Agilis Biotherapeutics Announces Orphan Designation Approval in Europe for the Treatment of Angelman Syndrome

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Agilis
Biotherapeutics, (Agilis), a biotechnology company advancing
innovative DNA therapeutics for rare genetic diseases that affect the
central nervous system (CNS), announced today that the European
Commission (EC) has granted Orphan Medicinal Product (OMP) designation
in the European Union (EU) to the Company’s gene therapy product
candidate, AGIL-AS. The EC’s approval follows a positive opinion in
March from the European Medicine Agency’s (EMA) Committee for Orphan
Medicinal Products (COMP). AGIL-AS is being developed for the treatment
of Angelman syndrome (AS), a neuro-genetic
disorder characterized by severe intellectual and developmental
disability. AGIL-AS is the first therapeutic to receive orphan
designation for Angelman syndrome in Europe.
The orphan designation provides 10 years of market exclusivity for the
product upon approval, and potential incentives for Agilis including
Protocol Assistance (scientific advice for orphan products), and reduced
filing and review fees with the EMA.
“Receiving orphan status from the EC, in conjunction with the previous
orphan drug designation from the US FDA, is another step on our path to
bringing this important new medicine to patients in need of an
effective, durable treatment,” said Mark Pykett, President and CEO of
Agilis. “The orphan designation in Europe provides important benefits
during development and commercialization, and represents important
progress as we seek to bring this novel treatment for AS to the market.”
Patricia Williams, Head of Regulatory Affairs at Agilis, said,
“Receiving orphan designation for Angelman syndrome in Europe,
represents an important regulatory milestone aimed at facilitating
development of novel treatments for this difficult to treat disease.”
AGIL-AS is an innovative gene therapy candidate being investigated to
treat AS by using an AAV vector to deliver a corrective UBE3A gene to
rescue neurological deficits in patients suffering from this disease. AS
is a rare genetic disorder caused by the deletion or mutation of the
UBE3A gene. UBE3A encodes the ubiquitin ligase E6-AP, a protein that
plays a critical role in the function of the central nervous system.
Characteristic features of AS include delayed development, intellectual
disability, severe speech impairment, seizures and ataxia. According to
The Foundation for Angelman Syndrome Therapeutics (FAST),
the disorder strikes an estimated one in 15,000 live births.
“Agilis’ receiving designation as an Orphan Medicinal Product is another
exciting milestone achieved for the Angelman syndrome community,” said
Paula Evans, Director of FAST. “We are thrilled with the news and are
looking forward to participating in clinical trials when the therapeutic
is available for evaluation.”
EC Orphan Designation is granted to drugs that are intended for the
treatment of life threatening or chronically debilitating rare diseases
where no therapeutic options either exist or are satisfactory. Rare
diseases are those defined as having a prevalence of less than five in
10,000 in Europe. The designation provides sponsors with development and
commercial incentives, including 10 years of market exclusivity,
prioritized consultation by EMA on the development of the drug,
including clinical studies, and certain exemptions from or reductions in
regulatory fees.
About Agilis Biotherapeutics, LLC
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 technologies are 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 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, Angelman syndrome, and Fragile X
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.
Safe Harbor Statement
Some of the statements made in this press release are forward-looking
statements. These forward-looking statements are based upon our current
expectations and projections about future events and generally relate to
our plans, objectives and expectations for the development of our
business. Although management believes that the plans and objectives
reflected in or suggested by these forward-looking statements are
reasonable, all forward-looking statements involve risks and
uncertainties and actual future results may be materially different from
the plans, objectives and expectations expressed in this press release.