BAP Pharma’s Post

📢 The Access to Medicine Index 2024 has just been released🎉 🧪Pharmaceutical innovations can save lives, but access is not guaranteed for all, especially in low- and middle-income countries🌍. 📄#ATMI24 evaluates 20 leading pharmaceutical companies on their efforts to expand access to their products in LMICs. It shows uneven progress, with many patients in LMICs still lacking access to essential medicines. 🎯The report identified key initiatives undertaken by pharmaceutical companies to support access to medicines in LMICs, but also identified notable gaps, particularly in addressing non-communicable diseases. 🫱🏽🫲🏾Some of these gaps could be addressed through partnerships with organisations like MPP. 🚀MPP plays a crucial role by facilitating voluntary licensing and technology transfer to make life-saving therapies more accessible and affordable. 📄 Access the full report: https://bit.ly/3YO9sKk #Access2Meds #GlobalHealth Access to Medicine Foundation

Update on #Orphan Registered Medicinal Products The UK government Medicines and Healthcare products Regulatory Agency has released the latest list of orphan registered medicinal products, highlighting significant advancements in the treatment of rare diseases. Here are the key points: 1. Comprehensive List: The document provides a detailed overview of orphan drugs that have received special designation for treating rare conditions. 2. Enhanced Accessibility: These drugs address unmet medical needs, offering new hope for patients with limited treatment options. 3. Regulatory Support: Orphan designation includes incentives such as market exclusivity and reduced fees, promoting innovation in the pharmaceutical industry. 4. Impactful Treatments: The list showcases a range of therapies, from genetic treatments to novel medications, underscoring the diversity of approaches in tackling rare diseases. [Read the Full Document] https://lnkd.in/dWRAy874 Let's continue to support and drive innovation in the field of #raredisease treatment! #Pharmaceuticals #RareDiseases #OrphanDrugs #HealthcareInnovation #MedicalResearch #Pharmacovigilance #PatientCare #Healthcare

*****Positive CHMP opinion for UCB's BIMZELX® in moderate to severe hidradenitis suppurativa***** The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for the regulatory application of BIMZELX® (bimekizumab; anti-IL17A/17F monoclonal antibody) for the treatment of adult patients with moderate to severe hidradenitis suppurativa with inadequate response to conventional systemic therapy. The positive opinion is based on two Ph3 clinical trials, BE HEARD I (https://shorturl.at/akCHZ) and BE HEARD II (https://shorturl.at/xQY14). The data from the two trials has demonstrated statistically significant and clinically meaningful improvements over placebo in the signs and symptoms of HS, as measured by HiSCR50 at Week 16, the primary endpoint, with responses sustained to Week 48. The safety profile of bimekizumab in the clinical trials was consistent with data seen in previous studies with no new observed safety signals. The EMA is expected to deliver a decision regarding the approval of marketing authorization of BIMZELX® in hidradenitis suppurativa before the end of May 2024. Source: https://shorturl.at/cmyL3 #ucb #hidradenitissuppurativa #bimzelx #ema #emaapproval #strategy #competitiveintelligence #marketing #pharma #pharmaceutical #biotech #productmarketing #healthcare #lifesciences #lifesciencesconsulting

Adalimumab (anti-TNF alpha monoclonal antibody) and Novartis' Cosentyx® (secukinumab; anti-IL17A monoclonal antibody) are the only biologic therapies approved for use in hidradenitis suppurativa so far. Infliximab is being used off-label by healthcare providers in moderate to severe hidradenitis suppurativa. Once approved, UCB's BIMZELX® would pose a significant competitive threat to Cosentyx®. Hidradenitis suppurativa would represent the fourth marketing authorization for bimekizumab in three years in EU, adding to the existing indications in moderate to severe plaque psoriasis, active psoriatic arthritis and active axial spondyloarthritis. AS per UCB 2023 full-year financial results, BIMZELX® has reported strong revenue growth (+323%) in FY 2023 and is expected to be a major growth driver in 2024-2025 as well. The company anticipates it to become the IL-17 class leader and market leader in its approved indications. #ucb #hidradenitissuppurativa #bimzelx #ema #emaapproval #strategy #competitiveintelligence #marketing #pharma #pharmaceutical #biotech #productmarketing #healthcare #lifesciences #lifesciencesconsulting

