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Roche makes risdiplam available globally through a pre-approval access programme

Today Roche announced that it is making risdiplam available through a global Pre-Approval Access/Compassionate Use Programme. Under the programme, the company will provide risdiplam for patients who cannot receive an approved treatment.

Upon clinician’s request, the company will provide this innovative oral drug, free of cost, for eligible patients who cannot be treated with other available drugs for medical or regulatory reasons.

As with the Early Access Programme of nusinersen, the risdiplam programme will initially be open to people with spinal muscular atrophy type 1 irrespective of their age and functional status. The programme will be run globally, including in the UK. There is no package limit in the programme and Roche intends to fulfil every medically justified request that comes from the treating clinician.

Until regulatory approval is received, risdiplam will remain an experimental drug whose safety and efficacy are being investigated in clinical trials. If data generated through these studies is robust enough, Roche say they will expand the programme to include people with SMA type 2 upon filing for regulatory approval of risdiplam. As the risdiplam safety and efficacy data is already very promising, we expect the regulatory filing to happen in the next few months.

We have to acknowledge that Roche has made a bold step towards eradicating this severe disease across the world. Even though not all suggestions have been incorporated, we applaud that the company has keenly listened to the patient voice when designing the programme.

This is great news that moves everyone another step closer to having a safe and effective treatment for all – an ultimate ambition of TreatSMA.

Below is an official update from Roche.

Following your recent request to receive more information about Roche’s Pre-Approval Access / Compassionate Use (PAA/CU) plans for the investigational medicine risdiplam, we would like to provide you with the below information.

While there have been very important treatment advances for patients with SMA in the last few  years,  we recognise that there continues to be high unmet medical need within the community and patients experiencing life-threating or severe conditions may not have access or be eligible to satisfactory treatments or to ongoing clinical trials.

As a result of this remaining need, we are pleased to inform you that Roche has initiated a global PAA/CU programme for risdiplam in countries where applicable laws and regulations allow  such  programmes  and which fulfill the criteria based on applicable company policy. A PAA/CU programme enables patients who are facing the most urgent medical need and have no other treatment options, to access our investigational therapies before these receive regulatory approval.

In countries where the PAA/CU programme already is or will be implemented, Roche is currently offering patients with the most urgent medical need, Type 1 SMA, the opportunity for access to risdiplam based  on their treating physician’s decision. Being mindful, however, that patients with other types of SMA are also facing life-endangering situations, the programme will be expanded to patients with Type 2 SMA at the moment of filing of the regulatory application for risdiplam in each country. This means that in participating countries within the European Union, the programme is already available for patients with Type 1 SMA and will expand to patients with Type 2 SMA upon filing of our Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA), which is currently planned for mid 2020.

It is important to highlight that PAA/CU for investigational medicines must always comply with applicable country-specific laws and regulations, which differ across countries, and as a result, local  variations in  the programme will occur. We recommend that people interested in accessing risdiplam via PAA/CU, discuss their options with their treating physician. The decision to apply for the programme is one that should be made by the treating physician. If the risdiplam PAA/CU programme is deemed to be the best path forward, requests must be submitted by the physician to the local Roche affiliate.

Achieving broad and sustainable access through regulatory approval and reimbursement is our key priority, and we are collaborating with Health Authorities, government agencies and other key stakeholders around the world with the aim to make risdiplam available to all patients who can benefit from the treatment as soon as possible.

We want to thank you for your collaboration and we look forward to providing further updates about our programme as they become available.


We will shortly disclose how the Risdiplam Pre-Approval Access Programme is going to be implemented in the UK. Stay tuned!

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