By James George, MD
2008-05-01
Dr. George attended this meeting as a consultant for Amgen and
has also been an investigator for the clinical trials related to the
development of romiplostim.
On March 12, 2008, the FDA's Oncology Drug Advisory Committee (ODAC)
unanimously recommended approval of the first of a new class of
treatments for ITP ― romiplostim ― developed by Amgen. These agents
stimulate platelet production by mimicking the effect of
thrombopoietin. There was no issue about efficacy; platelet counts are
increased to safe levels even in patients who are refractory to many
previous treatments, including splenectomy. However, there was concern
about safety. In studies of ITP, several patients developed an increase
of marrow reticulin; this appeared to be reversible with
discontinuation of romiplostim. Data were also presented from a
preliminary, non-randomized study in patients with myelodysplasia.
Several patients developed an increase of myeloblasts that also
appeared to be reversible with discontinuation of romiplostim.
Therefore, the FDA had recommended that romiplostim be approved with a
strict risk-management assessment program that would restrict off-label
use. The preliminary outline of the plan includes direct shipment of
romiplostim to physicians for each patient, with monitoring of the
patient's course by a case manager. With current concerns that the FDA
has not adequately supervised new drugs following approval, this type
of restricted access may become more common.
back to top
