FDA Grants Accelerated Approval to Novartis' Vijoice (alpelisib) for PIK3CA-Related Overgrowth Spectrum Based on Real-World Evidence

On April 6, Novartis announced that the U.S. FDA granted accelerated approval for Vijoice (alpelisib) to treat adult and pediatric patients aged 2 years and older with PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy and exhibit severe manifestations. The press release noted that Vijoice is the first PROS-targeted treatment approved by the U.S. FDA, addressing the underlying cause of PROS in specific patients.

PROS refers to the disease spectrum caused by PIK3CA gene mutations, characterized by atypical overgrowth and abnormalities in blood vessels, lymphatic systems, and other tissues, estimated to affect 14 people per million. Specific conditions associated with PROS include CLOVES syndrome, macrodactyly, muscular HH, epidermal nevi, benign lichenoid keratosis, or seborrheic keratosis, among others. PROS can impact quality of life and pose a range of physical, emotional, and social challenges for patients and their families, ranging from functional impairments and developmental delays to chronic pain, mobility issues, and feelings of isolation. Managing PROS can be challenging, requiring collaboration from multidisciplinary teams, with interventions available focusing primarily on symptom management for patients and physicians.

FDA Approval Based on Real-World Evidence from EPIK-P1, a retrospective, non-interventional medical record study, which showed that patients treated with Vijoice experienced reductions in target lesion volume and improvements in PROS-related symptoms. The primary endpoint analysis at week 24 showed that 27% of patients (10/37) achieved confirmed therapeutic response, defined as a reduction of more than 20% in the sum of target lesion volumes related to PROS. Among patients who underwent imaging at baseline and week 24, nearly three-quarters (74%, 23/31) showed a decrease in target lesion volume, with an average reduction of 13.7%, and no patients had disease progression at the time of the primary analysis.

Moreover, at week 24, the investigators observed improvements from baseline in patients' pain (90%, 20/22), fatigue (76%, 32/42), vascular malformations (79%, 30/38), limb asymmetry (69%, 20/29), and disseminated intravascular coagulation (55%, 16/29).

Executive Director of the patient organization CLOVES Syndrome Community, Kristen Davis, stated: "The approval of the first treatment for PROS today offers hope for a better quality of life to those affected by these rare conditions and their families. PROS conditions can be debilitating and disabling, often disrupting daily activities. Until now, the only treatment options for patients have often been surgery or interventional radiology procedures."

President of Novartis US Innovative Medicines, Victor Bulto, stated: "The approval of Vijoice marks a turning point for patients who, until now, have not had an approved therapy specifically addressing their condition. We are grateful to the doctors, patients, and families involved in the EPIK-P1 trial. We continue to invest in research to advance the scientific understanding of PROS and to explore the full potential of Vijoice."

Reference Source:

[1] FDA approves Novartis Vijoice® (alpelisib) as first and only treatment for select patients with PIK3CA-Related Overgrowth Spectrum (PROS). Retrieved 2022-04-06, from https://www.globenewswire.com/news-release/2022/04/06/2417163/0/en/FDA-approves-Novartis-Vijoice-alpelisib-as-first-and-only-treatment-for-select-patients-with-PIK3CA-Related-Overgrowth-Spectrum-PROS.html

▽Follow 【WuXi AppTecGermany】WeChat Official Account