Clinical Trial News
Palbociclib Plus Letrozole Approved for First-Line ER+, HER2- Metastatic Breast Cancer
- Palbociclib, a CDK4/6 inhibitor, received FDA accelerated approval in combination with letrozole for postmenopausal women with ER+, HER2- metastatic breast cancer.
- The approval was based on the PALOMA-1 trial, which demonstrated improved progression-free survival compared to letrozole alone in previously untreated patients.
- Common adverse events associated with the combination therapy include neutropenia, leukopenia, and fatigue, requiring careful monitoring and dose adjustments.
- Clinicians should consider toxicities, quality-of-life issues, and cost when making treatment decisions, as alternative regimens with different toxicity profiles exist.
Highlighted Clinical Trials:
Washington University School of Medicine
Posted 6/17/2014
German Breast Group
Posted 10/30/2013
Pfizer
Posted 9/15/2008
University of California, San Francisco
Posted 9/1/2010
FDA Approves Trofinetide for Rett Syndrome Treatment
The US Food and Drug Administration (FDA) has approved trofinetide, a synthetic analog of glycine-proline-glutamate (GPE), for the treatment of Rett syndrome (RTT) in adults and pediatric patients aged 2 years and older. This approval marks a significant milestone in the treatment of RTT, a rare neurodevelopmental disorder, following years of scientific and clinical research.
India Modernizes Clinical Trial Process with Digital System and Mandatory AV Consent Recording
- India's Central Drugs Standard Control Organisation (CDSCO) launches new IT-enabled system to streamline clinical trial applications, improving transparency and efficiency in trial registration process.
- Mandatory audiovisual recording of informed consent has been implemented across 90% of Indian trial sites, addressing previous concerns about participant protection and ethical standards.
- The initiatives aim to bridge the gap between Indian and international clinical trial standards, leveraging India's vast treatment-naïve population of over a billion people across 500+ sites.
Smoking Poses Heightened Health Risks for HIV Patients, Studies Show
- Recent studies reveal that people living with HIV who smoke lose more years of life from smoking than from HIV itself, with 50-70% of HIV-positive individuals being smokers.
- Smoking significantly increases HIV patients' risk of opportunistic infections, cardiovascular disease, and medication complications, with up to 60% higher mortality rates observed in smokers.
- Public health initiatives like Stoptober and e-cigarette adoption are being promoted as harm reduction strategies, with evidence showing e-cigarettes carry only 5% of tobacco's health risks.
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.
Highlighted Clinical Trials:
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
- 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.
Highlighted Clinical Trials:
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.