Identified cases of agranulocytosis, possibly drug-related, in some
cases associated with sepsis and death
Increased blood cell count monitoring to weekly in ongoing Phase 3
- Discussions with FDA and DSMB ongoing
- Conference call today at 8:30 a.m.
Acorda Therapeutics, Inc. (NASDAQ:ACOR) today announced that it has
increased the frequency of blood cell count monitoring for participants
to weekly in its Phase 3 program of tozadenant for Parkinson’s disease.
The Company took this action in response to cases of agranulocytosis,
possibly drug-related, and in some cases associated with sepsis and
death. Agranulocytosis is the absence of white blood cells, which fight
infection. The Company also has paused new enrollment in the long-term
safety studies, pending further discussion with the independent Data
Safety Monitoring Board (DSMB) and the United States Food and Drug
The Phase 3 program includes an ongoing pivotal efficacy and safety
study (CL05) and two long-term safety studies (CL05 extension and CL06).
Including the previously conducted Phase 2b study, approximately 890
patients have been exposed to tozadenant and 234 have been exposed to
placebo. This corresponds to approximately 300 patient years of
tozadenant exposure and 75 patient-years of placebo. There have been
seven cases of sepsis, all in the tozadenant groups, five of which were
fatal. Four of the sepsis cases were associated with agranulocytosis,
two had no white blood cell counts available at the time of the event
and one had a high white blood cell count.
“We have taken these steps in the best interests of the safety of
patients in the tozadenant studies, which is our top priority,” said Ron
Cohen, M.D., Acorda’s President and CEO. “Contingent on further input
from the DSMB and FDA, we continue to expect to report efficacy and
safety results of the double-blind Phase 3 study in the first quarter of