Rare Daily Staff

The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical’s MEPSEVII, the first treatment for children and adults with Mucopolysaccharidosis VII, an ultra-rare metabolic disease that causes progressive damage to organs throughout the body.

MPS VII, also known as Sly syndrome, is one of the rarest MPS disorders, with an estimated 200 patients in the developed world. It is a lysosomal storage disease that results from the inability of the body to produce adequate amounts of an enzyme needed to rid the body of waste products from the breakdown of carbohydrates. As improperly broken-down fragments of waste accumulate in cells through the body, they cause a breakdown in function. MEPSEVII is an enzyme replacement therapy designed to replace the deficient enzyme beta-glucuronidase in MPS VII patients.

“The approval of MEPSEVII is a pivotal moment for Ultragenyx and for patients suffering from ultra-rare genetic diseases for which the investment and development of treatments has not happened yet,” said Emil Kakkis, CEO and President of Ultragenyx. “Our development program sought to create a new paradigm in study design and endpoint evaluations to help accommodate the difficulties of studying extremely heterogeneous ultra-rare diseases to fulfill the promise that the science we have all invested in over many years actually becomes something available for patients.”

The FDA granted Ultragenyx priority review for MEPSEVII. The agency also issued Ultragenyx a Rare Pediatric Disease Priority Review Voucher with the approval. The voucher confers priority review to a subsequent drug application that would not otherwise qualify for priority review. It can be potentially lucrative because the voucher is transferrable. Past vouchers have been sold for as much as $350 million.

In Europe, the European Medicines Agency is currently reviewing the Marketing Authorization Application for the therapy. An opinion from the Committee for Medicinal Products for Human Use is expected in the first half of 2018.

Ultragenyx expected to make MEPSEVII available to patients in the United States later this month. In order to support patients, Ultragenyx has launched UltraCare, a comprehensive support service that will provide ongoing support to patients and caregivers. UltraCare will help patients obtain coverage and assist with financial support for both medication and administration of medication. 

November 16, 2017