Novartis has received accelerated approval from the US Food and Drug Administration (FDA) for its Vijoice (alpelisib) to treat severe PIK3CA-Related Overgrowth Spectrum (PROS).
The drug was indicated for the treatment of adult and paediatric patients, aged two years and above with severe symptoms of PROS, who require systemic therapy.
Vijoice is a kinase inhibitor intended for the treatment of rare overgrowth conditions caused by the effects of PIK3CA mutations in adults and children with PROS.
According to the Swiss drugmaker, the drug works by inhibiting the PI3K pathway, mostly the PI3K-alpha isoform, and is the first FDA approved treatment for PROS conditions.
PROS is characterised by a spectrum of rare conditions including overgrowths and blood vessel anomalies that affect the quality of life of the patients.
The US FDA approval was based on positive results from EPIK-P1, a retrospective chart review study of Vijoice in patients with severe manifestations of PROS.
In the study, treatment using Vijoice resulted in the reduction of target lesion volume and improvement in PROS-related symptoms and manifestations.
CLOVES Syndrome Community executive director Kristen Davis said: “Today’s approval of the first treatment for PROS offers hope for a better quality of life to patients and families affected by these rare conditions.
“PROS conditions can be debilitating and disabling and can result in disruptions to everyday activities. Until today, often the only treatment options for patients were surgical or interventional radiology procedures.”
In a separate development, Novartis’ subsidiary Sandoz has extended its existing strategic collaboration with Ares Genetics, a subsidiary of OpGen, until 31 January 2025.
The expanded partnership is aimed at driving advanced digital solutions in the global fight against antimicrobial resistance (AMR).
As part of the existing collaboration, Ares has developed a digital anti-infectives platform to support the identification of effective antimicrobial compounds to target critical pathogens.
The partnership combined established microbiology laboratory practices with advanced bioinformatics and AI methods for the platform.
In the next phases, the collaboration will focus on antibiotics by enabling genomic surveillance, leveraging Ares’ next-generation sequencing (NGS) and bioinformatics technologies.
Sandoz CEO Richard Saynor said: “This collaboration is a key step towards that goal, allowing us to take the fight directly to AMR by using cutting-edge big data and AI approaches combined with traditional data sets.
“By combining our respective skill sets, we believe we can not only use diagnostic and surveillance data to help healthcare professionals make better diagnoses, but also expand our options to treat AMR in the future, by targeted repurposing of key antibiotics.”
