The phase III win in March testing oral CFTR corrector tezacaftor and approved CFTR potentiator Kalydeco (ivacaftor) in cystic fibrosis (CF) patients left investors and analysts predicting success in early stage testing for triple-combo regimens featuring ivacaftor, tezacaftor and one of four possible next-generation CF correctors. But the phase I and phase II data unveiled late Tuesday by Vertex Pharmaceuticals Inc. simply blew away expectations. (See BioWorld Today, March 30, 2017.)
A cardiovascular signal that earned a second complete response letter (CRL) for Dynavax Technologies Corp.'s hepatitis B vaccine, Heplisav, was determined by members of the FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC) largely to be a "spurious" finding that should be investigated more fully in a required postmarketing study.
Having spent the last several years advancing its potentially disease-modifying nitrated fatty acid cell signaling approach targeting inflammatory and fibrotic indications, privately held Complexa Inc. is gearing up for phase II proof-of-concept trials after closing a $62 million series C round, by far its most substantial fundraising to date.
Despite some skepticism heading toward the July 21 PDUFA date, Puma Biotechnology Inc.'s breast cancer drug, Nerlynx (neratinib), won FDA approval a few days early, marking the first treatment designated as an extended adjuvant therapy in patients with early stage, HER2-positive disease.
Emmaus Medical Inc.'s pharmaceutical-grade L-glutamine cleared the FDA on its July 7 PDUFA date, becoming the first new treatment for sickle cell disease (SCD) in the U.S. since chemotherapeutic agent hydroxyurea gained approval in 1998, and the first treatment indicated for pediatric patients.
The widespread Ebola outbreak in West Africa in 2013 to 2016 drew worldwide attention and assistance to the continent and calls for local and global collaboration to target neglected tropical diseases.
SAN DIEGO – Drug pricing: Often drawing groans and sighs of resignation, those two words have managed to creep to the forefront of nearly every panel discussion as the biopharma industry gathered for the annual BIO International Convention amid increasing scrutiny by the U.S. government, expectations of an executive order from the White House – though a draft review by the New York Times indicated the latter tended more toward eliminating regulatory hurdles than actual price curbing – and the anticipated unveiling of a health care bill Thursday from Senate Republicans.
In one of the largest private financings of the year, 3-year-old Rubius Therapeutics Inc. raised $120 million in an oversubscribed round to build out the company and advance its lead Red-Cell Therapeutics candidates into the clinic.
Coherus Biosciences Inc.'s CHS-1701 has become the latest biosimilar candidate for Neulasta (pegfilgrastim) to hit a regulatory snag, earning a complete response letter (CRL) that is expected to delay for at least a year approval of its version of Amgen Inc.'s long-acting G-CSF drug.
For those working in the area of cancer today, it’s impossible to attend a meeting or scientific session without hearing about genetically targeted therapies or efforts in the increasingly hot immuno-oncology space. But a small firm out of Boston has a different view when it comes to cancer therapy.
