VOYDEYA

What it is

VOYDEYA is a film-coated tablet containing danicopan, available in 50 mg and 100 mg strengths. Danicopan binds reversibly to complement Factor D and selectively inhibits the alternative complement pathway, preventing the cleavage of complement Factor B which is required for formation of the C3 convertase.

Approved indications

— Add-on to ravulizumab or eculizumab for the treatment of the signs or symptoms of extravascular haemolysis (EVH) in adult patients with paroxysmal nocturnal haemoglobinuria (PNH).

Dosing overview

The recommended starting dose is 150 mg three times a day administered orally, approximately 8 hours apart (± 2 hours). Depending on clinical response the dose can be increased to 200 mg three times a day. VOYDEYA must not be administered as monotherapy as the efficacy has not been established. It should only be prescribed as an add-on to ravulizumab or eculizumab. VOYDEYA can be taken with or without food. PNH is a chronic disease and add-on treatment with VOYDEYA is recommended to continue for the patient's lifetime, unless the discontinuation of VOYDEYA is clinically indicated.

Key safety warnings

Patients receiving complement inhibitor therapy may have increased susceptibility to meningococcal infections (Neisseria meningitidis). Patients must be up to date on their meningococcal vaccines according to current national guidelines for vaccination use, prior to receiving the first dose of VOYDEYA. Patients must be vaccinated against serogroups A, C, Y, and W135 to prevent the commonly pathogenic meningococcal serogroups. Vaccination against serogroup B, where available, is also recommended. All patients treated with VOYDEYA should be monitored for early signs of meningococcal infection and sepsis, evaluated immediately if infection is suspected, and treated with appropriate antibiotics. Danicopan selectively blocks the activation of the complement alternative pathway; therefore, patients may have increased susceptibility to serious infections (other than Neisseria meningitidis). In clinical trials, laboratory abnormalities related to elevations in alanine aminotransferase (ALT) levels were observed in 14.0% of patients on VOYDEYA compared to 3.4% of patients on placebo. ALT elevations > 3 × the upper limit of normal (ULN) and ≤ 5 × ULN occurred in 8.8% of patients and > 5 × ULN and ≤ 10 × ULN in 5.3% of patients. There were no elevations > 10 × ULN. All patients were asymptomatic, and all elevations were transient. It is recommended that liver enzyme tests be performed before treatment begins. Following initiation of treatment, routine chemistry laboratory monitoring as per PNH management is recommended.

Contraindications

Hypersensitivity to the active substance or to any of the excipients listed in Section 6.1 List of Excipients. Do not initiate VOYDEYA therapy in patients with unresolved Neisseria meningitidis infection. Do not initiate VOYDEYA therapy in patients with unknown history of vaccination or who are not up to date on their meningococcal vaccines as per local guidelines, unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination.

Regulatory history

VOYDEYA was approved by the TGA on 11 November 2024, leading to its registration in the ARTG on 25 November 2024. Four ARTG registration records exist: ARTG 416091 (50 + 100 mg composite bottle pack), ARTG 416093 (100 mg blister pack), ARTG 416090 (100 mg bottle), and ARTG 416092 (50 + 100 mg composite blister pack), all first listed 2024-11-25. This medicinal product is subject to additional monitoring in Australia.