D2D Pharma inSIGHTS’ Post

In a ground-breaking development for the pharmaceutical industry and patients with haemophilia B, Hemgenix (etranacogene dezaparvovec) has become the first treatment to enter NHS England’s Innovative Medicines Fund (IMF) with managed access. ⚕️ This arrangement allows patients to receive promising treatments that might not be recommended otherwise due to uncertain clinical or cost-effectiveness evidence. This milestone follows a commercial agreement between NHS England and manufacturer CSL Behring. Hemgenix is a gene therapy that involves a one-time infusion lasting approximately 1-2 hours, offering a potentially curative treatment for haemophilia B. This therapy represents a significant advancement in managing this genetic disorder by reducing the frequency of bleeding episodes and decreasing the need for regular infusions of clotting factors, which are typically administered once or twice a week for severe cases. 🩸 The #InnovativeMedicinesFund is designed to fast-track promising treatments addressing unmet clinical needs, providing patients quicker access while data on their efficacy and safety is gathered. An estimated 2,000 people in the UK have haemophilia B, and about 250 of them will be eligible for this new treatment. Hemgenix’s inclusion in this fund marks a major step forward in adopting advanced therapies within the NHS framework, offering hope for individuals with rare and severe medical conditions. 🧬 🗣 Professor Sir Stephen Powis, NHS National Medical Director, commented, “It is a one-time therapy that could be truly life-changing for some, as it could help people avoid the need for regular hospital visits.” Discover more insights here ➡ https://lnkd.in/ee7u5_gQ #marketaccess #patientaccess #imf #therapies #nhsengland #pharma #healthcare #haemophilia * 𝒘𝒓𝒊𝒕𝒕𝒆𝒏 𝒃𝒚 Rahul V.

PharmaCircle has published our Special Report: Eyeing Up the Ophthalmic Disease Market 2024. This is a 27-page special report that provides an in-depth view into the eye disease market, including top product sales, key approvals, drug delivery technologies, formulation/excipients, epidemiology and much more. The special report references hand-curated content from our subscription database. This is PharmaCircle’s first special report designed to cover an important niche area of pharmaceutical development and specifically tailored to companies active in this space.   Special Report’s Table of Contents:   •      Introduction •      Key Product Sales •      Epidemiology •      NME Pipeline •      Cell And Gene Therapy •      Formulation And Excipients •      Recent Key Approvals •      Delivery Beyond Topicals •      Pestel •      Strategic Deals, Partnerships and Acquisitions •      CMO/CDMO Landscape •      The Future   PharmaCircle has been a leading information provider to the life sciences industry for more than 20 years, providing hand-curated data on drug, biologics and combination product development that is comprehensive, reliable and analysis-ready.  Many life sciences innovators and manufacturers are PharmaCircle clients and rely on our full complement of research, development, clinical, business intelligence, supply chain and regulatory content and analytics for mission-critical projects.   To learn more about how to order this Special Report, please contact us at info@pharmacircle.com or go to https://lnkd.in/e2xWQhzT. 

🧬 📰 Latest News in Life Sciences & Healthcare - quick recap of some significant developments from the past weeks👇 🧠 Lilly’s Alzheimer’s Drug - Eli Lilly has received FDA approval for its Alzheimer's drug, which is noted for its cost advantage over competitors. ❌ FDA Rejects Novo Nordisk's Insulin - The FDA has rejected Novo Nordisk's weekly basal insulin, insulin icodec, requiring more data on how well it performs in people with type 1 diabetes. 🔍 Ozempic & Wegovy Study - A new study links popular diabetes drugs Ozempic and Wegovy to a sight-threatening disorder. The authors acknowledge that the findings are speculative and do not prove causality, but add they could warrant further study. 💉 Moderna’s Bird Flu Vaccine - The US government has allocated $176 million to Moderna for the development of a bird flu vaccine, supporting pandemic preparedness. 👁 Roche's Eye Implant Comeback - The FDA has given the green light for Roche’s eye implant medication, Susvimo, to return to the market. 🔗 Ipsen's ADC Alliance - Ipsen has formed a new ADC alliance, continuing its strategic expansion in the antibody-drug conjugate space. ⚖️ Boehringer and Medicare Pricing - Boehringer Ingelheim continues the pharmaceutical industry's legal battle over Medicare price negotiations, facing setbacks in court. 🌿 Arcutis' Zoryve Approval - After an FDA delay, Arcutis has received approval for Zoryve, a treatment for atopic dermatitis. 🔬 uniQure's Gene Therapy - uniQure's gene therapy for Huntington’s disease has shown promising results, propelling the company forward in the field of neurodegenerative diseases. Sources : pharmaphorum, Fierce Pharma 👍 If you found this insightful, don’t forget to like and share! ——— I am Alexis - French pharmacist turned entrepreneur. My company BTHT is connecting senior industry experts, ex-payors, and KOLs with consultants and investors in Life Sciences and Healthcare for expert calls and senior advisory. 👇 For more content like this Join 14K+ followers by clicking my name + follow 🔔 Join 1.3K+ subscribers to my newsletter 💌

