Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader,
today announced the Swiss Agency for Therapeutic Products
(Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a
hematopoietic stem cell (HSC) gene therapy, for the treatment of
early-onset metachromatic leukodystrophy (MLD).
MLD is a rare, fatal genetic disorder caused by
a mutation in the gene responsible for encoding the enzyme
arylsulfatase A (ARSA) leading to neurological damage and
developmental regression. In its most severe form, babies develop
normally but in late infancy start to rapidly lose the ability to
walk, talk and interact with the world around them. These children
may require 24-hour care, and the majority pass away within five
years of disease onset, creating an enormous burden on patients,
their families and health care systems.
Libmeldy aims to correct the underlying genetic
cause of MLD by inserting a working copy of the ARSA gene into the
genome of a patients’ own HSCs. The genetically modified cells are
infused back into the patient, where they can naturally migrate
across the blood-brain barrier into the central nervous system,
engraft, and express the functional enzyme. This approach has the
potential to persistently restore enzymatic function with a single
treatment. In clinical trials, treatment with Libmeldy resulted in
the preservation of cognitive development and maintenance of motor
function past ages at which untreated patients showed severe
cognitive and motor impairments. With more than a cumulative 250
patient-years of follow-up, Libmeldy was generally well-tolerated,
with no treatment-related serious adverse events or deaths. Most
adverse events were associated with busulfan conditioning or
background disease.
“Today’s approval of Libmeldy in Switzerland
opens up tremendous new possibilities for eligible children with
MLD who previously had no approved treatment options beyond
supportive care,” said Leslie Meltzer, Ph.D., chief medical officer
of Orchard Therapeutics. “We are encouraged that regulatory
authorities in the European region continue to recognize the
clinical impact of Libmeldy, and we remain committed to broadening
access to this important therapy in new geographies for the benefit
of patients and their families.”
Libmeldy was previously approved by the European
Commission (EC) and UK Medicines and Healthcare products Regulatory
Agency (MHRA). Libmeldy is known as OTL-200 in the U.S., where it
is currently under Priority Review by the Food and Drug
Administration (FDA) with a Prescription Drug User Fee Act (PDUFA)
goal date of March 18, 2024.
About MLDMLD is a rare and
life-threatening inherited disease of the body’s metabolic system
estimated to occur in approximately one in every 100,000 live
births based on existing literature. MLD is caused by a mutation in
the arylsulfatase-A (ARSA) gene that results in the
accumulation of sulfatides in the brain and other areas of the
body, including the liver, gallbladder, kidneys, and/or spleen.
Over time, the nervous system is damaged, leading to neurological
problems such as motor, behavioral and cognitive regression, severe
spasticity and seizures. Patients with MLD gradually lose the
ability to move, talk, swallow, eat and see. In its late infantile
form, mortality at five years from onset is estimated at 50 percent
and 44 percent at 10 years for juvenile patients.i
About Libmeldy /
OTL-200Libmeldy (atidarsagene autotemcel), also known as
OTL-200, has been approved by the European Commission for the
treatment of metachromatic leukodystrophy (MLD) in patients
characterized by biallelic mutations in the ARSA gene leading to a
reduction of the ARSA enzymatic activity in children with i) late
infantile or early juvenile forms, without clinical manifestations
of the disease, or ii) the early juvenile form, with early clinical
manifestations of the disease, who still have the ability to walk
independently and before the onset of cognitive decline. Libmeldy
is the first therapy approved for eligible patients with
early-onset MLD.
The most common adverse reaction attributed to
treatment with Libmeldy was the occurrence of anti-ARSA antibodies.
In addition to the risks associated with the gene therapy,
treatment with Libmeldy is preceded by other medical interventions,
namely peripheral blood mobilization and apheresis, followed by
myeloablative conditioning, which carry their own risks. During the
clinical studies of Libmeldy, the safety profiles of these
interventions were consistent with their known safety and
tolerability.
