Nusinersen Treatment Information leaflets- SMA UK
SMA UK have produced leaflets aimed to provide a summary for anyone affected by SMA Types 1, 2 or 3 and considering the possibility of treatment with nusinersen for their child, or themselves. They're intended to be used in your discussions with you / your child's medical team. Please refer to link below.
https://smauk.org.uk/treatment-information-leaflets
SMA UK have produced leaflets aimed to provide a summary for anyone affected by SMA Types 1, 2 or 3 and considering the possibility of treatment with nusinersen for their child, or themselves. They're intended to be used in your discussions with you / your child's medical team. Please refer to link below.
https://smauk.org.uk/treatment-information-leaflets
NICE / NHS England Announces Changes to the Nusinersen Managed Access Agreement (MAA)
17th July 2019
The MAA for nusinersen (Spinraza) will now include paediatric patients who have recently (in the previous 12 months) lost the ability to walk independently. It also commits to considering any significant evidence being made available by Biogen in relation to all with SMA Type 3 who are non-ambulant, that may impact on the eligibility criteria of the MAA. These are just two changes following many confidential discussions which we have been unable to report on until now. Read more on our website.
17th July 2019
The MAA for nusinersen (Spinraza) will now include paediatric patients who have recently (in the previous 12 months) lost the ability to walk independently. It also commits to considering any significant evidence being made available by Biogen in relation to all with SMA Type 3 who are non-ambulant, that may impact on the eligibility criteria of the MAA. These are just two changes following many confidential discussions which we have been unable to report on until now. Read more on our website.
Guide to the 2017 international standards of care for SMA
27 June 2019
TREAT-NMD have coordinated a project to develop an accessible version of 'Diagnosis and management of spinal muscular atrophy', that was published in two parts in Neuromuscular Disorders, 2017. It is called 'A Guide to the 2017 International Standards of Care for SMA' and was written to provide people with information about the type of medical care someone with SMA might receive or for which they could advocate.
The guide was developed by clinicians, allied healthcare professionals and patient representatives from SMAUK, Cure SMA, MDUK and SMA Europe. It has been rigorously reviewed by eight academics and eight people who either have SMA or care for someone who has SMA.
You can access it here on the TREAT-NMD website: https://treat-nmd.org/care-overview/2017-standards-of-care-for-spinal-muscular-atrophy-sma/the-guide-to-the-2017-international-standards-of-care-for-sma/
27 June 2019
TREAT-NMD have coordinated a project to develop an accessible version of 'Diagnosis and management of spinal muscular atrophy', that was published in two parts in Neuromuscular Disorders, 2017. It is called 'A Guide to the 2017 International Standards of Care for SMA' and was written to provide people with information about the type of medical care someone with SMA might receive or for which they could advocate.
The guide was developed by clinicians, allied healthcare professionals and patient representatives from SMAUK, Cure SMA, MDUK and SMA Europe. It has been rigorously reviewed by eight academics and eight people who either have SMA or care for someone who has SMA.
You can access it here on the TREAT-NMD website: https://treat-nmd.org/care-overview/2017-standards-of-care-for-spinal-muscular-atrophy-sma/the-guide-to-the-2017-international-standards-of-care-for-sma/
Patient Advocacy Groups & Clinicians write to NICE & NHSE about the Nusinersen MAA and the impact this has had on the SMA community
14 June 2019
Letter from SMA REACH Clinicians and Patient Advocacy Groups (PAGs)
This first letter has been led by Professor Francesco Muntoni with input from clinicians and PAGs.
We have been advised that an appeal will halt the final publication of NICE/NHSE guidance and therefore the progress of the roll out of the programme for all. This will add many months of delay to the possibility of anyone other than infants with SMA Type 1 receiving the treatment. To try to avoid this we are exploring all options to secure some changes to the MAA. In summary we have asked NICE and NHS England to consider the following:
Letter from the Patient Advocacy Groups (PAGs)
This second letter to NICE and NHS England raises concerns about the way decisions have been communicated and the impact of this on the SMA Community. It asks for this to be taken into consideration when NICE and NHSE are making their next decisions and communicating them. The PAGs want to make it clear that they want to be able to work with them positively and collaboratively on the next steps of the MAA.
