TIDMGSK
RNS Number : 5861Z
GlaxoSmithKline PLC
27 May 2016
Issued: Friday 27 May London UK - LSE Announcement
Strimvelis(TM) receives European marketing authorisation to
treat very rare disease, ADA-SCID
GSK, Fondazione Telethon and Ospedale San Raffaele gain approval
to provide life-saving gene therapy to patients
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and
Ospedale San Raffaele (OSR) today announced that the European
Commission has approved Strimvelis, the first ex-vivo stem cell
gene therapy to treat patients with a very rare disease called
ADA-SCID (Severe Combined Immunodeficiency due to Adenosine
Deaminase deficiency). A child born with ADA-SCID does not have a
healthy, fully-functioning immune system and as a consequence, is
unable to fight off everyday infections. Strimvelis (autologous
CD34+ cells transduced to express ADA) is the first corrective gene
therapy for children to be awarded regulatory approval anywhere in
the world. It is indicated for the treatment of patients with
ADA-SCID for whom no suitable human leukocyte antigen (HLA)-matched
related stem cell donor is available.
ADA-SCID affects an estimated 15 children per year in Europe and
following today's approval, patients with the condition who are
referred for treatment will be able to receive the gene therapy at
Ospedale San Raffaele in Milan.
Martin Andrews, Head of the Rare Disease Unit, GSK said:
"Today's approval is the result of many years' work with our
collaborators in Milan and is the next step towards bringing
life-changing treatment to patients with ADA-SCID and their
families. This is the start of a new chapter in the treatment of
rare genetic diseases and we hope that this therapeutic approach
could also be used to help patients with other rare diseases in the
future."
The marketing authorisation decision was based on data collected
from 18 children treated with Strimvelis. A 100% survival rate at
three years post-treatment with Strimvelis was observed for all
children in the pivotal study (n=12) and every child receiving the
treatment who contributed to the marketing authorisation data
package is alive today (n=18), with a median follow-up duration of
approximately seven years. Full results of the analysis have
recently been published in BLOOD(1) .
Professor Alessandro Aiuti, Clinical Research Coordinator at San
Raffaele Telethon Institute for Gene Therapy (SR-Tiget), a joint
research collaboration between OSR and Telethon, said: "We are
delighted with today's news, which marks the culmination of more
than 20 years of research and development at Tiget. This
innovative, individualised treatment approach uses a patient's own
gene modified stem cells to correct the root cause of the disease.
It has been gratifying for all of us to see patients affected by
this severe immune deficiency growing over the years, being able to
play with other children and going to school. Working alongside
GSK, we can now make Strimvelis available to ADA-SCID patients and
transform the lives of children who so desperately need it."
Nicola Bedin, CEO of Ospedale San Raffaele, said: "This great
achievement would not have been possible without the effective
collaboration between OSR, Telethon and GSK, which has brought
together years of scientific research, first-class medical practice
and expertise in product development. Going forward we hope to
build on our shared mission to develop and deliver more much-needed
new medicines to patients with rare diseases."
Francesca Pasinelli, General Manager of Fondazione Telethon,
said: "This is a memorable day, not only for us, but overall for
the people we work for: with Strimvelis we can keep our promise to
patients. We can say that we have pioneered a model whereby the
charity organisation acts not only as a funding agency, but plays a
primary role in managing the development of research to ensure that
each step of the process leads to the ultimate goal, which is to
provide accessible therapy to patients."
About ADA-SCID
ADA-SCID is a very rare disorder caused by a faulty gene
inherited from both parents. This faulty gene stops the production
of an essential protein called adenosine deaminase (ADA), which is
required for the production of lymphocytes (a type of white blood
cell). Children born with ADA-SCID do not develop a healthy immune
system so cannot fight off everyday infections, which results in
severe and life-threatening illness. Without prompt treatment, the
disorder often proves fatal within the child's first year of life.
ADA-SCID is estimated to occur in approximately 15 patients per
year in Europe.
About Strimvelis
Strimvelis is only administered once and does not rely on a
third-party donor, so there is no risk of immune incompatibility
causing rejection (graft versus host disease), which is a common
side effect of bone marrow transplant treatment. With Strimvelis,
the patient's own bone marrow cells are removed, and a vector is
used to insert a normal copy of the ADA gene into the cells. This
step is known as transduction. The gene-corrected cells are then
re-introduced to the patient via an intravenous infusion, after
which some of the cells home back to the bone marrow. In order to
improve the engraftment of the gene-modified cells in the patient's
bone marrow, patients are also pre-treated with low dose
chemotherapy.
Within the primary data package which formed the basis of
marketing authorisation, a 100% survival rate at 3 years
post-treatment with Strimvelis (primary endpoint) was observed for
all 12 children in the pivotal study, with 92% having
intervention-free survival (i.e. did not require enzyme replacement
therapy for a period of >3 months post-treatment or
hematopoietic stem cell transplantation). All 18 children treated
with Strimvelis who contributed data to the marketing authorisation
application are alive today with a median follow-up duration of
approximately 7 years, with the first of these having received this
gene therapy over 13 years ago. Intervention-free survival within
the evaluable population (n=17) was 82%.
