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 The National Institute for Health and Care Excellence (NICE), which
had turned down Strensiq as too expensive in December, said on
Thursday it had decided the drug could offer a lifeline for a
handful of babies and young children.
But it urged the U.S. rare diseases specialist, which is already
offering the National Health Service a confidential discount to cap
costs for each patient, to find further ways to reduce the price.
Strensiq costs 58.80 pounds per milligram, meaning it would cost
165,000 pounds a year for babies weighing up to 9 kg and as much as
1.5 million pounds for adults and young people up to 80 kg, NICE
said.
The NICE recommendation covers the medicine's use in perinatal- and
infantile-onset hypophosphatasia, a condition affecting the
development of bone. It is estimated that only one to seven people
are diagnosed each year with this form of hypophosphatasia in
England.
Left untreated, hypophosphatasia causes weakening of bones and can
lead to chronic debilitating pain, muscle weakness, generalized
seizures because of vitamin B6 deficiency, as well as
life-threatening renal and respiratory complications.
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($1 = 0.7654 pounds)
(Reporting by Ben Hirschler; editing by David Clarke)
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