Chordia Therapeutics Secures Orphan Drug Designation for Rogocekib in the U.S.
Chordia Therapeutics Inc. has announced that its CLK inhibitor, rogocekib, for treating Refractory or Relapsed Acute Myeloid Lymphoma (AML), has received Orphan Drug Designation from the FDA, offering developmental and financial benefits.
FDA Accepts Abeona Therapeutics' BLA Resubmission for Prademagene Zamikeracel in RDEB Treatment
• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB).
• Pz-cel, an autologous cell-based gene therapy, aims to address the unmet needs of RDEB patients by providing collagen VII expression at wound sites.
• The BLA is supported by data from the Phase 3 VIITAL study and a Phase 1/2a study with up to 8 years of follow-up, showcasing clinical efficacy and safety.
• The FDA has set a PDUFA target action date of April 29, 2025, with potential for Abeona to receive a Priority Review Voucher upon approval.
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