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First treatment recommended for infants with Wolman Disease

European Pharmaceutical Review

The National Institute for Health and Care Excellence (NICE) has recommended enzyme replacement therapy sebelipase alfa (Kanuma ® ▼) in final draft guidance. This ultra-rare metabolic disease occurs due to lysosomal acid lipase (LAL) enzyme deficiency. How does the enzyme replacement therapy work?

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Utrogestan accepted as HRT in Scotland

European Pharmaceutical Review

According to the National Institute for Health and Care Excellence (NICE), an individualised approach should be adopted when treating women with HRT. Researchers have considered it to be associated with fewer risks compared to synthetic hormones. The SMC approval of Utrogestan is limited within NHS Scotland.

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First treatment recommended for infants with Wolman Disease

European Pharmaceutical Review

The National Institute for Health and Care Excellence (NICE) has recommended enzyme replacement therapy sebelipase alfa (Kanuma ® ▼) in final draft guidance. This ultra-rare metabolic disease occurs due to lysosomal acid lipase (LAL) enzyme deficiency. How does the enzyme replacement therapy work?

article thumbnail

Utrogestan approved as HRT in Scotland

European Pharmaceutical Review

According to the National Institute for Health and Care Excellence (NICE), an individualised approach should be adopted when treating women with HRT. Researchers have considered it to be associated with fewer risks compared to synthetic hormones. The SMC approval of Utrogestan is limited within NHS Scotland.

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Pegunigalsidase alfa receives NICE approval for Fabry disease

Hospital Pharmacy Europe

The use of pegunigalsidase alfa in the treatment of adults with Fabry disease (alpha-galactosidase deficiency) has been granted approval by the UK’s National Institute for Health and Care Excellence (NICE). This approval makes it the first ERT treatment recommended for routine NHS use by NICE.

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MHRA grants bluebird ‘innovation passport’ for sickle cell therapy

pharmaphorum

The UK innovation passport adds an additional layer however, as it involves input from the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC), meaning that NHS funding deliberations are bundled into the review.

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ILAP: Improving access to innovative oncology treatments

European Pharmaceutical Review

In November 2022, the Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport was awarded to Boehringer Ingelheim’s investigational BI 907828 in de-differentiated liposarcoma (DDLPS), a rare and aggressive oncology indication for which there has been no new first-line treatment in over forty years.