FDA submission
Dizal Pharmaceutical Seeking FDA Approval for Sunvozertinib in EGFR Exon 20 Insertion-Mutated NSCLC
Chinese regulators approved the drug for the same subset of advanced, previously treated EGFR-mutated NSCLC patients in 2023.
Verastem Seeking FDA Approval for Avutometinib, Defactinib in KRAS-Mutant Ovarian Cancer
The FDA granted priority review to the company's avutometinib-defactinib application and expects to decide on approval in June 2025.
Ultragenyx Submits BLA for Sanfilippo Syndrome Type A Gene Therapy, Seeks Accelerated Approval
The company separately said it has started treating patients within a Phase III trial testing an ASO drug for Angelman syndrome.
Rocket Pharma Starts Rolling BLA Submission for Fanconi Anemia Gene Therapy
RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.
Eisai Completes Rolling BLA Submission for Subcutaneous Leqembi Maintenance Treatment
Eisai has argued that maintenance dosing with Leqembi will prolong treatment benefits by targeting protofibrils, a toxic form of beta-amyloid.