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FDA Approves Datopotamab Deruxtecan for Previously Treated EGFR-Mutated NSCLC

Drug ApprovalOncology
  • The FDA granted accelerated approval to datopotamab deruxtecan-dlnk (Dato-DXd; Datroway) for patients with previously treated EGFR-mutated non-small cell lung cancer who have progressed on both EGFR-targeted therapies and chemotherapy.
  • Clinical trials TROPION-Lung05 and TROPION-Lung01 demonstrated a 45% overall response rate and median duration of response of 6.5 months in this patient population.
  • The approval addresses a significant unmet medical need in a treatment setting with limited therapeutic alternatives for patients with advanced EGFR-mutated NSCLC.
  • While the drug requires monitoring for adverse effects including stomatitis and interstitial lung disease, the safety profile is considered manageable by oncologists.

A Study of Nivolumab, Nivolumab Plus Ipilimumab, or Investigator's Choice Chemotherapy for the Treatment of Participants With Deficient Mismatch Repair (dMMR)/Microsatellite Instability High (MSI-H) Metastatic Colorectal Cancer (mCRC)

NCT04008030Active, Not RecruitingPhase 3
Bristol-Myers Squibb
Posted 8/5/2019

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

A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Participants With Myelofibrosis

NCT05371964RecruitingPhase 1
Geron Corporation
Posted 5/4/2022

Study to Compare the Combination of Ribociclib Plus Goserelin Acetate With Hormonal Therapy Versus Combination Chemotherapy in Premenopausal or Perimenopausal Patients With Advanced or Metastatic Breast Cancer

NCT03839823CompletedPhase 2
Novartis Pharmaceuticals
Posted 2/25/2019

Systemic Therapy With or Without Local Consolidative Therapy in Treating Patients With Oligometastatic Solid Tumor

NCT03599765Active, Not RecruitingPhase 2
M.D. Anderson Cancer Center
Posted 7/31/2018

A Study of the Combination of Ibrutinib Plus Venetoclax Versus Chlorambucil Plus Obinutuzumab for the First-line Treatment of Participants With Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL)

NCT03462719Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 4/17/2018

Ibrutinib Plus Venetoclax in Subjects With Treatment-naive Chronic Lymphocytic Leukemia /Small Lymphocytic Lymphoma (CLL/SLL)

NCT02910583CompletedPhase 2
Pharmacyclics LLC.
Posted 9/28/2016

Relacorilant in Combination With Nab-Paclitaxel in Advanced, Platinum-Resistant, High-Grade Epithelial Ovarian, Primary Peritoneal, or Fallopian-Tube Cancer

NCT05257408Active, Not RecruitingPhase 3
Corcept Therapeutics
Posted 6/29/2022

A Study Evaluating the Efficacy and Safety of Inavolisib + Palbociclib + Fulvestrant vs Placebo + Palbociclib + Fulvestrant in Participants With PIK3CA-Mutant, Hormone Receptor-Positive, HER2-Negative, Locally Advanced or Metastatic Breast Cancer

NCT04191499Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 1/29/2020

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

Study of NXC-201 CAR-T in Patients With Light Chain (AL) Amyloidosis

NCT06097832RecruitingPhase 1
Nexcella Inc.
Posted 6/5/2024

Tirzepatide Emerges as Most Effective GLP-1 Agonist for Diabetes and Weight Loss, Rapidly Overtaking Traditional Therapies

Drug Approval
  • Tirzepatide, a dual GLP-1 and GIP receptor agonist, ranked first among FDA-approved agents for both glycemic control and weight loss in a meta-analysis of 76 randomized trials involving 39,000 adults with type 2 diabetes.
  • The drug demonstrated superior efficacy with mean reductions of 2.1% in HbA1c, 3.11 mmol/L in fasting blood glucose, and 8.5 kg in body weight compared to placebo, outperforming semaglutide which ranked second.
  • Following its May 2022 market launch, tirzepatide achieved rapid uptake, reaching 12.3% of all glucose-lowering medication use by December 2023 and surpassing traditional therapies like metformin in incident prescriptions.
  • The medication's swift adoption represents a significant shift in diabetes and obesity treatment patterns, with implications for clinical practice guidelines and healthcare policy.

CMS Policy Changes Create New Reimbursement Challenges for 505(b)(2) Oncology Drugs

Drug ApprovalOncology
  • CMS has begun issuing unique J-codes to 505(b)(2) drugs as of January 2023, fundamentally changing how these medications are reimbursed and potentially affecting 943 approved drugs dating back to 2003.
  • The policy shift stems from a 2022 CMS reevaluation that now considers 505(b)(2) drugs that are not therapeutically interchangeable as sole-source products requiring individual billing codes.
  • This regulatory change creates significant operational challenges for oncology infusion centers while providing pharmaceutical manufacturers with competitive advantages including potential 7-year market exclusivity.
  • The transformation affects numerous oncology drugs approved through the 505(b)(2) pathway, including albumin-bound paclitaxel, bendamustine, bortezomib, and pemetrexed.

