Orchard Therapeutics, a global gene therapy leader, today announced
the company has reached an agreement with the Beneluxa Initiative
on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to
Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell
(HSC) gene therapy, approved for the treatment of early-onset
metachromatic leukodystrophy (MLD). The agreement covers several
member countries, including Belgium, The Netherlands, and Ireland.
It follows similar arrangements made with reimbursement authorities
in the United Kingdom (UK), Italy, Germany, Sweden, Iceland,
Finland and Norway, and is a key component of the company’s
geographic expansion plans.
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 repaired 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.
“Following its approval, health technology
assessment authorities across Europe have continued to recognize
the impact of Libmeldy commensurate with its clinical, economic and
societal value,” said Robin Kenselaar, senior vice president, EMEA
at Orchard Therapeutics. “We are pleased to have reached an
agreement with the Beneluxa consortium that enables sustainable
access to treatment. The agreement came after the acceptance of
additional long-term data and marks a reversal of the outcome from
earlier negotiations with the consortium which ended without
reaching an arrangement. We remain committed to broadening access
to this important therapy in new geographies for the benefit of
eligible patients and their families.”
Libmeldy is 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.
Additional European Commercial Updates
Multiple eligible MLD patients are currently in
the treatment process and expected to receive Libmeldy in the
coming months. This includes the first patient treated at the
company’s qualified center in Utrecht, The Netherlands. Following
administration, patients will have been treated at all six of the
company’s initially qualified treatment centers in Europe.
Additional centers in Spain and Saudi Arabia are currently in the
final stages of qualification and are expected to be ready to treat
MLD patients with Libmeldy in the near future.
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
TherapeuticsOrchard Therapeutics, a Kyowa Kirin company,
is a global gene therapy leader focused on ending the devastation
caused by genetic and other severe diseases 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 with a single
treatment.
Founded in 2015, Orchard’s roots go back to some
of the first research and clinical developments involving HSC gene
therapy. Our team has played a central role in the evolution of
this technology from a promising scientific idea to a potentially
life-transforming reality. Today, Orchard is advancing a pipeline
of 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.
For more information, please
visit www.orchard-tx.com, and follow us on X (Twitter)
and LinkedIn.
_____________________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
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