It's not just for cancer anymore. Imbruvica (ibrutinib), the blockbuster BTK inhibitor for hematological cancers, won FDA approval today as the first therapy for patients with chronic graft-vs.-host disease (cGVHD) who have failed first-line corticosteroid treatment.
Spark Therapeutics Inc. is having a very good summer. Its BLA for Luxturna (voretigene neparvovec) in RPE65-mediated inherited retinal disease was accepted for priority review, as well as receiving a rare pediatric disease designation, while its hemophilia B gene therapy wowed with interim data at the International Society on Thrombosis and Haemostasis (ISTH) meeting in Berlin.
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.