READ THE LETTER HERE
14 June 2019
Letter from SMA REACH Clinicians and Patient Advocacy Groups (PAGs)
This first letter has been led by Professor Francesco Muntoni with input from clinicians and PAGs.
We have been advised that an appeal will halt the final publication of NICE/NHSE guidance and therefore the progress of the roll out of the programme for all. This will add many months of delay to the possibility of anyone other than infants with SMA Type 1 receiving the treatment. To try to avoid this we are exploring all options to secure some changes to the MAA. In summary we have asked NICE and NHS England to consider the following:
- Based on the strong clinical evidence available, amend the MAA criteria now to include access for paediatric patients with SMA Type 3 who have lost ambulation.
- As part of the MAA, introduce regular reviews of new evidence for groups of patients currently excluded from the MAA and a commitment to amend the MAA if there is clear evidence of the clinical benefit.
- Establishing an SMA REACH steering committee of clinicians and academics to ensure a uniform interpretation of the guidance and to discuss complex cases.
Letter from the Patient Advocacy Groups (PAGs)
This second letter to NICE and NHS England raises concerns about the way decisions have been communicated and the impact of this on the SMA Community. It asks for this to be taken into consideration when NICE and NHSE are making their next decisions and communicating them. The PAGs want to make it clear that they want to be able to work with them positively and collaboratively on the next steps of the MAA.
READ THE LETTER HERE
PATIENT ADVOCACY GROUPS AND CLINICIANS WRITE TO NICE & NHSE ABOUT THE NUSINERSEN MAA AND THE IMPACT THIS HAS HAD ON THE SMA COMMUNITY
NICE Announces Guidance on Nusinersen will be Published on 26th June
15 May 2019
NICE have now announced that their guidance on access to nusinersen will be published on 26th June - see announcement here.
This means that until then Biogen will fund currently untreated infants with SMA Type 1 via a “bridging solution”. Access will be subject to the programme start criteria – including, most importantly, patients must not be permanently ventilated and there must be full consultation between the family and the treating clinician. All patients already on the EAP will continue to have access to treatment.
After June 26th, those with SMA Types 2 and 3 will start to get access. This will be subject to the Managed Access Agreement (MAA) starting criteria which we understand will be included in the published guidance and as soon as services are able to cope with the patient load. We imagine this will depend on individual hospital resources and capacity. Clinicians will be doing all they can to make this happen but we are aware this is a huge additional workload and there will be a need for further understanding and patience during this time.
We will keep you posted as soon as we hear more.
15 May 2019
NICE have now announced that their guidance on access to nusinersen will be published on 26th June - see announcement here.
This means that until then Biogen will fund currently untreated infants with SMA Type 1 via a “bridging solution”. Access will be subject to the programme start criteria – including, most importantly, patients must not be permanently ventilated and there must be full consultation between the family and the treating clinician. All patients already on the EAP will continue to have access to treatment.
After June 26th, those with SMA Types 2 and 3 will start to get access. This will be subject to the Managed Access Agreement (MAA) starting criteria which we understand will be included in the published guidance and as soon as services are able to cope with the patient load. We imagine this will depend on individual hospital resources and capacity. Clinicians will be doing all they can to make this happen but we are aware this is a huge additional workload and there will be a need for further understanding and patience during this time.
We will keep you posted as soon as we hear more.
5/01/2017
FDA News Release
FDA approves first drug for spinal muscular atrophy
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm
FDA News Release
FDA approves first drug for spinal muscular atrophy
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm
21/12/2016
Update on Nusinersen Expanded Access Programme in the UK
SMA Support UK, Muscular Dystrophy UK and The SMA Trust report on progress towards an Expanded Access Programme of the ENDEAR trial of nusinersen which treats eligible infants with Spinal Muscular Atrophy Type 1.
Families may recall reading the last update in September 2016 which was released by the Principal Investigators from the two trial centres at Newcastle Neuromuscular Centre and Great Ormond Street Hospital (GOSH). You can read this here.
