MILAN & MORRISTOWN, N.J.--(BUSINESS WIRE)--Newron Pharmaceuticals S.p.A. (“Newron”) (SIX: NWRN), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, today announced its support of this year’s Rare Disease Day®. Observed on February 28, Rare Disease Day helps raise awareness of rare diseases, and in turn improve access to treatments.
As part of its commitment to the rare disease patient community, Newron is partnering with the global Rett community to work on the first Burden of Disease (BOD) study. The study aims to deliver data and analytics to quantify the physical, emotional and financial challenges of Rett syndrome. The learnings accrued will identify and guide improved intervention programs and services designed to complement the Rett care pathway.
Newron, in collaboration with industry and university leaders, is advocating the development of a standardized evaluation method for the health economic assessment of orphan drugs. Most recently, for example, the Company hosted a roundtable discussion on the opportunity to develop quality clinical, economic and real-world evidence (RWE: data collected in routine clinical practice on how patients are being managed for a disease) on orphan drugs to inform policy decisions. The lack of an accepted method to assess the value of rare disease treatments can be an unsurmountable barrier to their development and/or access once approved.
“There are considerable unmet needs as only 5% of rare diseases have an approved treatment,” stated Dennis Dionne, Vice President Commercial Affairs, Newron. “For orphan drugs being developed, there are significant challenges in assessing the clinical and economic value. Understanding the real value of such drugs in terms of clinical benefit and improving quality-of-life (QoL) for patients and their families is crucial for payers to be able to assess their overall benefit against the cost of treatment. We are proud to engage in a meaningful way in Rare Disease Day, and to help advocate for improved methodologies and access.”
For more information
See
interviews with leading Rett-experts: http://www.newron.com/ENG/Default.aspx?PAG=215
STARS Study
Newron has also
initiated the Sarizotan Treatment of Apneas in Rett Syndrome (STARS)
study, a potentially pivotal clinical study to evaluate the efficacy,
safety and tolerability of sarizotan in patients with Rett syndrome
suffering from respiratory symptoms. Among the core symptoms of Rett,
breathing disturbances may affect the whole person body; they can have a
marked effect on biochemistry, influence emotions, circulation and
digestive function as well as musculoskeletal structures in the
respiratory process.
About Rare Disease Day®
Rare
Disease Day takes place on the last day of February each year, thus on
February 28, 2018. The main objective of Rare Disease Day is to raise
awareness amongst the general public and decision-makers about rare
diseases and their impact on patients' lives. The campaign targets
primarily the general public and also seeks to raise awareness amongst
policy makers, public authorities, industry representatives,
researchers, health professionals and anyone who has a genuine interest
in rare diseases. The campaign started as a European event and has
progressively become a world phenomenon, with the USA joining
in 2009 and participants in over 80
countries throughout the world in 2016.
For more
information, please visit: www.rarediseaseday.org.
About Rett Syndrome
Rett syndrome is a severe
neurodevelopmental disorder primarily affecting females, with an
estimated prevalence of one in 10,000 females. There are no approved
treatments available. Rett syndrome is characterized by a loss of
acquired fine and gross motor skills and the development of
neurological, cognitive and autonomic dysfunction, which leads to loss
of ability to conduct daily life activities, walk or communicate. Rett
syndrome also is associated with a reduced life expectancy.
Approximately 25 percent of the deaths in patients with Rett syndrome
are possibly related to multiple cardio-respiratory dysrhythmias that
result from brain stem immaturity and autonomic failure. More than 95
percent of these patients have a random mutation in the MeCP2 gene.
Episodes of apnea, hyperventilation and disordered breathing are found
in approximately 70 percent of patients with Rett syndrome at some stage
of their life.
For more information on Rett Syndrome, visit http://www.rettsyndrome.org.
About Newron Pharmaceuticals
Newron (SIX: NWRN) is a
biopharmaceutical company focused on the development of novel therapies
for patients with diseases of the central and peripheral nervous system.
The Company is headquartered in Bresso near Milan, Italy. Xadago®
(safinamide) has received marketing authorization for the treatment of
Parkinson’s disease in the European Union, Switzerland and the USA, and
is commercialized by Newron’s Partner Zambon. US WorldMeds holds the
commercialization rights in the USA. Meiji Seika has the rights to
develop and commercialize the compound in Japan and other key Asian
territories. In addition to Xadago® for Parkinson’s disease, Newron has
a strong pipeline of promising treatments for rare disease patients at
various stages of clinical development, including sarizotan for patients
with Rett syndrome and ralfinamide for patients with specific rare pain
indications. Newron is also developing Evenamide as the potential first
add-on therapy for the treatment of patients with positive symptoms of
schizophrenia. For more information, please visit: www.newron.com
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