AKANTIOR® Receives UK Marketing Authorization as First Approved Treatment for Acanthamoeba Keratitis
• SIFI's AKANTIOR® (polihexanide 0.08%) has received both Marketing Authorization and Promising Innovative Medicine designation from the UK's MHRA, marking it as the first approved treatment for Acanthamoeba keratitis.
• The approval confirms AKANTIOR's Orphan Drug Designation and New Active Substance status, recognizing its efficacy against an ultra-rare corneal infection that can lead to blindness if untreated.
• Following its European approval in August 2024, this UK authorization represents a significant advancement for patients with this devastating eye infection, with SIFI planning to file for NICE reimbursement by June 2025.
Colorectal Cancer Leads in Real-World Data Volume, But Oncology Trials Need Modernization
• Phesi's analysis of 167 million patient records reveals colorectal cancer has the largest volume of real-world data with nearly six million records, outpacing breast, lung, liver, and prostate cancers.
• Despite the wealth of available data, oncology clinical trials continue to suffer from inefficiencies, with trial design and execution failing to keep pace with advances in biomarker science and patient profiling.
• Experts urge a shift toward "precision oncology" using AI and clinical data science to optimize patient profiles, programs, protocols, and operations plans to accelerate drug development.
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
Moderna's UK mRNA Vaccine Facility Now Fully Operational in Oxfordshire
• Moderna's Innovation and Technology Centre in Harwell, Oxfordshire has received Good Manufacturing Practice certification, enabling commercial production of mRNA vaccines for pandemic preparedness.
• The facility is part of a ten-year partnership between Moderna and the UK government, with plans to produce at least 250 million vaccine doses during future pandemics for the NHS.
• The flexible manufacturing setup allows rapid switching between different vaccine types within weeks, with the first priority being a new COVID-19 vaccine for the NHS next winter.
Takeda's Adzynma Becomes First UK-Approved Treatment for Rare Blood Clotting Disorder cTTP
• The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has approved Takeda's Adzynma (rADAMTS13) as the first treatment for congenital thrombotic thrombocytopenic purpura (cTTP) in the UK for patients of all ages.
• Adzynma is an enzyme replacement therapy that provides a recombinant form of ADAMTS13, addressing the underlying enzyme deficiency that causes this ultra-rare inherited blood clotting disorder.
• In clinical trials, no patients experienced acute thrombotic thrombocytopenic purpura events while receiving Adzynma as preventative treatment, demonstrating significant efficacy compared to plasma-based therapies.
AstraZeneca's Trixeo Aerosphere Becomes First Inhaled Respiratory Medicine with Near-Zero Climate Impact Propellant
• UK regulatory authorities have approved AstraZeneca's Trixeo Aerosphere as the first pressurized metered-dose inhaler using a next-generation propellant with 99.9% lower Global Warming Potential than current options.
• The COPD treatment maintains clinical bioequivalence to the original formulation while achieving a carbon footprint comparable to propellant-free inhalers, addressing both patient needs and environmental concerns.
• This approval marks the first step in AstraZeneca's commitment to transition its entire pressurized metered-dose inhaler portfolio to near-zero climate impact propellants by 2030 as part of its Ambition Zero Carbon strategy.
FDA Grants Fast Track Designation to Givinostat for Polycythemia Vera Treatment
• The U.S. FDA has granted Fast Track designation to Italfarmaco's givinostat for treating polycythemia vera, a rare hematologic cancer with limited treatment options.
• Givinostat, an oral histone deacetylase inhibitor, targets abnormal gene expression to control excessive cell proliferation driven by mutations like JAK2V617F commonly found in PV patients.
• A Phase III clinical trial for givinostat in polycythemia vera is currently enrolling patients across Europe, UK, Israel, and North America, with the drug already approved for Duchenne muscular dystrophy.
Shilpa Medicare's Bengaluru Facility Receives EMA GMP Certification for Advanced Drug Delivery Systems
• Shilpa Medicare's Unit VI in Bengaluru has secured Good Manufacturing Practice (GMP) certification from the European Medicines Agency following a successful inspection with only minor observations.
