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Regeneron Navigates EYLEA Decline with Pipeline Diversification and Regulatory Wins

Drug ApprovalOncology
  • Regeneron reported challenging Q1 2025 results with total revenues declining 4% to $3.029 billion, primarily driven by EYLEA franchise headwinds from biosimilar competition and pricing pressures.
  • The company's diversification strategy shows promise with Dupixent global sales rising 19% to $3.67 billion and EU approval of Lynozyfic for multiple myeloma, demonstrating pipeline strength beyond ophthalmology.
  • Strategic moves including a $7 billion infrastructure investment, inaugural dividend of $0.88 per share, and $1.05 billion share buyback program signal management confidence in long-term growth prospects.
  • Key regulatory catalysts ahead include FDA decisions on EYLEA HD expanded indications with August 2025 target date and potential approvals for odronextamab in follicular lymphoma.

FDA Maintains Record-Breaking Approval Pace Despite Organizational Upheaval, Highlighting Three Breakthrough Therapies

Drug ApprovalOncology
  • The FDA has approved 84 drugs in 2025 so far, marking the second-highest total for this period despite major organizational changes including 3,500 staff layoffs.
  • Gilead's Yeztugo represents a potential game-changer for HIV prevention with 100% efficacy in phase 3 trials and twice-yearly dosing, though priced at $28,218 annually.
  • Daiichi Sankyo's ADC portfolio expansion continues with Datroway's lung cancer approval, while GSK's Blujepa becomes the first new urinary tract infection antibiotic in three decades.

Swissmedic Launches Fast-Track Clinical Trial Review to Accelerate Patient Access to Life-Saving Treatments

Drug Approval
  • Swissmedic introduces a pilot fast-track procedure for clinical trial applications, reducing review times from 30 to 20 days for known investigational drugs and from 60 to 40 days for first-in-human studies.
  • The accelerated process prioritizes studies addressing high medical need where no approved treatment options exist in Switzerland, positioning the country ahead of EU standards.
  • The pilot program runs from July 2025 through 2026, with potential permanent implementation by 2027, while maintaining all safety requirements and regulatory standards.
  • This initiative aims to strengthen Switzerland's position as a leading clinical research location and provide patients faster access to innovative, potentially life-saving therapies.

Complement-Based Drug Development Gains Momentum Following FDA Approvals and Pipeline Advances

Drug ApprovalRare Disease
  • The complement-based therapeutics field is experiencing significant growth following recent FDA approvals, including Novartis' alternative complement inhibitor for PNH and SYFOVRE approval.
  • Multiple complement inhibitors are advancing through clinical trials across diverse therapeutic areas including nephrology, ophthalmology, neurodegeneration, and inflammation.
  • Industry focus is expanding beyond rare diseases to larger patient populations, with companies exploring combination therapies and indication expansion strategies.
  • The 8th Annual Complement-Based Drug Development Summit will convene 100+ industry leaders to address key challenges in complement pathway targeting and clinical trial design.

FDA Approves Streamlined Access for Bristol Myers Squibb CAR T Cell Therapies

CAR-TDrug Approval
  • The FDA has approved label updates for Bristol Myers Squibb's CAR T cell therapies Breyanzi and Abecma, reducing patient monitoring requirements and removing Risk Evaluation and Mitigation Strategy (REMS) programs.
  • Key changes include reducing driving restrictions from 8 weeks to 2 weeks post-treatment and decreasing the requirement to stay near healthcare facilities from 4 weeks to 2 weeks following infusion.
  • These updates reflect growing real-world evidence from over 30,000 patients treated with CAR T cell therapy, with studies showing most serious adverse events occur within the first two weeks of infusion.
  • Currently only about 2 in 10 eligible patients receive cell therapy treatment due to logistical and geographic barriers, which these label changes aim to address.

A Study to Evaluate the Efficacy and Safety of JCAR017 in Adult Subjects With Relapsed or Refractory Indolent B-cell Non-Hodgkin Lymphoma (NHL)

NCT04245839RecruitingPhase 2
Celgene
Posted 7/14/2020

A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

NCT03575351CompletedPhase 3
Celgene
Posted 10/23/2018

Study Evaluating Safety and Efficacy of JCAR017 in Subjects With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

NCT03331198RecruitingPhase 1
Juno Therapeutics, a Subsidiary of Celgene
Posted 11/27/2017

Lisocabtagene Maraleucel (JCAR017) as Second-Line Therapy (TRANSCEND-PILOT-017006)

NCT03483103CompletedPhase 2
Juno Therapeutics, a Subsidiary of Celgene
Posted 7/27/2018

FDA Rejects Accelerated Approval for Edgewise's Sevasemten in Becker Muscular Dystrophy

Drug Approval
  • The FDA declined to consider Edgewise Therapeutics' sevasemten for accelerated approval in Becker muscular dystrophy, citing insufficient data from the Phase II CANYON trial.
  • The agency affirmed that North Star Ambulatory Assessment scores can serve as a clinically meaningful endpoint for traditional approval, providing a clear regulatory pathway forward.
  • Edgewise must now rely on its ongoing Phase III GRAND CANYON trial, which is expected to read out in Q4 2026 and is designed to demonstrate statistical significance versus placebo.
  • The company's stock dropped 10% following the announcement, though analysts noted the decision was not unexpected given the FDA's tightened stance on accelerated approval requirements.

