Novartis Augments Transformational Week with US Approval, European Milestones

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Days after Novartis announced a transformational restructuring, the Swiss pharma giant continues to showcase its capabilities in treating diseases with the regulatory approval of Vijoice (alpelisib) for the treatment of adult and pediatric patients who have severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS).

With the accelerated approval from the U.S. Food and Drug Administration, Vijoice becomes the first treatment approved in the United States for PROS, which is a spectrum of rare conditions characterized by overgrowths and blood vessel anomalies. PROS is estimated to impact approximately 14 million people. 

Approval was based on real-world evidence from EPIK-P1, a retrospective chart review study that showed patients who were treated with Vijoice experienced reduced target lesion volume and improvement in PROS-related symptoms and manifestations. According to the data, after 24 weeks, 27% of patients achieved a confirmed response to the treatment, which was defined as a 20% or greater reduction in the sum of PROS target lesion volume. Also, approximately three-fourths of patients achieved some reduction in target lesion volume, and no patients experienced disease progression at the time of primary analysis.

Victor Bulto, president of Novartis Innovative Medicines US, said the approval of Vijoice is a turning point for PROS patients who, until now, have not had an approved therapy. The company will continue to assess Vijoice in order to understand its full potential and advance the scientific understanding of PROS conditions. 

In addition to its FDA approval of Vijoice, Novartis saw the acceptance of multiple marketing authorization applications in Europe for its checkpoint inhibitor tislelizumab as a first-line treatment for patients with locally advanced or metastatic, squamous or non-squamous non-small cell lung cancer (NSCLC). Additionally, tislelizumab will be reviewed as a monotherapy following chemotherapy for locally advanced or metastatic NSCLC as monotherapy, as well as a post-chemotherapy monotherapy for unresectable, recurrent, locally advanced or metastatic esophageal squamous cell carcinoma.

Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody, which the company licensed from BeiGene last year. The acceptance of the authorization applications are significant for cancer patients in the countries where it will be available, Jeff Legos, global head of oncology and hematology at Novartis, said. 

“We look forward to working with the EMA to make tislelizumab available to people with these aggressive cancers, while continuing to expand our development program to investigate the potential of novel, synergistic combinations,” he said. Legos added that the European application acceptance builds on the FDA’s filing acceptance of tislelizumab in esophageal cancer.

The European Medicines Agency accepted the applications for tislelizumab for these indications based on data from the Phase III RATIONALE 302 trial. In that study, tislelizumab demonstrated a significant improvement in overall survival compared to chemotherapy in ESCC patients.

Also, data from the pivotal Phase III RATIONALE 303 trial showed that tislelizumab significantly improved overall survival compared to chemotherapy in people with NSCLC. Other data provided to the EMA included results from RATIONALE 304 and 307, which showed that tislelizumab and chemotherapy significantly improved progression-free survival in patients with untreated squamous and non-squamous NSCLC.

Beyond its regulatory wins and milestones, Novartis also announced that its Sandoz business unit extended a strategic collaboration with Ares Genetics, an OpGen subsidiary, until Jan. 31, 2025. The collaboration was forged in order to drive cutting-edge digital solutions in the global fight against antimicrobial resistance (AMR). The collaboration will allow the companies to harness Ares’ next-generation sequencing and bioinformatics technologies in order to dive into antibiotic stewardship through the use of genomic surveillance for resistant pathogens.

“Antibiotics are the cornerstone of modern medicine. AMR, which is now estimated to directly account for nearly 1.3 million deaths worldwide every year, is an unprecedented threat to global public health. As the world’s leading provider of generic antibiotics, our goal at Sandoz is to play a key role in overcoming this growing threat,” Sandoz Chief Executive Officer Richard Saynor said in a statement.