Therapeutics, Inc., announced today that the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation to the company's
most advanced product candidate, HT-100
(delayed-release halofuginone), an orally available, small molecule drug
candidate intended to reduce fibrosis and inflammation and promote
healthy muscle regeneration in boys with DMD.
Fast track designation is granted by the FDA to facilitate the
development and expedite the review of new drugs that are intended to
treat serious or life-threatening conditions and that demonstrate the
potential to address unmet medical needs.
"We are pleased that the FDA supports our application for fast track
designation for HT-100 for the treatment of DMD. This, along with the
previously granted orphan drug designation for HT-100, is an important
regulatory milestone for the program," said Marc
B. Blaustein, CEO of Akashi Therapeutics. "We will continue to work
closely with the FDA as we advance HT-100 through clinical development
and the associated regulatory processes."
Akashi is currently treating patients with DMD in a phase 1b/2a
multi-center clinical program to evaluate HT-100 safety and tolerability
and assess trends in a range of exploratory biomarkers and efficacy
(delayed-release halofuginone) is an orally available, small molecule
drug candidate designed to reduce fibrosis and inflammation and promote
healthy muscle fiber regeneration in DMD patients. HT-100 has been
granted orphan designation for DMD in both the U.S. and EU, and fast
track designation in the U.S.
About Duchenne muscular dystrophy (DMD)
Affecting approximately 1 in 3,600 boys worldwide, DMD
is the most common of the muscular dystrophies and the most lethal
genetic disorder of childhood. It is caused by a genetic mutation that
renders boys unable to make functional dystrophin, a protein critical
for normal muscle function. Young men with DMD show progressive signs of
physical impairment as early as age three, lose the ability to walk in
their teens, and die of cardiac or respiratory failure in their late
twenties or early thirties.
About Akashi Therapeutics
Therapeutics is a clinical stage biopharmaceutical company whose
mission is to develop treatments for Duchenne
muscular dystrophy and other rare pediatric diseases. Akashi was
founded by leading patient organizations and biotechnology industry
veterans and is managed by a seasoned team of drug development experts
to impact a central problem in rare diseases: rapid therapy development.
Akashi is developing a pipeline
of therapies with the goal of transforming Duchenne from a 100%
fatal, aggressive muscle-wasting disease to a chronic, manageable
condition. For more information, please visit www.akashirx.com.
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