The ENDEAR study results haven’t yet been fully analysed and there is only limited knowledge about the efficacy of nusinersen across the full spectrum of infants and children with SMA Type 1. The latest report can be read here. However, results have been sufficiently positive for Biogen to work on opening an Expanded Access Programme (EAP).
An Expanded Access Programme (EAP) is designed to provide access to treatment for eligible infants with SMA Type 1 who didn’t participate in a clinical trial. The programme is aimed at providing access prior to any European Medicines Agency approval.
On Wednesday 14 December, representatives of the two Centres met with Muscular Dystrophy UK, SMA Support UK, The SMA Trust and Biogen. The questions and answers below cover the discussions about the progress so far and the next steps, and are being publicised by the three charities on their websites and through their social media channels.
All involved with the discussions understand how frustrating and distressing the process can be for families. We are all doing all we can to move things forward and will continue to update you as soon as more information is available.
Q. Have any decisions been made about which children will be on the EAP?
A. In countries with higher numbers of eligible infants, it is impossible to recruit all children at the same time. There is therefore ongoing discussion on how to prioritise the recruitment of children into the EAP. This discussion is held in collaboration with other countries in which similar issues are being faced; and with the local ethics committees at the sites involved. Factors that will be taken into account include the medical need based on the information available so far about the risks and benefits there have been to the children treated in the ENDEAR study. Therefore, this may involve prioritising babies likely to receive the greatest benefit from the treatment. Other criteria that are being considered include the distance from individual centres/ travel; the severity of the condition and the age of the patient.
Q. Where will the treatment be offered in the UK?
A. An EAP has opened in Belfast for those living in Northern Ireland as the number of infants currently eligible for treatment there are so small. GOSH and Newcastle are in active discussion as to the best way to open the EAP at their individual centres. A number of other UK centres have expressed interest in being involved and in the next few weeks there will be discussions with Biogen to see if this is possible. We will provide an update on this as soon as this information becomes available.
Q. When will the EAP actually start at Newcastle and GOSH?
A. At the moment it is not possible to give an exact date when the EAP will begin at Newcastle and GOSH. The EAP could only start there once the children originally recruited to the randomised placebo part of the ENDEAR study had been transitioned to the nusinersen treatment. This process has now been completed so the process of initiating the EAP at those two centres is starting now.
Q. How does a family get on to the programme?
A. Families who are resident in the UK may apply by contacting the coordinators in London: a.bellin@ucl.ac.uk and in Newcastle Anna.Peel1@newcastle.ac.uk Families living in Northern Ireland may contact Belfast Children’s Hospital on 028 9032 9241 though we understand there is no capacity to enrol more infants in Belfast at the moment. Families can also contact Biogen by email: Patientcenter@biogen.com to obtain more information.
Q. Can you tell us exactly what the treatment involves?
A. The drug needs to be administered using a lumbar puncture. In the first year there are 4 injections in the first 2 months, and a further 2 injections in the remaining 10 months. From the second year onwards it is anticipated children will require 3 lumbar punctures each year.
The lumbar punctures require special precautions and, especially in older children, will require either sedation or general anaesthetic. These are particularly complex procedures in children with SMA Type 1.
Q. Are there any possible side effects / problems with the treatment we should be aware of?
A. The information available so far appears to indicate no drug related adverse event. However, as the drug requires repeated lumbar punctures, common problems relating to this procedure, such as headaches, have been reported in several of the children studied.
Q. What if a child was on the EAP and became unwell at the time of one of their scheduled treatments – would they still be able to carry on with the programme?
A. The lumbar puncture is a complex procedure which can only be performed if children are well. If a child is too unwell, the lumbar puncture will need to be rescheduled.
Q. Who will pay for the drug and any other costs to do with the treatment?
A. The drug will be made freely available by Biogen. The cost of the assessment and administration of the drug will be covered by the NHS and the local hospitals.
Q. Would there be help with travel and accommodation costs if needed?
A. This will be the same as for any hospital appointment.
Q. We know Biogen has applied to National Institute for Clinical Excellence (NICE) to begin the process of applying for the treatment to be approved in England. How does this fit in with the EAP and who gets involved in this licensing decision?
