NORTH CHICAGO, Ill. — AbbVie (NYSE: ABBV) today announced submission of a new Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of investigational Pivekimab sunirine (PVEK) for the treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The submission is based on data from the...
treatment News
NORTH CHICAGO, Ill. — AbbVie (NYSE: ABBV) announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the fixed-duration, all-oral combination regimen of VENCLEXTA® (venetoclax) and acalabrutinib in previously untreated patients with CLL, offering patients another VENCLEXTA combination regimen with the potential for time-limited...
NEWTON, Mass. — Abcuro, Inc., a late-stage clinical biotechnology company developing potentially first-in-class immunotherapies designed to benefit people living with debilitating and progressive rare autoimmune diseases and for other indications where certain cytotoxic T cells are pathogenic, today presented interim data from the ongoing Phase 1/2 clinical trial evaluating ulviprubart...
CLEVELAND, Ohio — Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the first commercial patient treatment with FDA-approved ZEVASKYN (prademagene zamikeracel), a first-of-its-kind, autologous gene therapy for treating wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). ZEVASKYN was administered at Lucile Packard Children’s Hospital Stanford in Palo...
CLEVELAND, Ohio — Abeona Therapeutics Inc. (Nasdaq: ABEO), a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases, today announced that its ABO-503 gene therapy for X-linked retinoschisis (XLRS) has been selected to participate in the U.S. Food and Drug Administration (FDA) Rare Disease Endpoint Advancement (RDEA) Pilot...
TOULOUSE, France – ABIONYX Pharma (FR0012616852 – ABNX – PEA PME eligible), a new generation biotech company dedicated to innovative therapies for patients, today announces positive clinical results from CER-001 in the LCAT (lecithin-cholesterol acyltransferase) deficiency disease published in the Annals of Internal Medicine. The patient with inherited mutations in the lecithin-cholesterol...
LUND, Sweden — Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage company developing drugs for the treatment of rare and severe primary mitochondrial disease, today announced a positive outcome of the interim analysis for FALCON, the potentially registrational study evaluating KL1333 in patients with primary mitochondrial disease. The study evaluates fatigue...
SAN DIEGO, Calif. – Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has approved DAYBUE® STIX (trofinetide) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients two years of age and...
– Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the Clinical Global Impression of Improvement (CGI-I) (p=0.0030) – Trofinetide met key secondary endpoint demonstrating statistically significant improvement over placebo in CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to communicate...
SAN DIEGO, Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadia’s submission. “Rett syndrome is a profoundly debilitating and complex...