f whom 47 (41%) had prior therapy for SM, and 93 (81%) had a documented D816V mutation at baseline. Table 8 provides the results of this analysis. Overall response by IWG-MRT-ECNM criteria was reported for 16 (17%) of 93 patients with a documented D816V mutation, and in 8 (17%) of 47 patients having prior therapy for SM.
Table 8: Efficacy of RYDAPT in Systemic Mastocytosis per IWG-MRT-ECNM Consensus Criteria Using an Algorithmic Approach (Study 2)
All patients
eva luated
(N = 115) b, c ASM
(N = 16)
SM-AHN
(N = 72)
MCL
(N = 21)
Subtype not
established
(N = 6)
Overall Response in 6 cycles, n a
(95% CI) 19 (17%)
(10, 25) 5 (31%)
(11, 59) 8 (11%)
(5, 21) 4 (19%)
(5, 42) 2 (33%)
(4, 78)
Best Overall Response, n
Complete Remission
Partial Remission 2 (2%)
17 (15%) 1 (6%)
4 (25%) 0 (0%)
8 (11%) 1 (5%)
3 (14%) 0 (0%)
2 (33%)
Duration of Response (months) d
Range 6.8+, 60.5+ 10.2+, 36.4+ 6.8+, 51.8+ 8.6+, 55.9+ 27.3+, 60.5+
a Determined with 12-week confirmation. Patients who received high-dose corticosteroids were considered eva luable for response.
b Median exposure to midostaurin was 11.5 (range: 0.3, 68.3) months.
c 31 patients were not assessable for MCL on central review, and 15 patients with unconfirmed AHN were classified as not having AHN.
d Median DOR was not reached in any subtype. Median follow up for DOR, among all responders, was 35.0 months.
Study 3
Study 3 was a single-arm, multicenter, open-label trial of 26 patients with advanced SM. RYDAPT was administered orally at 100 mg twice daily with food. The median age in this group was 64 years, 58% of patients were male and most were Caucasian (81%). Eligibility criteria were similar to Study 2. By Valent criteria per investigator assessment, of 17 patients with SM-AHN, 10 achieved a response (1 partial, 9 major) by 2 cycles that was sustained for at least 8 weeks. Patients who received concomitant corticosteroids were included. Of the 6 patients with MCL, 1 achieved partial response and 1 achieved major response. Median DOR for either group had not been reached, with DOR ranging from 3.4+ to 79.2+ months in patients with SM-AHN and 28.6+ to 32.1+ months in patients with MCL. The subtype of SM in the remaining 3 patients was unconfirmed.
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16 HOW SUPPLIED/STORAGE AND HANDLING
RYDAPT 25 mg capsules
Pale orange oblong soft capsule with red ink imprint ‘PKC NVR’; available in:
56 soft capsules………………………………………………………………………………………NDC 0078-0698-99
Contents: Each carton contains two inner packs, each with 28 capsules (7 blister cards with 4 capsules each)
112 soft capsules……………………………………………………………………………………..NDC 0078-0698-19
Contents: Each carton contains four inner packs,
