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● Recent Highlights
Blueprint Medicines
Blueprint received approval from the U.S. Food and Drug Administration (FDA) for AYVAKIT (avapritinib), the first approved precision therapy for patients with gastrointestinal stromal tumors (GIST) and the only highly active treatment for a specific mutation of GIST. This approval brings forward a new standard of care for a genomically defined population of GIST patients that previously had very limited treatment options.
Blueprint Medicines
GBT
GBT received accelerated approval from the U.S. Food and Drug Administration (FDA) for Oxbryta™ (voxelotor) for the treatment of sickle cell disease (SCD). The once-daily pill is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD.
GBT
Thrive Earlier Detection
Cancer is often detected too late, when it has become challenging to treat and may have spread from one area in the body to others. Thrive is developing a high performance, highly targeted, blood-based screening approach, called CancerSEEK, designed to be implemented into routine medical care with the goal of identifying cancer at earlier stages across multiple cancer types.
THRIVE EARLIER DETECTION
bluebird bio
bluebird received conditional marketing authorization from the European Commission for ZYNTEGLO™, the first gene therapy approved for the treatment of transfusion-dependent β-thalassemia. This novel therapy has the potential to transform lives by offering patients the possibility of a transfusion-free future.
BLUEBIRD BIO
Sage Therapeutics
Sage received approval from the U.S. Food and Drug Administration (FDA) for ZULRESSO™ (brexanolone) injection for the treatment of postpartum depression (PPD), the most common medical complication of childbirth. As the first and only treatment specifically indicated for PPD, new mothers and their families impacted by PPD now have a therapeutic option for this critical time in their lives.
SAGE THERAPEUTICS
Agios Pharmaceuticals
Agios has leveraged its core capabilities in cellular metabolism and precision medicine to build a product engine that is focused in the therapeutic areas of cancer and rare genetic diseases. With remarkable speed - about 10 years - Agios brought two therapeutics to the market. And now patients with acute myeloid leukemia have treatment options they didn’t have before.
AGIOS PHARMACEUTICALS