EMA CHMP recommends AstraZeneca’s Voydeya for PNH treatment

27 Feb 2024
www.pharmaceutical-technology.com
Phase 3AcquisitionClinical Result
AstraZeneca’s Voydeya is an additional treatment to standard-of-care for adult paroxysmal nocturnal haemoglobinuria patients. Credit: Anna in Sweden / Shutterstock.com.
AstraZenecaee Voydeyaicinal Products for Human Use (CHMP) of the European Medicparoxysmal nocturnal haemoglobinuriaeca‘s oral Factor D inhibitor, Voydeya (danicopan), for marketing authorisation to treat paroxysmal nocturnal haemoglobinuria (PNH).
The product is an additional treatment to standard-of-care UltomiEuropean Medicines Agencyris (eculizumab) for adult PNH patients who have residual haemolytic anaemia.paroxysmal nocturnal haemoglobinuria (PNH)
The positive opinion is based on data from the ALPHA Phase UltomiriscaravulizumabglobaSolirisleeculizumabltiple-dose,PNHacebo-controlled study desighaemolytic anaemiae safety and efficacy of Voydeya as an add-on to a C5 inhibitor therapy in PNH patients with clinically significant extravascular haemolysis (EVH).
Patients in the trial were randomised to receive either Voydeya or a placebo in a 2:1 ratio, in addition to their ongoing Soliris or Ultomiris therapy, for 12 weeks.VoydeyaC5inhibitorPNH
Voydeya met the primary endpoint of variation in haemoglVoydeyavels from baseline to week 12, and all key secondary endpoiSolirisUltomiris
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Transfusion avoidance and variation in patients’ Functional Assessment of Chronic Illness Therapy – Fatigue score were among the secondary endpoints.
Voydeya FDA generally well tolerated with noIL-17 inhibitorIL-17safety concerns reported.
AstraZeneca Rare Disease group Alexion CEO Marc Dunoyer said: “The positive CHMP recommendation recognises the promise of Voydeya as an add-on to standard-of-care to address signs and symptoms of clinically significant EVH for this small subset of patients.
“As we saw in the pivotal ALPHA Phase III trial, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for these patients.”
Voydeyaest development comes after AstraZeneca concluded the acquisition of China-based clinical-stage biopharmaceutical company Gracell Biotechnologies for $1.2bn.
AstraZenecation, initially agreAlexionecember 2023, positions Gracell as a wholly owned subsidiary of AstraZeneca with opeVoydeya continuing in the US and China.
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