A. Before NICE can formally review the drug for approval in England, a decision on the drug’s licence must be taken by the European Medicines Agency (EMA). The EMA’s process for assessing the drug has just begun, and it is expected a decision will be reached later in 2017. In the meantime, NICE is currently conducting what is called a ‘scoping’ process. This means it is gathering evidence about SMA and nusinersen, to help decide whether to review the drug should the EMA grant Biogen a licence. An initial meeting to consider this will take place in January 2017. Muscular Dystrophy UK and representatives from SMA Support UK and The SMA Trust will be there.
The EAP therefore provides the most immediate prospect of access to the drug for children meeting the criteria. However, in the long term it will be important to receive approval from the EMA and NICE.
Q. What are the processes in Scotland, Wales and Northern Ireland?
A. In Scotland, decisions on approval after an EMA licence is granted are taken by the Scottish Medicines Consortium (SMC). Wales and Northern Ireland also have separate processes – although often these two countries follow the decisions made by NICE.
Q. We have heard that the drug may soon be licenced in the US. Will that affect what happens in the UK?
A. No – the United States has a separate process for approving new treatments.
Q. Is there any other way families can get the treatment now – either privately or by going to another country?
A. No – the treatment can only currently be accessed via the EAP in the UK.
Q. What else is happening to help speed things up?
A. The EAP was discussed at Muscular Dystrophy UK’s Services Development Committee meeting in Parliament this month. MD UK has also highlighted the need for access to nusinersen with NHS England’s paediatric neurosciences clinical reference group (CRG). CRGs are groups of clinicians, commissioners, public health experts, patients and carers who use their specific knowledge and expertise to advise NHS England on the best ways that specialised services should be provided. Data will be presented to the group in January. At the next meeting in January, the group will also consider the multidisciplinary resources needed to support centres involved in the centres to deliver the treatment.
Q. What can families do to help?
A. It is important that decision makers understand SMA Type 1 and nusinersen. If you would like to help to raise awareness of the issue with your MP – so that they can raise the issue on your behalf – please contact Peter Sutton at MD UK on: p.sutton@musculardystrophyuk.org
If you have any other questions, families can contact Biogen by email at: Patientcenter@biogen.com
Physicians can contact: medinfo@biogen.com
SMA Support UK will continue to work with MD UK, The SMA Trust, NICE, clinicians and Biogen in order to move things forward. We will keep you informed of progress via our website and social media. You can also contact us on 01789 267520 or email: supportservices@smasupportuk.org.uk
The SMA Trust can be contacted on 01789 801155 or email: info@smatrust.org
Update on Nusinersen Expanded Access Programme in the UK
SMA Support UK, Muscular Dystrophy UK and The SMA Trust report on progress towards an Expanded Access Programme of the ENDEAR trial of nusinersen which treats eligible infants with Spinal Muscular Atrophy Type 1.
Families may recall reading the last update in September 2016 which was released by the Principal Investigators from the two trial centres at Newcastle Neuromuscular Centre and Great Ormond Street Hospital (GOSH). You can read this here.
The ENDEAR study results haven’t yet been fully analysed and there is only limited knowledge about the efficacy of nusinersen across the full spectrum of infants and children with SMA Type 1. The latest report can be read here. However, results have been sufficiently positive for Biogen to work on opening an Expanded Access Programme (EAP).
An Expanded Access Programme (EAP) is designed to provide access to treatment for eligible infants with SMA Type 1 who didn’t participate in a clinical trial. The programme is aimed at providing access prior to any European Medicines Agency approval.
On Wednesday 14 December, representatives of the two Centres met with Muscular Dystrophy UK, SMA Support UK, The SMA Trust and Biogen. The questions and answers below cover the discussions about the progress so far and the next steps, and are being publicised by the three charities on their websites and through their social media channels.
All involved with the discussions understand how frustrating and distressing the process can be for families. We are all doing all we can to move things forward and will continue to update you as soon as more information is available.
