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Clinical Trial News

Real-World Data Supports rpFVIII Use in Acquired Hemophilia A Treatment

  • Real-world data supports the use of recombinant porcine factor VIII (rpFVIII) as a first-line treatment for bleeding events in adults with acquired hemophilia A.
  • The study reported no new safety signals, hypersensitivity reactions, or thromboembolic events, aligning with previous Phase 2/3 clinical trial findings.
  • A higher proportion of patients receiving first-line rpFVIII achieved resolution of initial bleeding events compared to those receiving it as a second-line therapy.
  • The study suggests that a shorter time from initial bleed to first rpFVIII infusion may favor bleed resolution in acquired hemophilia A patients.

FDA Commissioner Defends Approval Standards Amidst Trial Failures

  • FDA Commissioner Robert Califf addressed concerns that the agency is lowering its standards for drug approvals, even when clinical trials fail to meet endpoints.
  • The FDA's decision to expand Sarepta Therapeutics' Elevidys approval for Duchenne muscular dystrophy, despite a failed Phase 3 trial, has drawn criticism.
  • Travere Therapeutics' Filspari, a drug for IgAN, also received full approval despite failing its Phase 3 confirmatory trial, sparking debate about biomarker-based approvals.

MHRA Approves Sanofi/Regeneron's Dupixent for Uncontrolled COPD

  • The MHRA has approved Dupixent (dupilumab) as an add-on maintenance treatment for adults with uncontrolled COPD and raised blood eosinophils.
  • Dupixent is the first targeted biologic approved in the UK for uncontrolled COPD, offering a new treatment option for eligible patients.
  • The approval is based on Phase 3 trials (BOREAS and NOTUS) demonstrating significant reductions in COPD exacerbations and improved lung function.
  • NICE and SMC will now review Dupixent for reimbursement, potentially expanding access to this treatment option within the NHS.

Tusamitamab Ravtansine Fails to Meet Survival Endpoints in NSCLC Trial

  • The Phase 3 CARMEN-LC03 trial evaluated tusamitamab ravtansine, an antibody-drug conjugate targeting CEACAM5, against docetaxel in advanced nonsquamous NSCLC patients.
  • The trial did not demonstrate a statistically significant improvement in progression-free survival (PFS) or overall survival (OS) compared to docetaxel.
  • Tusamitamab ravtansine showed a positive trend in time to deterioration in health-related quality of life, particularly in disease-related symptoms.
  • Fewer grade 3 or higher adverse events and treatment discontinuations were observed in the tusamitamab ravtansine arm compared to the docetaxel arm.

SAR408701 Versus Docetaxel in Previously Treated, Carcinoembryonic Antigen-related Cell Adhesion Molecule 5 (CEACAM5) Positive Metastatic Non-squamous Non-small-cell Lung Cancer Patients

NCT04154956Active, Not RecruitingPhase 3
Sanofi
Posted 2/6/2020

Pathologists Call for Reform in HER2 Testing Standards as Breast Cancer Treatment Landscape Evolves

  • Leading pathologists highlight critical challenges in distinguishing between HER2-low and HER2-negative breast cancer cases, raising concerns about treatment decisions worth $100,000 or more.
  • Current immunohistochemistry (IHC) testing, regulated as a stain rather than a diagnostic assay, lacks standardization across laboratories, potentially leading to inconsistent results affecting patient care.
  • Recent clinical trials, including DESTINY-Breast06, demonstrate the growing importance of precise HER2 testing as new targeted therapies emerge for HER2-low and ultralow breast cancer patients.

Study of Trastuzumab Deruxtecan (T-DXd) vs Investigator's Choice Chemotherapy in HER2-low, Hormone Receptor Positive, Metastatic Breast Cancer

NCT04494425Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/24/2020

Trastuzumab Deruxtecan (DS-8201a) Versus Investigator's Choice for HER2-low Breast Cancer That Has Spread or Cannot be Surgically Removed [DESTINY-Breast04]

NCT03734029Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 12/27/2018

Enterprise Therapeutics Appoints Annabella Amatulli as Head of Regulatory Affairs

  • Enterprise Therapeutics has appointed Annabella Amatulli as Head of Regulatory Affairs to bolster its strategic leadership team.
  • Amatulli will spearhead regulatory strategy, focusing on the Phase 2 clinical trial of ETD001 for cystic fibrosis (CF) patients not benefiting from CFTR modulators.
  • Her extensive experience includes securing Orphan Drug Designation and Breakthrough Therapy approvals from EMA, FDA, MHRA, and NMPA.
  • Amatulli's role will also encompass defining regulatory pathways for Enterprise’s pipeline of novel low molecular weight compounds.

Optimizing Lung Cancer Clinical Trials: Technology, Endpoints, and Patient Relationships

  • Emerging endpoints like event-free survival (EFS) and pathologic complete response offer advantages over overall survival in lung cancer trials, providing earlier efficacy signals.
  • Standardizing radiotherapy through advanced technology, such as AI-driven autosegmentation, is crucial for minimizing variations and ensuring the validity of clinical trial findings.
  • Prioritizing relationships with investigators, patients, and their families is essential for improving trial adherence and achieving better outcomes in lung cancer research.
  • Integrating patient perspectives and preferences into trial design enhances patient empowerment and fosters a collaborative partnership between clinicians and patients.

PharmAla Biotech to Supply LaNeo MDMA for Clinical Trials at Mt. Sinai, Harvard, and Johns Hopkins

  • PharmAla Biotech will supply its GMP LaNeo MDMA to Mt. Sinai Health System for a clinical trial, supporting research into MDMA-assisted therapy.
  • Harvard Medical School's McLean Hospital has contracted PharmAla to provide MDMA for a clinical trial focused on borderline personality disorder.
  • Johns Hopkins Medicine has also contracted with PharmAla Biotech to supply MDMA for clinical trials, expanding the reach of their MDMA supply.
  • PharmAla Biotech emphasizes its commitment to regulatory compliance and high-quality MDMA production for clinical research purposes.

FDA Grants Orphan Drug and Rare Pediatric Disease Designations for Duchenne Muscular Dystrophy Therapies

  • Ractigen's RAG-18, an RNA activation agent targeting utrophin, received orphan drug designation for Duchenne and Becker muscular dystrophy, offering development incentives.
  • Neu-REFIX Beta glucan, an oral food supplement, also obtained rare pediatric disease and orphan drug designations for DMD treatment, facilitating clinical trials in the US.
  • Nippon Shinyaku's NS-050/NCNP-03, an antisense oligonucleotide for exon 50 skipping, previously received rare pediatric disease designation for DMD treatment.

Theradaptive Treats First Patient in OASIS Trial for Spinal Fusion with OsteoAdapt SP

  • Theradaptive has initiated its Phase I/II OASIS trial, evaluating OsteoAdapt SP for spinal fusion, with the first patient successfully treated.
  • The OASIS trial is a multicenter, blinded, controlled study assessing the safety and efficacy of OsteoAdapt SP as an alternative to autologous bone graft in TLIF procedures.
  • OsteoAdapt SP contains AMP2, a next-generation protein designed to enhance bone formation without the off-target effects associated with rhBMP-2.
  • The trial addresses the increasing demand for spinal fusion and the limitations of current treatments like autologous bone grafts and artificial implants.

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