South Korea Approves World's First Recombinant Anthrax Vaccine BARYTHRAX

Drug Approval
  • The Korean Ministry of Food and Drug Safety approved BARYTHRAX, the world's first recombinant protein-based anthrax vaccine developed by GC Biopharma and the Korea Disease Control and Prevention Agency.
  • The vaccine eliminates toxin exposure risks associated with traditional anthrax vaccines by using protective antigen proteins produced through genetic recombination techniques.
  • BARYTHRAX demonstrated high safety and efficacy in clinical trials, with vaccinated subjects generating sufficient neutralizing antibodies and maintaining high survival rates in animal studies.
  • The approval enables South Korea to achieve self-sufficiency in anthrax vaccine production, ending reliance on imports and potentially opening export markets globally.

Myasthenia Gravis Market Set to Reach $10.3 Billion by 2034 Driven by Seven Late-Stage Pipeline Therapies

Monoclonal AntibodyDrug Approval
  • The myasthenia gravis market across seven major markets is projected to grow from $6.1 billion in 2024 to $10.3 billion by 2034, representing a 5.3% compound annual growth rate.
  • Seven late-stage pipeline products including CRD-1, Descartes-08, Imaavy, gefurulimab, inebilizumab, pozelimab + cemdisiran combination therapy, and telitacicept are expected to drive market expansion.
  • The combination therapy of pozelimab and cemdisiran shows particular promise for treating seronegative myasthenia gravis patients, addressing a significant unmet medical need.
  • Despite growth potential, generic erosion and biosimilar competition pose barriers, particularly affecting high-grossing disease-modifying therapies that generated an estimated $5 billion in combined sales in 2024.

Once-Nightly Sodium Oxybate Shows Safety Profile in Narcolepsy Patients with Sleep Apnea Concerns

Drug Approval
  • Post-hoc analysis of the phase 3 REST-ON trial demonstrates that once-nightly sodium oxybate (Lumryz) does not worsen apnea-hypopnea index in narcolepsy patients with mild or no sleep apnea.
  • The study found minimal respiratory-related adverse drug reactions, with only 0.9% of treated patients experiencing sleep apnea and 0.9% reporting snoring.
  • Mean apnea-hypopnea index values remained consistently low throughout the 13-week treatment period, with no significant differences between treatment and placebo groups.
  • Additional analysis revealed the drug effectively reduced hypnagogic and hypnopompic hallucinations, with significant improvements observed across multiple time points compared to placebo.

An Open Label Study of FT218 in Subjects With Narcolepsy

NCT04451668CompletedPhase 3
Avadel
Posted 6/12/2020

Once-Nightly Sodium Oxybate for Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy

NCT02720744CompletedPhase 3
Avadel
Posted 11/17/2016

PharmaMar Expands Global Reach of Zepzelca with Argentina Approval and Merck Partnership for Japan

Drug ApprovalOncology
  • PharmaMar and partner Adium Pharma have secured conditional marketing approval for Zepzelca (lurbinectedin) in Argentina for treating metastatic small cell lung cancer, extending the drug's availability to 18 territories globally.
  • The company has simultaneously formed an exclusive license agreement with Merck for Zepzelca's development and commercialization in Japan, receiving an initial payment of €22 million with potential for €31 million more in milestone payments.
  • These strategic moves strengthen PharmaMar's position in the oncology market while providing new treatment options for patients with an aggressive form of lung cancer across multiple regions.

Mesoblast's Ryoncil Receives US FDA Orphan Drug Approval, Driving 15% Share Price Surge

Orphan DrugDrug Approval
  • Mesoblast received US FDA orphan drug approval for Ryoncil, granting seven years of market exclusivity and driving a 15% share price increase over one week.
  • The approval significantly strengthens Mesoblast's competitive positioning, with expanded insurance coverage now reaching 104 million US lives.
  • Despite the regulatory milestone, Mesoblast remains unprofitable with A$47.93 million in net losses, though analysts forecast 56.7% annual revenue growth.
  • The company's shares still trade approximately 88% below consensus analyst price targets, suggesting potential for further market adjustments.

Japan Prioritizes 14 "Drug Loss" Products for Urgent Domestic Development to Address Critical Medical Needs

Drug Approval
  • Japan's Ministry of Health, Labor and Welfare has categorized 78 "drug loss" products into four priority tiers, identifying 14 as highest priority for domestic development.
  • The regulatory council determined that 11 drug loss products have high medical needs, enabling development requests or sponsor invitations to be issued.
  • This initiative represents Japan's systematic approach to addressing pharmaceutical access gaps where overseas-approved drugs remain unavailable domestically.
  • The prioritization scheme aims to facilitate partnerships between domestic firms and overseas originators to accelerate drug availability in Japan.

CRISPR Gene Editing Advances from Laboratory to Clinic with 250+ Active Trials Across Multiple Therapeutic Areas

Gene EditingCAR-TDrug Approval
  • CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas.
  • CASGEVY became the first approved CRISPR-based therapy in 2023, receiving regulatory clearance in multiple regions for treating sickle cell disease and beta thalassemia through foetal haemoglobin induction.
  • Gene editing for blood disorders continues to lead the clinical landscape, with the majority of Phase 3 trials targeting sickle cell disease and beta thalassemia, while Phase 3 trials are also underway in hereditary amyloidosis and immunodeficiencies.
  • Clinical applications now span 15+ therapeutic areas including blood cancers, viral diseases, metabolic disorders, autoimmune diseases, inherited eye diseases, cardiovascular disease, and neurological conditions.

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