Novartis has secured accelerated approval from the US Food and Drug Administration (FDA) for vijoice (alpelisib) to treat PIK3CA-related overgrowth spectrum (PROS) patients.
A kinase inhibitor, vijoice is intended for the treatment of rare overgrowth conditions which are caused by the effects of PIK3CA mutations in adults and children aged two years and above with PROS.
It is claimed to be the first treatment to receive FDA approval for PROS, which is a spectrum of rare conditions that are characterised by overgrowths and anomalies in blood vessels.
Novartis Innovative Medicines US president Victor Bulto said: “The approval of Vijoice marks a turning point for patients who, until now, have not had an approved therapy to specifically address their disease.
“We are grateful to the physicians, patients and families who participated in the EPIK-P1 trial. We are continuing to invest in studies to advance the scientific understanding of PROS conditions and to understand the full potential of Vijoice.”
The regulatory approval was based on the real-world data obtained from the retrospective chart review EPIK-P1 study.
The findings showed that the patients who received treatment with Vijoice had reduced target lesion volume and PROS-related symptoms and manifestations improvement.
About 27% of patients treated with the therapy achieved a confirmed response to treatment during the primary endpoint analysis which was conducted at week 24.
The most common adverse events observed in the EPIK-P1 study include stomatitis, diarrhea, and hyperglycemia.
In December last year, Novartis received the US FDA approval for its Cosentyx (secukinumab) for the treatment of enthesitis-related arthritis and psoriatic arthritis in children and adolescents.