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FDA Expands Vonvendi Approval to Include Pediatric von Willebrand Disease Patients

Drug ApprovalRare Disease
  • The FDA has expanded approval of recombinant von Willebrand factor (Vonvendi) to include pediatric patients with von Willebrand disease for acute bleeding treatment and surgical procedures.
  • This marks the first non-plasma-derived VWF therapy available for children in the United States, offering more consistent pharmacokinetics and eliminating plasma-based therapy risks.
  • The approval also extends routine prophylactic use to adults with all types of VWD, previously limited to only severe Type 3 cases.
  • Clinical data showed most non-surgical bleeds in pediatric patients were effectively managed with a single infusion, with a half-life of 14.3 hours in children.

rVWF IN PROPHYLAXIS

NCT02973087CompletedPhase 3
Takeda
Posted 11/16/2017

A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)

NCT03879135CompletedPhase 3
Baxalta now part of Shire
Posted 4/1/2019

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

NCT02932618RecruitingPhase 3
Baxalta now part of Shire
Posted 11/6/2017

Dupilumab and Remibrutinib Emerge as New Treatment Options for Chronic Spontaneous Urticaria

Monoclonal AntibodyDrug Approval
  • Recent phase 3 trial data reveals dupilumab and remibrutinib as promising alternatives to omalizumab for chronic spontaneous urticaria patients inadequately controlled with antihistamines.
  • Dupilumab achieved complete response rates of 13-31% at week 24, while remibrutinib demonstrated 28-31% complete response rates at week 12, increasing to 36% at week 24.
  • Real-world pilot data shows dupilumab provides modest symptom improvement in omalizumab-resistant patients, with mean UCT scores rising from 6.5 to 8.4 after one month of treatment.
  • All three biologics showed favorable safety profiles with rare serious adverse events and infrequent treatment discontinuations due to side effects.

Avadel Presents New LUMRYZ Data at World Sleep 2025, Demonstrating Real-World Efficacy in Narcolepsy Treatment

Drug ApprovalReal World EvidenceOrphan Drug
  • Avadel Pharmaceuticals presented 17 abstracts at World Sleep 2025, including new data from the REFRESH real-world study showing clinically meaningful improvements in narcolepsy symptoms with once-nightly LUMRYZ treatment.
  • The REFRESH study demonstrated that participants experienced improvements in excessive daytime sleepiness scores to within normal range and reduced severity of narcolepsy symptoms from moderate to mild.
  • LUMRYZ, the first and only once-at-bedtime sodium oxybate treatment, showed benefits for both patients switching from twice-nightly oxybates and those new to oxybate therapy.
  • Additional research revealed that patients with narcolepsy have significantly higher rates of sleep, pain, and psychiatric comorbidities compared to matched controls, highlighting the broader disease burden.

RYBREVANT Plus LAZCLUZE Demonstrates Superior Survival in First-Line EGFR-Mutated NSCLC

Drug Approval
  • The combination of RYBREVANT (amivantamab-vmjw) and LAZCLUZE (lazertinib) achieved 61% three-year survival versus 53% with osimertinib in first-line EGFR-mutated NSCLC treatment.
  • The chemotherapy-free regimen extended intracranial progression-free survival significantly, with 38% of patients remaining progression-free at three years compared to 18% on osimertinib.
  • Johnson & Johnson presented updated MARIPOSA trial data at WCLC 2025, showing the combination prevents common EGFR and MET resistance mechanisms.
  • New subcutaneous formulation and once-monthly dosing options are being developed to improve patient convenience and treatment experience.

Premedication to Reduce Amivantamab Associated Infusion Related Reactions

NCT05663866Active, Not RecruitingPhase 2
Janssen Research & Development, LLC
Posted 5/18/2023

A Study of Lazertinib With Subcutaneous Amivantamab Compared With Intravenous Amivantamab in Participants With Epidermal Growth Factor Receptor (EGFR)-Mutated Advanced or Metastatic Non-small Cell Lung Cancer

NCT05388669Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 8/5/2022

A Study of Combination Therapy With Amivantamab and Cetrelimab in Participants With Metastatic Non-small Cell Lung Cancer

NCT05908734Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 5/18/2023

A Study of Amivantamab in Combination With Lazertinib, or Amivantamab in Combination With Platinum-Based Chemotherapy, for Common Epidermal Growth Factor Receptor (EGFR)-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC)

NCT06667076RecruitingPhase 2
Janssen Research & Development, LLC
Posted 12/16/2024

A Study of Amivantamab Subcutaneous (SC) Administration for the Treatment of Advanced Solid Malignancies

NCT04606381Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 11/10/2020

A Study of Amivantamab in Participants With Advanced or Metastatic Solid Tumors Including Epidermal Growth Factor Receptor (EGFR)-Mutated Non-Small Cell Lung Cancer

NCT05498428RecruitingPhase 2
Janssen Research & Development, LLC
Posted 11/11/2022

A Study of Amivantamab and Capmatinib Combination Therapy in Unresectable Metastatic Non-small Cell Lung Cancer

NCT05488314Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 12/13/2022

A Study of Combination Amivantamab and Carboplatin-Pemetrexed Therapy, Compared With Carboplatin-Pemetrexed, in Participants With Advanced or Metastatic Non-Small Cell Lung Cancer Characterized by Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions

NCT04538664Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 10/13/2020

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

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

Study of Amivantamab, a Human Bispecific EGFR and cMet Antibody, in Participants With Advanced Non-Small Cell Lung Cancer

NCT02609776Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 5/24/2016

A Study of Combination Therapy With Amivantamab and Docetaxel in Participants With Metastatic Non-small Cell Lung Cancer

