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How Treatment May Help

RYDAPT

RYDAPT® (midostaurin) capsules is the only FDA-approved treatment for advanced systemic mastocytosis (SM), including those patients with a certain mutation known as the KIT D816V mutation.

What are the potential benefits of taking RYDAPT?

In a clinical trial of 116 patients with advanced SM, 89 patients were evaluated to assess their response to treatment with RYDAPT.

 

Overall, 21% of these patients (19 of 89) responded to RYDAPT; response included disease improvement in at least 1 organ. In half of these patients, RYDAPT began to work in about 2 weeks.

 

Patients evaluated included those with and without a KIT D816V mutation:

Disease improvement based on mutation status Disease improvement based on mutation status Disease improvement based on mutation status
About 66% of previously treated advanced SM patients responded to RYDAPT treatment About 66% of previously treated advanced SM patients responded to RYDAPT treatment About 66% of previously treated advanced SM patients responded to RYDAPT treatment

How effective is RYDAPT in the different subtypes of advanced SM?

In the clinical trial, patients were evaluated to determine whether or not they responded to RYDAPT, how long their responses lasted, and how quickly they began to respond.

 

The results below show the effectiveness of RYDAPT in all patients in the clinical trial (regardless of what type of SM they had) and in patients according to their specific subtype of SM.

 

All patients evaluated (89 patients)

  • 21% (19 of 89 patients) achieved a complete or incomplete remission of disease after receiving 6 treatment cycles with RYDAPT
  • The median duration (the time point) at which half of the patients continued to have a complete or incomplete response to RYDAPT ranged from 6.6 months to 65.8 months. A specific median duration of response was not reported because, at the time of analysis, more than half of patients receiving RYDAPT were still responding to treatment
  • The median time (the time point) at which half of the patients began to respond to RYDAPT was 0.5 months. The median time to response ranged from 0.1 months to 3 months

 

Patients with aggressive systemic mastocytosis (ASM) (16 patients)

  • 38% (6 of 16 patients) achieved a complete or incomplete remission of disease after receiving 6 treatment cycles with RYDAPT
  • The median duration (the time point) at which half of the patients continued to have a complete or incomplete response to RYDAPT ranged from 12.1 months to 36.8 months. A specific median duration of response was not reported because, at the time of analysis, more than half of patients receiving RYDAPT were still responding to treatment
  • The median time (the time point) at which half of the patients began to respond to RYDAPT was 0.7 months. The median time to response ranged from 0.3 months to 1.9 months

 

Patients with systemic mastocytosis with associated hematological neoplasm (SM-AHN) (57 patients)

  • 16% (9 of 57 patients) achieved a complete or incomplete remission of disease after receiving 6 treatment cycles with RYDAPT
  • The median duration (the time point) at which half of the patients continued to have a complete or incomplete response to RYDAPT ranged from 6.6 months to 52.1 months. A specific median duration of response was not reported because, at the time of analysis, more than half of patients receiving RYDAPT were still responding to treatment
  • The median time (the time point) at which half of the patients began to respond to RYDAPT was 0.5 months. The median time to response ranged from 0.1 months to 3 months

 

Patients with mast cell leukemia (MCL) (16 patients)

  • 25% (4 of 16 patients) achieved a complete or incomplete remission of disease after receiving 6 treatment cycles with RYDAPT
  • The median duration (the time point) at which half of the patients continued to have a complete or incomplete response to RYDAPT ranged from 19.1 months to 65.8 months. A specific median duration of response was not reported because, at the time of analysis, more than half of patients receiving RYDAPT were still responding to treatment
  • The median time (the time point) at which half of the patients began to respond to RYDAPT was 0.3 months. The median time to response ranged from 0.1 months to 0.5 months