NICE has published draft guidance recommending the drug asfotase alfa (also called Strensiq and made by Alexion Pharma UK) for people with paediatric-onset hypophosphatasia.
The extremely rare inherited condition affects between 1 and 7 babies each year in England.
A new agreement between the company and NHS England on price and access to the drug means NICE can now widen the group of people eligible for treatment on the NHS.
Previous NICE draft guidance recommended the treatment for only babies with the most severe form.
Now an improved deal includes a 5-year managed access agreement which reduces the cost of the drug to the NHS and enables people with the highest unmet need to be identified.
Described as a ‘step-change’ in the management of the condition, the drug offers a ‘lifeline’ for people that could enable them to have a good quality of life but without which they would not survive. The drug is the first therapy that specifically targets the underlying cause of hypophosphatasia.
Professor Carole Longson MBE, director of the centre for health technology evaluation at NICE, said: “Asfotase alfa is an important development in the treatment of this devastating condition that has been shown to save lives and prevent or delay its progression.
“However, up until now the committee felt the benefits of the drug were too uncertain in the whole population in relation to its very high price for them to be able to recommend it for any but the most seriously affected.
“The new deal, which includes a managed access agreement, between the company and NHS England means that people with the greatest clinical need for treatment can be identified and the costs and risks to the NHS have been reduced.
“We are very pleased therefore to be able to recommend it as an option for treating paediatric-onset hypophosphatasia.”
The agreement means asfotase alfa will be made available on the NHS to eligible children and adults for 5 years initially. During this time more information will be gathered on how long treatment should continue, and when treatment could be stopped or the dose reduced.
Hypophosphatasia disrupts the process in which minerals such as calcium and phosphorus are deposited in developing bones. Infants with perinatal - and infantile-onset hypophosphatasia have a high mortality rate, with up to 100% dying within the first year of life.
Asfotase alfa provides long term enzyme replacement therapy to restore normal development of bones and teeth.
Asfotase alfa has been considered as part of NICE’s Highly Specialised Technologies programme that looks at treatments for very rare diseases that are commissioned nationally by NHS England.
The draft guidance is now with consultees, including the company, healthcare professionals and patient/carer organisations who now have the opportunity to appeal against the draft recommendations.
The positive news about asfotase alfa follows the publication of NICE guidance on 28 June approving eliglustat (Cerdelga, Genzyme Therapeutics) for treating Type 1 Gaucher disease following an improved patient access scheme from the company.