United States

FDA halts two multiple myeloma Keytruda trials

Tuesday, September 5, 2017

Based on data from two recently halted clinical trials, the FDA is issuing this statement to inform the public, healthcare professionals and oncology clinical investigators about the risks associated with the use of Keytruda (pembrolizumab) in combination with dexamethasone and an immunomodulatory agent (lenalidomide or pomalidomide) for the treatment of patients with multiple myeloma. Keytruda (pembrolizumab) is not approved for treatment of multiple myeloma.

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Abbott addresses pacemaker hacking fears

Tuesday, September 5, 2017

On August 23, 2017, the FDA approved a firmware update that is now available and is intended as a recall, specifically a corrective action, to reduce the risk of patient harm due to potential exploitation of cybersecurity vulnerabilities for certain Abbott (formerly St. Jude Medical) pacemakers. “Firmware” is a specific type of software embedded in the hardware of a medical device (e.g. a component in the pacemaker).

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ProQR’s QRX-421 for Usher syndrome receives FDA/EMA Orphan Designation

Tuesday, September 5, 2017

ProQR Therapeutics announced that investigational drug QRX-421 for Usher syndrome has received orphan drug designation (ODD) from the FDA and EMA. This marks the third drug candidate in the company’s ophthalmology pipeline and the fourth drug in the broader pipeline to receive ODD from the FDA and EMA. QR-421 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Usher syndrome is the leading cause of combined deafness and blindness. 

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NIH’s All of Us Research Program expands medical centers network

Wednesday, August 30, 2017

Three sets of healthcare provider organizations will add to a growing network of trusted leaders charged with implementing the NIH’s All of Us Research Program, an ambitious effort to advance research into precision medicine. Combined, the new awardees will receive $13.8 million to enroll interested individuals, gather participant health information and help retain participants in the program through ongoing engagement efforts. These awardees will extend the geographic coverage of the program and strengthen its reach within underserved communities, including lower-income, Hispanic and Latino, African American, American Indian and rural communities.

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Abeona receives FDA breakthrough designation for epidermolysis bullosa

Wednesday, August 30, 2017

Abeona Therapeutics, a clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced that the FDA has granted Breakthrough Therapy designation status to the company’s EB-101 gene therapy program for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). The designation from the FDA enables collaborative discussions with senior FDA personnel, priority review and an expedited approval process to drug candidates where preliminary clinical trials indicate that a therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases.

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