🚨 𝐈𝐦𝐩𝐨𝐫𝐭𝐚𝐧𝐭 𝐅𝐃𝐀 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐦𝐞𝐧𝐭 🚨 The FDA made a significant decision regarding Pepaxto (melphalan flufenamide), a medication previously approved for use in combination with dexamethasone to treat certain patients with multiple myeloma. The agency has officially withdrawn its approval for Pepaxto due to two crucial reasons: 1️⃣ The confirmatory study, which was required as a condition of accelerated approval, did not confirm Pepaxto's clinical benefit. 2️⃣ Available evidence demonstrates that Pepaxto is not proven to be safe or effective under its conditions of use. This final decision, issued by FDA's Center for Biologics Evaluation and Research Director Peter Marks, M.D., Ph.D., is effective immediately. In alignment with the amended procedures for withdrawal of accelerated approval enacted in 2023 under the Food and Drug Omnibus Report Act of 2022 (FDORA), the FDA provided Oncopeptides with a notice of proposed withdrawal, an explanation for the proposed withdrawal, and an opportunity for appeal. Following Oncopeptides' appeal and meeting with the Commissioner's designee, this withdrawal decision was finalized. It's crucial to note that this marks the first instance the FDA has utilized these amended withdrawal procedures. The agency will publish a Federal Register notice announcing today's decision and remove Pepaxto from the Orange Book. Additionally, the FDA has alerted patients and healthcare professionals about clinical trial results showing an increased risk of death associated with Pepaxto. The Oncologic Drugs Advisory Committee (ODAC) reviewed the results of the OCEAN confirmatory trial and voted against the favorable benefit-risk profile of melphalan flufenamide for the indicated patient population. At present, Pepaxto is not being marketed in the United States. We encourage healthcare professionals to stay informed and discuss alternative treatments with their patients, while patients currently receiving Pepaxto should consult their healthcare providers regarding the risks and benefits of continuing treatment. For more information, please refer to the public docket and relevant FDA publications. Contact  BIOBOSTON CONSULTING today or visit our  website to learn more about how we can support your organization. #pharmaceuticals #biotechnology #medicaldevices #consulting #fda  #quality #compliance #FDA #Pepaxto #MultipleMyeloma #Healthcare #MedicalNews

PBAC prioritizes high-impact drug submissions for March 2025, ensuring timely, equitable access to essential therapies amid high demand. #Pharma #PharmaceuticalBenefits #RegulatoryAgencies #GlobalMarketAccess #HealthcareInnovation #MarketAccessToday #MarketAccess

Regulatory Update: EMA’s PRIME Initiative 🌟 The European Medicines Agency (EMA) has developed the PRIME (PRIority MEdicines) program in alignment with the European Commission’s priorities and the 2020 strategy for the European medicines regulatory network. This initiative aims to accelerate the research and development of innovative medicines for patients with unmet medical needs, offering them better treatment options and a chance at healthier lives. Discover more about how PRIME is paving the way for promising medicines in the latest factsheet: PRIME Factsheet https://zurl.co/9ZpY #RegulatoryUpdate #EMA #PRIME #HealthcareInnovation #PatientCare #Pharmaceuticals #LifeScience #Pharma #PV #Innovation

🚨 Breaking News: G-BA Ends Data Collection Requirement for Marstacimab! 🚨 In a significant move, Berlin's Federal Joint Committee (G-BA) has officially halted the mandate for application-related data collection on Marstacimab, following its approval as an orphan drug. This decision underscores the stringent criteria surrounding data collection mandates and highlights how orphan drug classification can streamline regulatory processes. Key Highlights: ✅ Marstacimab approved for routine prophylaxis in patients aged 12+ with severe hemophilia A or B. ✅ G-BA's decision reflects a broader trend in pharmaceutical strategies to optimize market access. ✅ Patients can now expect quicker access to essential treatments, enhancing health outcomes. This development not only benefits pharmaceutical companies but also paves the way for improved patient care. For more insights on this pivotal decision and its implications, click on the link! #G-BA #HealthcareInnovation #Marstacimab #OrphanDrug #Pharma #PharmaceuticalNews #RegulatoryAgencies #RegulatoryUpdates #MarketAccess #MarketAccessToday

🌟 Exciting Milestone Alert for #Galactosemia Treatment 🌟 Applied Therapeutics, Inc. has reached a pivotal moment with the FDA's acceptance of the New Drug Application #NDA for #govorestat (AT-007), offering a beacon of hope for Classic #Galactosemia patients. With Priority Review status and a PDUFA date set for August 28, 2024, we are one step closer to addressing this high unmet medical need. #Galactosemia, affecting thousands worldwide, has long awaited an effective treatment. #Govorestat represents not just a breakthrough in therapy but also in patient care, as it aims to be the first medication indicated for Galactosemia #treatment. This achievement is a testament to the relentless pursuit of innovation and the power of collaboration between patients, families, and the medical community. Connect, Repost & Follow for more info on the pharmaceutical industry 🌍 #pharma #pharmaceutical #pharmaindustry #europe #pharmaceuticalindustry #healthcare

Next week, PharmaCompass will bring to you an overview on new drug approvals in H1 2024. Meanwhile, stay informed on the surge in FDA approvals last year. [Free Excel Download at https://lnkd.in/gizBWR5s] FDA approvals rose 49% in 2023. Of the 55 new drugs approved by the CDER in 2023, 36 percent were considered first-in-class. The EMA granted marketing authorization to 32 novel drugs. As usual, oncology topped the list of drug approvals. Read more at: https://lnkd.in/gAKJazPc #PharmaFlow - Pharma Data Compilations by PharmaCompass.com #FDA #medicine #oncology #genetherapy #healthcare #pharmaceuticals #pharmanews #pharmaceuticalindustry #innovation #management #digitalmarketing #technology #startups #marketing #sales #strategy #networking #pharma #biotechnology #pharmacompass