Q. Have any decisions been made about which children will be on the EAP?
A. In countries with higher numbers of eligible infants, it is impossible to recruit all children at the same time. There is therefore ongoing discussion on how to prioritise the recruitment of children into the EAP. This discussion is held in collaboration with other countries in which similar issues are being faced; and with the local ethics committees at the sites involved. Factors that will be taken into account include the medical need based on the information available so far about the risks and benefits there have been to the children treated in the ENDEAR study. Therefore, this may involve prioritising babies likely to receive the greatest benefit from the treatment. Other criteria that are being considered include the distance from individual centres/ travel; the severity of the condition and the age of the patient.
Q. Where will the treatment be offered in the UK?
A. An EAP has opened in Belfast for those living in Northern Ireland as the number of infants currently eligible for treatment there are so small. GOSH and Newcastle are in active discussion as to the best way to open the EAP at their individual centres. A number of other UK centres have expressed interest in being involved and in the next few weeks there will be discussions with Biogen to see if this is possible. We will provide an update on this as soon as this information becomes available.
Q. When will the EAP actually start at Newcastle and GOSH?
A. At the moment it is not possible to give an exact date when the EAP will begin at Newcastle and GOSH. The EAP could only start there once the children originally recruited to the randomised placebo part of the ENDEAR study had been transitioned to the nusinersen treatment. This process has now been completed so the process of initiating the EAP at those two centres is starting now.
Q. How does a family get on to the programme?
A. Families who are resident in the UK may apply by contacting the coordinators in London: a.bellin@ucl.ac.uk and in Newcastle Anna.Peel1@newcastle.ac.uk Families living in Northern Ireland may contact Belfast Children’s Hospital on 028 9032 9241 though we understand there is no capacity to enrol more infants in Belfast at the moment. Families can also contact Biogen by email: Patientcenter@biogen.com to obtain more information.
Q. Can you tell us exactly what the treatment involves?
A. The drug needs to be administered using a lumbar puncture. In the first year there are 4 injections in the first 2 months, and a further 2 injections in the remaining 10 months. From the second year onwards it is anticipated children will require 3 lumbar punctures each year.
The lumbar punctures require special precautions and, especially in older children, will require either sedation or general anaesthetic. These are particularly complex procedures in children with SMA Type 1.
Q. Are there any possible side effects / problems with the treatment we should be aware of?
A. The information available so far appears to indicate no drug related adverse event. However, as the drug requires repeated lumbar punctures, common problems relating to this procedure, such as headaches, have been reported in several of the children studied.
Q. What if a child was on the EAP and became unwell at the time of one of their scheduled treatments – would they still be able to carry on with the programme?
A. The lumbar puncture is a complex procedure which can only be performed if children are well. If a child is too unwell, the lumbar puncture will need to be rescheduled.
Q. Who will pay for the drug and any other costs to do with the treatment?
A. The drug will be made freely available by Biogen. The cost of the assessment and administration of the drug will be covered by the NHS and the local hospitals.
Q. Would there be help with travel and accommodation costs if needed?
A. This will be the same as for any hospital appointment.
Q. We know Biogen has applied to National Institute for Clinical Excellence (NICE) to begin the process of applying for the treatment to be approved in England. How does this fit in with the EAP and who gets involved in this licensing decision?
A. Before NICE can formally review the drug for approval in England, a decision on the drug’s licence must be taken by the European Medicines Agency (EMA). The EMA’s process for assessing the drug has just begun, and it is expected a decision will be reached later in 2017. In the meantime, NICE is currently conducting what is called a ‘scoping’ process. This means it is gathering evidence about SMA and nusinersen, to help decide whether to review the drug should the EMA grant Biogen a licence. An initial meeting to consider this will take place in January 2017. Muscular Dystrophy UK and representatives from SMA Support UK and The SMA Trust will be there.
The EAP therefore provides the most immediate prospect of access to the drug for children meeting the criteria. However, in the long term it will be important to receive approval from the EMA and NICE.
Q. What are the processes in Scotland, Wales and Northern Ireland?
A. In Scotland, decisions on approval after an EMA licence is granted are taken by the Scottish Medicines Consortium (SMC). Wales and Northern Ireland also have separate processes – although often these two countries follow the decisions made by NICE.
