Madrid, February 28 of 2019 – In conjunction with the World Day of Rare Diseases, Biogen Inc. The company pharmaceutical company celebrates its first year in the Spanish market with nusinersen, the first and only muscular therapy in the world of Atrophic Spinal Treatment (AME)1 Rare diseases, according to clinical experts, would be between 300 and 350 patients.2
In the last decade, Biogen has had a pharmaceutical company that has developed and developed therapeutic diseases that have been investigated and developed in the study of rare neuromuscular diseases. In this way, the company has been the first and only one to market SMA's treatment for patients' treatment (nusiners) worldwide.1 This landmark has changed the history of pathological history and improved prognosis for many patients. The head of the Ministry of Health, Social Services and Equality, a year ago, gave birth to a great step towards changing the history of the disease at the Atrophia Muscular Espinal Foundation (FundAME). Spain became one of the first countries to access this drug in pharmaceutical services.3
Today, nusiners has already been approved in 40 countries around the world, and 30 of them already make money.4 More than 6,600 people are treated worldwide with this drug.4
Nusiners results in SMA patients
This therapy has improved the survival and functioning of patients with SMA, the main problems that the disease presents.1 In this sense, Spain is based on the results of two pivotal studies: ENDEAR5 (AME home from the beginning) and CHERISH6 (AME postponed occurrence), with clinically meaningful efficacy and a good safety profile.1
In SMA's initial appearance (including ENDEAR study), 89 SHINE continued to be studied,7 It measured the safety and long-term effectiveness of Nusiners.7 The results showed additional improvements in the total and precise functions of the engines (for example, for controlling the head and seat), together with the general function of CHOP INTEND measured engines.7 The discovery of security was consistent with what was previously reported in the nusiners.7 These preliminary data increase the profile of the risk profile of nusiners in early illness patients and demonstrate that improvements in the engine may be improved without taking into account the age of the treatment.7
With regard to the CHERISH study,6 Nusinersen also showed that the delay in AME showed that patients had a clinically significant improvement in motor function compared to those who did not treat them.6 The drug has been progressively improved and patient movements are maintained over time.6
In addition, Biogen has other clinical trials with AME, such as EMBRACE8 and NURTURE.9 In the midst of the NURTURE trial in patients with presymptomatic diagnostic genetic diagnosis, the treatment was compatible with the development of adequate age development for the patient and the development of healthy patient motors.9 In this sense, 100% of the patients who participated in the study could sit without help, 88% managed to help and only 77% were able to walk. In addition, all the participants continued their analysis without proof of sustainability.10
Getting an adult handle with SMA and improved treatment
This neuromuscular disease can be severe in its severe forms, especially the SMA type 1 in childhood.11 According to specialists, this type of patient is usually not reached for more than two years, due to illness caused by motor and respiratory complications.
However, patients with a progressive disease of disease have percentages in order to reach adults.12 In this group of people, the disease causes many limitations when it is particularly important to carry out basic activities for walking and breathing, undermining the muscles.11 This reduces your expectation and quality of life.
At the beginning of this treatment, adult patients help stop or improve the progress of the disease.13 Additionally, the results obtained in clinical trials suggest that in the long run, life expectancy can be increased to dampen motor and respiratory problems caused by SMA.1
As explained above Marta ValenteBiogen Medical Director in Spain, "The start of the nurses has been an important step and has been a change in the SMA patient's disease, but there is still a lot to do. Initial detection is essential, but we can not forget about all those who forget about it, and this treatment can be beneficial. The challenge is to ensure that health professionals have up-to-date information on the treatment and ensure that decisions are made as much as possible, something that we are cooperating, sharing the same goal: improving the quality of life of the patients and their families, together with the commitment made by AMN to commit ourselves to rare diseases such as Progressive Supranukaride Paralysis, or Amyloctic Lateral Sclerosis, also works. Patients throughout the world also help to find hopeful hope. "14
What is muscular atrophy? 15,16,17
SMA is a rare disease in the spinal cord and cerebral cells, resulting in severe and progressive muscular atrophy. In the end, people with the most severe type of SMA lose motor function and perform basic difficult functions, such as swallowing or breathing.
SMN1 gene loss or mutation People with SMA do not produce enough brain neuron survival protein (NMS) enough to keep motor neurons. Almost equally identical gene (SMN2) is able to produce the same protein, but in smaller quantities. In patients with SMA, the severity of the disease is associated with the number of copies of the SMN2 gene and, therefore, in the ability of SMN functional proteins capable of producing.
