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Clinical Trial News

FDA Draft Guidance Clarifies Requirements for Confirmatory Trials Under Accelerated Approval

  • The FDA has issued draft guidance on accelerated approval, focusing on when confirmatory trials must be 'underway'.
  • This guidance interprets 'underway' and details factors the FDA will consider before granting accelerated approval.
  • The guidance aims to ensure timely completion of confirmatory trials, as mandated by the Consolidated Appropriations Act of 2023.
  • Stakeholders can submit comments on the draft guidance until March 10, 2025, for consideration by the FDA.

Fulvestrant Shows Overall Survival Benefit Over Anastrozole in First-Line Advanced Breast Cancer

• A phase II trial (FIRST) comparing fulvestrant 500 mg to anastrozole 1 mg in postmenopausal women with ER-positive advanced breast cancer showed a statistically significant improvement in overall survival (OS). • Patients treated with fulvestrant 500 mg had a median OS of 54.1 months compared to 48.4 months with anastrozole, representing a 30% reduction in mortality risk (HR 0.70, P=0.04). • The OS benefit with fulvestrant was generally consistent across pre-specified subgroups, and no new safety concerns were identified during the follow-up period. • These findings suggest fulvestrant 500 mg could be considered as a first-line treatment option for ER-positive advanced breast cancer, especially where access to other therapies is limited.

A Study of Palbociclib (PD-0332991) + Letrozole vs. Letrozole For 1st Line Treatment Of Postmenopausal Women With ER+/HER2- Advanced Breast Cancer (PALOMA-2)

NCT01740427CompletedPhase 3
Pfizer
Posted 2/22/2013

A Global Study to Compare the Effects of Fulvestrant and Arimidex in a Subset of Patients With Breast Cancer.

NCT01602380Active, Not RecruitingPhase 3
AstraZeneca
Posted 10/17/2012

NICE Provisionally Approves BioMarin's Vimizim for Ultra-Rare Morquio Disease with Real-World Data Requirements

• NICE has issued a provisional approval for BioMarin's Vimizim (elosulfase alfa), priced at £395,000 per patient annually, for treating the ultra-rare Morquio disease affecting 88 patients in England.
• The approval is contingent on implementing real-world data collection and establishing a price cap, marking NICE's innovative approach to managing high-cost rare disease treatments.
• The MPS Society welcomes the decision while expressing concerns about further delays in the approval process, as patients urgently await access to this life-changing therapy.

FDA Approves Two New Ustekinumab Biosimilars, Bringing Total US Biosimilar Count to 65

Monoclonal AntibodyDrug Approval
  • The FDA has approved Biocon's Yesintek (ustekinumab-kfce) and Celltrion's Steqeyma (ustekinumab-stba) in November and December 2024, expanding treatment options for autoimmune conditions including Crohn's disease and psoriasis.
  • Of the 65 FDA-approved biosimilars, 14 have received interchangeable designation, allowing pharmacists to substitute them for reference products without prescriber intervention, enhancing patient access to more affordable treatments.
  • The biosimilar landscape continues to evolve with products spanning five major classes: insulin, granulocyte colony-stimulating factors, monoclonal antibodies, TNF-alpha inhibitors, and VEGF inhibitors.

Ropeginterferon Alfa-2b Shows Promise in Polycythemia Vera Treatment

  • Ropeginterferon alfa-2b demonstrates a high overall response rate of 90% in polycythemia vera (PV) patients, including complete and partial hematologic responses.
  • The study found no dose-limiting toxicities with ropeginterferon alfa-2b, supporting its favorable safety profile and potential for long-term use.
  • Treatment with ropeginterferon alfa-2b led to significant reductions in JAK2 V617F allelic burden, indicating molecular responses in PV patients.
  • The research suggests that ropeginterferon alfa-2b could effectively normalize blood cell counts and prevent thromboembolic complications in PV patients.

Safety Study of Pegylated Interferon Alpha 2b to Treat Polycythemia Vera

NCT01193699CompletedPhase 1
AOP Orphan Pharmaceuticals AG
Posted 8/1/2010

AstraZeneca Expands Immuno-Oncology Portfolio with Three Strategic Partnerships

• AstraZeneca secures rights to Inovio's INO-3112, a DNA vaccine targeting HPV-related cancers, in a deal worth up to $727.5 million, planning combination studies with durvalumab.
• MedImmune partners with Heptares on adenosine A2A receptor antagonist HTL-2071 for $510 million, aiming to enhance T-cell anti-cancer activity.
• A collaboration with Mirati Therapeutics will explore combining durvalumab with HDAC inhibitor mocetinostat in non-small cell lung cancer trials starting next year.

Europe Emerges as Leading Hub for Precision Medicine Innovation and Implementation

  • Europe's healthcare systems are rapidly adopting genomic medicine and personalized therapies, driven by collaboration among providers, payers, and pharmaceutical companies across the continent.
  • The convergence of advanced diagnostics, genetic analytics, and companion diagnostics has created a mature ecosystem for precision medicine delivery in everyday clinical practice.
  • European institutions' willingness to share platforms and data while maintaining security standards positions the continent to potentially surpass the US in precision medicine commercialization.

