The Quinze-Vingts Hospital & GenSight Biologics Announce a First Temporary Authorization for Use (ATU) for LUMEVOQ™ (GS010)...
December 09 2019 - 1:30AM
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The National Eye Hospital (CHNO) of the Quinze-Vingts in Paris
and GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN:
FR0013183985, PEA-PME eligible), a biopharma company focused on
discovering and developing innovative gene therapies for retinal
neurodegenerative diseases and central nervous system disorders,
today announced that the French Competent Authority, the National
Drug Safety Agency (Agence Nationale de Sécurité du Médicament or
ANSM), granted a named patient Temporary Authorization for Use
(“ATU nominative”) for LUMEVOQ™ (GS010) to the CHNO of the
Quinze-Vingts. Dr Catherine Vignal, who as the prescribing
physician originated the request, will be able to use LUMEVOQ™ to
treat a patient recently affected by Leber Hereditary Optic
Neuropathy (LHON). GenSight Biologics committed to provide the drug
for a bilateral injection.
“As we are preparing to file for Marketing Approval in Europe in
2020, this ATU testifies to the strong interest of the medical
community for LUMEVOQ™ and to the impact on patients,” said
Bernard Gilly, Co-founder and Chief Executive Officer of
GenSight. “We are of course willing to provide available products
if more requests are authorized.”
Leber Hereditary Optic Neuropathy (LHON) is a rare, maternally
inherited mitochondrial genetic disease, characterized by the
degeneration of retinal ganglion cells that results in precipitous
and irreversible vision loss typically leading to legal blindness.
The disease mainly affects adolescents and young adults. LHON is
associated with painless, dramatic and sudden loss of central
vision in the first eye, with the second eye becoming irreversibly
involved shortly after. 97% of patients have bilateral involvement
within one year of onset of vision loss, and in 25% of cases,
vision loss occurs in both eyes simultaneously. LHON causes the
blindness of an estimated 1,400 to 1,500 new patients each year in
the United States and Europe.
“France’s ATU program is a powerful means of providing LHON
patients with the ND4 mutation, with a therapeutic solution,
LUMEVOQ gene therapy,” said Dr. Catherine Vignal, Principal
Investigator for LUMEVOQ™ trials at the Department of Ophthalmology
at Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
(Department led by Dr José-Alain Sahel) and Head of the department
of Neuro-Ophthalmology at the Rothschild Foundation, Paris. “To me
as a clinician, it is important that I can now offer my patients a
treatment for their condition, in close collaboration with the
Regulatory Competent Authorities, while waiting for official
marketing authorization.”
The temporary authorization is the outcome of a close
partnership between physicians and pharmacists from the CHNO of the
Quinze-Vingts, the “Ouvrir les yeux” (Open the eyes) patient
advocacy group and GenSight Biologics, to the benefit of patients
affected by LHON.
“Access to treatment by gene therapy to people affected with
LHON is the result a decade of research at the Institut de la
Vision and a successful partnership with the teams at GenSight
Biologics,” noted Dr. José-Alain Sahel, Director of
the Institut de la Vision (Sorbonne-Université/Inserm/CNRS), Paris;
Chairman of the Department of Ophthalmology at Centre Hospitalier
National d'Ophtalmologie des XV-XX, Paris; Professor and Chairman
of the Department of Ophthalmology at University of Pittsburgh
School of Medicine and UPMC (University of Pittsburgh Medical
Center); and co-founder of GenSight.
“Our association is deeply involved in all the steps that can
improve the lives of patients with inherited neuropathy and their
families. Gene therapy aims to bring about an improvement in one of
the 3 main mutations of LHON,” said Maryse Roger, President
of the “Ouvrir les yeux” patient advocacy group. “We hope for the
rapid completion of the various ongoing research so that all
patients can benefit from treatment as soon as possible. In the
meantime, Ouvrir les yeux will continue to enable the voices of
patients brutally struck by blindness to be heard.”
In France, use of pharmaceutical products not yet approved with
a Marketing Authorization (AMM) and not recruiting for a clinical
trial requires first obtaining an ATU from the ANSM.
Named patient ATUs are granted by the ANSM under the following
conditions:
- The product is meant to treat, prevent or diagnose a severe or
rare disease,
- No other appropriate treatment is available in France,
- The product’s efficacy and safety are presumed in the state of
scientific knowledge,
- The ATU is requested by and remains under the responsibility of
the prescribing physician when the drug has the potential to
benefit the patient.
“The Quinze-Vingts Hospital, the world's oldest institution for
fighting blindness, fully supports the innovation strategy led by
the hospital's clinical teams and those of the Institut de la
Vision,” said Jean-François Ségovia, Director of the Centre
Hospitalier National d'Ophtalmologie (National Eye Hospital) des
Quinze-Vingts.
About the Centre hospitalier national d’ophtalmologie des
Quinze-Vingts (National Eye Hospital)
GenSight Biologics S.A. is a clinical-stage biopharma company
focused on discovering and developing innovative gene therapies for
retinal neurodegenerative diseases and central nervous system
disorders. GenSight Biologics’ pipeline leverages two core
technology platforms, the Mitochondrial Targeting Sequence (MTS)
and optogenetics, to help preserve or restore vision in patients
suffering from blinding retinal diseases. GenSight Biologics’ lead
product candidate, GS010, is in Phase III trials in Leber
Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease
that leads to irreversible blindness in teens and young adults.
Using its gene therapy-based approach, GenSight Biologics’ product
candidates are designed to be administered in a single treatment to
each eye by intravitreal injection to offer patients a sustainable
functional visual recovery.
About GenSight Biologics
GenSight Biologics S.A. is a clinical-stage biopharma company
focused on discovering and developing innovative gene therapies for
retinal neurodegenerative diseases and central nervous system
disorders. GenSight Biologics’ pipeline leverages two core
technology platforms, the Mitochondrial Targeting Sequence (MTS)
and optogenetics, to help preserve or restore vision in patients
suffering from blinding retinal diseases. GenSight Biologics’ lead
product candidate, GS010, is in Phase III trials in Leber
Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease
that leads to irreversible blindness in teens and young adults.
Using its gene therapy-based approach, GenSight Biologics’ product
candidates are designed to be administered in a single treatment to
each eye by intravitreal injection to offer patients a sustainable
functional visual recovery.
About GS010
GS010 targets Leber Hereditary
Optic Neuropathy (LHON) by leveraging a mitochondrial targeting
sequence (MTS) proprietary technology platform, arising from
research conducted at the Institut de la Vision in Paris, which,
when associated with the gene of interest, allows the platform to
specifically address defects inside the mitochondria using an AAV
vector (Adeno-Associated Virus). The gene of interest is
transferred into the cell to be expressed and produces the
functional protein, which will then be shuttled to the mitochondria
through specific nucleotidic sequences in order to restore the
missing or deficient mitochondrial function.
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version on businesswire.com: https://www.businesswire.com/news/home/20191208005050/en/
Quinze-Vingts Hospital Sylvie Renier General Affairs and
Communication srenier@15-20.fr +33 (0)1 40 02 11 01 GenSight
Biologics Thomas Gidoin Chief Financial Officer
tgidoin@gensight-biologics.com +33 (0)1 76 21 72 20
RooneyPartners Media Relations Marion Janic
mjanic@rooneyco.com +1-212-223-4017 Solebury Trout US
Investor Relations Chad Rubin crubin@troutgroup.com
+1-646-378-2947
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