When Douglas Alexander’s prostate cancer showed signs of re-emerging after years in remission, he didn’t have any viable treatment options available. The radiation therapy he’d had when he was first diagnosed with a stage I tumor five years earlier couldn’t be repeated, and surgery also wasn’t an option: Despite rising levels of prostate-specific antigen (PSA),…
CHICAGO—In the eyes of Richard Pazdur, a breakthrough cancer drug should be “transformative.” His view is important because Dr. Pazdur is the head of the U.S. Food and Drug Administration’s office of hematology and oncology products, which helps decide whether experimental cancer drugs are sufficiently safe and effective to go on the market. Under a…
A new regulatory pathway established last year allows drugs with dramatic early clinical promise to be expedited to the market quicker than ever before. To date, most of these ‘breakthrough’ designations have gone to cancer agents, raising the prospect of faster access to the latest lifesaving therapies for the estimated 4,500 people newly diagnosed with…
CHICAGO–The FDA’s oncology chief, Richard Pazdur, used his turn in front of the press mob at ASCO today to make some very public vows about his commitment to the newly launched breakthrough-drug program, that intriguing new classification for certain stellar therapeutic programs that will be extended VIP status at the agency. Pazdur stressed that any…
Whether she’s tackling the complexities of science or style, the medical philanthropist, fashion icon and social mover Deeda Blair approaches all facets of life with quiet but unwavering discipline. Slide Show Most scientists are astonished by Deeda Blair’s style, and the style mavens are surprised by her scientific expertise. That is obvious to even the…
The FDA has added another regulatory coup for Alexion’s ($ALXN) late-stage program for asfotase alfa, an enzyme replacement therapy for extremely rare cases of a metabolic disease known as hypophosphatasia. Already handed an orphan drug designation alongside fast-track status, the agency has added the highly sought after “breakthrough drug” designation for the therapy, which could…
Synageva Biotech Corp., a Lexington biopharmaceutical company developing therapies for rare diseases, said Monday that the Food and Drug Administration has granted breakthrough therapy designation to its drug candidate for the treatment of the early onset form of LAL deficiency. The drug candidate is called sebelipase alfa. Lysosomal acid lipase deficiency (or LAL deficiency) is…
The U.S. Food and Drug Administration today approved Xofigo (radium Ra 223 dichloride) to treat men with symptomatic late-stage (metastatic) castration-resistant prostate cancer that has spread to bones but not to other organs. It is intended for men whose cancer has spread after receiving medical or surgical therapy to lower testosterone. Prostate cancer forms…
While Novartis ($NVS) is beginning to test just how valuable the FDA’s new “breakthrough” status is, AbbVie ($ABBV) has now joined the ranks of pharma companies that can lay claim to boasting rights for the designation. The company says that the FDA has picked its antiviral combo–with and without ribavirin–as a new breakthrough therapy worthy…
A Johnson & Johnson (JNJ) unit said that the U.S. Food and Drug Administration has designated its investigational myeloma treatment daratumumab a “breakthrough therapy,” paving the way for its speedier development. Janssen Research and Development LLC said the FDA granted the designation for daratumumab to treat patients with multiple myeloma who have received at least…
