April 29 (Reuters) - X4 Pharmaceuticals ( XFOR ) said on
Monday the U.S. FDA has approved its therapy to treat a rare
genetic immunodeficiency disease in patients 12 years of age and
older, sending its shares up 16% before the bell.
X4's mavorixafor, which will be sold under the brand name
Xolremdi, is the first therapy to get U.S. approval specifically
for the treatment of WHIM syndrome.
WHIM is characterized by disorders in which the body's
immune system does not function properly. It is an acronym for
warts, hypogammaglobulinemia or low antibody levels, infections,
and myelokathexis or low white blood cell count, which are all
symptoms of the disorder.
The decision is based on a late-stage study where the
therapy showed 60% reduction in infection rate compared to
placebo when tested in patients with WHIM syndrome.
Less than 1,000 people in the United States have WHIM
syndrome, according to government data.