NICE recommends elosulfase alfa for Morquio A syndrome
The UK's National Institute for Health and Care Excellence (NICE) has recommended Vimizim (elosulfase alfa) for the treatment of a very rare life-limiting genetic disorder, it has been announced.
The final guidance recommends the drug for the treatment of Morquio A syndrome, also known as mucopolysaccharidosis type IVa and MPS IVa.
It follows the development of a managed access scheme by US biotech company BioMarin Pharmaceutical (Nasdaq: BMRN) and NHS England that fulfils the conditions for the drug’s use specified by NICE in its previous guidance.
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