New FDA Approval: VOYDEYA™ (danicopan)
The Food and Drug Administration (FDA) recently approved VOYDEYA™, the first-in-class, oral, Factor D inhibitor based on results from pivotal ALPHA Phase III trial.
| Drug Name |
VOYDEYA™ |
| Active Ingredient |
danicopan |
| Date of Approval |
3/29/24 |
| Manufacturer |
AstraZeneca |
| Approval Pathways and Indications |
Approval Pathway: Orphan Drug Designation and Breakthrough Therapy Designation (FDA); PRIority MEdicines (PRIME) status by the European Medicines Agency
Indication: Indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH)
|
| Therapeutic Class |
Complement Factor D Inhibitor; Complement Inhibitor |
| Formulation |
Tablet |
| MoA |
Binds to the complement factor D. It prevents the cleavage into Ba and Bb fragments required for the formation of an enzyme involved in extravascular hemolysis (EVH) |
| Dosing and Administration |
Usual starting dose: 150mg by mouth three times a day. It can be taken with or without food - Strengths available: 50mg and 100mg tablets |
| Place in Therapy |
VOYDEYA™ is a first-in-class, oral, Factor D inhibitor developed as an add-on therapy to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant EVH while treated with a C5 inhibitor. Voydeya treats by inhibiting complement factor D while Ultomiris and Soliris both work to remove C5 from the bloodstream and help prevent the uncontrolled complement activity.
|
| Expected Market Launch Date |
Unavailable |
| New Molecular Entity (NME) or Existing Formulation |
NME |
| Expected Cost |
AstraZeneca has not released pricing yet but it is currently being estimated to be around $12,300 and $13,100 a year by the Institute for Clinical and Economic Review. |
| Product Discontinuation |
N/A |
| Clinical Trial Information |
The approval of VOYDEYA™ was based off of the Phase III ALPHA. The trial evaluated the efficacy and safety of VOYDEYA™ and demonstrated a statistically significant and clinically meaningful increase in haemoglobin levels and maintained disease control in patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience clinically significant extravascular haemolysis (EVH), defined as haemoglobin ≤9.5 g/dL and absolute reticulocyte count levels ≥120x109/L, compared to placebo plus established C5 inhibitor therapy (Soliris and Ultomiris).
Results showed that VOYDEYA™ met the primary endpoint of change in haemoglobin from baseline to week 12 and all key secondary endpoints, including transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score. |
Sources:
1. ICER publishes evidence report on treatments for paroxysmal nocturnal hemoglobinuria. ICER. (2024, February 1). https://icer.org/news-insights/press-releases/icer-publishes-evidence-report-on-treatments-for-paroxysmal-nocturnal-hemoglobinuria/
2. Voydeya approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular haemolysis in adults with the rare disease PNH. (1AD). https://www.astrazeneca.com/media-centre/press-releases/2024/voydeya-approved-in-us.html/
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