Novartis' Breast Cancer PI3K Drug, Scores FDA Approval For Ultra-Rare Indication

The FDA has granted accelerated approval to Novartis AG's NVS Vijoice (alpelisib) for 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.
• Under the Accelerated Approval Program, continued approval may be contingent upon verification and description of clinical benefit from confirmatory evidence.
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• Alpelisib is already marketed as Piqray to treat a subset of advanced or metastatic breast cancer that is HR-positive, HER2-negative, and PIK3CA-mutated. 
• Novartis ran a real-world study dubbed EPIK-P1, reviewing the medical charts of 57 patients. It found that those treated with Vijoice saw a reduction in the size of PROS lesions and improvements in related signs and symptoms.
• The Company giant said it would conduct two additional Phase 2 studies: EPIK-P2 to assess safety, efficacy, and pharmacokinetics, and EPIK-P3 to look into long-term safety and efficacy for patients in EPIK-P1.
• Price Action: NVS shares are up 0.77% at $88.85 during the premarket session on the last check Wednesday.