• The certification covers manufacturing, packaging, testing, storage, and distribution of two complex dosage forms: Oral Mouth Dissolving Films and Transdermal Systems, expanding the company's European market access.
• The Bengaluru facility, already approved by UK's MHRA and Australia's TGA, currently supplies Oral Film products to the US and other international markets with a robust pipeline of products in various stages of development.
Stablepharma Begins Phase 1 Trial of World's First Fridge-Free Tetanus-Diphtheria Vaccine
• UK-based Stablepharma has commenced a Phase 1 clinical trial for SPVX02, the world's first fridge-free tetanus and diphtheria vaccine, with the first participant dosed on April 15, 2025.
• The company's StablevaX™ technology enables vaccines to remain stable at room temperature for up to 18 months and withstand extreme temperature fluctuations from -20°C to +40°C, potentially eliminating cold chain requirements.
• With WHO reporting that over 50% of vaccines are wasted annually due to cold chain failures, this innovation could significantly improve global vaccine access, reduce waste, and decrease carbon emissions from temperature-controlled transportation.
NHS Expands Cancer Vaccine Launch Pad to Include Promising Melanoma Treatment
• The NHS Cancer Vaccine Launch Pad program is expanding to include trials of a new melanoma vaccine, aiming to improve treatment outcomes for patients with advanced skin cancer.
• The iSCIB1+ (Immunobody) vaccine works by helping the immune system recognize and attack cancer cells, potentially boosting the effectiveness of existing immunotherapy treatments.
• With melanoma cases projected to increase by 33% by 2040, the needle-free vaccine will be available at seven initial hospital sites across England, with first patient referrals expected in May 2025.
FDA Approves Pre-Filled Syringe for Self-Injection of Efgartigimod in gMG and CIDP Patients
• The FDA has approved a new pre-filled syringe formulation of efgartigimod (VYVGART Hytrulo) for self-injection in adults with generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy.
• The self-injection option provides patients greater independence and flexibility, allowing treatment at home or while traveling, reducing the need for frequent clinic visits while maintaining the medication's established safety and efficacy profile.
• Developed through argenx's partnership with Halozyme's ENHANZE drug delivery technology, the pre-filled syringe enables rapid 20-30 second subcutaneous administration, with regulatory decisions expected in the EU, Japan, and Canada by 2025.
4basebio Secures UK Regulatory License for GMP-Grade Synthetic DNA Production
• 4basebio has received Good Manufacturing Practice certification from the UK's Medicines and Healthcare products Regulatory Agency, enabling the supply of synthetic DNA for clinical applications in cell and gene therapies.
• The company's proprietary platform offers faster production and improved safety profiles compared to traditional plasmid DNA, providing scalable solutions for clinical trials and commercial products.
• This regulatory milestone follows a £40 million investment announced in 2024, positioning 4basebio as an emerging leader in the synthetic DNA space for advanced therapeutic development.
UK Government Unveils Life Sciences Strategy to Strengthen Post-Brexit Industry
• The UK government has revealed a comprehensive life sciences strategy led by Professor Sir John Bell, aiming to strengthen the sector after Brexit through increased funding and policy reforms.
• Key proposals include £160 million in new funding, improved clinical trial capabilities, implementation of the Accelerated Access Review, and benchmarking the UK to be in the top quartile for innovative medicine adoption by 2023.
• Industry leaders from pharmaceutical companies, biotech firms, and healthcare organizations collaborated on the strategy, which addresses challenges in NHS funding constraints while seeking to create a globally competitive life sciences ecosystem.
EU Approves Aurobindo Pharma's Dyrupeg: New Pegfilgrastim Biosimilar for Chemotherapy Patients
• The European Commission has granted marketing authorization for Dyrupeg™, a pegylated filgrastim biosimilar developed by CuraTeQ Biologics, a subsidiary of Aurobindo Pharma.
• Dyrupeg™ is indicated for reducing neutropenia duration and febrile neutropenia incidence in adult cancer patients undergoing cytotoxic chemotherapy, offering a cost-effective alternative to existing treatments.
