Novartis announces NEJM publication of landmark PARADIGMS study demonstrating significant benefit of Gilenya® in children and adolescents with MS|
Novartis International AG /
Novartis announces NEJM publication of landmark PARADIGMS study demonstrating
significant benefit of Gilenya® in children and adolescents with MS
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The issuer is solely responsible for the content of this announcement.
* Treatment with Gilenya (fingolimod) substantially reduced the debilitating
impact of MS, with significant decreases in key measures of disease activity
vs. interferon beta-1a
* MS severely affects the everyday lives of children and adolescents with the
disease and carries a significant impact throughout their lifetime
* Gilenya, a leading oral therapy for relapsing MS, is the only treatment
approved by the US FDA for patients from 10 years of age through to
The digital press release with multimedia content can be accessed here:
Basel, September 12, 2018 - Novartis today announced that The New England
Journal of Medicine (NEJM) has published full results from the landmark Phase
III Gilenya(® )(fingolimod) PARADIGMS study, the first-ever controlled,
randomized study specifically designed for children and adolescents (aged 10 to
17) with relapsing forms of MS (RMS). Children and adolescents with MS
experience more frequent and often more severe relapses than those seen in
adults with MS. The negative effect of relapses on movement, memory and
thinking prevents patients from enjoying their childhood and adolescent years to
the full, often leaving them feeling isolated and anxious. PARADIGMS met the
primary endpoint of significantly reducing the rate of relapses when compared to
interferon beta-1a intramuscular injections over a period of up to two years.
The study also met several secondary clinical and imaging endpoints.
"I'd like to thank all the children who participated in the PARADIGMS study, and
their families, who have helped transform the outlook for pediatric patients
living with relapsing MS," said Dr. Tanuja Chitnis, Principle Investigator for
PARADIGMS and Director of the Partners Pediatric Multiple Sclerosis Center,
Massachusetts General Hospital, Boston, US, and Scientist, Ann Romney Center,
Brigham and Women's Hospital, Boston, US. "These data, published today, will go
a long way in helping to advance knowledge and understanding amongst the MS
community of how to evaluate and treat pediatric patients with MS."
Results from PARADIGMS show that, compared to interferon beta-1a, Gilenya:
* Significantly reduced relapse rates by 82% (p<0.001) and delayed the time to
first relapse; an estimated 85.7% of patients treated with Gilenya were
relapse-free at 24 months, versus 38.8% of patients treated with interferon
* Significantly reduced the number of new or newly enlarged T2 lesions up to
24 months by 53% (p<0.001). Also, it significantly reduced the average
number of gadolinium enhancing T1 (Gd+) lesions per scan at 24 months by
66.0% (p<0.001). The number and volume of lesions are associated with
increased relapse rates and disability progression
* In additional analyses, significantly reduced the annualized rate of brain
volume loss (brain shrinkage) by 40%
The safety profile of Gilenya in this study was overall consistent with that
seen in previous clinical trials in adults.
"PARADIGMS exemplifies Novartis' commitment to reimagining care for young
patients with neurological conditions," said Danny Bar-Zohar, Global Head,
Neuroscience Development for Novartis. "It is pioneering in every sense of the
word, demonstrating the collaborative approach taken with all stakeholders and
disciplines to bring the understanding of the unique attributes of pediatric MS
to the next level. Our priority now is to continue discussions with worldwide
health authorities to bring Gilenya to young patients in need, as soon as
Gilenya is a well-established treatment for MS in the adult population, having
been used to treat more than 255,000 patients in both clinical trials and the
post-marketing setting, with approximately 566,000 years of patient
About the Phase III PARADIGMS study
The Phase III PARADIGMS study (NCT01892722) is a flexible duration (up to two
years), double-blind, randomized, multi-center study to evaluate the safety and
efficacy of oral Gilenya(®) (fingolimod) compared to interferon beta-1a in
children and adolescents with a confirmed diagnosis of multiple sclerosis (MS),
followed by a five-year open label extension phase. The study enrolled 215
children and adolescents with MS, 10 to less than 18 years of age with an
Expanded Disability Status Scale (EDSS) score between 0 and 5.5. Patients
were randomized to receive once-daily oral Gilenya (0.5 mg or 0.25 mg, dependent
on patients' body weight) or intramuscular interferon beta-1a once weekly.
