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Clinical Trial News

FDA Approves Henlius-Organon Denosumab Biosimilars BILDYOS and BILPREVDA for Bone Health Conditions

Drug ApprovalMonoclonal AntibodyOncology
  • The FDA has approved BILDYOS (denosumab-nxxp) and BILPREVDA (denosumab-nxxp), biosimilars to PROLIA and XGEVA respectively, developed by Shanghai Henlius Biotech and commercialized by Organon.
  • BILDYOS is indicated for multiple osteoporosis conditions including postmenopausal women at high fracture risk, while BILPREVDA targets cancer-related bone complications and giant cell tumor of bone.
  • The approvals are based on comprehensive data demonstrating the biosimilars are highly similar to reference products with no clinically meaningful differences in safety, purity, and potency.
  • This milestone expands Organon's biosimilars portfolio and represents Henlius's continued success in bringing self-developed biosimilars to the US market.

FDA Issues Complete Response Letter for Telix's Kidney Cancer Imaging Agent TLX250-CDx

  • Telix Pharmaceuticals received a Complete Response Letter from the FDA regarding TLX250-CDx, a novel PET imaging agent for diagnosing clear cell renal cell carcinoma, citing manufacturing and quality control deficiencies.
  • The FDA requested additional data on scaled-up manufacturing processes and flagged deficiencies at two third-party manufacturing partners that require remediation before resubmission.
  • Despite the setback, TLX250-CDx maintains its Breakthrough Therapy and Priority Review status, and Telix believes the concerns are readily addressable within a reasonable timeframe.
  • The company's stock dropped 20.7% following the announcement, though the CRL does not impact Telix's 2025 revenue guidance as the product was not included in projections.

BioNTech's Cancer Drug Trastuzumab Pamirtecan Meets Primary Endpoint in Phase 3 Breast Cancer Trial

Oncology
  • BioNTech and Duality Biologics announced their antibody-drug conjugate trastuzumab pamirtecan met its primary endpoint of progression-free survival in a phase 3 trial for unresectable or metastatic breast cancer.
  • The head-to-head study compared the experimental ADC treatment against trastuzumab emtansine in patients who had previously received chemotherapy.
  • This marks BioNTech's first late-stage oncology drug to achieve its primary endpoint in a phase 3 study, with plans to file for regulatory approval in China, the EU, and the United States.
  • The positive results drove BioNTech's stock up 10% as investors recognized the potential in a large addressable market with over 350,000 new breast cancer cases annually in China alone.

FDA Grants Breakthrough Therapy Designation for GSK's Osteosarcoma Treatment

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for GSK's GSK'227, a B7-H3-targeted antibody-drug conjugate, for treating relapsed or refractory osteosarcoma in adults. This decision is based on promising data from the ARTEMIS-002 study, highlighting the drug's potential in addressing a significant unmet medical need.

Vir Biotechnology's Tobevibart and Elebsiran Receive FDA and EMA Designations for Chronic Hepatitis Delta Treatment

Vir Biotechnology has announced that its drugs, tobevibart and elebsiran, have been granted Breakthrough Therapy designation by the FDA and Priority Medicines designation by the EMA for treating chronic hepatitis delta (CHD). This decision is backed by positive safety and efficacy data from the Phase 2 SOLSTICE trial. The Phase 3 ECLIPSE registrational program is set to begin in the first half of 2025, aiming to provide a much-needed treatment option for CHD, a severe form of chronic viral hepatitis with no approved treatment in the U.S. and limited options globally.

HyBryte™: A Promising Photodynamic Therapy for Cutaneous T-Cell Lymphoma (CTCL)

HyBryte™, a photodynamic therapy using synthetic hypericin and visible fluorescent light, shows promise as a safe and effective treatment for Cutaneous T-Cell Lymphoma (CTCL), particularly in early-stage disease. With no FDA-approved first-line therapy for CTCL, HyBryte™ aims to reduce disease progression and toxic side effects, offering a new hope for approximately 20,000 patients in the US affected by this chronic condition.

Gene Therapy Breakthrough Effectively Cures 'Bubble Boy' Disease in Infants

  • A groundbreaking gene therapy developed by US scientists has effectively cured infants with X-linked severe combined immunodeficiency (X-SCID), a rare and fatal genetic disorder that leaves children without functioning immune systems.
  • The treatment involves collecting patients' bone marrow, inserting a corrected copy of the mutated gene into their blood stem cells using a specially engineered virus, and returning the cells to the body after low-dose chemotherapy.
  • Within three months of treatment, seven of eight infants developed normal levels of multiple immune cell types, allowing them to fight off infections and lead normal lives without the need for ongoing treatment or protective isolation.

QGel's Synthetic Matrix Breakthrough Aims to Revolutionize Organoid-Based Precision Medicine

  • QGel has developed a synthetic extracellular matrix to replace animal-based gels in organoid development, potentially enabling large-scale precision medicine applications in cancer treatment.
  • Clinical studies show organoid-based approaches can match patients with effective cancer treatments with up to 90% accuracy, significantly outperforming current genomic methods that benefit only 7% of patients.
  • The company is working to integrate standardized organoid testing into clinical workflows, with initial plans to treat hundreds of patients annually while addressing regulatory, reimbursement, and implementation challenges.

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