Novartis gets USFDA accelerated approval for PIK3CA-Related Overgrowth Spectrum treatment Vijoice
Basel: Novartis has recently announced that the U.S. Food and Drug Administration (USFDA) has granted accelerated approval to Vijoice (alpelisib) for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy.
Vijoice is the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowths and blood vessel anomalies impacting an estimated 14 people per million. In accordance with the Accelerated Approval Program, continued approval may be contingent upon verification and description of clinical benefit from confirmatory evidence.
"The approval of the first treatment for PROS offers hope for a better quality of life to patients and families affected by these rare conditions," said Kristen Davis, Executive Director of CLOVES Syndrome Community. "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."
PROS conditions can affect quality of life and pose a range of physical, emotional and social challenges for patients and their families, ranging from functional impacts and developmental delays to chronic pain, mobility issues, and feelings of isolation. PROS management can be challenging, requiring collaboration from a multidisciplinary team, and patients and physicians have only had access to interventions focused on symptom management.
"I am proud of this outstanding achievement for the PROS community. The EPIK-P1 study results build on our earlier pre-clinical findings and demonstrate the efficacy of Vijoice for select PROS conditions, effectively reducing PROS growths," said Guillaume Canaud, MD, PhD, Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, Inserm (INEM Institute Necker Enfants Malades – Centre for Molecular Medicine). "This is a significant advancement in therapy for PROS with the potential to positively change the treatment trajectory and outcomes for patients."
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