Q. We have heard that the drug may soon be licenced in the US. Will that affect what happens in the UK?
A. No – the United States has a separate process for approving new treatments.
Q. Is there any other way families can get the treatment now – either privately or by going to another country?
A. No – the treatment can only currently be accessed via the EAP in the UK.
Q. What else is happening to help speed things up?
A. The EAP was discussed at Muscular Dystrophy UK’s Services Development Committee meeting in Parliament this month. MD UK has also highlighted the need for access to nusinersen with NHS England’s paediatric neurosciences clinical reference group (CRG). CRGs are groups of clinicians, commissioners, public health experts, patients and carers who use their specific knowledge and expertise to advise NHS England on the best ways that specialised services should be provided. Data will be presented to the group in January. At the next meeting in January, the group will also consider the multidisciplinary resources needed to support centres involved in the centres to deliver the treatment.
Q. What can families do to help?
A. It is important that decision makers understand SMA Type 1 and nusinersen. If you would like to help to raise awareness of the issue with your MP – so that they can raise the issue on your behalf – please contact Peter Sutton at MD UK on: p.sutton@musculardystrophyuk.org
If you have any other questions, families can contact Biogen by email at: Patientcenter@biogen.com
Physicians can contact: medinfo@biogen.com
SMA Support UK will continue to work with MD UK, The SMA Trust, NICE, clinicians and Biogen in order to move things forward. We will keep you informed of progress via our website and social media. You can also contact us on 01789 267520 or email: supportservices@smasupportuk.org.uk
The SMA Trust can be contacted on 01789 801155 or email: info@smatrust.org
16/11/2016: Interim Analysis of CHERISH
Details of the interim analysis of CHERISH, the Phase 3 study evaluating nusinersen in later-onset (consistent with Type 2) SMA, are now available publically via a press-release and a statement has been made to the patient associations, which you can find here:
http://www.sma-europe.eu/news/biogen-announces-positive-interim-results-for-cherish-trials/
26/09/2016: SMA REACH UK - Part 2
Today it was announced that The SMA Trust have partnered with Muscular Dystrophy UK to fund the SMA REACH UK project for a further two years. We are very grateful for their funding and excited for the next phase of the project.
Hear more about SMA REACH UK from Principle Investigator Professor Francesco Muntoni in the video below, and read more about the grant on The SMA Trust website and the Muscular Dystrophy UK website. |
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16/09/2016 Latest Clinical Trial News:
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You may have seen the recent announcements from pharmaceutical companies Biogen and Ionis about the interim results of the clinical trial called ENDEAR Phase 3. This involved infants with SMA Type 1. The drug being trialled was Nusinersen.
Investigators from our two specialist centres were involved in this important study and are delighted with its positive outcome.
Biogen is now working to open an Expanded Access Program (EAP) in the autumn of 2016. Such a programme is designed to provide eligible others outside a trial with access to the successfully trialed treatment prior to potential regulatory approval.
Since the announcement made by Biogen about a proposed EAP for Nusinersen, we have received many requests from families with an infant with SMA Type 1, both from the UK and other countries, who, understandably, wish their children to participate. We are therefore wanting to make sure everyone making enquiries has access to the same, correct information.
The first step for Biogen will be to ensure that all the children from the current ENDEAR study have access to Nusinersen. This is via what is called their extension studies.
Next, Biogen needs to ensure that the proposed EAP can in fact take place in the UK according to UK laws and regulations. A third party called Idis/Clingen will help with the regulatory issues and practical steps that should allow the EAP to start in the UK. It is anticipated that the approval process is likely to take several months.
Because of our experience in administering Nusinersen, initially only our two existing clinical trial sites could potentially be able to participate in the proposed EAP. However, the expectation is that other sites could also be opened in the future.
It is clear that there is an urgent need to agree how to prioritise the requests of families with infants with SMA Type 1 whose child could potentially be enrolled in the EAP for Nusinersen. With this in mind, we are currently in discussion with other European and US sites to agree on the most rational and equitable approach. This discussion will take into consideration all the available information on the potential risks and benefits of Nusinersen for infants with SMA Type 1.
At this time, we are therefore unable to predict if and when we will be able to start the enrolment of infants with SMA Type 1 in an EAP. We do though know that children who are not eligible to use the UK National Health Service (NHS), will be unable to access the proposed EAP in the UK.
