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Alexion's rare bone disorder therapy rejected by NICE

Draft guidance rules Strensiq does not have a compelling enough evidence base

Alexion 

The UK's cost-effectiveness watchdog has issued draft guidance for Alexion's Strensiq suggesting it is not minded to approve the drug for use on the NHS.

The National Institute for Health and Care Excellence (NICE) has said evidence for the drug's benefits in paediatric-onset hypophosphatasia (HPP) - a rare, inherited disorder of the bones - is not compelling enough to justify its £367,000 (around $550,000) annual cost.

Strensiq (asfotase alfa) is an enzyme replacement therapy that is designed to address the underlying deficiency in HPP, which leads to weakening of bones, chronic pain and muscle weakness and other life-threatening complications respiratory and renal, as well as vitamin B6 deficiency that can cause seizures.

At the moment there are no approved therapies for HPP, with patients generally receiving calcium and vitamin B6 supplements and medicines such as non-steroidal anti-inflammatory drugs (NSAIDS) to control pain. There are around seven cases of HPP diagnosed every year in England, and in severe cases infants die within the first year.

In a statement, NICE said that it believed Strensiq improved the chances of survival in patients when added to supportive care, with studies suggesting it could reduce the need for respiratory support and the severity of bone symptoms such as rickets.

However, NICE's technology appraisals director Meindert Boysen said the agency was "unable to estimate from the clinical evidence the precise size of these benefits that are relevant for patients in the longer term".

He also said Alexion had not provided sufficient evidence to justify the price of the drug - the £367,000 figure is based on an average-weight infant - and concluded that at that level it "does not represent value for money for the NHS".

Alexion paid $610m upfront to buy Enobia in 2012 in order to gain control of Strensiq, which was approved by the FDA in October. 

In the US the drug has been priced at an average $285,000 a year - well below the level expected by analysts - with some suggesting the company may have been spooked by increased scrutiny or pharma pricing policies by Congress. The US decision may signal that Alexion is prepared to be flexible on Strensiq's price in England.

The draft guidance is open for public consultation until January 7, and NICE's appraisal committee is due to consider Strensiq at a meeting later that month.

Article by
Phil Taylor

7th December 2015

From: Sales, Regulatory

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