SMA-type people who require intensive care, in most cases one or two copies, do not sit without help or have no respiratory support for more than two years. SMA types 2 and 3, in most cases with two to four specimens, generate a greater number of SMN proteins and therefore have the most severe forms of SMA, but at the same time they have a progressive nature and change it and condition the quality of life.
In Biogen, our mission is clear: we are pioneers in neuroscience. Biogen develops and develops innovative treatments for neurological and neurodegenerative diseases. Biogen was founded in 1978 by Bioeng, Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, and nowadays he has a remarkable drug. He has presented the first and only approved muscular atrophy treatment program for Alzheimer's and dementia, multiple sclerosis and neuroimmunology programs, movement disorders, neuromuscular disorders, acute neurology and neurocognitive disorders, pain and ophthalmology. Biogen produces and markets biologically advanced biological products.
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- Technical card in Nursiners. https://ec.europa.eu/health/documents/community-register/2017/20170530137918/anx_137918_en.pdf [Fecha de último acceso: febrero 2019]
- Therapeutic Positioning Report. https://www.aemps.gob.es/medicamentosUsoHumano/informesPublicos/docs/IPT-nusinersen-Spinraza-atrofia-muscular-spinal.pdf [Fecha de último acceso: febrero de 2019]
- Press release of the Ministry of Health. https://www.mscbs.gob.es/gl/gabinete/notasPrensa.do?id=4292 [Fecha de último acceso: febrero 2019]
- Biogen Inc (BIIB) Q4 2018 Earnings Transcript of the Congress Call. https://m.nasdaq.com/article/biogen-inc-biib-q4-2018-earnings-conference-call-transcript-cm1089241 [Fecha de último acceso: febrero 2019]
- Finkel RS, Mercury E, Darras BT, et al. Nusiners versus sham control under the atrophy of the initial spinal cord atrial child. N Engl J Med 2017; 377: 1723-32
- M Mercuri et al. Nusinersen versus Sham Control in Later-Onset-in Spinal Muscular Atrophy. N Engl J Med 2018; 378: 625-35
- With Castro, D A detailed report on the safety and efficacy of long-term treatment, when Nusiners begins in childhood, accelerating atrophy of the muscles (SMA): SHINE Study results. Presented at the 23th International Mus Tournament of the World Muscle Association. October 2 to 6 2018. Mendonza, Argentina. P.170
- Acsadi et al. Nusiners' Safety and Efficiency Infant / Infant Wound Atrophy of the Spinal Cord (SMA): Phase 2 EMBRACE Study's first part. _22nd World Congress of the World Muscle Association. 3-7 October 2017. Saint Malo, France. P.380
- Crawford TA et al; Nusiners' Early Childhood Treatment at the Early Bone Atrophy Atrophy (SMA) in a Presymptomatic Stage: Sustainable Efficacy and Safety Results from the NURTURE Study Phase 2. P 146 Child Neurology Recalling the 47th Annual Meeting of the Society I October 15-18, 2018 I Chicago, IL
- Swoboda KJ and all. Treatments that begin in childhood at Nusiners at the muscular atrophy of the presymptomatic phase of the spine (SMA): NURTURE Examination Phase 2 efficacy and safety periods. Presented at the 23th International Mus Tournament of the World Muscle Association. October 2 to 6 2018. Mendonza, Argentina.
- Kolb, Stephen J., et al. "Natural history of early childhood muscular atrophy". 82.6 (2017) year of neurology: 883-891
- Mercuri, Eugenio, et al. Models of the progression of diseases of type 2 and type 3: the effects of clinical trials ". Neuromuscular disorders 26.2 (2016): 126-131
- Walter M. et all; Nusiners treatment mature adult SMA type 3; P502 ICNMD Vienna 2018
- Full Biogen Q4 and 2018 (Economic Results and Business Update). http://investors.biogen.com/static-files/c0d4fbe2-d4f2-404b-be17-19e770386d6e [Fecha de último acceso: enero 2019]
- Faravelli I, et al. Nat Rev Neurol. 2015; 11: 351-9;
- Lunn MR, Wang CH. Lancet. 2008; 371: 2120-33;
- von Gontard A, Rudnik-Schoneborn S, Zerres K. Stress and the atrophy of children and adolescents' backbone arteries. Klin Padiatr. Jul 2012; 224 (4): 247-51.