Making of Mepsevii

Why Ultragenyx developed Mepsevii for ultra-rare indication MPS VII

It may take a few years for Ultragenyx Pharmaceutical Inc. to recoup its investment in Mepsevii vestronidase alfa-vjbk, but the company expects to continue to reliably churn out treatments for ultra-rare diseases alongside a portfolio of products for larger Orphan indications.

FDA approved Mepsevii on Nov. 15 to treat mucopolysaccharidosis VII (MPS VII; Sly syndrome), an indication with only 200 patients worldwide.

It was the first approval for the indication, and for Ultragenyx. EMA is evaluating an MAA for the therapy in the same indication; a CHMP opinion is expected next half.

On a Nov. 15 conference call, CFO and EVP Shalini Sharp said after discounts, Ultragenyx expects the average net annual cost of Mepsevii in the U.S. to be $375,000 for a 25-kg patient. For that same patient, Mepsevii would have a wholesale acquisition cost (WAC) of about $549,900 per year.

She noted the price is comparable to enzyme replacement therapies (ERTs) for other MPS disorders, which affect

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