CLINICAL NEWS
16
Latest & Greatest
New Indication for
the JAK Inhibitor
Ruxolitinib
Ruxolitinib, a JAK1 and JAK2 inhibitor
approved for the treatment of intermediate or high-risk myelofibrosis, is now
approved by the FDA for the treatment
of polycythemia vera. This new indication is intended to treat the condition in
patients who have an inadequate response
to or are intolerant of hydroxyurea –
another medicine often prescribed to
reduce the number of red blood cells and
platelets in the blood. The approval was
based on results from a clinical study
of 222 participants who had polycythemia vera for at least 24 weeks and
had undergone a phlebotomy procedure and exhibited an enlarged spleen.
Participants were randomly assigned to
receive ruxolitinib or the best available
therapy, as determined by the investigator on a participant-by-participant basis.
At 32 weeks of follow-up, 21 percent of
ruxolitinib-treated patients experienced a
reduction in the need for phlebotomy and
a reduction in spleen volume, compared
to 1 percent of participants who received
best available therapy. The most common
side effects of ruxolitinib were anemia
and thrombocytopenia; the most common non–blood-related side effects were
dizziness, constipation, and shingles.
The FDA reviewed ruxolitinib’s use for
polycythemia vera under the agency’s
priority review program, because, at
the time the application was submitted,
the drug demonstrated the potential to
be a significant improvement in safety
or effectiveness over available therapy
in the treatment of a serious condition.
The drug also received orphan product
designation because it is intended to treat
a rare disease. ●
Source: FDA press release, December 4, 2014