BIOGEN Optimistic for Rapid and Broad Patient Access Following Invitation to Submit SPINRAZA(R) (nusinersen) to National Institute for Health and Care Excellence (NICE) for Single Technology Appraisal (STA)
MAIDENHEAD, England, January 22, 2018 /PRNewswire/ --
Managed Access Agreement to be developed in parallel to urgently provide effective new treatment to patients in England
The National Institute for Health and Care Excellence (NICE) has formally invited Biogen to submit SPINRAZA (nusinersen), the first and only disease-modifying treatment for 5q spinal muscular atrophy (SMA), for assessment via the Single Technology Appraisal (STA) route. Whilst on its own this choice of route could have signalled a setback for access, the openness demonstrated by NICE and NHS England to commence development of a Managed Access Agreement straight away has reassured the company that there is still a viable, long-term reimbursement path for this significant therapeutic advancement in England.
Terry O'Regan, Vice President and Managing Director of Biogen UK and Ireland, comments,
"We now have an opportunity for SMA patients in England & Wales who deserve access to nusinersen, a medicine which has the potential to address unmet medical need in a devastating disease. Whilst we have been disappointed by the delays in the NICE process so far, we have been encouraged by a recent meeting with NICE and NHS England, where the need to respond to specific STA and access challenges in appraising nusinersen was expressly acknowledged, and an immediate plan for a Managed Access Agreement that can work alongside the STA process was agreed. Now is the time for all stakeholders to work together to ensure as many patients as possible get rapid access to this life saving medicine."
Nusinersen received European marketing authorisation following accelerated assessment in May 2017, and since that time NICE has been reviewing the best route for the appraisal. So far, nusinersen is reimbursed in Italy and Sweden with ongoing negotiations taking place across Europe.
Due to the significant clinical improvements shown with nusinersen in clinical trials and the urgent need for a treatment where before there was none, Biogen opened one of the largest global expanded access programmes in rare disease (since Autumn 2016) as an interim measure for those with infantile-onset SMA (consistent with type 1). To date, all eligible children in the UK have been treated with nusinersen following a Biogen / NHS collaboration in which it was agreed that Biogen would provide the medicine free of charge, and the NHS would devise an urgent policy to administer the treatment.
What happens now?
Biogen will submit to NICE in March 2018 (in line with the submission request date), and the NICE appraisal committee will outline their initial recommendation in June 2018 - at this point, feedback will be invited from relevant stakeholders including clinicians and patient representatives. NICE then provides its recommendation on whether routine funding can be made available to patients in England via the NHS, currently scheduled for November 2018. The STA will consider nusinersen for all appropriate patients who may benefit, which extends beyond those affected by infantile-onset SMA (consistent with type 1).
The Biogen expanded access programme remains open to all eligible children at the current time, however cannot be a replacement for health services, and urgent funding is needed in order to make nusinersen available long-term to the whole patient population who may benefit.
What is SMA?
SMA is an autosomal recessive neuromuscular, serious, debilitating, and life-threatening rare disease - it is the leading genetic cause of death in infants. SMA is progressive, and patients often decline over time affecting their ability to move (i.e. sit or stand), swallow and breathe. At any one time, it is thought that there are between 650 and 1,300 children and adults in the UK living with SMA. Children in the UK with the most severe form of SMA rarely live to see their second birthday.
About SPINRAZA® (nusinersen)
SPINRAZA is being developed globally for the treatment of 5q SMA.
SPINRAZA is an antisense oligonucleotide (ASO), using Ionis' proprietary antisense technology that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein. ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, SPINRAZA has the potential to increase the amount of full-length SMN protein in individuals with SMA.
SPINRAZA must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord, where motor neurons degenerate in individuals with SMA due to insufficient levels of SMN protein.
Following the assessment of nusinersen in patients affected by SMA, results from the ENDEAR end of study analysis indicate that some infants achieved motor milestones including full head control, ability to roll, sit, and stand. Additionally, infants treated with SPINRAZA (nusinersen) demonstrated a statistically significant reduction in the risk of death or permanent ventilation.
SPINRAZA demonstrated a favourable benefit-risk profile. The most common adverse events were headache vomiting and back pain, considered related to the lumbar puncture procedure. The incidence and severity of these events were consistent with events expected to occur with lumbar puncture. Thrombocytopenia and coagulation abnormalities, including acute severe thrombocytopenia, have been observed after administration of some ASOs. Renal toxicity has been observed after administration of some ASOs. The primary route of elimination is expected via urinary excretion of nusinersen and its metabolites.
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases. Founded in 1978 as one of the world's first global biotechnology companies by Charles Weissman, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, today Biogen has the leading portfolio of medicines to treat multiple sclerosis; has introduced the first and only approved treatment for spinal muscular atrophy; and is focused on advancing neuroscience research programmes in Alzheimer's disease and dementia, neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and acute neurology. Biogen also manufactures and commercialises biosimilars of advanced biologics. To learn more, please visit www.biogen.uk.com .
- Committee for Medicinal Products for Human Use (CHMP), Assessment report - Spinraza, International non-proprietary name: nusinersen, 21 April 2017. Procedure No. EMEA/H/C/004312/0000.
- NHS Choices. Spinal Muscular Atrophy. Available at: www.nhs.uk/conditions/spinal-muscular-atrophy-sma/. Last accessed: January 2018.
- The SMA Trust, About SMA. Available at: www.smatrust.org/what-is-sma/about-sma/. Last accessed: January 2018.
- Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010;24(15):16344-44.
- Evers MM, Toonen LJ, van Roon-Mom WM. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev. 2015;87:90-103.
- Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.
- SPINRAZA Summary of Product Characteristics (SmPC). November 2017.
Date of Preparation: January 2018
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