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Clinical Trial News

Vorasidenib Approved in China's Boao Lecheng for IDH-Mutant Glioma Treatment

  • Vorasidenib, a dual inhibitor of IDH1 and IDH2, has been approved for clinical use in the Boao Lecheng International Medical Tourism Pilot Zone in China.
  • This approval allows Chinese patients with IDH-mutant diffuse glioma access to the first-in-class targeted therapy before its official registration in China.
  • A real-world study is underway in Boao Lecheng to assess the safety and efficacy of Vorasidenib in Chinese patients, potentially accelerating research by 1-2 years.
  • Vorasidenib has already been approved in the US, Canada, Australia, Israel, Switzerland and UAE, marking its global recognition as an important treatment option.

EMA Grants Orphan Drug Designation to Ocugen's OCU410ST for ABCA4-Associated Retinopathies

  • The European Medicines Agency (EMA) has granted orphan medicinal product designation to OCU410ST for treating ABCA4-associated retinopathies, including Stargardt disease.
  • OCU410ST previously received orphan drug designation from the U.S. FDA in April 2023, highlighting its potential in addressing unmet needs.
  • Phase 1/2 GARDian trial data showed an 84% reduction in atrophic lesion growth in treated eyes compared to untreated eyes after six months.
  • Ocugen plans to pursue an accelerated marketing authorization application (MAA) for OCU410ST, aiming to expedite its availability to patients.

FDA Reviewing RP1 and Nivolumab Combination for Advanced Melanoma After PD-1 Inhibitor Failure

• The FDA is evaluating a biologics license application for RP1 plus nivolumab to treat advanced melanoma in adults who have previously received a PD-1 inhibitor. • The application is supported by data from the phase 1/2 IGNYTE trial, which demonstrated an overall response rate of 32.7% in patients with anti-PD-1-failed melanoma. • The RP1 and nivolumab combination has been granted breakthrough therapy designation by the FDA for this indication, expediting its potential approval. • A phase 3 trial, IGNYTE-3, is currently enrolling patients to further evaluate the combination's efficacy in patients who have progressed on anti-PD-1 and anti-CTLA-4 therapy.

VO and Nivolumab vs Physician's Choice in Advanced Melanoma That Progressed on Anti-PD-1 & Anti-CTLA-4 Drugs (IGNYTE-3)

NCT06264180RecruitingPhase 3
Replimune Inc.
Posted 7/11/2024

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab

NCT03767348RecruitingPhase 2
Replimune Inc.
Posted 9/20/2017

FDA Approves BridgeBio's Attruby for Rare Heart Condition ATTR-CM

• The FDA has approved BridgeBio's Attruby, offering a new treatment option for ATTR-CM, a rare heart condition, challenging Pfizer's previous dominance in this therapeutic area. • Research indicates that healthy breast cells may exhibit genetic changes similar to invasive breast cancer, raising concerns about diagnostic accuracy in breast cancer screening. • Canadian health officials have confirmed the country's first case of clade I mpox amid an ongoing outbreak, highlighting the continued global spread of the virus.

Vorasidenib Approved in China's Boao Lecheng Pilot Zone for IDH-Mutant Glioma

  • Vorasidenib, a dual inhibitor of IDH1 and IDH2, has been approved for clinical use in the Boao Lecheng International Medical Tourism Pilot Zone in China.
  • This approval allows Chinese patients with IDH-mutant diffuse glioma access to the drug before its official registration in China, aligning with global availability.
  • A real-world study is underway in Boao Lecheng to evaluate vorasidenib's safety and efficacy in Chinese patients, potentially accelerating research by 1-2 years.
  • Vorasidenib has already been approved in the US, Canada, Australia, Israel, Switzerland and UAE, marking a significant advancement in glioma treatment.

Enveda Biosciences Secures $130 Million to Advance AI-Powered Drug Discovery

• Enveda Biosciences has raised $130 million in a Series C funding round to advance its AI-driven drug discovery platform and clinical pipeline. • The funding will support clinical trials for multiple drug candidates, including ENV-294, an oral anti-inflammatory agent for atopic dermatitis. • Enveda's platform combines metabolomics data, machine learning, and high-throughput experiments to identify and develop novel therapeutics from nature. • The company's AI platform has characterized over 1 million natural compounds, creating a large dataset for drug discovery workflows.

Vorasidenib Approved for Clinical Use in China's Boao Lecheng Pilot Zone

  • Vorasidenib, a dual inhibitor of IDH1 and IDH2, has been approved for clinical use in the Boao Lecheng International Medical Tourism Pilot Zone in China.
  • This approval marks the first use of Vorasidenib in Asia, preceding its availability in Europe, for treating Chinese patients with IDH-mutant diffuse glioma.
  • A real-world study is underway in Boao Lecheng to evaluate Vorasidenib's safety and efficacy in Chinese patients, potentially accelerating research by 1-2 years.
  • Vorasidenib has already been approved in the US, Canada, Australia, Israel, Switzerland and UAE, and is in preparation for official registration in China.

MEMBRANE Trial Demonstrates Benefits of MMA Embolization in Chronic Subdural Hematoma Treatment

  • The MEMBRANE trial demonstrated that middle meningeal artery (MMA) embolization with Trufill n-BCA, as an adjunct to standard care, significantly improves outcomes in chronic subdural hematoma (cSDH) patients.
  • The study met its primary effectiveness endpoint, showing a significantly lower failure rate in the embolization group compared to standard care alone, with an odds ratio of 0.529.
  • MMA embolization was statistically non-inferior to standard of care in achieving good functional outcomes at three months and almost achieved superiority based on mRS scores.
  • Subgroup analysis revealed the most significant treatment effect in surgical patients over 75, suggesting a substantial benefit in this vulnerable population.

Larotrectinib Shows High Efficacy in Pediatric Solid Tumors with NTRK Fusion

  • Larotrectinib demonstrates high effectiveness in pediatric patients with newly diagnosed infantile fibrosarcoma (IFS) and other solid tumors harboring NTRK gene fusions.
  • The study marks the first Children's Oncology Group (COG) trial to assess precision medicine in a front-line setting based on genetic biomarkers.
  • Results indicate larotrectinib could replace chemotherapy as a first-line treatment for NTRK fusion-positive tumors, reducing potential harm to developing organs.
  • The objective response rate within six cycles was 94% for IFS and 60% for other solid tumors, highlighting the drug's potential in pediatric cancer care.

A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children

NCT02637687Active, Not RecruitingPhase 1
Bayer
Posted 12/16/2015

A Study to Test the Effect of the Drug Larotrectinib in Adults and Children With NTRK-fusion Positive Solid Tumors

NCT02576431Active, Not RecruitingPhase 2
Bayer
Posted 9/30/2015

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