For more information about Libmeldy, please see
the Summary of Product Characteristics (SmPC) available on the EMA
website.
Libmeldy is approved in the European Union, UK,
Iceland, Switzerland, Liechtenstein and Norway. OTL-200 is an
investigational therapy in the U.S.
Libmeldy was developed in partnership with the
San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in
Milan, Italy.
About Orchard TherapeuticsAt
Orchard Therapeutics, our vision is to end the devastation caused
by genetic and other severe diseases. We aim to do this by
discovering, developing and commercializing new treatments that tap
into the curative potential of hematopoietic stem cell (HSC) gene
therapy. In this approach, a patient’s own blood stem cells are
genetically modified outside of the body and then reinserted, with
the goal of correcting the underlying cause of disease in a single
treatment.
In 2018, the company acquired GSK’s rare disease
gene therapy portfolio, which originated from a pioneering
collaboration between GSK and the San Raffaele Telethon Institute
for Gene Therapy in Milan, Italy. Today, Orchard is advancing a
pipeline spanning pre-clinical, clinical and commercial stage HSC
gene therapies designed to address serious diseases where the
burden is immense for patients, families and society and current
treatment options are limited or do not exist.
Orchard has its global headquarters
in London and U.S. headquarters in Boston. For
more information, please visit www.orchard-tx.com, and follow
us on X (Twitter) and LinkedIn.
Availability of Other Information About
OrchardInvestors and others should note that Orchard
communicates with its investors and the public using the company
website (www.orchard-tx.com), the investor relations website
(ir.orchard-tx.com), and on social media
(Twitter and LinkedIn), including but not limited to
investor presentations and investor fact sheets, U.S.
Securities and Exchange Commission filings, press releases,
public conference calls and webcasts. The information that Orchard
posts on these channels and websites could be deemed to be material
information. As a result, Orchard encourages investors, the media,
and others interested in Orchard to review the information that is
posted on these channels, including the investor relations website,
on a regular basis. This list of channels may be updated from time
to time on Orchard’s investor relations website and may include
additional social media channels. The contents of Orchard’s website
or these channels, or any other website that may be accessed from
its website or these channels, shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933.
Forward-looking StatementsThis
press release contains forward-looking statements, which are made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. All statements that are not
statements of historical facts are, or may be deemed to be,
forward-looking statements. These statements are neither promises
nor guarantees and are subject to a variety of risks and
uncertainties, many of which are beyond Orchard’s control, which
could cause actual results to differ materially from those
contemplated in these forward-looking statements. In particular,
these risks and uncertainties include, without limitation, the risk
that prior results, including signals of safety and efficacy, will
not be replicated or will not continue in ongoing or future studies
and the risk that long-term adverse safety findings may be
discovered. Given these uncertainties, the reader is advised not to
place any undue reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard
include those identified under the heading "Risk Factors" in
Orchard’s most recent annual or quarterly report filed with the
U.S. Securities and Exchange Commission (SEC), as well as
subsequent filings and reports filed with the SEC. The
forward-looking statements contained in this press release reflect
Orchard’s views as of the date hereof, and Orchard does not assume
and specifically disclaims any obligation to publicly update or
revise any forward-looking statements, whether as a result of new
information, future events or otherwise, except as may be required
by law.______________________________iMahmood et
al. Metachromatic Leukodystrophy: A Case of Triplets with the Late
Infantile Variant and a Systematic Review of the Literature.
Journal of Child Neurology 2010,
DOI: http://doi.org/10.1177/0883073809341669
Contact
Benjamin Navon
+1 857-248-9454
Benjamin.Navon@orchard-tx.com
Orchard Therapeutics (NASDAQ:ORTX)
過去 株価チャート
から 5 2024 まで 6 2024
Orchard Therapeutics (NASDAQ:ORTX)
過去 株価チャート
から 6 2023 まで 6 2024