Novartis today announced that FDA approved Fabhalta® (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune…
Bristol Myers Squibb K.K. announced that the company has received manufacturing and marketing approval of the supplemental New Drug Application for an additional indication for Abecma®, a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for patients…
Sernova Corp., a clinical-stage company and leader in cell therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company’s Hemophilia A program. The FDA…
AstraZeneca’s Truqap (capivasertib) in combination with Faslodex (fulvestrant) has been approved in the US for the treatment of adult patients with hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations (PIK3CA, AKT1 or…
Vertex Pharmaceuticals and CRISPR Therapeutics announced that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and…
Astellas Pharma and Pfizer today announced that the companies received an approval by the FDA of a supplemental New Drug Application for XTANDI® (enzalutamide), following FDA expedited development and review programs (Priority Review designation, Fast Track designation, Real-time Oncology Review),…
Bristol Myers Squibb announced that FDA approved Augtyro™ (repotrectinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). Administered as an oral therapy, Augtyro is a tyrosine kinase inhibitor (TKI) targeting ROS1…
Boehringer Ingelheim announced that FDA has granted Orphan Drug Designation to BI 1819479 for the potential treatment of idiopathic pulmonary fibrosis (IPF). BI 1819479 is an investigational compound that may address pulmonary fibrosis—a scarring of the lung tissue that negatively…
FDA approved Eli Lilly and Company’s Zepbound™ (tirzepatide) injection, the first and only obesity treatment of its kind that activates both GIP (glucose-dependent insulinotropic polypeptide) and GLP-1 (glucagon-like peptide-1) hormone receptors. Zepbound is indicated for adults with obesity (with a…
Takeda announced that FDA has approved FRUZAQLA™ (fruquintinib), an oral targeted therapy for adults with metastatic colorectal cancer (mCRC) who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type and medically appropriate,…