PharmaCircle has published our Special Report: Eyeing Up the Ophthalmic Disease Market 2024. This is a 27-page special report that provides an in-depth view into the eye disease market, including top product sales, key approvals, drug delivery technologies, formulation/excipients, epidemiology and much more. The special report references hand-curated content from our subscription database. This is PharmaCircle’s first special report designed to cover an important niche area of pharmaceutical development and specifically tailored to companies active in this space.   Special Report’s Table of Contents:   •      Introduction •      Key Product Sales •      Epidemiology •      NME Pipeline •      Cell And Gene Therapy •      Formulation And Excipients •      Recent Key Approvals •      Delivery Beyond Topicals •      Pestel •      Strategic Deals, Partnerships and Acquisitions •      CMO/CDMO Landscape •      The Future   PharmaCircle has been a leading information provider to the life sciences industry for more than 20 years, providing hand-curated data on drug, biologics and combination product development that is comprehensive, reliable and analysis-ready.  Many life sciences innovators and manufacturers are PharmaCircle clients and rely on our full complement of research, development, clinical, business intelligence, supply chain and regulatory content and analytics for mission-critical projects. To learn more about how to order this special report, please contact us at  info@pharmacircle.com or go to https://lnkd.in/e8343Jfx.

Gene&Cell Therapy >> FDA's Marks, Woodcock talk regulatory flexibility for new rare disease drugs: The FDA’s top biologics chief and a former deputy commissioner on Tuesday discussed the need for regulatory flexibility for rare disease therapies and underscored the need to bolster gene therapy manufacturing. Peter Marks, the FDA’s biologics center director, and Janet Woodcock, former FDA deputy commissioner who retired earlier this year, discussed how the agency should be able to balance following regulations with exercising some flexibility for rare diseases. “What we’ve seen is a lot of cognitive dissonance internally,” Woodcock said. “People are being asked to, on one hand, enforce the standard, on the other hand be flexible. You can be flexible, flexible, flexible, and then pretty soon, you’re going to break.” Marks added that he thinks the FDA already can leverage a fair amount of regulatory flexibility but that the culture of the agency has historically prioritized strict adherence to the regulations. “We have people that are smart enough that we can focus on the patient and actually still stay on the right side of the regs, and I think it’s just a matter of, in some cases, just changing how we think about things and how we approach them,” he said. Marks, who’s been pushing for more accelerated approvals for rare disease therapies since at least August 2023, warned that changing the current framework could also exacerbate issues with insurance companies hesitating to cover the expensive new therapies, saying that that has already been an issue with some accelerated approvals. Biogen’s former Alzheimer’s drug Aduhelm won an accelerated approval in 2021 but failed to muster any sales as CMS declined to cover it, and eventually Biogen sold it. “We may see with Medicaid and gene therapies for sickle cell disease just to call that out, I’m kind of worried about how that’s going to roll out,” he said. “But you can imagine it happening on a broader scale if we change a standard.” Marks and Woodcock both emphasized that investing in manufacturing for gene therapies is key to bringing down their cost. “This would be something that the US could invest in,” Woodcock said. “I’ve been trying for continuous manufacturing for the last 20 years.” #lucidquest #genetherapy #celltherapy

𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐚𝐧𝐝 𝐏𝐡𝐚𝐫𝐦𝐚 𝐔𝐩𝐝𝐚𝐭𝐞𝐬: October 3 - 6, 2024 (Get the full list of 23 news pieces in today's TLDR Biotech newsletter: https://lnkd.in/gJ72_XgF) • Sanofi makes $875M sale of rare disease asset to Recordati • Editas Medicine diverts Cas9 licensing fees to DRI Healthcare for $57M upfront • Ultragenyx ups dosage as Wilson disease gene therapy shows promise • Alnylam Pharmaceuticals drops COVID-19 delivery tech lawsuit against Moderna • BeiGene launches Tevimbra with a 10% discount compared to Merck & Co.’s Keytruda • Texas sues big pharmas and PBMs over insulin price “conspiracy” • Johnson & Johnson continues infectious disease retreat with Dengue fever program cut • EMA investigates connection between hair loss, enlarged prostrate meds & “suicidal ideation” risks

