The US Food and Drug Administration (FDA) has approved Apellis Pharmaceuticals’  Empaveli injector (Enfuse) for the subcutaneous delivery of Empaveli (pegcetacoplan) for adults with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare disease that is characterised by red blood cells breaking apart prematurely, causing symptoms such as haemolytic anaemia and shortness of breath.

The injector is a single-use, wearable device that allows patients to self-administer 20ml of subcutaneous therapy, enabling at-home treatment.

Empaveli generated $65.1m in sales last year, according to Apellis’ 2022 annual report. It is not the only treatment for PNH that is delivered by intravenous infusion (IV). Ultomiris (ravulizumab) marketed by Alexion Pharmaceuticals generated $1.96bn in sales last year, according to AstraZeneca’s 2022 annual report. Ultomiris must be administered by a healthcare provider, unlike Empaveli, which can now be administered at home by the patient themselves.

According to GlobalData, Empaveli is forecast to exceed $2bn by 2030.

The injector is developed in collaboration with Enable Injections, a Cincinnati, US-based developer of drug delivery systems.  

Chief technology officer and executive vice-president of business development at Enable Injections Matthew Huddleston said: “The Empaveli Injector is the first purely mechanical, large-volume, on-body subcutaneous drug delivery device.”