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FDA Approves Roche's PATHWAY HER2 Test as Companion Diagnostic for Biliary Tract Cancer

• The FDA has approved Roche's PATHWAY HER2 (4B5) test as a companion diagnostic for identifying biliary tract cancer (BTC) patients eligible for ZIIHERA (zanidatamab-hrii) treatment. • This marks the first FDA-approved companion diagnostic for HER2-positive BTC, addressing a critical unmet need in selecting patients for targeted therapy. • The PATHWAY HER2 test expands its clinical utility, building upon its existing use in breast cancer to now include identifying HER2-positive BTC patients. • ZIIHERA is the first FDA-approved treatment for adults with previously-treated, unresectable or metastatic HER2-positive biliary tract cancer.

FDA Approves BridgeBio's Attruby (acoramidis) for ATTR-CM

• The FDA has approved Attruby (acoramidis) by BridgeBio Pharma for treating transthyretin amyloid cardiomyopathy (ATTR-CM) in adults, marking a significant advancement in cardiac care. • Attruby, an orally administered TTR stabilizer, is designed to reduce cardiovascular death and cardiovascular-related hospitalization by targeting the destabilization of the TTR tetramer. • Clinical trials demonstrated that Attruby significantly reduced cardiovascular mortality and hospitalization rates, alongside improvements in patient quality of life, showcasing its therapeutic potential. • BridgeBio is also planning to pursue approvals globally, next in Europe, Japan, and Brazil, to extend the availability of Attruby to more patients.

Citius Pharma Gains Clarity from FDA on Mino-Lok's Path to Approval for CRBSI

  • Citius Pharmaceuticals completed a Type C meeting with the FDA to discuss the Phase 3 Mino-Lok program for catheter-related bloodstream infections (CRBSI).
  • The FDA provided clear and actionable guidance, supporting a potential New Drug Application (NDA) submission for Mino-Lok.
  • Mino-Lok, if approved, would be the first FDA-approved treatment to salvage central venous catheters, potentially reducing healthcare costs and improving patient outcomes.
  • Citius Pharma is committed to advancing the Mino-Lok program and will provide updates on regulatory and clinical developments.

NRx Pharmaceuticals Enhances Clinical Trial Reliability in Depression Studies

  • NRx Pharmaceuticals' methodology for training and monitoring study site raters demonstrates higher interrater reliability (IRR) on the MADRS depression scale.
  • The enhanced reliability in psychometric ratings has positive implications for future registration trials of NRX-101 and similar medications targeting depression.
  • In the NRX-101 study, 94.5% concordance was achieved, defined by no more than 3 points of disagreement between site and central raters.
  • The methodology enabled statistical significance with fewer participants, potentially reducing study costs and risks of failure in antidepressant drug trials.

Citius Pharma Advances Mino-Lok Approval Pathway Following FDA Meeting

  • Citius Pharmaceuticals recently met with the FDA to discuss the NDA pathway for Mino-Lok, an antibiotic lock solution for infected central venous catheters.
  • The FDA provided constructive feedback on regulatory considerations and clinical data needed to support the NDA submission for Mino-Lok.
  • Mino-Lok aims to offer an alternative to catheter removal, potentially improving the management of catheter-related bloodstream infections and reducing healthcare costs.
  • Citius Pharma remains dedicated to advancing the Mino-Lok program and will continue to update on regulatory and clinical developments.

GSK Seeks FDA Approval for Blenrep Combination Therapy in Multiple Myeloma

  • GSK's Blenrep, previously withdrawn, aims for re-approval as a combination therapy for multiple myeloma after at least one prior line of treatment.
  • The FDA has accepted GSK's application for Blenrep combined with bortezomib plus dexamethasone, or pomalidomide plus dexamethasone, with a decision expected by July 2025.
  • Phase III trials DREAMM-7 and DREAMM-8 demonstrated statistically significant improvements in progression-free survival compared to standard-of-care regimens.
  • Blenrep's potential approval could introduce competition to BCMA-targeted therapies, offering a simpler administration route than CAR-T therapy.

Evaluation of Efficacy and Safety of Belantamab Mafodotin, Bortezomib and Dexamethasone Versus Daratumumab, Bortezomib and Dexamethasone in Participants With Relapsed/Refractory Multiple Myeloma

NCT04246047Active, Not RecruitingPhase 3
GlaxoSmithKline
Posted 5/7/2020

Belantamab Mafodotin Plus Pomalidomide and Dexamethasone (Pd) Versus Bortezomib Plus Pd in Relapsed/Refractory Multiple Myeloma

NCT04484623Active, Not RecruitingPhase 3
GlaxoSmithKline
Posted 10/1/2020

Citius Pharma Gains FDA Guidance on Mino-Lok Phase 3 Program for CLABSI

  • Citius Pharmaceuticals received constructive feedback from the FDA regarding its Phase 3 Mino-Lok program, aimed at salvaging central venous catheters in patients with CLABSI.
  • The FDA's guidance supports a potential New Drug Application (NDA) submission for Mino-Lok, addressing critical aspects like in-vitro data, clinical efficacy, safety, and regulatory considerations.
  • Mino-Lok, combining minocycline, ethanol, and edetate disodium, offers a novel approach to treating catheter-related bloodstream infections, potentially reducing healthcare costs and improving patient outcomes.
  • Citius Pharma is committed to advancing the Mino-Lok program and will provide updates on regulatory and clinical developments.

Citius Pharma Gains Clarity on Mino-Lok's Regulatory Path After FDA Meeting

  • Citius Pharmaceuticals reports a productive Type C meeting with the FDA regarding its Mino-Lok program for catheter-related bloodstream infections (CRBSI).
  • The FDA provided clear and actionable guidance, supporting a potential New Drug Application (NDA) submission for Mino-Lok.
  • Mino-Lok demonstrated compelling clinical outcomes in a Phase 3 trial, offering a potential alternative to catheter removal and reducing healthcare costs.
  • Citius Pharma is committed to advancing the Mino-Lok program and will provide updates on regulatory and clinical developments.

NRx Pharmaceuticals' Methodology Achieves High Accuracy in Depression Trial Ratings

  • NRx Pharmaceuticals' novel training and monitoring methodology demonstrated a 94.5% interrater reliability in a Phase 2b/3 clinical trial for NRX-101, surpassing previous industry standards.
  • The improved rater reliability enabled the identification of statistically significant improvements in suicidality and akathisia with a smaller patient cohort.
  • The enhanced methodology is expected to reduce the costs and risks of failure in future clinical trials for NRX-101 and similar medications targeting depression.

Long COVID Patients Frustrated by Lack of FDA-Approved Treatments Despite Federal Research Funding

• Long COVID patients express frustration over the lack of FDA-approved treatments despite $1.15 billion in federal research funding. • The NIH's RECOVER initiative is criticized for prioritizing observational studies over clinical trials for potential therapies. • An additional $662 million in funding, including $300 million for clinical trials, aims to address the urgent need for effective treatments. • Researchers and patient advocates emphasize the importance of understanding biological mechanisms and prioritizing patient-centered research.

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