The primary endpoint of the study was the frequency of relapses in patients
treated up to 24 months (annualized relapse rate). Secondary endpoints
include the number of new or newly enlarged T2 lesions, gadolinium-enhancing T1
lesions, safety and the pharmacokinetic properties of Gilenya, all measured
throughout the treatment period.
The Phase III PARADIGMS study was conducted in 80 centers in 25 countries, and
was designed in partnership with the US Food and Drug Administration, the
European Medicines Agency and the International Pediatric Multiple Sclerosis
About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic disorder of the central nervous system
(CNS) that disrupts the normal functioning of the brain, optic nerves and spinal
cord through inflammation and tissue loss. In adults, there are three types
of MS: relapsing-remitting MS (RRMS), secondary progressive MS (SPMS) and
primary progressive MS (PPMS). Approximately 85% of people with MS have RRMS,
where the immune system attacks healthy tissue. In children and adolescents,
RRMS accounts for nearly all cases (approximately 98 percent).
The evolution of MS results in an increasing loss of both physical and cognitive
(e.g. memory) function. This has a substantial negative impact on the lives of
the approximately 2.3 million people worldwide affected by MS, of which between
three and five percent are estimated to be children or adolescents,.
About Gilenya (fingolimod)
Gilenya(®) (fingolimod) is an oral disease-modifying therapy (DMT) that is
highly efficacious at controlling disease activity in relapsing multiple
sclerosis (RMS). Gilenya has a reversible lymphocyte redistribution effect
targeting both focal and diffuse central nervous system (CNS) damage caused by
MS,. Long-term clinical trial and real-world evidence and experience has
shown Gilenya treatment to be convenient for individuals to incorporate into
everyday life, leading to high treatment satisfaction, long-term persistence,
and ultimately, improved long-term outcomes for people with RMS,.
Gilenya impacts four key measures of RMS disease activity: relapses, MRI
lesions, brain shrinkage (brain volume loss) and disability
progression,. Its effectiveness on all of these measures has been
consistently shown in multiple controlled clinical studies and in the real-world
setting. Studies have shown its safety and high efficacy to be sustained over
the long term, demonstrating that switching to Gilenya treatment as early in the
disease course as possible can be beneficial in helping to preserve individuals'
Gilenya is approved in the US for the first-line treatment of relapsing forms of
MS in adults, and children and adolescents ages 10 to less than 18 years of
age. In the EU, Gilenya is approved for adult patients with highly-active
relapsing-remitting MS (RRMS) defined as either high disease activity despite
treatment with at least one DMT, or rapidly-evolving severe RRMS. Gilenya is
currently under review with the European Medicines Agency as a treatment for
children and adolescents with MS.
About Novartis in Multiple Sclerosis
Alongside Gilenya(®) (fingolimod, a modulator of the S1P receptor subtypes
1,3,4 and 5), Novartis' multiple sclerosis (MS) portfolio includes Extavia(®)
(interferon beta-1b for subcutaneous injection) which is approved in the US for
the treatment of relapsing forms of MS. In Europe, Extavia is approved to treat
people with relapsing-remitting MS, secondary progressive MS (SPMS) with active
disease and people who have had a single clinical event suggestive of MS.
Investigational compounds include siponimod (BAF312, a selective modulator of
the S1P receptor subtypes 1 and 5), for SPMS, and ofatumumab (OMB157), a fully
human monoclonal antibody in development for relapsing MS. Ofatumumab targets
CD20, and is currently being investigated in two Phase III pivotal studies.
In the US, the Sandoz Division of Novartis markets Glatopa(®) (glatiramer
acetate injection) 20 mg/mL and 40 mg/mL, generic versions of Teva's
*Copaxone(®) is a registered trademark of Teva Pharmaceutical Industries Ltd.
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