We hope to have more clarity on the expected timeline after we meet with the FDA to discuss the complete response
letter," stated Paul Hamelin, President of Savient Pharmaceuticals.
In the February Complete Response
Letter, the FDA acknowledged objective and subjective efficacy at the beginning and end of treatment in elderly patients, and at the beginning of treatment for non-elderly adult patients, and the most recent Complete Response
Letter did not alter those conclusions," continued Pascoe.
The Company believes that data and information submitted to FDA in May 2009, which the Agency acknowledges were not reviewed before sending the Complete Response
letter, may address several of the deficiencies raised by FDA and may limit the need for some of the actions specified in the letter.
In previous studies involving MDS and CMML patients who received other chemotherapies, the rate of complete response
to treatment has been disappointing.
The primary endpoint of the trial was complete response
rate, which was determined by CT scan, the conventional methodology, and PET scan.
(CR) rate is the primary study endpoint.
The objective response rate (ORR) was 73 percent, including a 34 percent complete response
(CR) rate, 1 percent partial response (PR) rate, 24 percent marrow CR rate with or without other hematologic improvement (HI) responses, and a 14 percent HI rate.
After receiving therapy, of 27 evaluable patients, a partial response was seen in 13 patients and a complete response
in three patients.
One complete response
, confirmed to be PET-negative, was deemed by the independent radiologists' assessments to be qualified because the index lesion was in an area that had received prior radiation (a technical protocol violation).
Of the 75 currently evaluable patients, 31% (23 of 75) demonstrated a partial or complete response
Both clinical trials successfully met the primary efficacy endpoint of complete response
for the 0-24 hour time period following surgery for the selected dose of 0.
Among the 12 evaluable patients treated with a dose of 75 mg/m2, one patient demonstrated a complete response
and six patients showed partial responses, giving a response rate of 58 percent (95 percent Cl 27-84 percent).