- CSL (OTC:CSLLY) announced topline Phase 3 results for garadacimab (CSL312) for hereditary angioedema (HAE).
- HAE is a rare genetic condition that causes painful, debilitating, unpredictable episodes of swelling of the abdomen, larynx, face, and extremities, among other body areas.
- The study met its primary and secondary efficacy objectives and demonstrated favorable safety and tolerability.
- CSL aims to begin filing with global health authorities at the end of the current fiscal year for full approval.
- Full results from the study will be presented at an upcoming scientific congress and published in a peer-reviewed journal.
- Garadacimab is a novel Factor XIIa-inhibitory monoclonal antibody (FXIIa mAb) currently in Phase 3 clinical development as a new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE.
- Garadacimab uniquely inhibits the plasma protein FXIIa. When FXIIa is activated, it initiates the cascade of events leading to edema formation. By targeting FXIIa, garadacimab inhibits the HAE cascade.
- The drug has received orphan-drug designation from the FDA and European Medicines Agency.
- CSL is also investigating garadacimab for other indications beyond HAE, where FXIIa inhibition may play an essential role in improving clinical outcomes, including pulmonary fibrosis.
- Price Action: CSLLY shares are trading lower by 3.18% at $100.82 on the last check Wednesday.
Rocket Pharmaceuticals Provides Update on Anticipated Registration Path for RP-A501 in Danon Disease Following End-of-Phase 1 FDA Meeting
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces updates from the Company's end-of-Phase 1 meeting with the U.S.