• The approval follows a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in January 2025, marking a significant expansion of Aurobindo's biosimilar portfolio in Europe.
Regulators Adapt to AI Revolution in Healthcare: MHRA Leads with New Guidance and Sandbox Initiative
• The UK's MHRA has published comprehensive guidance on regulating AI in healthcare while exploring ways to leverage the technology to improve its own regulatory efficiency.
• A new regulatory sandbox called "AI-Airlock" will launch in spring 2024, creating a controlled environment for testing AI as Medical Device (AIaMD) products and identifying potential regulatory challenges.
• Regulatory bodies worldwide are developing frameworks to balance innovation with safety concerns, with the EU finalizing its AI Act and the FDA implementing pre-authorized software change processes for AI/ML systems.
FDA Expands Pluvicto Approval for Earlier Treatment of Metastatic Prostate Cancer
• Novartis's radioligand therapy Pluvicto received FDA approval for earlier use in PSMA-positive metastatic castration-resistant prostate cancer patients after ARPI therapy but before chemotherapy, tripling the eligible patient population.
• In the Phase III PSMAfore trial, Pluvicto reduced the risk of disease progression or death by 59% and more than doubled median radiographic progression-free survival compared to alternative hormone therapy.
• The expanded indication addresses a critical treatment gap, as approximately half of mCRPC patients do not survive long enough to receive second-line therapy, highlighting the importance of effective earlier intervention options.
Yaqrit Secures £2.2 Million NIHR Grant for Pivotal DIALIVE Liver Support Device Trials
• Yaqrit has received a £2.2 million ($2.85 million) grant from the UK's National Institute for Health and Care Research to fund pivotal trials of its DIALIVE liver support device across 13 UK centers.
• The DIALIVE device treats Acute-on-Chronic Liver Failure (ACLF) by removing disease-causing toxins from blood and exchanging dysfunctional albumin, showing twice the resolution rate compared to standard care in previous studies.
• The randomized trial will enroll approximately 70 high-risk ACLF grade 2-3 patients, with results expected in Q1 2027, potentially offering new hope for a condition that carries 40-80% mortality within 28 days.
UK Government Commits £121 Million to Revitalize Clinical Trial Sector Following O'Shaughnessy Review
• The UK government has pledged £121 million over three years to implement recommendations from Lord O'Shaughnessy's review of commercial clinical trials, aiming to double trial recruitment by 2025 and again by 2027.
• Key commitments include reducing approval times to 60 days, establishing a mandatory national approach to contracting, and developing clinical trial acceleration networks for infectious diseases, cancer, and dementia.
• Recent data shows early signs of recovery with a 4.3% increase in commercial trials in 2022 compared to 2021, though numbers remain below historical levels following a 41% decline between 2017-2021.
GSK's Blenrep Secures UK Approval for Multiple Myeloma Treatment in Combination Therapy
• The UK's medicines regulatory body has approved GSK's Blenrep (belantamab mafodotin) in combination with other drugs for multiple myeloma patients whose first treatment failed or caused severe side effects.
• This approval marks a significant comeback for Blenrep, which was withdrawn from markets in 2022 after failing to outperform existing treatments when used as monotherapy.
• Clinical trials demonstrated Blenrep's combination therapy extended progression-free survival and overall survival compared to standard care regimens, including those based on Darzalex (daratumumab).
EU Approves WAINZUA (Eplontersen) for Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
• The European Commission has approved WAINZUA (eplontersen), developed by Ionis Pharmaceuticals and AstraZeneca, for treating hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adult patients with stage 1 or 2 disease.
• WAINZUA is the only EU-approved treatment for ATTRv-PN that can be self-administered monthly via an auto-injector, offering consistent TTR protein suppression and improvements in neuropathy impairment and quality of life.
• The approval follows positive results from the NEURO-TTRansform Phase 3 trial, which demonstrated sustained benefits through 66 weeks on serum TTR concentration, neuropathy impairment, and quality of life compared to placebo.
© Copyright 2025. All Rights Reserved by MedPath