Novartis' blood cancer drug bags a 'breakthrough' tag on the way to FDA filing

Novartis oncology chief Alessandro Riva

Novartis ($NVS) picked up an FDA breakthrough therapy designation for its in-development treatment for blood cancer, guaranteeing the company preferred access to top regulators as it prepares to file for approval.

The agency has added PKC412 to its breakthrough program, which is meant to accelerate the process for treatments that could change the standard of care in particularly underserved diseases.

Novartis' drug is an oral therapy designed to treat patients with a form of acute myeloid leukemia, or AML. PKC412, also known as midostaurin, works by blocking multiple enzymes key to cancer growth in the roughly one-third of AML patients who have mutations in their FLT3 genes. In Phase III data presented at the American Society of Hematology meeting last year, the drug charted a median overall survival of 74.7 months, beating out the 25.6 months seen on placebo alone.

Novartis said it's on track to submit PKC412 for global regulatory approvals in the first half of this year, working with Invivoscribe Technologies to develop a companion diagnostic that can screen AML patients for FLT3 mutations.

AML accounts for about 25% of all adult leukemias, according to Novartis, and has the lowest survival rate.

"For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged," Novartis oncology chief Alessandro Riva said in a statement. "We look forward to working closely with the FDA to bring PKC412 (midostaurin), the first potential AML targeted therapy, to patients as quickly as possible."

Novartis is also developing PKC412 in mast cell leukemia and myelodysplastic syndrome.

- read the statement