The Food and Drug Administration officially cleared Novartis AG's Rydapt (midostaurin), which is a drug for treating Acute Myeloid Leukemia or AML. The FDA announced the drug's clearance on Friday, April 28.
AML is basically a type of "rapidly progressing cancer." It affects the bone marrow, causing increased white blood cell count in the blood stream of the infected person.
"Rydapt represents a remarkable advance as the first and only targeted therapy approved for patients who had limited options for many years," Novartis Oncology's CEO Bruno Strigin remarked.
Rydapt: What Does It Do?
According to the FDA, the now-approved drug is effective in patients who are diagnosed with early stages of AML and possess a particular gene mutation named FLT3. However, Rydapt can only be used along with a companion diagnostic dubbed LeukoStrat CDx FLT3 Mutation Assay.
"The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment," Richard Pazdur, who is the acting director of the Office of Hematology and Oncology Products in the FDA, shared.
Rydapt is essentially a kinase inhibitor and blocks the enzymes responsible for cell growth. If AML is detected in a patient after being tested with the LeukoStrat CDx FLT3 Mutation Assay, Rydapt treatment can be considered.
The drug was also cleared to be used on adults with certain blood diseases like ASM or aggressive systemic mastocytosis and mast cell leukemia.
How Effective Is Rydapt?
To understand the effectiveness of the drug, 717 random patients with AML were given Rydapt along with chemotherapy. The results showed that patients who were administered both treatment, namely chemotherapy and Rydapt, lived longer than those whose treatment only comprised chemotherapy.
It was also established that patients who were administered Rydapt got through an average of 8.2 months without any complications, such as progression of leukemia or failure to achieve remission. This timeframe was much longer vis-à-vis patients who only received chemotherapy.
Previous studies from Novartis also concluded that use of Rydapt in AML patients decreased the possibility of death from the disease by 23 percent.
Cost Of Rydapt Treatment
For a 14-day treatment with the drug, the listed price is $7,495. A 28-day treatment would set one back by $14,990. A late stage trial of Rydapt showed that an average 42-day treatment was required for AML and, therefore, the cost of the drug comes to $22,485.
For people with ASM, the list price of the drug is $32,121 for a 30-day treatment. The average time for treatment for ASM cases is around 11.4 months.
AML cases are quite common in the United States and the American Cancer Society predicted that it will affect 21,380 people in 2017 and have a mortality count of 10,590. With the approval of the new drug, there is a ray of hope for those suffering from AML. Rydapt will become available soon and the price will differ according to dosage and indication.