LEQVIO Emerges as Leading PCSK9 Inhibitor in Cholesterol Management Across Major Markets
• Novartis's LEQVIO (inclisiran), the first FDA-approved siRNA therapy for LDL-C reduction, is projected to reach USD 2.2 billion in US market size by 2034, offering a novel mechanism with biannual dosing that enhances patient adherence.
• The PCSK9 inhibitor market, valued at USD 2 billion across seven major markets in 2023, is expected to grow substantially due to expanding applications in statin-intolerant patients and preventive cardiovascular strategies.
• Emerging competitors including Lerodalcibep, Merck's oral MK-0616, and Verve Therapeutics' gene-editing candidates are advancing through clinical trials, potentially disrupting LEQVIO's market position after 2027.
FDA Advisory Committee Discusses Columvi Combination for Relapsed or Refractory DLBCL
• Genentech's Columvi (glofitamab-gxbm) in combination with gemcitabine and oxaliplatin demonstrated a 41% reduction in risk of death for patients with relapsed or refractory diffuse large B-cell lymphoma in the Phase III STARGLO study.
• The FDA Oncologic Drugs Advisory Committee met to discuss the supplemental Biologics License Application for this combination therapy, focusing on the applicability of global trial results to U.S. patients.
• If approved, this first-of-its-kind bispecific antibody combination could provide a much-needed, off-the-shelf, fixed-duration treatment option for transplant-ineligible patients who currently face poor prognosis.
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.
Integra Therapeutics and Factor Bioscience Showcase Advanced Gene-Editing Technologies for CAR-T Cell Therapies at ASGCT 2025
• Integra Therapeutics presented new pre-clinical data demonstrating their FiCAT gene-writing platform's ability to create CAR-T cells with complex CARs in a single DNA donor, showing improved efficacy against B-ALL tumors and autoimmune diseases.
• Factor Bioscience revealed advancements in non-viral transgene insertion technology for next-generation allogeneic CAR-T cell therapies, presenting four studies highlighting their UltraSlice gene-editing platform and nucleic acid delivery innovations.
• Both companies' presentations at the 28th Annual Meeting of the American Society of Gene and Cell Therapy represent significant progress in developing safer, more efficient cell engineering technologies for oncological and autoimmune diseases.
Ipsen's Elafibranor Shows Promise in Phase II Trial for Primary Sclerosing Cholangitis
• Elafibranor demonstrated a favorable safety profile and significant dose-dependent efficacy in the Phase II ELMWOOD trial for primary sclerosing cholangitis (PSC), a rare liver disease with no currently approved treatments.
• Patients treated with elafibranor showed significant improvements in liver biochemical parameters, including alkaline phosphatase, with stabilization of non-invasive markers of liver fibrosis compared to placebo.
• The 120mg dose of elafibranor significantly improved pruritus symptoms, offering potential relief for a common and distressing symptom experienced by PSC patients.
Vertex's Cystic Fibrosis Drug Alyftrek Receives CHMP Recommendation for EU Approval
• The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended Vertex Pharmaceuticals' Alyftrek for EU-wide approval for cystic fibrosis treatment.
• Alyftrek is one of six orphan drugs among a total of 16 medications that received positive opinions from the CHMP, setting them on track for European Commission marketing authorization.
• The recommendation marks another potential addition to Vertex's portfolio of cystic fibrosis treatments, addressing the significant unmet needs that remain for patients with this rare genetic disorder.
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.
Patient Organizations Emerge as Critical Partners in Drug Development and Clinical Research
• Patient organizations have evolved from support groups to influential stakeholders in drug development, providing crucial insights into patient needs, trial design, and research priorities.
• These organizations now generate significant revenue—with US patient groups earning over $62.5 billion in the past five years—enabling them to fund research, create patient registries, and establish community advisory boards.
• The World Duchenne Organization exemplifies this evolution by forming advisory boards that work directly with pharmaceutical companies to optimize clinical trial designs and accelerate development of safe, effective treatments.
EMA Proposes Streamlined Approval Process for Biosimilars to Enhance Patient Access
• The European Medicines Agency (EMA) has released a draft reflection paper outlining a simplified approval pathway for biosimilars that could reduce clinical data requirements while maintaining safety standards.
• The proposed approach focuses on structural and functional comparability along with pharmacokinetic data, potentially eliminating the need for extensive clinical efficacy studies in biosimilar development.
• If implemented in 2026 after the consultation period ends in September 2025, the streamlined process aims to increase market competition and improve patient access to critical biological treatments across the EU.
European Regulators Reject Eli Lilly's Alzheimer's Drug Kisunla Over Safety Concerns
• The European Medicines Agency's advisory committee has recommended against approving Eli Lilly's Alzheimer's treatment Kisunla, citing safety concerns related to brain swelling.
• European regulators determined that the risks of amyloid-related imaging abnormalities (ARIA) outweigh the potential benefits of the drug, creating a notable divergence from US regulatory decisions.
• This rejection marks another instance where European and American regulators have reached different conclusions on new Alzheimer's treatments, highlighting regional differences in benefit-risk assessment.
European Pharma Giants Push for US-Level Drug Pricing Amid Tariff Tensions
• Novartis and Sanofi CEOs have urged the European Commission to increase spending on medicines and vaccines, citing artificially low drug prices in Europe that discourage innovation.
