NICE wants cost data on BioMarin's Vimizim

The Highly Specialised Technologies (HST) Evaluation Committee of the U.K.'s NICE said it is "minded not to recommend" Vimizim elosulfase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to treat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome) and requested additional information demonstrating the drug's cost effectiveness.

NICE asked for an explanation of the drug's cost and further economic analyses including "more plausible" rates of disease progression, alternative estimates for the drug's benefits, alternative models of mortality and the exclusion of the value-added tax (VAT) waiver for home care. The committee also requested information on the budget impact of Vimizim on NHS and details of criteria for starting and stopping treatment with the drug in clinical practice. The European Commission approved Vimizim last April (see BioCentury Extra, April 28, 2014). ...