NCT06532032Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 7/23/2024

A Study of Lazertinib as Monotherapy or in Combination With Amivantamab in Participants With Advanced Non-small Cell Lung Cancer

NCT04077463Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 9/4/2019

Enhanced Dermatological Care to Reduce Rash and Paronychia in Epidermal Growth Factor Receptor (EGRF)-Mutated Non-Small Cell Lung Cancer (NSCLC) Treated First-line With Amivantamab Plus Lazertinib

NCT06120140RecruitingPhase 2
Janssen Research & Development, LLC
Posted 2/16/2024

Shield Therapeutics' ACCRUFeR Receives FDA Priority Review for Pediatric Iron Deficiency Anemia

Drug Approval
  • The FDA has granted Priority Review status to Shield Therapeutics' ACCRUFeR (ferric maltol) for treating iron deficiency anemia in adolescents aged 10 years and above, with approval anticipated in 2026.
  • The regulatory submission follows positive results from the Phase 3 FORTIS pediatric clinical trial that demonstrated efficacy, safety, and tolerability of the new oral liquid pediatric formulation.
  • Shield plans to further extend the indication to children as young as 1 month old through a separate NDA submission for a pediatric suspension formulation.
  • The company's EU partner Norgine has also filed for regulatory approval with the EMA for the adolescent population, targeting 2026 approval.

Biohaven Prepares for Potential First-in-Class Spinocerebellar Ataxia Drug Launch as FDA Decision Looms

Rare DiseaseDrug ApprovalOrphan Drug
  • Biohaven's Vyglxia could become the first approved treatment for spinocerebellar ataxia, a rare genetic brain disorder affecting movement and coordination, with FDA decision expected between October and December.
  • The company has established comprehensive launch infrastructure including patient hubs, insurance specialists, and sales teams, with CEO Vlad Coric stating they are "ready to ship on Day 1" following potential approval.
  • Approximately 400 patients are already receiving Vyglxia through expanded access programs, and the company estimates 6,000-7,000 of the 15,000 U.S. patients will be reachable at launch through specialized treatment centers.
  • Unlike previous competitive launches, Vyglxia faces no direct competition in the spinocerebellar ataxia market, positioning Biohaven for a streamlined commercial strategy with a sales team less than 10% the size used for their previous migraine drug.

FDA Clears Medtronic's MiniMed 780G System for Abbott Sensor Integration and Type 2 Diabetes Use

Drug Approval
  • The FDA cleared Medtronic's SmartGuard algorithm as an interoperable automated glycemic controller, enabling integration with Abbott's Instinct sensor for type 1 diabetes patients.
  • The MiniMed 780G system received FDA approval for use in adults 18 and older with insulin-requiring type 2 diabetes, making it the first automated insulin delivery system with Meal Detection technology available to this population.
  • Clinical trials demonstrated significant improvements in glycemic control, with participants experiencing a 0.7% reduction in HbA1c and increased Time in Range to 81% from a baseline of 72%.
  • The regulatory clearance completes Medtronic's FDA pre-market approval pathway for Instinct sensor integration, with ordering expected to begin following completion of compliance documentation in the coming weeks.

Hengrui Medicine Receives China's First EZH2 Inhibitor Approval for Hematological Malignancies

Drug ApprovalTargeted Therapy
  • Hengrui Medicine's Zemetostat received conditional approval from China's NMPA, becoming the country's first EZH2 inhibitor for treating relapsed or refractory peripheral T-cell lymphoma.
  • The drug demonstrated a 34% objective response rate in Phase II trials for patients with relapsed/refractory follicular lymphoma and EZH2 mutations, addressing a critical unmet medical need.
  • Hengrui's regulatory milestone positions the company to capture significant market share in the global EZH2 inhibitors market, projected to reach $9.59 billion by 2034.
  • The approval represents a strategic breakthrough for Hengrui's transition from generic drugs to innovative therapies, with innovative drug revenue accounting for 61% of total income.

Tazemetostat for the Treatment of Relapsed/Refractory Follicular Lymphoma

NCT05467943CompletedPhase 2
Hutchmed
Posted 7/29/2022

Shanghai Henlius Biotech Receives FDA Approval for Two Denosumab Biosimilars for Osteoporosis Treatment

Drug ApprovalFDA Approval
  • Shanghai Henlius Biotech has received FDA approval for two denosumab biosimilars, BILDYOS and BILPREVDA, marking the company's sixth overseas product approval.
  • The biosimilars are approved for eight indications related to osteoporosis and bone conditions, specifically targeting postmenopausal women at high risk for fractures.
  • FDA approval was based on comprehensive comparative studies demonstrating similarity to the reference product Prolia in quality, safety, and efficacy.
  • The products will be commercialized outside China through a partnership with Organon LLC, expanding the company's global market reach.

FDA Approves Weekly Autoinjector for Eisai's Alzheimer's Drug Leqembi, Enabling At-Home Treatment

Drug Approval
  • The FDA has approved a once-weekly autoinjector version of Eisai's Alzheimer's drug Leqembi, branded as Leqembi IQLIK, marking the first at-home dosing option for amyloid-based Alzheimer's therapy.
  • The autoinjector will be available for early Alzheimer's patients transitioning to maintenance dosing after 18 months of intravenous treatment, launching in the US in October 2025.
  • The approval reduces injection time from one hour for IV infusions to just 15 seconds, while potentially improving patient adherence and freeing up infusion capacity for new patients.
  • Phase III CLARITY AD study results demonstrated no efficacy difference between patients switching to the autoinjector after 18 months of initial IV dosing.

A Study to Confirm Safety and Efficacy of Lecanemab in Participants With Early Alzheimer's Disease

NCT03887455Active, Not RecruitingPhase 3
Eisai Inc.
Posted 3/27/2019

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