd.in/eQFVRUU8

Antimicrobial resistance (AMR) is a global health crisis—and we can’t wait for new drugs to save the day. Antimicrobials are expensive to develop and can take 10-15 years from discovery to approval. That’s why it’s crucial to preserve the efficacy of drugs we rely on today. Read more about #AMR and new drug development on our Living Diagnostics blog: https://bit.ly/3QKigg8

Thank you Rajanikanth (Raj) Bandaru for simplifying the guiance for general consumption. I am very encouraged to see the guidance putting emphasis on the involvement of patients, their caregivers, and advocates in drug development. In the absence of their input, development programs can miss important information about patient experiences, perspectives, needs, and priorities. Another aspect that is equally important is HCP input. It is key to determine their experience and ability to deliver care with ease - clinic readiness for commercial scale. When the treatment protocol and delivery capabilities are not designed for clinic readiness, patient and HCP journeys require special considerations for commercial phase. It can potentially limit where care is available for patients and create treatment center deserts.