February 19, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Basel, Switzerland-based Novartis AG announced today that the U.S. Food and Drug Administration (FDA) granted its drug PKC412 (midostaurin) Breakthrough Therapy Designation.
PKC412 is an oral, multi-targeted kinase inhibitor for the treatment of patients with acute myeloid leukemia (AML) with FLT3 mutations. It is also being studied to treat aggressive systemic mastocytosis/mast cell leukemia. FMS-like tyrosine kinase-3 (FLT3) is a cell-surface receptor for tyrosine kinase, which has a role in the increase of certain blood cell types.
The compound’s status was primarily based on positive data from the Phase III RATIFY (CALGB 10603) clinical trial, which was conducted in partnership with the Alliance for Clinical Trials in Oncology. Data was presented at the 57th American Society of Hematology (ASH) Annual Meeting.
In the study, patients receiving PKC412 with standard induction and consolidation chemotherapy showed significant improvement in overall survival (OS) compared to patients receiving standard induction and consolidation chemotherapy. For patients in the PKC412 treatment group, the median OS was 74.7 months. The median OS for patients in the placebo group was 25.6 months.
“For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged,” said Alessandro Riva, Global Head, Novartis Oncology Development and Medical Affairs, 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.”
It’s been a big week for drugs receiving Breakthrough Designation status. On Feb. 17, London-based AstraZeneca and its global biologics research and development unit, MedImmune , announced that its MEDI4736 (durvalumab) was given Breakthrough Therapy Designation. That compound is a human monoclonal antibody that targets programmed death ligand-1 (PD-L1), and is being investigated for treatment of PD-L1 positive inoperable or metastatic urothelial bladder cancer when the tumor has progressed during or after treatment by a platinum-based therapy.
On the same day, Swiss-based Roche Group (RHHBY) announced that the FDA gave its drug Ocrevus (ocrelizumab) Breakthrough Therapy Designation for the treatment of primary progressive multiple sclerosis (PPMS).
“Ocrelizumab is the first investigational medicine for MS to be granted Breakthrough Therapy Designation by the FDA,” said Sandra Horning, chief medical officer and head of Global Product Development for Roche , in a statement. “With no approved treatments for primary progressive MS, ocrelizumab has the potential to address an important unmet need. We are committed to working with the FDA to bring ocrelizumab to people with primary progressive MS as quickly as possible.”
Regarding PKC412, Novartis stated, “Since PKC412 (midostaurin) is investigational at this time and is expected to be submitted for FDA approval, Novartis opened a Global Individual Patient Program (compassionate use program) and a US Expanded Treatment Protocol (ETP) to enable PKC412 (midostaurin) access. Patients 18 years of age and older with newly-diagnosed FLT3-mutated AML and able to receive standard induction and consolidation therapy will be considered.”
In addition, to help identify patients with the FLT3 mutation, Novartis has a deal with San Diego-based Invivoscribe Technologies, Inc., which is working on regulatory submissions for a companion diagnostic test.