Overall the safety findings are in line with those expected in
children with ADA-SCID who have undergone treatment with low-dose
chemotherapy and who are undergoing immune recovery. A significant
reduction in severe infections has been documented and no leukaemic
events have been observed to date.
About the GSK / Telethon / OSR collaboration
The gene therapy for the treatment of ADA-SCID was originally
developed in Milan by Ospedale San Raffaele (OSR) and Fondazione
Telethon (Telethon), through their joint San Raffaele Telethon
Institute for Gene Therapy (SR-Tiget) and was taken forward by GSK
through a strategic collaboration formed in 2010 between GSK, OSR
and Telethon. Within the collaboration GSK, working with the
biotechnology company MolMed S.p.A, has applied its expertise in
product development to optimise, standardise and characterise a
manufacturing process that was previously only suitable for
clinical trials into one that has been demonstrated to be robust
and suitable for commercial supply.
Important Safety Information for Strimvelis in the European
Union
Overall the safety findings in the study were in line with those
expected in children with ADA-SCID who have undergone treatment
with low-dose chemotherapy and who are undergoing immune recovery.
Adverse events were reported for all 18 patients; the most
frequently reported being usual childhood infections including
upper respiratory tract infection, gastroenteritis and rhinitis. Of
the 39 serious adverse events which were reported post-GT, 62% were
infections, with the most common being device-related infections,
for example, from the central venous catheter (CVC) used during the
treatment. Five patients reported SAEs due to CVC infection, three
due to gastroenteritis and three due to pneumonia. A number of
patients also experienced neurologic, CNS or hearing impairments
which continued post-GT. No leukaemic events have been observed to
date.
GSK - one of the world's leading research-based pharmaceutical
and healthcare companies - is committed to improving the quality of
human life by enabling people to do more, feel better and live
longer. For further information please visit www.gsk.com.
Fondazione Telethon - Fondazione Telethon is a major biomedical
charity in Italy whose mission is to advance biomedical research
towards the cure of rare genetic diseases. Throughout its 26 years
of activity, the Telethon Foundation has invested over EUR450
million in funding over 2,500 projects to study 470 diseases,
involving more than 1,500 researchers. For further information,
visit www.telethon.it/en
Ospedale San Raffaele - Ospedale San Raffaele (OSR) is a
clinical-research-university hospital established in 1971 to
provide international-level specialised care for the most complex
and difficult health conditions. Since 2012 OSR is part of Gruppo
Ospedaliero San Donato, the leading hospital group in Italy. The
hospital is a multi-specialty centre with over 50 clinical
specialties and has over 1,300 beds. Research at OSR focuses on
integrating basic, translational and clinical activities to provide
the most advanced care to our patients. For further information,
visit: www.hsr.it .
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) -
Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene
Therapy (SR-Tiget) is a joint venture between the Ospedale San
Raffaele and Telethon. SR-Tiget was established in 1995 to perform
research on gene transfer and cell transplantation and translate
its results into clinical applications of gene and cell therapies
for different genetic diseases. For further information, visit
http://www.tiget.it/.
Strimvelis is a trade mark of the GSK group of companies.
GSK enquiries:
UK Media enquiries: Fiona McMillan +44 (0) 20 (London)
8047 5502
Claire Brough +44 (0) 20 (London)
8047 5502
US Media enquiries: Sarah Alspach +1 202 715 (Washington,
1048 DC)
Sarah Spencer +1 215 751 (Philadelphia)
3335
Analyst/Investor Ziba Shamsi +44 (0) 20 (London)
enquiries: 8047 5543
Tom Curry + 1 215 751 (Philadelphia)
5419
Gary Davies +44 (0) 20 (London)
8047 5503
James Dodwell +44 (0) 20 (London)
8047 2406
Jeff McLaughlin +1 215 751 (Philadelphia)
7002
Cautionary statement regarding forward-looking
statements
GSK cautions investors that any forward-looking
statements or projections made by GSK, including
those made in this announcement, are subject
to risks and uncertainties that may cause actual
results to differ materially from those projected.
Such factors include, but are not limited to,
those described under Item 3.D 'Risk factors'
in the company's Annual Report on Form 20-F for
2015.
References
1 Cicalese, MP et al. Update on the safety and efficacy of
retroviral gene therapy for immunodeficiency due to adenosine
deaminase deficiency. BLOOD. DOI 10.1182/blood-2016-01-688226
http://www.bloodjournal.org/content/early/2016/04/29/blood-2016-01-688226
Last accessed May 2016
Registered in England & Wales:
No. 3888792
Registered Office:
980 Great West Road
Brentford, Middlesex
TW8 9GS
This information is provided by RNS
The company news service from the London Stock Exchange
END
MSCZDLFLQEFZBBL
(END) Dow Jones Newswires
May 27, 2016 10:46 ET (14:46 GMT)
Gsk (LSE:GSK)
Historical Stock Chart
From Mar 2024 to Apr 2024
Gsk (LSE:GSK)
Historical Stock Chart
From Apr 2023 to Apr 2024