India Revamps Clinical Trial Regulations to Align with Global Standards

  • India has significantly reformed its clinical trial regulations, extending SAE reporting timelines from 10 to 14 days to match international standards and streamlining the approval process.
  • The regulatory framework now requires trials to be evaluated based on risk-benefit assessment, innovation value, and unmet medical needs in the country, ensuring greater transparency.
  • New draft standards for accreditation of sites, investigators, and ethics committees have been introduced by CDSCO, aiming to enhance operational standards across clinical research.

WHO Welcomes European Regulators' Historic Green Light for First Malaria Vaccine

  • The European Medicines Agency adopted a positive scientific opinion for Mosquirix, marking the first time a malaria vaccine has reached regulatory review by any authority.
  • WHO will conduct its own assessment in October 2015, evaluating public health aspects including affordability, cost-effectiveness, and field implementation in developing countries.
  • The vaccine addresses a critical unmet need, as malaria caused nearly 200 million cases and close to 600,000 deaths in 2013, with African children particularly affected.
  • WHO expects to issue its policy recommendation in November 2015 on whether to add the vaccine to existing malaria control tools.

Palbociclib Plus Letrozole Significantly Extends Progression-Free Survival in ER+ Breast Cancer

• Palbociclib, a CDK4/6 inhibitor, combined with letrozole, an aromatase inhibitor, significantly improved progression-free survival (PFS) in postmenopausal women with ER+/HER2- metastatic breast cancer. • The PALOMA-1/TRIO-18 phase II trial demonstrated a median PFS of 20.2 months with the palbociclib-letrozole combination, compared to 10.2 months with letrozole alone. • Palbociclib received accelerated FDA approval based on these results, pending confirmatory phase III trial outcomes, marking a significant advancement in targeted therapy for hormone receptor-positive breast cancer. • Common adverse events associated with the combination therapy included neutropenia and leukopenia, manageable through dose modification and blood count monitoring.

Palbociclib (PD-0332991) Combined With Fulvestrant In Hormone Receptor+ HER2-Negative Metastatic Breast Cancer After Endocrine Failure (PALOMA-3)

NCT01942135CompletedPhase 3
Pfizer
Posted 9/26/2013

PIPA: Combination of PI3 Kinase Inhibitors and PAlbociclib

NCT02389842Unknown StatusPhase 1
Royal Marsden NHS Foundation Trust
Posted 3/25/2015

A Study of Abemaciclib (LY2835219) in Participants With Breast Cancer, Non-small Cell Lung Cancer, or Melanoma That Has Spread to the Brain

NCT02308020CompletedPhase 2
Eli Lilly and Company
Posted 4/20/2015

PD 0332991 and Anastrozole for Stage 2 or 3 Estrogen Receptor Positive and HER2 Negative Breast Cancer

NCT01723774Active, Not RecruitingPhase 2
Washington University School of Medicine
Posted 4/10/2013

Palbociclib in Combination With Adjuvant Endocrine Therapy for Hormone Receptor Positive, HER2 Negative Invasive Breast Cancer

NCT02040857CompletedPhase 2
Dana-Farber Cancer Institute
Posted 1/1/2014

A Study of Palbociclib in Addition to Standard Endocrine Treatment in Hormone Receptor Positive Her2 Normal Patients With Residual Disease After Neoadjuvant Chemotherapy and Surgery

NCT01864746CompletedPhase 3
German Breast Group
Posted 10/30/2013

A Phase II Randomized Study Evaluating the Biological and Clinical Effects of the Combination of Palbociclib With Letrozole as Neoadjuvant Therapy in Post-Menopausal Women With Estrogen-Receptor Positive Primary Breast Cancer

NCT02296801CompletedPhase 2
NSABP Foundation Inc
Posted 1/1/2015

A Study Of Palbociclib (PD-0332991) + Letrozole VS. Placebo+ Letrozole For 1st Line Treatment Of Asian Postmenopausal Women With ER+/HER2- Advanced Breast Cancer [PALOMA-4]

NCT02297438Active, Not RecruitingPhase 3
Pfizer
Posted 3/23/2015

A Study Of Oral Palbociclib (PD-0332991), A CDK4/6 Inhibitor, As Single Agent In Japanese Patients With Advanced Solid Tumors Or In Combination With Letrozole For The First-Line Treatment Of Postmenopausal Japanese Patients With ER (+) HER2 (-) Advanced Breast Cancer

NCT01684215CompletedPhase 2
Pfizer
Posted 10/19/2012

Phase 1b Study of PD-0332991 in Combination With T-DM1(Trastuzumab-DM1)

NCT01976169CompletedPhase 1
University of Texas Southwestern Medical Center
Posted 1/24/2014

A Study of Palbociclib in Combination With Fulvestrant or Tamoxifen as Treatment for Metastatic Breast Cancer

NCT02384239CompletedPhase 2
University of California, San Francisco
Posted 10/19/2015

Phase III Palbociclib With Endocrine Therapy vs. Capecitabine in HR+/HER2- MBC With Resistance to Aromatase Inhibitors

NCT02028507CompletedPhase 3
Spanish Breast Cancer Research Group
Posted 3/13/2014

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