Zenocutuzumab Achieves 30% Response Rate in NRG1 Fusion-Positive Solid Tumors, Receives FDA Approval

Targeted TherapyDrug ApprovalPrecision Medicine
  • The Phase 2 eNRGy trial demonstrated zenocutuzumab's clinical efficacy with a 30% objective response rate and 11.1-month median duration of response in patients with NRG1 fusion-positive solid tumors.
  • Pancreatic cancer patients showed particularly promising results with a 42% response rate and 9.2-month median progression-free survival, offering hope for this historically difficult-to-treat malignancy.
  • Zenocutuzumab received accelerated FDA approval as the first targeted therapy for NRG1-positive non-small cell lung cancer and pancreatic adenocarcinoma.
  • The bispecific antibody demonstrated a favorable safety profile with treatment-related adverse events in 95% of participants but fewer than 10% experiencing grade 3 or higher toxicities.

Study of Seribantumab in Adult Patients With NRG1 Gene Fusion Positive Advanced Solid Tumors

NCT04383210TerminatedPhase 2
Elevation Oncology
Posted 9/29/2020

A Phase I/IIa Trial of HMBD-001 in Advanced HER3 Positive Solid Tumours

NCT05057013Active, Not RecruitingPhase 1
Cancer Research UK
Posted 11/10/2021

Patritumab Deruxtecan in Patients with Solid Tumor Harboring an NRG1 Fusion

NCT06383884RecruitingPhase 2
Samsung Medical Center
Posted 8/13/2024

Dose Escalation Study to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of GSK2849330 in Subjects With Advanced Her3-Positive Solid Tumors

NCT01966445CompletedPhase 1
GlaxoSmithKline
Posted 11/26/2013

TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer

NCT02693535RecruitingPhase 2
American Society of Clinical Oncology
Posted 3/14/2016

A Study of Zenocutuzumab (MCLA-128) in Patients With Solid Tumors Harboring an NRG1 Fusion (eNRGy)

NCT02912949Active, Not RecruitingPhase 2
Partner Therapeutics, Inc.
Posted 1/1/2015

First European Patient Receives Breakthrough Treatment for Ultra-Rare APDS Immune Disorder

Rare DiseaseTargeted TherapyDrug ApprovalOrphan Drug
  • Mary Catchpole, 19, becomes the first European patient to receive leniolisib (Joenja), a newly approved targeted treatment for activated PI3-kinase delta syndrome (APDS), a rare inherited immune disorder.
  • The drug works by inhibiting an overactive enzyme that disrupts immune function, offering a potential cure for patients who previously faced lifelong infections and invasive treatments.
  • APDS was discovered by Cambridge researchers in 2013 with help from Catchpole's family, who lost four members to the condition before this breakthrough treatment became available.
  • The NHS approved leniolisib at a list price of £352,000 per year with a confidential discount, potentially benefiting up to 50 patients over age 12 in England.

China Approves First Once-Daily Nebulized LAMA for COPD, Triggering $7.5M Milestone for Theravance

Drug Approval
  • China's NMPA has approved YUPELRI (revefenacin) as the first once-daily nebulized long-acting muscarinic antagonist for maintenance treatment of chronic obstructive pulmonary disease.
  • The regulatory approval triggers a $7.5 million milestone payment from Viatris to Theravance Biopharma, with additional sales milestones up to $37.5 million and tiered royalties of 14-20% on net sales.
  • Viatris assumes full responsibility for YUPELRI's launch and commercialization in China, while Theravance incurs no commercial costs in the region.
  • The approval strengthens Theravance's financial position, which includes $131 million in cash and potential near-term milestone payments from multiple revenue streams.

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic nOH in Participants With Multiple System Atrophy

NCT05696717Active, Not RecruitingPhase 3
Theravance Biopharma
Posted 6/27/2023

XEOMIN Receives First-Ever Approval in Japan for Chronic Sialorrhea Treatment

Drug ApprovalRare Disease
  • Teijin Pharma has received approval from Japan's Ministry of Health, Labor and Welfare for XEOMIN (incobotulinumtoxinA) to treat chronic sialorrhea, marking the first drug approved for this indication in Japan.
  • The approval was based on Phase III clinical trials conducted by Merz Therapeutics in Germany and Poland, as well as trials conducted by Teijin in Japan.
  • XEOMIN works by inhibiting acetylcholine release from cholinergic nerve endings, effectively suppressing saliva secretion and reducing water and electrolyte secretion from salivary glands.
  • Chronic sialorrhea significantly impacts patients' quality of life, causing social isolation and difficulties with speaking and eating, while placing heavy burdens on families and caregivers.

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Episodic Migraine

NCT07018700RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Chronic Migraine

NCT07018713RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

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