Voydeya Approved in the US as Add-On Therapy for PNH Patients with Extravascular Haemolysis

SciFocus/April 28, 2024 -- A significant milestone has been reached in the treatment of paroxysmal nocturnal haemoglobinuria (PNH) with the approval of Voydeya (danicopan) in the US as add-on therapy to ravulizumab or eculizumab for the management of extravascular haemolysis (EVH) in adults with this rare disease. Here are the key highlights and insights from this groundbreaking approval:

• Voydeya (danicopan) is a first-in-class, oral Factor D inhibitor developed to address the needs of PNH patients experiencing clinically significant EVH while on a C5 inhibitor.
• The approval by the US FDA is based on positive results from the pivotal ALPHA Phase III trial, showcasing Voydeya's efficacy and safety as an add-on therapy.

Key Highlights:

• Unique Treatment Approach: Voydeya acts as an add-on therapy to Ultomiris or Soliris to specifically target EVH in PNH patients, addressing a critical unmet need in this population.
• Clinical Efficacy: The ALPHA Phase III trial demonstrated Voydeya's effectiveness in improving haemoglobin levels, transfusion avoidance, and reducing fatigue scores.
• Safety Profile: Voydeya was generally well tolerated in the trial, with no new safety concerns identified, providing a favorable risk-benefit profile for patients.

Expert Insights:

• Dr. Bart Scott: Acknowledged Voydeya as offering a targeted add-on therapy for PNH patients with EVH, complementing the established disease control achieved with Ultomiris or Soliris.
• Marc Dunoyer, CEO of Alexion: Emphasized Voydeya's role as a first-in-class Factor D inhibitor, marking a significant advancement in PNH treatment and demonstrating a commitment to innovation in complement science.

About PNH and EVH:

• PNH is a rare, chronic, and potentially life-threatening blood disorder characterized by red blood cell destruction and increased risk of thrombosis.
• EVH, the removal of red blood cells outside blood vessels, can occur in PNH patients treated with C5 inhibitors, leading to ongoing symptoms of anaemia.

Future Prospects:

• Voydeya's approval marks a critical step in addressing the complexities of PNH, offering hope for improved outcomes and quality of life for patients.
• Ongoing research and development efforts by Alexion and AstraZeneca in rare diseases continue to drive innovation in PNH treatment and complement science.

This approval underscores the commitment of the healthcare community to advancing therapies for rare diseases like PNH, bringing new options to patients in need.

For more information about Alexion and AstraZeneca's rare disease initiatives, visit astrazeneca.com and follow @AstraZeneca on social media.

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