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Clinical Trial News

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough

Monoclonal AntibodyDrug ApprovalRare DiseaseOrphan Drug
  • The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
  • In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
  • The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
  • Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

NCT01818492CompletedPhase 2
Swedish Orphan Biovitrum
Posted 7/1/2013

VR-CAP Regimen Demonstrates Superior Overall Survival in Mantle Cell Lymphoma

  • The VR-CAP regimen (bortezomib, rituximab, cyclophosphamide, doxorubicin, and prednisone) significantly extended overall survival compared to R-CHOP in transplant-ineligible MCL patients.
  • After a median follow-up of 82 months, VR-CAP showed a median overall survival of 90.7 months versus 55.7 months for R-CHOP (HR 0.66, p=0.001).
  • The VR-CAP regimen presented a manageable safety profile, supporting its consideration as a frontline treatment option for mantle cell lymphoma.

Study of the Combination of Rituximab, Cyclophosphamide, Doxorubicin, VELCADE, and Prednisone or Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone in Patients With Newly Diagnosed Mantle Cell Lymphoma

NCT00722137CompletedPhase 3
Millennium Pharmaceuticals, Inc.
Posted 5/1/2008

FDA Approves First Direct-to-Consumer Pharmacogenetic Test from 23andMe

Drug Approval
  • The FDA approved 23andMe's Personal Genome Service Pharmacogenetic Reports, marking the first direct-to-consumer genetic test to help patients understand how their genetics may affect responses to over 50 common prescription and over-the-counter drugs.
  • The test analyzes 33 genetic variants associated with drug metabolism, including responses to blood thinners like clopidogrel and mental health treatments, but comes with strict limitations prohibiting its use for treatment decisions or medical advice.
  • Medical experts express caution about the clinical utility of pharmacogenetic testing for general consumers, noting that while genetic variants affect drug metabolism, the clinical interpretation and prescription implications remain unclear for most patients.

Novartis Launches Biome Innovation Lab Network to Accelerate Digital Health Solutions

  • Novartis establishes the Novartis Biome, a digital health innovation lab network headquartered in San Francisco, to foster collaboration with healthcare technology startups and accelerate digital transformation.
  • The initiative includes the HealthX World Series challenges, with recent competitions focusing on heart failure solutions, where startups Veta Health and Wavy emerged as winners.
  • Participating startups will receive workspace, mentorship, access to Novartis datasets, and opportunities for proof-of-concept studies, without mandatory company ownership requirements.

Pfizer Strengthens Digital Strategy with New Chief Digital Officer Appointment

• Pfizer has appointed Lidia Fonseca as its new Chief Digital and Technology Officer, joining the executive leadership team in January to accelerate the company's digital capabilities.
• The appointment comes as part of a broader executive restructuring following Albert Bourla's succession as CEO, reflecting the growing importance of digital technology in pharmaceutical R&D and operations.
• The leadership reorganization includes new roles for key executives, with Mikael Dolsten overseeing medical operations, Angela Hwang leading innovative medicines, and Freda Lewis-Hall becoming Chief Patient Officer.

FDA Launches Cybersecurity Playbook to Protect Medical Devices from Digital Threats

  • The FDA has released a comprehensive cybersecurity playbook in collaboration with Mitre Corporation to help healthcare organizations protect medical devices and prepare for potential security breaches.
  • FDA Commissioner Scott Gottlieb warns that cyber threats to medical devices are no longer theoretical, with potential impacts on network-connected equipment like radiologic imaging devices.
  • The agency is establishing new partnerships through memoranda of understanding and creating a Centre of Excellence for Digital Health to strengthen medical device cybersecurity measures.

J&J's Continuous Manufacturing Journey: Pioneering Success with HIV Drug Prezista

  • Johnson & Johnson achieved a historic FDA approval in 2016 to transition HIV medication Prezista from batch to continuous manufacturing, marking a significant advancement in pharmaceutical production methods.
  • The continuous manufacturing system demonstrates superior efficiency through real-time quality monitoring, reduced waste, and flexible production capabilities, while maintaining the highest quality standards.
  • J&J's strategic decision to implement continuous manufacturing with an established product like Prezista, rather than a new drug, has provided valuable insights and lessons for future pharmaceutical manufacturing innovation.

Survey Reveals Gap Between Patient-Centric Aspirations and Implementation in Pharma Industry

  • New industry survey shows 91% of pharma professionals value patient-focused missions, yet only 22% understand how to effectively engage with patients.
  • Significant trust gap emerges as only 36% of patients express strong trust in the pharmaceutical industry, with notable disparities between employee and patient perceptions of patient-centric initiatives.
  • Industry leaders emphasize that connecting employees to their sense of purpose and patient impact is key to achieving true patient centricity and sustainable business outcomes.

Life Molecular Imaging Secures FDA Fast Track Designation for [18F]florbetaben in Cardiac Amyloidosis Diagnosis

Orphan Drug
  • Life Molecular Imaging received FDA Fast Track Designation for [18F]florbetaben to diagnose cardiac amyloid light-chain (AL) and amyloid transthyretin-related (ATTR) amyloidosis.
  • The PET imaging agent is already approved as Neuraceq® for detecting brain amyloid plaques and has demonstrated capability to identify amyloid deposits in the heart.
  • A Phase 3 trial (NCT05184088) is currently underway to validate [18F]florbetaben's efficacy in cardiac amyloidosis diagnosis.
  • The designation aims to streamline the complex diagnostic process for cardiac AL amyloidosis, potentially providing earlier access to therapy and improved patient monitoring.

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088RecruitingPhase 3
Life Molecular Imaging GmbH
Posted 1/13/2023

EU Approves Sanofi's Cablivi as First Targeted Therapy for Rare Blood Clotting Disorder aTTP

  • The European Commission has authorized Cablivi (caplacizumab) as the first specific treatment for acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder.
  • Clinical trials involving 220 adults demonstrated Cablivi significantly reduced platelet count normalization time and decreased aTTP-related complications when added to standard care, with no deaths reported in the treatment group.
  • The approval marks a significant advancement in aTTP treatment, where current standard therapy still carries up to 20% mortality rate, with Cablivi also under priority review by the FDA.

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