• Pharmaceutical companies are advocating for medical goods to be excluded from potential US-EU tariff wars to prevent price spikes on popular European-made drugs like Wegovy and Keytruda.
• Major pharmaceutical firms including Roche ($50bn) and Novartis ($23bn) have announced significant US investments following Trump's return to power, while industry leaders warn of accelerated company departures from Europe without urgent action.
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).
Navigating the Orphan Drug Landscape in Europe: HTA Challenges and the Rising Role of Real-World Evidence
• European orphan drug approvals have grown significantly, with 192 approvals through 2022, yet approximately 95% of rare diseases still lack approved treatments, highlighting persistent market access challenges despite regulatory progress.
• HTA outcomes for orphan drugs vary dramatically across European countries, with approval rates ranging from 98% in Germany to 67% in Scotland, creating inconsistent patient access and requiring tailored market entry strategies.
• Real-world evidence (RWE) is emerging as a critical solution to bridge the evidence gap in orphan drug development, with pharmaceutical companies increasingly investing in RWE infrastructure to support regulatory approvals and strengthen value propositions.
AbbVie's RINVOQ Receives Positive CHMP Opinion for Giant Cell Arteritis Treatment
• AbbVie's upadacitinib (RINVOQ) receives CHMP recommendation as the first oral advanced therapy for giant cell arteritis, with European Commission decision expected in first half of 2025.
• Phase 3 SELECT-GCA trial demonstrated sustained remission in GCA patients through week 52, with consistent safety profile compared to other approved indications.
• Giant cell arteritis, primarily affecting adults over 50, can lead to severe complications including blindness and stroke if left untreated, highlighting the significance of this new treatment option.
Advancing Rare Disease Research: How mRNA Technology and Synthetic Control Arms Are Transforming Clinical Trials
• Rare diseases affect approximately 300 million people globally, with over 90% lacking approved therapies and patients facing significant diagnostic delays averaging 4-5 years.
• Innovative approaches like synthetic control arms (SCAs) are revolutionizing rare disease clinical trials by reducing placebo groups, accelerating recruitment, and maintaining scientific integrity despite small patient populations.
• mRNA technology shows promising potential for treating rare diseases by instructing cells to produce functional versions of missing or defective proteins, with clinical trials underway for conditions like propionic acidemia and methylmalonic acidemia.
Celltrion's Phase 3 Study Validates Yuflyma's Interchangeability with Humira for Psoriasis Treatment
• A Phase 3 trial involving 367 Estonian patients demonstrated pharmacokinetic equivalence between Yuflyma and Humira in treating moderate to severe plaque psoriasis, with comparable efficacy and safety profiles.
• Celltrion has submitted a supplemental application to the FDA in January 2024 seeking interchangeability status for Yuflyma, which could streamline the prescription switching process.
• As the world's first high-concentration Humira biosimilar, Yuflyma offers advantages including reduced injection volume and citrate-free formulation, targeting a market where Humira generated $14.4 billion in global sales in 2024.
Ipsen's Sohonos Becomes First Approved Treatment for FOP in Canada
• Health Canada has approved Sohonos (palovarotene), making it the first-ever treatment for fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder that turns soft tissue into bone.
• The approval follows regulatory challenges in the US, where Ipsen withdrew its marketing application in 2022 after the FDA requested additional phase 3 trial data.
• The drug is approved for both chronic use and flare-ups in patients aged 8+ (females) and 10+ (males), marking a significant milestone following Ipsen's $1.3 billion acquisition of Clementia Pharma.
FDA Approves Novel Antibiotic Combination Emblaveo for Complex Abdominal Infections
• AbbVie's Emblaveo, a groundbreaking aztreonam-avibactam combination, receives FDA approval for treating complicated intra-abdominal infections with limited treatment options.
• The novel therapy combines a monobactam antibiotic with a β-lactamase inhibitor, effectively targeting resistant Gram-negative bacteria including those producing metallo-β-lactamases.
• Commercial availability is scheduled for Q3 2025, offering healthcare providers a crucial new tool against antimicrobial-resistant pathogens in complicated intra-abdominal infections.
Lantheus to Present New PYLARIFY Data for Early Prostate Cancer Detection at ASCO GU 2025
• Lantheus will showcase new data demonstrating PYLARIFY's effectiveness in detecting recurrent prostate cancer in patients with low PSA levels at the 2025 ASCO Genitourinary Cancers Symposium.
• PYLARIFY has emerged as the leading PSMA PET imaging agent in the U.S., with over 400,000 scans conducted across 48 states, demonstrating significant clinical adoption.
• Real-world evidence from the PYLARIFY Registry will be presented, offering insights into the diagnostic agent's performance in both newly-diagnosed and recurrent prostate cancer cases.
Libtayo Becomes First EU-Approved Immunotherapy for Advanced Cervical Cancer
• The European Commission has approved Regeneron's Libtayo (cemiplimab) as the first immunotherapy for advanced cervical cancer treatment in Europe, regardless of PD-L1 expression or tumor histology.
• Phase 3 EMPOWER-Cervical 1 trial demonstrated significant survival benefits, showing a 31% reduced risk of death compared to chemotherapy in patients with recurrent or metastatic cervical cancer.
• The approval expands Libtayo's European indications to four cancer types, including advanced SCC, BCC, CSCC, and NSCLC, marking a significant advancement in treatment options.
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