The U.S. Food and Drug Administration (FDA) granted accelerated approval to Novartis’ Vijoice (alpelisib) for the treatment of severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS), according to Novartis.

The drug has been approved for adult and pediatric patients 2 years of age and older with PROS – a group of rare disorders that causes overgrowth of parts of the body due to mutations in the PIK3CA gene. It is estimated to affect 14 people per million.

Vijoice is the first FDA-approved treatment for PROS. Its active drug alpelisib is a kinase inhibitor, which treats rare overgrowth conditions caused by the effects of PIK3CA mutations in adults and children with PROS. The treatment is not approved for use outside the United States.

The most common side effects of Vijoice are diarrhea, stomatitis, hyperglycemia, and cellulitis.

Kristen Davis, Executive Director of CLOVES Syndrome Community, said, “Today’s approval of the first treatment for PROS offers hope for a better quality of life to patients and families affected by these rare conditions.”

“PROS conditions can be debilitating and disabling and can result in disruptions to everyday activities,” she added. “Until today, often the only treatment options for patients were surgical or interventional radiology procedures.”

Specific disorders in PROS include fibroadipose hyperplasia, CLOVES syndrome, megalencephaly-capillary malformation syndrome, hemihyperplasia‐multiple lipomatosis syndrome, hemimegalencephaly, and facial infiltrating lipomatosis.

The signs and symptoms include having a larger-than-normal brain (megalencephaly), low muscle tone (hypotonia), seizures, intellectual disability, changes in the blood vessels (vascular system), and overgrowth of one area of the body (focal overgrowth) or of multiple areas of the body (segmental overgrowth), with normal growth elsewhere, according to GARD (Genetic and Rare Disease Information Center).

Dr. Guillaume Canaud of Necker-Enfants Malades Hospital, France, said, “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.”

“This is a significant advancement in therapy for PROS with the potential to positively change the treatment trajectory and outcomes for patients,” he added.

Victor Bulto, President, Innovative Medicines US, Novartis, said, “The approval of Vijoice marks a turning point for patients who, until now, have not had an approved therapy to specifically address their disease.”

“We are grateful to the physicians, patients, and families who participated in the EPIK-P1 trial,” he added. “We are continuing to invest in studies to advance the scientific understanding of PROS conditions and to understand the full potential of Vijoice.”