Thoughts on this? >> Ultragenyx reports serious adverse effects in gene therapy trial (NASDAQ:RARE) - Seeking Alpha >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #competitivemarketing #healthcare #biotech #pharmaceutical

𝗥𝗲𝗰𝗼𝗺𝗯𝗶𝗻𝗮𝗻𝘁 𝗖𝗼𝗮𝗴𝘂𝗹𝗮𝘁𝗶𝗼𝗻 𝗙𝗮𝗰𝘁𝗼𝗿𝘀 𝗠𝗮𝗿𝗸𝗲𝘁 𝘁𝗼 𝗯𝗲 𝗪𝗼𝗿𝘁𝗵 $𝟮𝟲.𝟮𝟵 𝗕𝗶𝗹𝗹𝗶𝗼𝗻 𝗯𝘆 𝟮𝟬𝟯𝟭 The Recombinant Coagulation Factors Market is expected to reach $26.29 billion by 2031, growing at a CAGR of 8.6% from 2024 to 2031. Recombinant coagulation factors, produced through #RecombinantDNA technology, minimize the risk of blood-borne infections like #hepatitisC and #HIV, making them essential for treating bleeding disorders such as #hemophilia. Market growth is fueled by the increasing prevalence of hemophilia, rising #biotech R&D efforts, and growing awareness of the benefits of recombinant factors. However, high costs and limited accessibility in developing countries remain significant challenges. Untapped markets in emerging economies and increasing #healthcareexpenditure offer growth opportunities. Yet, challenges in production, stringent #regulatoryrequirements, and limited reimbursement in some regions continue to impact market expansion. #PharmaIndustry #GlobalHealth 𝗗𝗼𝘄𝗻𝗹𝗼𝗮𝗱 𝗣𝗗𝗙 𝗕𝗿𝗼𝗰𝗵𝘂𝗿𝗲 𝗼𝗳 𝗥𝗲𝗰𝗼𝗺𝗯𝗶𝗻𝗮𝗻𝘁 𝗖𝗼𝗮𝗴𝘂𝗹𝗮𝘁𝗶𝗼𝗻 𝗙𝗮𝗰𝘁𝗼𝗿𝘀 𝗠𝗮𝗿𝗸𝗲𝘁 :- https://lnkd.in/gvAQ93X7 ➤𝗕𝘆 𝗞𝗲𝘆 𝗣𝗹𝗮𝘆𝗲𝗿𝘀 :- Baxter International Inc., Grifols, CSL, Octapharma, Novo Nordisk, Biogen., Bayer, Kedrion Biopharma, Emergent BioSolutions, Pfizer. ➤𝗦𝗲𝗴𝗺𝗲𝗻𝘁 𝗖𝗼𝘃𝗲𝗿𝗲𝗱 𝗕𝘆 𝗧𝘆𝗽𝗲 Recombinant Factor VIII Recombinant Factor IX Other Types 𝗕𝘆 𝗦𝗼𝘂𝗿𝗰𝗲 Chinese Hamster Ovary (CHO) Cell Line Human Embryonic Kidney (HEK) Cell Line Other Sources 𝗕𝘆 𝗔𝗽𝗽𝗹𝗶𝗰𝗮𝘁𝗶𝗼𝗻 Hemophilia A Hemophilia B Other Applications 𝗕𝘆 𝗘𝗻𝗱 𝗨𝘀𝗲𝗿 Hospitals & Clinics Clinical Research Laboratories

Medicines Australia and its members take a lead role in Australia’s pharmaceutical horizon scanning to keep policy makers informed of innovative new classes of medicines that have the potential to significantly impact the way conditions are treated or the way that medicines are assessed and funded.    In Australia, horizon scanning is maturing with concerted efforts by industry and the Commonwealth to strengthen frameworks and processes so that our system is ready for new technologies ahead of their assessment and approval for use in Australia by the TGA. Last week, many of Australia's leading scientists, researchers, policy makers, regulators and patient groups attended our horizon scanning forum to learn more about cell and gene therapies, new antibiotics, vaccine advances and diagnostics.   The HTA Review has highlighted the importance of robust horizon scanning as an indicator of a world-class health system and the importance of advanced notice for policy makers to be able to plan and prepare the health system for disruptive medical advances. Over the last decade, the arrival of cell and gene therapies and genetic testing has revolutionised the diagnostics and treatment practices of many life-threatening conditions.    But our system to assess and fund these medicines was not prepared, and today patients are still waiting to access these and other new medicines far longer than in other countries.    This cannot continue.     Medicines Australia and its members support the recommendation for a stronger horizon scanning process.     We look forward to co-designing with the Commonwealth, patient groups and other stakeholders, a world-class horizon scanning system that enables greater flexibility and informed decision-making to bring breakthrough medical technologies to Australia faster. Find out more about >> https://lnkd.in/gcczfeey #StrongerPBS

