As more cancer drugs are approved and prices continue to rise, questions have circulated on whether these drugs are extending patient survival or improving quality of life (QoL). A research article published in the BMJ last week reviewed the approval of drugs by the European Medicines Agency (EMA) from 2009 to 2013 and noted that most drugs entered the market without evidence of survival benefits or QoL. "When there were survival gains over existing treatment o...

California Gov. Jerry Brown this week signed into law a bill that requires health providers to post notices in their offices when they are administering stem cell treatments that have not been approved by the US Food and Drug Administration (FDA). The law is part of efforts to crack down on the use and sale of unapproved stem cell treatments, some of which are being hawked as cures for a range of diseases with few or no treatment options, like ALS, autism and spina...

The US Food and Drug Administration (FDA) on Thursday finalized its 2015 draft guidance on the types of studies necessary to establish the performance characteristics of diagnostics used to detect human papillomavirus (HPV) when screening for cervical cancer. Background HPV is the leading cause of cervical cancer, with fourteen genotypes of the virus considered to be carcinogenic or high-risk. According to the Centers for Disease Control, cervical cancer was the leading...

The US Food and Drug Administration (FDA) on Friday approved Mylotarg (gemtuzumab ozogamicin) for the treatment of adults with newly diagnosed acute myeloid leukemia whose tumors express the CD33 antigen (CD33-positive AML) and for patients aged two years and older with CD33-positive AML who have experienced a relapse or who have not responded to initial treatment.  In 2010, Pfizer voluntarily withdrew the treatment, 10 years after it won accelerated approval, wh...

The US Food and Drug Administration's (FDA) Commissioner Scott Gottlieb said Monday the agency will advance a new framework to better regulate stem cell therapies this fall. "This comprehensive policy will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency's current authority, and then define an efficient process for how these products should be evaluated for safety and effectiveness," Gottli...

The US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee on Wednesday voted unanimously, 10 to 0, in favor of the benefit-risk profile for the first of a new kind of cancer therapy, known as a Chimeric Antigen Receptor T-cell (CAR-T) therapy. Although the outside panel of experts raised questions about concerns with the safety and manufacturing of the Novartis treatment, known as CTL019 (tisagenlecleucel-T), the panel did not question the effi...

The US Food and Drug Administration (FDA) on Friday granted approval to Emmaus Medical’s Endari (L-glutamine oral powder), the first treatment approved for patients with sickle cell disease in almost 20 years. Endari is approved for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. Endari is the 24th new drug approved by FDA in 2017, already two more than all of 2016 . Richard Pazdur...

With a new Texas law now in the books to allow companies to sell unproven stem cell treatments without US Food and Drug Administration (FDA) approval, some experts wonder when FDA will step in to shut down companies and clinics exposing people to unapproved medical products. Similar to the “Right to Try” laws spreading across the US and attempting to undercut FDA’s regulation of investigational products, the Texas law , which had been brewing in some form since...

The US Food and Drug Administration (FDA) and National Cancer Institute (NCI) on Monday unveiled a memorandum of understanding (MOU) to further cooperate on understanding the molecular biology of cancer as it relates to diagnostic measurements, product development and treatment. "The purpose of this MOU is to engage in scientific and programmatic collaborations in clinical proteogenomics cancer research, such as statistical experimental design, instrument/technolo...

The US Food and Drug Administration (FDA) on Monday expanded the approved use of Roche’s Actemra (tocilizumab) to treat adults with giant cell arteritis, making it the first FDA-approved therapy for this condition. The efficacy and safety of subcutaneous (injected under the skin) Actemra for giant cell arteritis were established in a double-blind, placebo-controlled study with 251 patients with giant cell arteritis. A greater proportion of patients receiving subcuta...

Peter Marks, director of the US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research (CBER) on Thursday said his office is equipped to support the development of cutting edge treatments, such as cell and gene therapies. Speaking at the Food and Drug Law Institute's annual conference, Marks said the 21st Century Cures Act added tools to FDA's regulatory arsenal, including the regenerative medicine advanced therapy (RMAT) designation to provi...

The US Food and Drug Administration (FDA) on Tuesday released warning letters sent to 14 US-based companies for selling dozens of unapproved cancer treatments online and through social media. The companies, FDA says, have 15 days to respond with plans to address the violations or face possible criminal prosecution, including up to a year in federal prison and fine of $100,000 or twice the companies' gains on the fraudulent products. The agency is also advising patients ...