NICE recommends Novartis Rydapt to treat systemic mastocytosis

CompilationFan Dongdong

A few days ago, the National Institute of Health and Care Excellence (NICE) has decided to recommend Novartis Rydapt (midostaurin) for the treatment of aggressive systemic mastocytosis, systemic mastocytosis with related blood tumors or mast cell leukemia ( Collectively referred to as AdvSM)
.

After being recommended by NICE, Rydapt became the first and only licensed AdvSM treatment in the UK.
Previously, the drug has been proven to provide therapeutic benefits to adults with the disease
.

The previous clinical treatment options for British patients can only improve symptoms, but the approved Rydapt is a targeted therapy that can inhibit KIT signaling, cell proliferation and histamine release, and induce mast cell apoptosis

According to the test results published in the New England Journal of Medicine in January this year, Rydapt can inhibit KIT D816V, which promotes the progression of mastocytosis
.

Trial data showed that the overall response rate of patients in the Rydapt group was 60%, and 45% of the patients experienced complete regression of at least one hypertrophy-related organ damage

The drug is an oral multi-kinase inhibitor capsule containing 25 mg of midostaurin
.

After the patient takes it, the drug can inhibit a variety of key kinases that regulate cell growth, including Flt3, thereby interfering with the growth and proliferation of cancer cells in the patient's body

In April 2017, the U.
S.
Food and Drug Administration (FDA) approved the use of Rydapt in combination with chemotherapy to treat newly diagnosed adult patients with acute myeloid leukemia with specific gene mutation FLT3.
This also made Rydapt the first in the United States at that time.
A targeted drug used in combination with chemotherapy to treat acute myeloid leukemia
.

Compared with other patients with acute myeloid leukemia, patients with FLT3 gene mutations are more likely to relapse and have a lower survival rate

In addition, the FDA also approved the drug for the treatment of invasive systemic mastocytosis, a rare blood disease
.

Previously, the FDA also granted priority review qualifications for Rydapt listing applications, fast-track review qualifications (for mastocytosis), and breakthrough therapy formulation (for acute myeloid leukemia)

In terms of competitors, Ayvakit (avapritinib) developed by Blueprint was approved by the FDA in June this year to treat adult patients with advanced systemic mastocytosis (SM), including aggressive SM, SM with related blood tumors, and mast cell leukemia
.

The results of the trial showed that in patients who received or did not receive previous treatment, Ayvakit treatment produced a durable clinical response including complete remission

Reference source: NICE recommends Novartis' Rydapt for AdvSM treatment