𝗙𝗗𝗔 𝗡𝗼𝘃𝗲𝗹 𝗗𝗿𝘂𝗴 𝗔𝗽𝗽𝗿𝗼𝘃𝗮𝗹𝘀 𝗳𝗼𝗿 𝟮𝟬𝟮𝟯 ✓ What is a novel drug by FDA: Novel drugs are often innovative products that serve previously unmet medical needs or otherwise significantly help to advance patient care and public health. new molecular entities (“NMEs”) have chemical structures that have never been approved before. These products frequently provide important new therapies for patients. ✓ Every year, FDA CDER approves a wide range of new drugs and biological products.below listed 55 number's of new molecular entities and new therapeutic biological products that CDER approved in 2023. ✓ This listing 55 drugs does not contain vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or other products that the CBER 2023.Others are the same as, or related to, previously approved products, and they will compete with those products in the marketplace. S.No. Drug Name - Active Ingredient - Treatment 1. Leqembi - Lecanemab-irmb - Alzheimer’s 2. Brenzavvy- Bexagliflozin- #glycemic control. 3. Jaypirca- Pirtobrutinib- #Mantle cell lymphoma 4. Orserdu - Elacestrant- #breastcancer 5. Jesduvroq- Daprodustat - #anemia 6. Lamzede -Velmanase alfa-tycv- #nervoussystem 7. Filspari -Sparsentan - #proteinuria 8. Skyclarys -Omaveloxolone - Friedrich’s ataxia 9. Zavzpret -Zavegepant - Migraine 10. Daybue -Trofinetide - Rett syndrome 11. Zynyz -Retifanlimab - Treat metastatic 12. Rezzayo- Rezafungin - Bloodstream infection. 13. Joenja- Leniolisib -#Immunodeficiency 14. Qalsody - Tofersen 15. Elfabrio - Pegunigalsidase alfa-iwxj 16. Veozah - Fezolinetant-Menopause 17. Miebo - Perfluorhexyloctane - #eyecare 18. Epkinly - Epcoritamab-bysp 19. Xacduro - Sulbactam, durlobactam 20. Paxlovid - Nirmatrelvir, ritonavir - #covid 21. Posluma- Flotufolastat F 18 22. Inpefa - Sotagliflozin -#heartfailure 23. Columvi - Glofitamab-gxbm 24. Litfulo - Ritlecitinib 25. Rystiggo- Rozanolixizumab-noli 26. Ngenla - Somatrogon-ghla 27. Beyfortu- Nirsevimab-alip 28. Vanflyta - Quizartinib 29. Xdemvy - Lotilaner 30. Zurzuvae- Zuranolone 31. Izervay - Avacincaptad pegol 32. Talvey - Aalquetamab-tgvs 33. Elrexfio - Elranatamab-bcmm 34. Sohonos - Palovarotene 35. Veopoz - Pozelimab-bbfg 36. Aphexda -Motixafortide 37. Ojjaara - Momelotinib 38. Exxua - Gepirone - #Depressive disorder 39. Pombiliti - Cipaglucosidase alfa-atga 40. Rivfloza - Nedosiran 41. Velsipity - Etrasimod 42. Zilbrysq - Zilucoplan 43. Bimzelx - Bimekizumab 44. Agamree - Vamorolone 45. Omvoh - Mirikizumab-mrkz 46. Loqtorzi - Toripalimab-tpzi 47. Fruzaqla - Fruquintinib 48. Defencath -Taurolidine. 49. Augtyro - Repotrectinib 50. Ryzneuta -Efbemalenograstim alfa-vuxw 51. Truqap - Capivasertib 52. Ogsiveo - Nirogacestat 53. Fabhalta - Iptacopan 54. Filsuvez - Birch triterpenes 55. Wainua - Eplontersen #drug #healthcare #patients #pharmaceuticalindustry #pharma #pharmaceuticals #innovation #inspection #safety