Alnylam has completed the submission of marketing applications for lumasiran on both sides of the Atlantic for the ultra-rare inherited disease primary hyperoxaluria type 1 (PH1).
The firm said it had completed a rolling submission to the US Food and Drug Administration and marketing authorisation application to the European Medicines Agency (EMA) for use of the RNAi therapeutic.
If approved, lumasiran would be the first approved therapeutic treatment option for PH1 patients, according to the firm.
The filings include data from the pivotal ILLUMINATE-A Phase III study, in which lumasiran met all primary and secondary endpoints, demonstrating a substantial reduction in urinary oxalate excretion – a key factor in the underlying pathophysiology of PH1, “with an encouraging safety and tolerability profile”.
PH1 is characterised by painful and recurrent kidney stones, resulting from excess oxalate production. This excess oxalate can lead to compromised kidney function, resulting in subsequent accumulation of oxalate crystals in bones, eyes, skin, and heart, leading to severe illness and death.
