Factor D

VOYDEYA™ approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular hemolysis in adults with the rare disease PNH

Retrieved on: 
Monday, April 1, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Factor D, Arthralgia, Chest pain, Confusion, Neuropsychological test, Infection, Eculizumab, Risk, Headache, ALPHA, PNH, C5, Patient, Orphan, Mitigation, Vaccine, Light, Paroxysmal nocturnal hemoglobinuria, EVH, Bacteria, University, Chills, MD, Breakthrough therapy, Cough, European Medicines Agency, Fatigue, Nausea, Rash, Fever, Priority, Safety, Immune system, REMS, Division, Vomiting, Hematology, Patient safety, Medicine

VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.

Key Points: 
  • VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.
  • You must complete or update meningococcal vaccine(s) and streptococcus vaccine(s) at least 2 weeks before your first dose of VOYDEYA.
  • If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting VOYDEYA.
  • Carry it with you at all times during treatment and for 1 week after your last VOYDEYA dose.

BioCryst’s Oral Factor D Inhibitor, BCX9930, Shows Clinical Benefit as Monotherapy Through 400 mg bid in Treatment-naïve PNH Patients

Retrieved on: 
Wednesday, September 30, 2020
Medical specialties, Hematology, Hemolysis, Blood, Factor D, Anatomy, Acquired hemolytic anemia, Paroxysmal nocturnal hemoglobinuria, Hematopathology

We are thrilled with the clinical benefits and safety profile of oral BCX9930 monotherapy that we continue to see in PNH patients with dosing up to 400 mg bid.

Key Points: 
  • We are thrilled with the clinical benefits and safety profile of oral BCX9930 monotherapy that we continue to see in PNH patients with dosing up to 400 mg bid.
  • Four patients had a history of compromised bone marrow function, and two had thrombotic, lung or kidney complications from PNH.
  • New data from the four treatment-nave PNH patients who have received more than six weeks of therapy at 400 mg bid show significant clinical benefits for these patients.
  • As a Factor D inhibitor, BCX9930 is designed as an oral monotherapy that can address both intravascular and extravascular hemolysis in PNH patients.

FDA Grants Fast Track Designation for BCX9930 in PNH

Retrieved on: 
Monday, August 3, 2020
Organ systems, Life sciences, Biotechnology, PNH, Paroxysmal nocturnal hemoglobinuria, BioCryst Pharmaceuticals, Fast track, Factor D, Food and Drug Administration, Paroxysmal attack, Eculizumab

RESEARCH TRIANGLE PARK, N.C., Aug. 03, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., Aug. 03, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
  • PNH patients have a tremendous need for therapy improvements and it is exciting for patients that the FDA has reviewed our proof of concept PNH data and granted Fast Track status.
  • We look forward to working closely with the FDA to fully utilize the opportunities presented by our Fast Track designation to advance this important medicine to patients as quickly as possible in PNH.
  • This press release contains forward-looking statements, including statements regarding potential benefits associated with an FDA Fast Track designation.