FDA Orphan Drug Designation For Ketamine To
Amyotrophic Lateral Sclerosis
2 clinical study in the U.S.
2021 -- InvestorsHub NewsWire -- PharmaTher Holdings Ltd. (the
"Company" or "PharmaTher") (OTCQB: PHRRF) (CSE: PHRM), a clinical-stage psychedelics biotech
company, is pleased
that the U.S. Food and Drug Administration (FDA) has granted
in the treatment of
Amyotrophic Lateral Sclerosis (ALS),
also known as Lou Gehrig's disease.
Fabio Chianelli, Chief Executive Officer of PharmaTher, said,
orphan drug designation is a massive validation for ketamine as a
potential treatment for ALS,
and it allows us to confidently proceed
in evaluating ketamine in
a phase 2 clinical study in patients suffering
this life-threatening disease. This
designation not only expedites
clinical and product development plans,
but also validates our belief in the potential of ketamine as a
PharmaTher has an exclusive license agreement with The University
of Kansas (KU) for the development and commercialization of the
intellectual property of ketamine in the treatment of
The FDA evaluated the research data licensed from KU in the ODD
application that ultimately served as the foundation to allow the
FDA to grant the designation.
The Company is also working with clinicians in finalizing a phase 2
study protocol to evaluate ketamine for ALS in the
Ketamine has the potential to effectively increase the life
expectancy of those with ALS at any stage and slow the progressive
loss of muscle associated with poor outcomes of the disease.
University of Kansas Medical Center researchers and inventors of
the potential use of ketamine to treat ALS, Dr. Richard J. Barohn,
M.D., John A. Stanford, Ph.D., and Dr. Matthew Macaluso, D.O., have
made the promising discovery that ketamine can be administered as
an effective treatment for ALS.
In addition, unpublished
and patent-pending preclinical research has shown that the
administration of ketamine preserves muscle function in advancing
ALS and increases life expectancy when given in the early stages of
Ketamine works by blocking the action of the ionotropic glutamate
receptor, the NMDA receptor.
other inhibitors of NMDA receptor function, such as riluzole,
dampens NMDA receptor-related glutamate excitotoxicity.
Further, ketamine can lower D-serine concentrations intracellularly
and also partially activates dopamine receptors.
mechanisms of ketamine contribute in part to the drug's
neuroprotective effects, which may extend to the motor neurons
targeted in ALS.
ALS is a progressive neuromuscular disease with a life expectancy
of only two to six years after diagnosis.
Currently, there is no known cure for ALS.
ALS affects approximately 50,000 people in the U.S. and Europe,
with over 5,000 new cases diagnosed annually.
As ALS advances, upper and lower motor neurons die, causing the
brain to lose its ability to control muscle movement.
As a result,
ALS patients experience progressive loss of voluntary muscle action
as an effect of the disease, resulting in the inability to speak,
eat, move and, eventually, breathe.
The FDA approved only three pharmaceuticals for the treatment of
ALS: riluzole, edaravone, and Nuedexta (dextromethorphan HBr and
These drugs are effective against disease mechanisms of ALS but
fail to have measurable effects on attenuating disease progression
or improve survival.
Therefore, there is an imperative need for new pharmacological
therapies that can stop or slow the muscle decline associated with
ALS progression and extend the life expectancy of the ALS
The Orphan Drug Act grants special status to a drug or biological
product to treat a rare disease or condition upon request of a
This status is referred to as orphan designation (or sometimes
The FDA grants orphan status to products that treat rare diseases,
providing incentives to sponsors developing drugs or
The FDA defines rare diseases as those affecting fewer than 200,000
people in the United States at any given time.
Orphan drug designation would qualify
for certain benefits and incentives, including seven years of
marketing exclusivity if regulatory approval is ultimately received
for the designated indication, potential tax credits for certain
clinical drug testing costs, activities, eligibility for orphan
drug grants, and the waiver of the FDA New Drug Application filing
fee of approximately $2.4 million.
PharmaTher Holdings Ltd.
PharmaTher Holdings Ltd. (OTCQB: PHRRF) (CSE: PHRM) is a
clinical-stage psychedelics biotech company focused on the
research, development and commercialization of novel uses,
formulations and delivery methods of
such as ketamine,
to treat mental
neurological and pain disorders.
PharmaTher is currently
an FDA approved phase 2 clinical study with ketamine to treat
Parkinson's disease and is developing a novel microneedle patch for
the intradermal delivery of psychedelics.
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