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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.

FDA Approves Roche's Polivy as First New Frontline DLBCL Treatment in Nearly 20 Years

Drug ApprovalOncology
  • The FDA has approved Roche's Polivy in combination with R-CHP chemotherapy as the first new treatment for previously-untreated diffuse large B-cell lymphoma in nearly 20 years.
  • The POLARIX study demonstrated a 27% reduction in the risk of disease progression, relapse, or death compared to standard R-CHOP therapy, with benefits maintained over 40 months of follow-up.
  • The approval addresses a critical unmet need, as approximately 40% of DLBCL patients relapse after initial therapy and face poor prognosis with median survival under two years.
  • Analysts project peak sales of $2.4 billion for Polivy in first-line DLBCL, while NICE has also recommended NHS funding for the drug in second-line treatment settings.

GSK's Oral Carbapenem Antibiotic Tebipenem HBr Succeeds in Phase 3 Trial, Setting Stage for FDA Filing

Drug Approval
  • GSK's phase 3 PIVOT-PO trial for tebipenem HBr was stopped early after an independent monitoring committee determined the oral carbapenem antibiotic was at least as effective as intravenous treatment for complicated urinary tract infections.
  • The drug could become the first oral carbapenem antibiotic approved for complicated UTIs in the US, offering patients home-based treatment instead of hospital-based IV therapy.
  • GSK plans to file for FDA approval in the second half of 2024, following a $66 million licensing deal with Spero Therapeutics after the FDA initially rejected the drug in 2022.
  • The success represents a significant advance in combating antimicrobial resistance, with an estimated 2.9 million complicated UTI cases treated annually in the US at a cost of approximately $6 billion per year.

China's Aphranel MagiCCrystal CaHA Filler Receives Global Regulatory Approvals, Setting New Standards in Regenerative Aesthetics

Drug Approval
  • Aphranel MagiCCrystal, developed by Shanghai Moyom Biotechnology, has become China's first Class III calcium hydroxylapatite (CaHA) microsphere-based dermal filler to receive NMPA certification.
  • The innovative filler features proprietary "raspberry-like" through-hole microsphere morphology and physically crosslinked gel matrix, offering superior biocompatibility with 40% increased porosity compared to competitors.
  • With regulatory clearances across Asia, Europe, and Latin America, including EU Medical Device certification and MDSAP compliance, the product marks China's transition from industry follower to global innovator in aesthetic medicine.

USDA Approves First Autologous Immunotherapy for Canine Osteosarcoma

Drug ApprovalOncology
  • ELIAS Cancer Immunotherapy (ECI) has received full approval from the USDA Center for Veterinary Biologics as the first autologous prescription product for treating canine osteosarcoma.
  • The innovative treatment works by conditioning the dog's immune system to recognize cancer cells, then delivering activated killer T cells that target and attack those specific cancer cells.
  • ECI is available at 100 authorized treatment centers across the United States, with the treatment process requiring cancer tissue collection prior to surgery for manufacturing the personalized therapy.

NHS England Denies Access to Life-Extending Prostate Cancer Treatments Despite Strong Clinical Evidence

OncologyDrug Approval
• Thousands of men with prostate cancer in England are being denied access to abiraterone, a life-extending drug available free on the NHS in Scotland and Wales for high-risk patients. • NHS England's refusal is based on "overall affordability" concerns, forcing English patients to pay £250 monthly for private prescriptions or go without treatment. • Separately, NHS England is ignoring breakthrough research showing docetaxel chemotherapy can add 22 months of life when used earlier in metastatic prostate cancer patients. • The docetaxel findings from the UK-funded STAMPEDE trial represent a 25% improvement in overall survival, but regulatory barriers prevent formal approval despite the drug being low-cost and off-patent.

Ascelia Pharma Advances Orviglance NDA Following Positive FDA Meeting, Submission Planned for Mid-2025

Drug ApprovalOncologyRare Disease
  • Ascelia Pharma has received positive guidance from the FDA regarding its New Drug Application for Orviglance, a novel contrast agent for MRI liver imaging in patients with impaired kidney function.
  • The company's Phase 3 SPARKLE study met its primary endpoint with statistical significance (p<0.001), demonstrating Orviglance's ability to improve visualization of focal liver lesions in the target patient population.
  • Following successful completion of nine clinical studies involving 286 participants, Ascelia plans to submit the Orviglance NDA by mid-2025, likely in early August, with a cash runway extending through at least the end of 2025.

FDA Accepts Merck's Application for Novel Two-Drug HIV Regimen Doravirine/Islatravir

Drug Approval
  • The FDA has accepted Merck's New Drug Application for doravirine/islatravir (DOR/ISL), a once-daily, oral, two-drug regimen for adults with virologically suppressed HIV-1 infection.
  • If approved, DOR/ISL would be the first FDA-approved two-drug regimen without an integrase inhibitor that demonstrated non-inferior efficacy to three-drug regimens in Phase 3 trials.
  • The application is based on 48-week data from two pivotal Phase 3 trials (MK-8591A-051 and MK-8591A-052) showing non-inferiority to baseline therapy and BIC/FTC/TAF respectively.
  • The FDA has set a target action date of April 28, 2026, under the Prescription Drug User Fee Act (PDUFA) for the application review.

A Switch to Doravirine/Islatravir (DOR/ISL) in Participants With Human Immunodeficiency Virus Type 1 (HIV-1) Who Are Virologically Suppressed on Bictegravir/Emtricitabine/Tenofovir Alafenamide (BIC/FTC/TAF) (MK-8591A-052)

NCT05630755Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 2/17/2023

A Switch to Doravirine/Islatravir (DOR/ISL) in Participants With Human Immunodeficiency Virus Type 1 (HIV-1) Who Are Virologically Suppressed on Antiretroviral Therapy (ART) (MK-8591A-051)

NCT05631093Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 2/20/2023

Roche's PiaSky Becomes First Subcutaneous PNH Therapy Approved in EU

Drug ApprovalMonoclonal AntibodyRare Disease
  • Roche's PiaSky (crovalimab) has received European Commission approval as the first subcutaneous therapy for paroxysmal nocturnal haemoglobinuria (PNH) that can be self-administered monthly at home.
  • The approval is based on COMMODORE 2 trial data showing PiaSky was equivalent to AstraZeneca's Soliris in controlling red blood cell destruction while reducing blood transfusion needs.
  • PiaSky now has regulatory clearance in the top four global pharmaceutical markets including China, Japan, the US, and EU, positioning it to compete with established intravenous therapies.
  • The drug faces emerging competition from oral therapies including Novartis' recently approved Fabhalta, which analysts predict could reach $3.6 billion in peak annual sales.

A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors

NCT04434092Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 10/8/2020

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