Topic: Breakthrough Therapy
Roche has been awarded breakthrough status for a drug that has shown some signs of activity in autism spectrum disorders, according to reports.
As the battle between Swiss major Novartis and biotech rival Kite Pharma heats up to be the first to market a CAR-T cancer med, the Big Pharma has been handed its second FDA "breakthrough" tag for its eagerly awaited JULIET trial.
The FDA has awarded Regeneron breakthrough status for evinacumab, a potentially first-in-class drug designed to help patients who struggle to meet cholesterol targets despite current therapies.
An FDA “Breakthrough” tag, which has been around since 2012, was once a highly coveted badge that in essence allows the regulator to help speed up development of new meds, and it was believed initially that these would be fairly rare occurrences held back for some truly innovative treatments.
Gene therapy player AveXis has gotten a Breakthrough Therapy Designation for its only clinical candidate, AVXS-101 to treat spinal muscular atrophy Type 1.
Shire has been given a breakthrough designation from the FDA for its two experimental gastrointestinal meds SHP621 and SHP625, which should speed up the regulatory review process.
The very people charged with prescribing medicines to patients don’t, as it turns out, know very much at all about how they are approved.
Roche and AbbVie won an accelerated approval to market venetoclax for chronic lymphocytic leukemia patients with a chromosomal abnormality called 17p deletion.