Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia (FA)

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Agilis Biotherapeutics, LLC (Agilis),
a biotechnology company advancing innovative DNA therapeutics for rare
genetic diseases that affect the central nervous system (CNS), announced
today that the United States Food and Drug Administration (FDA) has
granted Orphan Drug Designation to Agilis’ gene therapy product
candidate, AGIL-FA, being developed for the treatment of Friedreich’s
ataxia (FA), an inherited degenerative neuromuscular disorder resulting
in loss of motor coordination and strength, hearing, vision, speech and
often premature death. Agilis is the first company to receive orphan
designation from the FDA for a gene therapy to treat FA and it is the
fourth time this year that Agilis has been granted orphan designation in
the US and Europe for its gene therapies. The Company’s gene therapies
for AADC deficiency and Angelman syndrome have previously received
orphan status.
“Having the first gene therapy product candidate to receive orphan drug
designation from the FDA for the treatment of FA is an honor,” said Mark
Pykett, President and CEO of Agilis. “The orphan designation is another
step on our path to bringing this important new therapy to patients who
currently lack treatment options.” AGIL-FA is a gene-therapy product
consisting of a unique gene construct developed in partnership with
Intrexon Corporation (NYSE: XON) delivered with adeno-associated virus
technology.
“We are extremely pleased to receive this landmark designation, as we
move our FA development program forward,” said Christopher Silber, MD,
Agilis Chief Medical Officer. “With each of our IND and clinical stage
pipeline candidates having now received Orphan Designation, this
achievement highlights our efforts to advance innovative therapeutics
for patients with rare genetic diseases affecting the CNS.”
Friedreich’s ataxia (FA) is a rare and life-shortening neurodegenerative
disease caused by a defect in the FXN gene that reduces production of
the frataxin protein. Agilis’ FA gene therapy program is focused on
delivering corrective DNA to specific CNS cells to restore frataxin
protein levels. Agilis has worked closely with the Friedreich’s Ataxia
Research Alliance (FARA) to focus the development program on patient
needs. “FARA is delighted to continue our support of Agilis and their
innovative approach to the treatment of FA,” said Jennifer Farmer, MS,
Executive Director of FARA. “We look forward to continuing our
partnership to advance this important potential therapy, as well as
supporting research to identify biomarkers and clinical outcome
measures, which will advance the development of the product candidate
into clinical trials.”
FDA Orphan Drug Designation is evaluated for drugs from all classes
(e.g. small molecules, proteins, and gene or cell therapies) that are
intended for the treatment of rare diseases, defined as diseases
affecting fewer than 200,000 people in the United States. The
designation provides sponsors with development and commercial
incentives, including seven years of market exclusivity in the US,
prioritized consultation by FDA on clinical studies, and certain
exemptions from or reductions in regulatory fees.
About Friedreich’s ataxia
Friedreich’s ataxia (FA) is an inherited neuromuscular disorder most
commonly caused by a single genetic defect in the FXN gene that leads to
reduced production of frataxin, a mitochondrial protein that is
important for cellular metabolism and energy production. FA results in a
physically debilitating, life-shortening condition and is the most
common hereditary ataxia, with an estimated 5,000 to 10,000 patients in
the US (i.e., one in every 50,000 people). Both male and female children
can inherit the disorder. Symptoms of FA include progressive loss of
coordination and muscle strength, which lead to the full-time use of a
wheelchair; scoliosis (which often requires surgical intervention);
diabetes mellitus; hearing and vision impairment; serious heart
conditions; and premature death. Current FA therapies are primarily
focused on symptomatic relief, and there are no FDA-approved drugs to
treat the cause of FA. Visit www.curefa.org
for more information.
About Agilis Biotherapeutics
Agilis is advancing innovative gene therapies designed to provide
long-term efficacy for patients with debilitating, often fatal, rare
genetic diseases that affect the central nervous system. Our therapies
are engineered to impart sustainable clinical benefits, and potentially
a functional cure, by inducing persistent expression of a therapeutic
gene. The Company’s technology is aimed at the precise targeting and
restoration of a lost gene function, while avoiding unintended
off-target effects. Our integrated strategy increases the efficiency of
developing DNA therapeutics into safe, targeted gene therapies that
achieve long-term efficacy and enable patients to remain asymptomatic
without continuous invasive treatment. Agilis’ rare disease programs are
focused on gene therapy for AADC deficiency, Friedreich’s ataxia, and
Angelman syndrome, rare genetic diseases that include severe
neurological deficits and result in physically debilitating conditions.
We invite you to visit our website at: www.agilisbio.com.