NICE publish recommendation of hypophosphatasia drug

NICE has published further draft guidance as part of its highly specialised technologies programme recommending asfotase alfa (Strensiq, Alexion Pharma UK) for children with hypophosphatasia. A rare, and often fatal genetic condition. The drug is the first treatment to target the underlying cause of perinatal- and infantile-onset hypophosphatasia, Asfotase alfa provides long-term enzyme replacement therapy

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AZ initiate trials for using diabetes drug in kidney disease and heart failure

AstraZeneca has launched two new trials with its SGLT-2 inhibitor Forxiga with the aim of exploring its potential beyond the drug’s approved diabetes indication. AZ have initiated two randomised, placebo-controlled Phase IIIb outcome trials assessing Forxiga in the management of chronic kidney disease and chronic heart failure, within participants with and without type-2 diabetes. The

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NICE recommends PTC Therapeutics Duchenne muscular dystrophy treatment

PTC Therapeutics has announced that NICE has issued final guidance recommending Translarna for ambulatory patients aged five and over with nonsense mutation Duchenne muscular dystrophy in connection with a managed access agreement with NHS England. Duchenne muscular dystrophy, a rare genetic disease that primarily affects males, is a progressive muscular disorder caused by the lack

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