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Taiwan Administers First Alzheimer's Drug Targeting Amyloid Plaques

Monoclonal AntibodyDrug Approval
  • Far Eastern Memorial Hospital in New Taipei City administered Taiwan's first dose of Kisunla (donanemab), a new Alzheimer's drug that targets underlying disease causes rather than just symptoms.
  • The monoclonal antibody treatment, developed by Eli Lilly, was given to an 83-year-old woman and represents a breakthrough approach to reducing amyloid plaques in the brain.
  • Both Kisunla and Leqembi require 12-18 months of treatment to significantly reduce amyloid accumulation, with fewer than 100,000 of Taiwan's 350,000 dementia patients eligible for these therapies.
  • While promising, experts caution these drugs are not cures and require ongoing monitoring, with long-term effectiveness and side effects still under evaluation.

FDA Approves Novavax COVID-19 Vaccine with Restricted Use for High-Risk Groups

Drug Approval
• The FDA has granted full approval for Novavax's protein-based COVID-19 vaccine, but with unusual restrictions limiting its use to adults 65 and older or those 12-64 with health risk factors.
• Novavax's vaccine represents the only traditional protein-based coronavirus vaccine option in the US market, previously available under emergency authorization for anyone 12 and older.
• The restricted approval comes amid shifting vaccine policies under the Trump administration, with the FDA requiring Novavax to conduct additional post-approval studies on potential heart conditions and benefits for certain age groups.

First Biologic Drug for Pulmonary Sarcoidosis in 50 Years Administered at Oxford Hospital

Rare DiseaseDrug Approval
• Infliximab, a TNF-α inhibitor, has become the first new drug approved for pulmonary sarcoidosis in over 50 years, following NHS England commissioning with NICE support.
• The biologic therapy, administered to the first patient at Oxford's John Radcliffe Hospital, offers potential to reduce reliance on broad immunosuppressants that often cause significant side effects.
• Professor Ling-Pei Ho, who led the Oxford Sarcoidosis Service and chaired the initial NHS England working policy group, played a key role in securing the drug's approval for this rare condition affecting 1 in 10,000 UK residents.

Cardioplexol™ Demonstrates Safety and Efficacy in Phase 3 Trial for Cardiac Surgery

Drug Approval
  • A pivotal phase 3 non-inferiority trial has confirmed the safety and efficacy of Cardioplexol™, a new low-volume cardioplegic solution, compared to standard Buckberg blood cardioplegia in cardiac surgeries.
  • Cardioplexol™ showed significant advantages including faster cardiac arrest (11 vs 71 seconds), reduced cross-clamp time, lower defibrillation rates (10% vs 52%), and decreased need for post-operative inotropic support.
  • Following successful clinical validation, Cardioplexol™ received marketing authorization in Switzerland in September 2023 and in 10 European countries in April 2024.

China Approves First Domestically Developed Enzyme Replacement Therapy for Gaucher Disease

Orphan DrugDrug ApprovalRare Disease
  • CANbridge Pharmaceuticals has received NMPA approval for velaglucerase-beta (Gaurunning), China's first domestically developed enzyme replacement therapy for Type I and III Gaucher disease in patients aged 12 and above.
  • The pivotal clinical trial demonstrated statistically significant reductions in spleen volume at both 60 U/kg (P<0.0001) and 30 U/kg (P<0.001) doses, meeting its primary efficacy endpoint.
  • Developed in collaboration with WuXi Biologics, Gaurunning represents a breakthrough in rare disease treatment in China, potentially improving accessibility and affordability for the estimated 3,000 Chinese Gaucher disease patients.

Resistance Mechanisms Drive Personalized Treatment Selection After Osimertinib Failure in EGFR-Mutant NSCLC

Precision MedicineDrug Approval
  • Understanding specific resistance mechanisms to osimertinib is crucial for selecting optimal second-line therapies in EGFR-mutant non-small cell lung cancer patients.
  • MET amplification remains the most common resistance driver, with savolitinib plus osimertinib showing significant progression-free survival benefits in phase 2 and 3 trials.
  • Antibody-drug conjugates like datopotamab deruxtecan have demonstrated robust efficacy and received FDA accelerated approval for previously treated EGFR-mutant NSCLC.
  • The ORCHARD trial represents a precision medicine approach, assigning patients to targeted combination therapies based on their specific resistance biomarkers.

