Europe aims to create new regulations regarding hematological malignancies.
The EMEA is proposing to create new guidance on hematological malignancies. The agency's idea arises from the recent increased activity in developing new treatments for hematological malignancies, including rare disorders and small target populations. This has led to a rise in applications for marketing authorization, but above all to numerous requests for scientific advice from the agency, prior to submitting an application.
The new guidance will deal with special considerations such as allogeneic stem cell transplantation and its consequences in relation to designation of primary endpoints. It will also take account of the use of molecular techniques in response evaluations, treatments administered with curative or palliative intent, and progression or relapse off-therapy. It will focus on the design of confirmatory studies, and will provide separate discussion of major diagnoses such as the acute leukemias, myelodysplastic syndromes, low and high grade lymphomas, and malignant myeloma. Other, rarer conditions will be covered conceptually. Most of the work will be carried out during this year—and anyone wanting to give their views to the agency is invited to do so until the end of this month.—Peter O'Donnell
Merck Launches Phase III Trials of Once-Monthly Oral HIV PrEP Candidate MK-8527
July 14th 2025In the new EXPrESSIVE clinical trial program, Merck will evaluate MK-8527, a once-monthly, oral PrEP therapy, in populations at high risk of HIV-1 infection, including adolescent girls and women in sub-Saharan Africa.
Unifying Industry to Better Understand GCP Guidance
May 7th 2025In this episode of the Applied Clinical Trials Podcast, David Nickerson, head of clinical quality management at EMD Serono; and Arlene Lee, director of product management, data quality & risk management solutions at Medidata, discuss the newest ICH E6(R3) GCP guidelines as well as how TransCelerate and ACRO have partnered to help stakeholders better acclimate to these guidelines.
Baxdrostat Shows Significant Blood Pressure Reduction in Phase III BaxHTN Trial
July 14th 2025In the BaxHTN Phase III study, AstraZeneca’s baxdrostat demonstrated statistically significant and clinically meaningful reductions in systolic blood pressure in patients with uncontrolled or treatment-resistant hypertension.
New FDA Initiative Reveals Common Reasons for Drug Application Rejection
July 11th 2025By publishing more than 200 complete response letters, the FDA is offering new visibility into the issues that most often delay drug approvals, including safety concerns, trial design flaws, and manufacturing gaps.
Arcus’ Quemliclustat Earns Orphan Drug Designation as Phase III Pancreatic Cancer Trial Advances
July 11th 2025The FDA has granted orphan status to Arcus Biosciences’ CD73 inhibitor quemliclustat for metastatic pancreatic cancer, as the global PRISM-1 Phase III trial nears full enrollment following promising survival data from ARC-8.