As for any EAP, this proposal would anyway be a limited interim arrangement while full regulatory approval for Nusinersen is applied for in the UK.
We understand how frustrating and distressing this process can be for families. We are doing all we can to move things forward and will update you as soon as more information is available from Biogen, Ionis and the regulatory authorities.
Families can send a message to the following email address to obtain more information about Nusinersen Expanded Access Program (EAP): Patientcenter@biogen.com
Physicians can send a message or call the number below. medinfo@biogen.com
(+1) 866-633-4636
Francesco Muntoni Volker Straub
London ENDEAR Principal Investigator Newcastle ENDEAR Principal Investigator
For further information about the EAP please see: http://media.biogen.com/press-release/investor-relations/biogen-and-ionis-pharmaceuticals-report-Nusinersen-meets-primary-en)
Investigators from our two specialist centres were involved in this important study and are delighted with its positive outcome.
Biogen is now working to open an Expanded Access Program (EAP) in the autumn of 2016. Such a programme is designed to provide eligible others outside a trial with access to the successfully trialed treatment prior to potential regulatory approval.
Since the announcement made by Biogen about a proposed EAP for Nusinersen, we have received many requests from families with an infant with SMA Type 1, both from the UK and other countries, who, understandably, wish their children to participate. We are therefore wanting to make sure everyone making enquiries has access to the same, correct information.
The first step for Biogen will be to ensure that all the children from the current ENDEAR study have access to Nusinersen. This is via what is called their extension studies.
Next, Biogen needs to ensure that the proposed EAP can in fact take place in the UK according to UK laws and regulations. A third party called Idis/Clingen will help with the regulatory issues and practical steps that should allow the EAP to start in the UK. It is anticipated that the approval process is likely to take several months.
Because of our experience in administering Nusinersen, initially only our two existing clinical trial sites could potentially be able to participate in the proposed EAP. However, the expectation is that other sites could also be opened in the future.
It is clear that there is an urgent need to agree how to prioritise the requests of families with infants with SMA Type 1 whose child could potentially be enrolled in the EAP for Nusinersen. With this in mind, we are currently in discussion with other European and US sites to agree on the most rational and equitable approach. This discussion will take into consideration all the available information on the potential risks and benefits of Nusinersen for infants with SMA Type 1.
At this time, we are therefore unable to predict if and when we will be able to start the enrolment of infants with SMA Type 1 in an EAP. We do though know that children who are not eligible to use the UK National Health Service (NHS), will be unable to access the proposed EAP in the UK.
As for any EAP, this proposal would anyway be a limited interim arrangement while full regulatory approval for Nusinersen is applied for in the UK.
We understand how frustrating and distressing this process can be for families. We are doing all we can to move things forward and will update you as soon as more information is available from Biogen, Ionis and the regulatory authorities.
Families can send a message to the following email address to obtain more information about Nusinersen Expanded Access Program (EAP): Patientcenter@biogen.com
Physicians can send a message or call the number below. medinfo@biogen.com
(+1) 866-633-4636
Francesco Muntoni Volker Straub
London ENDEAR Principal Investigator Newcastle ENDEAR Principal Investigator
For further information about the EAP please see: http://media.biogen.com/press-release/investor-relations/biogen-and-ionis-pharmaceuticals-report-Nusinersen-meets-primary-en)
- SMA REACH UK features in the latest SMA Trust newsletter, find out about the latest developments in clinical trials in the newsletter.
- The SMA Trust gives an update on SMA REACH UK
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- SMA Awareness Week, 28th September 2015 - 4th October 2015: Danielle Ramsey will be presenting two posters on SMA Functional Scales at the 20th International World Muscle Society Congress in Brighton on Thursday 1st October 2015.
- For more information on SMA Awareness Week please visit the following websites:
SMA Support UK
- SMA Europe Meeting, 11th July 2015: Dr Mariacristina Scoto presented the ongoing work, aims and objectives of the SMA REACH project to SMA Europe stakeholders. Danielle Ramsey presented the work completed to date on revising outcome measures in SMA.
