Novartis scores accelerated FDA nod for Vijoice to treat rare overgrowth condition

Less than three years after launching a budding blockbuster in breast cancer drug Piqray (alpelisib), Novartis has scored an approval for the treatment in a vastly different disorder.

On Wednesday, the FDA signed off on the PI3K inhibitor to treat PIK3CA-related overgrowth spectrum (PROS), an umbrella term for the rare condition that causes blood vessel abnormalities and overgrowth of tissue that can present in a variety of ways.

To treat PROS, alpelisib is branded as Vijoice. It becomes the first treatment to address the root cause of the condition and is approved for select patients 2 years and older with a severe form of the disorder that requires systemic therapy. It's the first drug specifically approved for the condition.

The nod comes after a real-world study showed Vijoice reduced the size of PROS lesions. It also came under the FDA’s accelerated approval program, so the nod is contingent upon verification of clinical benefit in a confirmatory trial.  

“PROS conditions can be debilitating and disabling and can result in disruptions to everyday activities,” Kristen Davis, the executive director of CLOVES Syndrome Community, said in a release. “Until today, the only treatment options for patients were surgical or interventional radiology procedures.”

The mutation that causes PROS occurs during embryonic development. It is present in 14 people per one million. Complications from PROS depend on the site and the extent of the overgrowth and may include chronic pain, superficial infections, hemorrhages and embolisms, neurologic complications, functional impairments, and cardiac and renal abnormalities.

In some people, it can simply take the form of discolored skin in a mosaic pattern in a particular part of the body.

The EPIK-P1 real-world, retrospective chart review study of 37 PROS patients showed that 27% of patients on the drug saw a 20% or greater reduction in lesion volume, meeting the primary endpoint. In all, 74% of patients showed some reduction in lesion volume.

Additionally, in subsets of patients at week 24, there were improvements in pain for 90%, fatigue for 76%, vascular malformation for 79%, limb asymmetry for 69% and disseminated intravascular coagulation for 55%.

In May of 2019, the FDA blessed alpelisib (Piqray) for its original use, making it the first novel drug to be approved under the regulator’s Real-Time Oncology Review pilot program, allowing for faster evaluation of potential breakthrough drugs. Piqray is the first drug for HR+/HER2- breast cancer patients with a PIK3CA mutation. In its second full year on the market, the med rang up sales of $370 million.