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NICE approves life-changing medication for treating MPS IVA Morquio


We are delighted to announce that Elosulfase alfa (Vimizim®) has been recommended by NICE (National Institute for Health and Care Excellence) for treating MPS IVA Morquio in people of all ages in England and Wales. This recommendation applies to all patients, including newly diagnosed and those already receiving treatment.

Elosulfase alfa, manufactured by pharmaceutical company BioMarin, is the first disease modifying treatment recommended for routine NHS use by NICE following completion of a 5-year managed access programme. This recommendation and continued access to the treatment will give hope to people with MPS IVA, their families and carers of a better future.

Elosulfase alfa has been available as a treatment option as part of a managed access agreement since the original NICE highly specialised technology guidance for elosulfase alfa was published in 2015. This innovative approach of collecting long term clinical and cost effectiveness data was the first of its kind used in NICE’s review of Highly Specialised Technologies. The managed access agreement facilitated the collection of not only clinical data but ‘real world’ evidence for 69 people, parents and carers.

The NICE re-evaluation looked at clinical trial evidence, data from the managed access agreement and feedback from patient and carer experience were collected. The evidence showed some slowing down of disease progression and long-term benefits. The committee considered the health and quality of life benefits to patients to be substantial.

The managed access agreement included a number of ongoing assessments that patients had to adhere to for treatment to continue. The committee concluded that this level of review no longer applied and patients should have continued access as long as there was ongoing clinical benefit. Clinical management would follow simpler treatment criteria as appropriate.

NICE’s close collaboration with the MPS Society, Rare Diseases Research Partners, clinicians, NHS England and the company was key to the success of the data collection, which enabled new evidence to be presented on the benefits of this treatment to patients.

Bob Stevens, Group Chief Executive of the MPS Society, said:

“Today we celebrate the news that NICE has recommended elosulfase alfa for routine use as a therapy for MPS IVA Morquio. It has been a long and at times difficult journey, but due to the willingness of all parties to find innovative solutions, and the pivotal role the MPS Society has played, the long-term future of many within our MPS IVA community has been greatly improved. We want to thank the patients, clinicians and staff who have worked together to make this possible.”

Helen Knight, acting interim director of medicines evaluation at NICE, said: “The arrangement to give access to elosulfase alfa while further data was collected on its clinical and cost-effectiveness was the first of its kind to have been attempted in the NHS in England. Today’s announcement demonstrates the value of this approach, with patient groups, clinicians, academics, companies, NICE and NHSE all working together to improve the lives of people with rare diseases."

Commenting on the announcement, Jim Lennertz, Senior Vice President, Commercial Operations, EMEA said:

"BioMarin is pleased that MPS IVA patients in England will continue to be able to access treatment with elosulfase alfa, the only enzyme replacement therapy that addresses the underlying cause of the condition. We recognise and appreciate the significant effort and commitment of the MPS Society, clinicians and all stakeholders during the NICE evaluation process. BioMarin has been working to improve the health and quality of life of people with MPS for 25 years. Elosulfase alfa is widely available globally, and we are glad that patients in England will now have routine access to the therapy."

Katy Brown, patient expert and mum to a child on elosulfase alfa, said:

“I am delighted and relieved that NICE has finally made the decision to recommend Vimizim. Our eldest son, Sam, has received Vimizim for almost a decade and it has benefited him hugely. The possibility of this being taken away from him and our Morquio community was unthinkable. Alongside dealing with the day-to-day challenges of a very rare and complex condition, the Morquio community has had to endure uncertainty and anxiety for many years, not knowing if Vimizim would continue. Today, we all breathe a collective sigh of relief. I feel immensely proud of our community’s resilience, dedication, and strength to make sure that the patient voice has been truly heard and understood, and hope this now paves the way for a brighter future for the treatment of other very rare conditions.”

Dr Saikat Santra on behalf of Birmingham Women’s and children’s NHS Foundation Trust, said:

“We are really pleased that this journey, which started with the first patient in the World with Morquio A being treated here in Birmingham as part of clinical trials, has ended with NICE recommending that elosulfase alfa be provided on the NHS. This will open up a pathway to treatment for many patients across England and hopefully help many people with Morquio A disease to live a healthier life. We would like to pay particular thanks to the many families who have diligently taken part in clinical trials and data collection during the managed access agreement period whose patience has enabled today’s important decision.”

Today, we all breathe a collective sigh of relief. I feel immensely proud of our community’s resilience, dedication, and strength to make sure that the patient voice has been truly heard and understood, and hope this now paves the way for a brighter future for the treatment of other very rare conditions.

The final evaluation document (FED) is available on the NICE website.

You can view NICE’s press release here.

When NICE recommends a treatment, the NHS must make sure it is available within three months.

We would like to thank our MPS IVA community, clinicians, and patient groups for all their submissions, representation at committee meetings and information shared through the surveys and personal stories.

Read more about the our fight for Vimizim.


Bob Stevens (Group Chief Executive Officer)

Sophie Thomas (Senior Head of Patient Services and Clinical Liaisons)

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