Truseltiq is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR.
All articles by Brian Park, PharmD
Apitegromab is a selective inhibitor of the inactive form of myostatin to block its activation in the muscle.
Pamrevlumab is a first-in-class fully human antibody that works by inhibiting the activity of connective tissue growth factor.
Maralixibat is an oral apical sodium dependent bile acid transporter (ASBT) inhibitor.
Ziritaxestat is an investigational autotaxin inhibitor.
The FDA has approved Dojolvi™ (triheptanoin; Ultragenyx) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders.
The FDA has approved Qinlock™ (ripretinib; Deciphera) for the treatment of adults with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib.
Pemigatinib, a small molecule kinase inhibitor, selectively targets FGFR isoforms 1, 2 and 3, which, in preclinical studies, has resulted in anti-tumor activity in cells with FGFR alterations.
The FDA has approved Ayvakit (avapritinib; Blueprint Medicines Corporation), a tyrosine kinase inhibitor, for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.
The FDA has granted Fast Track designation to ARO-AAT an investigational treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency