Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader,
today announced it has reached an agreement with Gesetzliche
Krankenversicherung Spitzenverband (GKV-SV) which will result in
reimbursed access to Libmeldy® (atidarsagene autotemcel) for all
metachromatic leukodystrophy (MLD) patients in Germany who fall
within the scope of the European marketing authorization.
The agreement follows the completion of a
comprehensive assessment during which Libmeldy received the highest
possible therapeutic benefit rating for presymptomatic, early-onset
patients with MLD by the Federal Joint Committee, also known as
Gemeinsame Bundesausschuss (G-BA). To date, only four other
medicines have ever received this rating.
“Our agreement with GKV-SV for Libmeldy marks a
significant milestone for children with MLD, their caregivers and
the entire community,” said Braden Parker, chief commercial
officer. “Following similar agreements in the UK and Italy, Germany
is the third major European market to recognize the value of
Libmeldy commensurate with its clinical impact. We are encouraged
by the continued progress we are making across the region to help
eligible patients gain access to this therapy.”
As previously disclosed, one German patient has
already received Libmeldy at Tübingen University Hospital through
an interim reimbursed access arrangement.
Expansion of Patient Identification
EffortsTo help identify other eligible patients within the
treatment window, Orchard Therapeutics is supporting several
newborn screening and diagnostic testing initiatives in the
country, including a recently initiated prospective newborn
screening study in Hannover, Germany in collaboration with
ARCHIMEDlife. This study will expand to include Weiden,
Germany.
The company also partnered with ARCHIMEDlife to
support its leukodystrophy testing program, which provides
no-charge testing to patients in Germany to confirm the underlying
cause of symptoms suggestive of specific neurodegenerative
diseases, including MLD. This program also offers no-charge testing
to siblings of patients who receive a definitive disease
diagnosis.
About MLDMLD is a rare and
life-threatening inherited disease of the body’s metabolic system
occurring in approximately one in every 100,000 live births. 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 MLD in eligible early-onset 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 bone marrow harvest or peripheral blood mobilization and
apheresis, followed by myeloablative conditioning, which carry
their own risks. During the clinical studies, 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, 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 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 certain forward-looking statements about
Orchard’s strategy, future plans and prospects, which are made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. Such forward-looking statements may
be identified by words such as “anticipates,” “believes,”
“expects,” “plans,” “intends,” “projects,” and “future” or similar
expressions that are intended to identify forward-looking
statements. Except for statements of historical fact, information
contained herein constitutes forward-looking statements and may
include, but is not limited to, Orchard’s expectations regarding
the safety and efficacy of Libmeldy, Orchard’s ability to establish
the infrastructure necessary to enable the treatment of eligible
MLD patients, Orchard’s ability to identify eligible MLD patients,
and the adequacy of Orchard’s supply chain and ability to
commercialize Libmeldy. 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 Libmeldy
will not be successfully commercialized; the risk that any one or
more of Orchard’s product candidates, including OTL-200, will not
be approved, successfully developed or commercialized; the risk
that prior results, such as signals of safety, activity or
durability of effect, observed from preclinical studies or clinical
trials of Orchard’s product candidates will not be repeated or
continue in ongoing or future studies or trials involving its
product candidates; the risk that the market opportunity for
Libmeldy or its product candidates may be lower than estimated; and
the severity of the impact of the COVID-19 pandemic on Orchard’s
business, including on preclinical and clinical development, its
supply chain and commercial programs. Given these uncertainties,
the reader is advised not to place 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.
i Mahmood 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
Contacts
Investors
Renee Leck
Director, Investor Relations
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media
Benjamin Navon
Director, Corporate Communications
+1 857-248-9454
Benjamin.Navon@orchard-tx.com
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