fda announcement in us today, page-16

i have not read it

the American journalist sent it to me

page 6

Issues with the submission

FDA would like to seek advice from the ODAC members on the following issues:

Issue #1: Clinical significance of response rate and duration of response

Study CML-202 was planned to enroll 100 patients. However, the NDA submission only included the efficacy data from 66 patients. The Applicant continued to enroll additional patients after the data cut-off for these 66 patients.

The indication sought for omacetaxine is for the treatment of CML patients with Bcr-Abl T315I mutation. However, there is no commercially available assay for the detection of this mutation and the Applicant has not submitted any data related to the assay methods to FDAs CDRH.

Thirty-five percent of patients had no confirmation of Bcr-Abl T315I mutation status by the central laboratory at the time of enrollment, a required study entry criterion.

The response rate observed in the efficacy study was low. For the chronic phase cohort of 40 patients, the MCyR rate was 15% (10% CCyR + 5% PCyR) with a median duration of response of 7.7 months. For the accelerated phase cohort of 16 patients, the MCyR rate was 6.3% and CHR rate was 31.3% with a median of duration of response of 22 weeks. There were no responders in the BP cohort.

ODAC advice is sought on the adequacy of the sample size and the clinical significance of the observed responses and their duration for the intended patient