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Take a look at a selection of our recent media coverage:

NICE recommends sebelipase alfa as first NHS treatment for Wolman disease

1st December 2023

The enzyme replacement therapy sebelipase alfa (brand name Kanuma) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat Wolman disease, its manufacturer Alexion has announced.

The final draft guidance from NICE recommends sebelipase alfa for the treatment of infants who have the ultra-rare Wolman disease – a genetic, rapidly progressive lysosomal acid lipase deficiency that causes multi-organ damage – and are under two years of age at the start of treatment.

The enzyme replacement therapy involves weekly intravenous infusions, which can be given at home, and is used alongside a restricted diet. It can allow a haematopoietic stem cell transplant to be done, if appropriate.

This will be the first treatment available on the NHS for the treatment of Wolman disease. Standard care without sebelipase alfa is palliative.

Following the recommendation, NHS England said ’the life-saving treatment will be fast-tracked to be available to any eligible patients straight away’ and will be funded via the Innovative Medicines Fund.

The final guidance is expected to be published in early January 2024.

NICE’s recommendation is based on sebelipase alfa’s clinical effectiveness evidence from two single-arm, open-label, multicentre trials: LAL-CL08 and LAL-CL03.

A natural history study (LAL-1-NH01) was also used to estimate outcomes for clinical management without sebelipase alfa.

In LAL-CL08, some 90% of people who had sebelipase alfa were alive at 12 months and 80% alive at 24 months. In LAL-CL03, a total of 67% of people who had sebelipase alfa were alive at 12 months, with 56% alive at 24 and 60 months. In LAL-1-NH01, everyone who did not have treatment died before 12 months old.

Other disease-related outcomes such as improvements in weight and length of age, nutritional outcomes and important measures of liver damage were also identified.

While the evidence suggested sebelipase alfa increases how long people live, there remains uncertainty around its longer-term effectiveness with regards to how much longer people will live or how their long-term quality of life compares with people without the condition.

Professor Simon Jones, consultant in paediatric inherited metabolic disease, at Manchester University NHS Foundation Trust (MFT), said: ‘Having shown in clinical trials at the NIHR Manchester Clinical Research Facility at Royal Manchester Children’s Hospital (part of MFT) that we could treat a fatal disease of infancy with this lifesaving drug is extremely gratifying.

‘Moving forward to treat children on the NHS is however what we all really want and signifies a substantial step forward in our dedication to practical advancements in rare disease medicine and improved patient outcomes.’

Bob Stevens, group chief executive of the MPS Society, said: ‘Today’s announcement is positive news, giving babies born with Wolman disease and their parents access to a new treatment option.

’It remains challenging to get medicines for rare diseases reimbursed but our commitment to our community of MPS and rare disease families is unwavering. Moments like this are special and are only possible through a collaborative process where the patient voice is heard, valued, and acted upon.’

MHRA and NICE approve two-component therapy for late-onset Pompe disease

17th August 2023

A treatment combining cipaglucosidase alfa (Pombiliti) and miglustat (Opfolda) for adults living with late-onset Pompe disease has been granted marketing authorisation by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

Cipaglucosidase alfa is a long-term enzyme replacement therapy (ERT) used in combination with the enzyme stabiliser miglustat for adults with late-onset Pompe disease. This rare, inherited lysosomal disorder is caused by deficiency of the enzyme acid alpha-glucosidase (GAA).

The approval comes as the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Pombiliti + Opfolda for use within the NHS in England and Wales.

NICE concluded that the cost-effectiveness estimates for Pombiliti + Opfolda showed a positive net health benefit and recommended Pombiliti + Opfolda for adults with late-onset Pompe disease as first line and later lines of therapy.

The MHRA approval and NICE decisions were based on clinical data from the Phase 3 pivotal PROPEL study in which Pombiliti + Opfolda was associated with demonstrable improvements in both musculoskeletal and respiratory measures.

The time to market for Pombiliti + Opfolda was accelerated after it was granted an Innovation Passport under the Innovative Licensing and Access Pathway, a Priority Innovative Medicines designation and a positive scientific opinion under the Early Access to Medicines Scheme. This enabled healthcare professionals to prescribe the treatment prior to marketing authorisation based on clinical factors for patients with a clear unmet need.

In Pompe disease, reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues, leading to skeletal muscle weakness and progressive respiratory involvement.

Professor Mark Roberts, consultant neurologist at the Greater Manchester Neurosciences Unit at Salford Royal NHS Foundation Trust, commented: ‘From the positive uptake of the Early Access to Medicines Scheme, we have already seen the impact that this treatment is having for patients. Having widespread access to this treatment is an exciting development for the Pompe community, giving HCPs and patients a new option that exhibits a novel mode of action.’

Referring to this ‘major step forward’ for late-onset Pompe disease treatment, Bradley Campbell, president and chief executive officer of Amicus Therapeutics, said: ‘We are grateful to the global Pompe community who have helped advance this therapy, especially the patients, families, and physicians who participated in our clinical studies. I am proud of Amicus’ relentless commitment toward ensuring patient access to our innovative therapies, and we are working as quickly as possible to make Pombiliti and Opfolda commercially available.’

Pombiliti + Opfolda will both be added to the Orphan Register held by the MHRA and will benefit from 10 years of market exclusivity.

The European Commission granted approval for Opfolda for adults with late-onset Pompe disease in late June, following its approval of Pombiliti in March 2023.