SAN FRANCISCO – If 2017 marked a year of big wins for scientific innovation with the FDA approvals of the first three gene therapies – Novartis AG's Kymriah (tisagenlecleucel) CAR T immunotherapy for B-cell acute lymphoblastic leukemia, Kite Pharma Inc.-developed CAR T Yescarta (axicabtagene ciloleucel) for certain types of non-Hodgkin lymphoma and more "traditional" gene therapy Luxturna (voretigene neparvovec-rzyl) from Spark Therapeutics Inc. to treat RPE65 mutation-associated retinal dystrophy – then 2018 might be the year when the innovative capabilities on the payer side are put to the test.
ATLANTA – With last month's early FDA nod for bispecific antibody Hemlibra (emicizumab) – more than three months ahead of its PDUFA date – and a rapidly advancing gene therapy candidate, the treatment landscape for hemophilia A is poised for a dramatic change.
Shares of Acorda Therapeutics Inc. (NASDAQ:ACOR) took a hit Wednesday, falling $11.20 to close at $17, on reports of potentially treatment-related adverse events in its tozadenant program for Parkinson's disease. While reports of agranulocytosis that led to cases of sepsis and death prompted analysts to drop the drug from their models, the Ardsley, N.Y.-based firm has no plans at this time to discontinue work on the phase III oral adenosine A2a receptor antagonist it acquired in last year's buyout of Biotie Therapies Oyj.
Sage Therapeutics Inc. scored a big win Thursday, disclosing positive top-line data from its pivotal program testing brexanolone in postpartum depression (PPD), results that should support regulatory filings next year and position the intravenously administered drug (previously known as SAGE-547) as potentially the first treatment specifically for PPD.
Gilead Sciences Inc. beat earnings estimates for the third quarter, but analysts seeking to tease out stabilizing trends in the company's declining hepatitis C virus (HCV) franchise were largely disappointed, while overarching concerns about the paucity of promising late-stage programs also helped drive the stock down slightly Friday. Shares of Gilead (NASDAQ:GILD) closed at $77.07, down 81 cents.
CLEVELAND – Coming into the Cleveland Clinic's 15th annual Medical Innovation Summit, gene therapy had been making headlines, most recently the unanimous recommendation by the FDA's Cellular, Tissue and Gene Therapies Advisory Committee in favor of Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec) in RPE65-mediated inherited retinal disease. But back in the early 2000s, when Casey Cunningham, of Sante Ventures, was working as a clinical investigator, the gene-based technologies weren't exactly hot property.
CLEVELAND – A technology so promising it took the FDA barely 100 days to clear it for marketing approval topped the Cleveland Clinic's list of 10 innovations expected to have significant impacts on health care in 2018. Unveiled on the last day of the 15th annual Medical Innovation Summit, the list leads with the closed-loop insulin delivery system for type 1 diabetes and includes advances such as gene therapy and telehealth that have been years in the making.
CLEVELAND – A technology so promising it took the FDA barely 100 days to clear it for marketing approval topped the Cleveland Clinic's list of 10 innovations expected to have significant impacts on health care in 2018. Unveiled on the last day of the 15th annual Medical Innovation Summit, the list leads with the closed-loop insulin delivery system for type 1 diabetes and includes advances such as gene therapy and telehealth that have been years in the making.
