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…
Cancer medicines that cost more than $100,000 a year aren’t morally justifiable and may keep patients from getting life-saving treatments, a group of more than 100 leukemia doctors said. Of the 12 cancer medications approved by the Food and Drug Administration last year, 11 cost more than $100,000 annually, the physicians said in an article in Blood, the journal…
Earlier this year, the US Food and Drug Administration (FDA) granted its first ‘breakthrough therapy designations’ to a pair of cystic fibrosis drugs (see Nat. Med. 19, 116–117, 2013). But since then, it’s been all about the cancer agents. The New Jersey drug giant Merck announced this morning that its investigational cancer drug lambrolizumab…
Electric fans growl like airplanes taking off and banks of green lights wink in a basement at Mount Sinai’s medical school, where a new $3 million supercomputer makes quick work of huge amounts of genetic and other biological information. Just a couple of miles away, a competitor, Weill Cornell Medical College and NewYork-Presbyterian Hospital/Weill Cornell…
More than 50 Nobel laureates are urging Congress to spare the federal science establishment from the looming budget cuts known as the sequester, saying that research has endured years of budget reductions and that additional cuts could endanger “the innovation engine that is essential to our economy.” The open letter was written by Burton Richter, a 1976 Nobel…
Rep. Renee Ellmers (R-N.C.) has just introduced legislation that would reverse the sequester cuts that have led some cancer clinics to turn away Medicare patients. Medicare has already said it doesn’t have the authority on its own to reverse the automatic reductions, which federal law specifies must hit all programs equally. That essentially leaves it in Congress’s hands—legislators passed the…
A cancer drug being jointly developed by Pharmacyclics and Janssen has been awarded a third breakthrough designation, the companies announced Monday (April 8). FDA in February granted the drug, ibrutinib, two breakthrough designations for the treatment of two types of lymphoma. The third breakthrough designation for ibrutinib is for the treatment of chronic lymphocytic leukemia or small…