- 10th Annual North Star Clinical Network Meeting, 22nd April 2015: Isabelle Wilson gave an update to the network on SMA REACH UK, achievements to date and aims for 2015.
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- First SMA REACH UK Focus Group, 28th March 2015, University College London: A total of six families from the London SMA REACH UK cohort (children and parents) attended the group. Dr Mariacristina Scoto presented an update on the current clinical trials in SMA followed by an open discussion on clinical trials. The second half of the focus group was lead by Danielle Ramsey, this involved discussing each item of the Revised Hammersmith Scale for SMA, gaining insights into the scale from a patient/parent perspective.
- 8th UK Neuromuscular Translational Research Conference, 19-20th March 2015, Centre for Life, Newcastle Upon Tyne: Danielle Ramsey attended and presented a poster on SMA REACH UK One Year On: The Evolution of Robust Functional Outcome Measures for Spinal Muscular Atrophy Type 2 & 3
- ENMC SMA Outcomes and Clinical Trials Workshop, 7-9th November 2014 Heemskerk, Netherlands: Professor Muntoni, Danielle Ramsey and Marion Main attended this workshop together with an international team of experts, industry representatives and patient representatives. For further detail on the meeting please click here
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- The SMA REACH UK Project and recent developments in revised outcome measures in neuromuscular conditions was presented at the Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London Seminar Series by Danielle Ramsey
- Recruitment Update August 2014: 34 patients have been recruited at GOSH and 11 patients recruited in Newcastle. Congratulations to the teams
- August 2014: SMA Awareness Month
- Saturday June 28th 2014: Day to Day with SMA, Spinal Muscular Atrophy Support UK Conference, Stratford upon Avon
| SMA REACH UK Workshop 28th June 2014 |
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- 11th-12th June 2014: Danielle Ramsey attended the SO- SMART: Significant Outcomes-Spinal Muscular Atrophy Readies for Trials Workshop in Washington hosted by the National Institute of Neurological Disorders and Stroke. Progress to date on revising outcome measures in SMA was discussed. International physiotherapy experts met with members of the PNCR group to discuss work that has been conducted to date revising the Hammersmith scale
- 19th- 20th May 2014: 2nd SMA REACH UK International Workshop, UCL London
The workshop, chaired by Francesco Muntoni and Eugenio Mercuri, was attended by physicians and physiotherapists from Rome, London, Newcastle and US (PNCR) in addition to representatives of SMA Trust, MDC and SMA Foundation and representatives from three independent industrial partners: ROCHE, ISIS and BIOGEN. The meeting was joined via Skype teleconference by Richard Finkel (Chair of the SMA REACH Scientific Advisory Board) and Kate Bishop (ISIS), and Enrico Bertini was also present as representative of the Scientific Advisory Board
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- 30th April 2014: SMA REACH UK - National Neuromuscular Database Meeting
- Recruitment Update April 2014: 16 patients have been recruited at GOSH and 5 patients recruited in Newcastle. Congratulations to the teams.
- 4th April 2014: An update on the SMA REACH UK Project was presented by Dr Mariacristina Scoto at the NorthStar National Neuromuscular Database Clinical Network Meeting 4th April. The work on exploratory functional scales tools was presented to all physiotherpists in the National Clinical Network by Danielle Ramsey and Anna Mayhew.
- Research News March 2014: Trophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy.
For more details please visit: http://www.trophos.com/news/pr20140310.htm
- 5th March 2014: Recruitment opens at Newcastle, the first patient has been recruited
- 3rd & 4th March 2014: 7th Annual UK Neuromuscular
Translational Research Conference 3rd & 4th March 2014, UCL Institute of
Child Health, London
| Improving Standards of Care and Translational Research in SMA - Functional Scales |
- February 2014: Thank you to Tori Elliott Illustration for designing the SMA REACH UK Logo
- SMA REACH UK opens for recruitment: GOSH opens recruitment 5th February 2014, the first three patients have been recruited
- 6-7th December 2013: 1st SMA REACH UK International Workshop, London
GOSH Charity Thank You video
Celebrating 10 years of the SMA Trust
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Thank you to the SMA Trust for SMA REACH UK designs
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