HERTHENA-Lung02: A Study of Patritumab Deruxtecan Versus Platinum-based Chemotherapy in Metastatic or Locally Advanced EGFRm NSCLC After Failure of EGFR TKI Therapy

NCT05338970Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 7/8/2022

A Study of Amivantamab and Lazertinib Combination Therapy Versus Osimertinib in Locally Advanced or Metastatic Non-Small Cell Lung Cancer

NCT04487080Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 9/30/2020

Phase 2 Platform Study in Patients With Advanced Non-Small Lung Cancer Who Progressed on First-Line Osimertinib Therapy (ORCHARD)

NCT03944772Active, Not RecruitingPhase 2
AstraZeneca
Posted 6/25/2019

Study of DS-1062a Versus Docetaxel in Previously Treated Advanced or Metastatic Non-small Cell Lung Cancer With or Without Actionable Genomic Alterations (TROPION-LUNG01)

NCT04656652Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 12/21/2020

Study of DS-1062a in Advanced or Metastatic Non-small Cell Lung Cancer With Actionable Genomic Alterations (TROPION-Lung05)

NCT04484142Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 3/30/2021

A Study of Amivantamab and Lazertinib in Combination With Platinum-Based Chemotherapy Compared With Platinum-Based Chemotherapy in Patients With Epidermal Growth Factor Receptor (EGFR)-Mutated Locally Advanced or Metastatic Non- Small Cell Lung Cancer After Osimertinib Failure

NCT04988295Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 11/17/2021

A trial to learn how well osimertinib with savolitinib works in people with non-small cell lung cancer (NSCLC) that has gotten worse after initial treatment with osimertinib.

2024-512581-34-00Active, Not RecruitingPhase 2
AstraZeneca AB

HERTHENA-Lung01: Patritumab Deruxtecan in Subjects With Metastatic or Locally Advanced EGFR-mutated Non-Small Cell Lung Cancer

NCT04619004Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 2/2/2021

HER3-DXd (Patritumab Deruxtecan; U3-1402) in Combination With Osimertinib in Subjects With Locally Advanced or Metastatic EGFR-mutated Non-Small Cell Lung Cancer

NCT04676477Active, Not RecruitingPhase 1
Daiichi Sankyo
Posted 6/11/2021

Study on Savolitinib Combined With Osimertinib in Treatment of Advanced NSCLC With MET Amplification

NCT05015608Active, Not RecruitingPhase 3
Hutchison Medipharma Limited
Posted 11/22/2021

ATTR-CM Treatment Landscape Expands with Three FDA-Approved Disease-Modifying Therapies

Drug ApprovalFDA Approval
  • Transthyretin amyloid cardiomyopathy (ATTR-CM) has evolved from an untreatable condition to one managed with three FDA-approved disease-modifying agents: tafamidis, acoramidis, and vutrisiran.
  • Tafamidis demonstrated substantial survival benefit in the ATTR-ACT trial, while acoramidis showed similar efficacy in ATTRibute-CM, and vutrisiran represents a mechanistically distinct gene silencing approach validated in HELIOS-B.
  • Next-generation therapies targeting amyloid clearance from the myocardium are in development, including NI006, PRX004, and AT-02, potentially offering complementary treatment options.
  • The ACT-EARLY trial will test whether early treatment with acoramidis can prevent clinical disease onset in high-risk individuals with pathogenic TTR variants.

FDA Deputy Commissioner Signals Potential Overhaul of PDUFA Reauthorization Structure

Drug Approval
  • The FDA is preparing to renegotiate its multibillion-dollar, five-year user fee agreements with the pharmaceutical industry later this summer, with potential structural changes on the horizon.
  • A newly appointed FDA deputy commissioner suggested on Friday that the current PDUFA framework may need significant restructuring to better serve regulatory needs.
  • The Prescription Drug User Fee Act (PDUFA) represents a critical funding mechanism for FDA drug review processes, with negotiations set to impact future drug approval timelines and requirements.

Acadia Pharmaceuticals Secures Patent Victory for Parkinson's Drug Nuplazid Until 2038

Rare DiseaseDrug Approval
  • The U.S. District Court for the District of Delaware ruled in favor of Acadia Pharmaceuticals, upholding their '721 formulation patent for Nuplazid against Aurobindo Pharma's generic challenge.
  • Judge Gregory B. Williams determined that Aurobindo's proposed generic pimavanserin capsules would infringe Acadia's patent, which provides protection for the 34 mg capsule formulation until August 2038.
  • The ruling represents a significant win for Acadia, securing long-term market exclusivity for their FDA-approved treatment for hallucinations and delusions associated with Parkinson's disease psychosis.

Novartis Receives Swiss Approval for First Malaria Treatment Designed for Newborns and Infants

Drug Approval
  • Novartis' Coartem Baby (artemether-lumefantrine) has been approved by Swissmedic as the first malaria medicine specifically designed for newborns and infants.
  • The treatment was developed in collaboration with Medicines for Malaria Venture and eight African countries to address the critical gap in malaria care for the most vulnerable population.
  • Novartis plans to introduce the treatment to malaria-endemic areas, marking a significant milestone after three decades of the company's commitment to fighting malaria.
  • The approval represents a major breakthrough in pediatric malaria treatment, ensuring that even the smallest patients can receive clinically proven care.

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