Novartis' PI3K drug, already approved in breast cancer, nabs a speedy OK to treat ultra-rare condition
Half a year after Novartis showcased the potential of its PI3K inhibitor alpelisib among a group of young patients with an ultra-rare condition, the FDA has greenlighted the drug’s use based solely on real-world evidence.
Branded Vijoice, it is the first and only treatment aimed at the root cause of PIK3CA-related overgrowth spectrum (PROS), according to Novartis, a disease estimated to afflict 14 people per million.
The underlying mutations in the PIK3CA are just what alpelisib targets. The same drug is already marketed as Piqray to treat a subset of advanced or metastatic breast cancer that’s HR-positive, HER2-negative and PIK3CA-mutated. Novartis reported $390 million in sales for 2021.
With PROS, patients’ body parts can grow too large or have an unusual shape, and they may have blood vessel abnormalities.
“PROS conditions can be debilitating and disabling and can result in disruptions to everyday activities,” said Kristen Davis, executive director of CLOVES Syndrome Community, in a prepared statement. “Until today, often the only treatment options for patients were surgical or interventional radiology procedures.”
Through a compassionate use program, a number of PROS patients received the drug. Novartis then ran a real-world study dubbed EPIK-P1 reviewing the medical charts of 57 patients, including 39 pediatric and 18 adult patients, across five countries. It found that those treated with Vijoice saw a reduction in the size of PROS lesions as well as improvements in related signs and symptoms.
Since Vijoice was OK’d under the accelerated approval pathway, Novartis will be required to run a confirmatory trial.
The pharma giant said it will conduct two additional Phase II studies: EPIK-P2 to assess safety, efficacy and pharmacokinetics, and EPIK-P3 to look into long-term safety and efficacy for patients who were in EPIK-P1.