FAQs re Access to Roche’s risdiplam Compassionate Use Programme (CUP) in the UK

24 April 2020 / Posted in: Treatments & Research

The following FAQs have been checked for accuracy with Roche. We hope they will help to clarify further how their COVID-19 response measures affect pre-approval access to risdiplam in the UK, including practical aspects of the programme.

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Impact of COVID-19 on SMA UK

22 April 2020 / Posted in: Information, Support

We are still here for you and this will remain our key focus. We have, though, had to make some difficult decisions and cuts to survive our drop in income.

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Roche Updates SMA UK about their COVID-19 Response Measures and Pre-Approval Access to Risdiplam

17 April 2020 / Posted in: Treatments & Research

Following our enquiry, we received a community update today. This covers: maintaining risdiplam supply; ongoing risdiplam clinical trials and support services; Compassionate Use for risdiplam.

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Extension of the New Drug Application (NDA) Timetable for Risdiplam in the USA

14 April 2020 / Posted in: Treatments & Research

Roche has advised the SMA Community that the US FDA recently informed them that due to the volume of additional data submitted by Roche, they will need a three-month review extension of the New Drug Application (NDA) of risdiplam. Roche, therefore, expects a decision from the FDA by 24th August 2020.

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Easter Bank Holiday

08 April 2020 / Posted in: Information, Support

SMA UK staff won’t be working over the Easter Weekend - so will finish at the end of Thursday 9th April and will be back working at 9am on Tuesday 14th. Find out about where to find information, including re COVID-19, during this time.

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Continued Advocacy re: Access to Nusinersen For All Those Who Have SMA Type 3

07 April 2020 / Posted in: Treatments & Research

Please be assured that SMA UK, MDUK and TreatSMA are still actively working on this and continue to ask NICE and NHS England to address the concerns we have at the earliest possible time.

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NICE’s Scoping of Risdiplam Treatment Delayed due to COVID-19

07 April 2020 / Posted in: Treatments & Research

NICE has announced that as it has to prioritise work that is either therapeutically critical or relates to addressing COVID-19 diagnostic or therapeutic interventions, it will not go ahead with this stage of the appraisal process at this time.

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Information Statement about how Access to Nusinersen Treatment will be Affected by COVID-19 (coronavirus)

06 April 2020 / Posted in: Treatments & Research

Advice from clinical experts and discussions with the Patient Groups have informed this statement from NICE, NHS England and NHS Improvement. It concerns those who are already receiving treatment and those who have not yet started treatment.

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First Patient in Biogen DEVOTE Trial Treated with Higher Nusinersen Dose

06 April 2020 / Posted in: Treatments & Research

Biogen has announced that they have initiated SMA patient treatment in their DEVOTE study, a Phase 2/3 clinical trial of higher nusinersen doses. Although already approved for use in SMA, there is always room to improve the treatment regime of a successful therapy. With the DEVOTE trial, this is exactly what Biogen are attempting.

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NICE Confirms Appraisal of onasemnogene abeparvovec (Zolgensma) is Not Defined as Therapeutically Critical

03 April 2020 / Posted in: Treatments & Research

NICE has decided that the HST evaluation of onasemnogene abeparvovec is not defined as therapeutically critical. The HST evaluation will progress as planned with company and Evidence Review Group input into the next phase of the timeline. NICE will provide further updates on the progress of the evaluation when known.

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