BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that The National Institute for Health and Care Excellence (NICE), NHS England and BioMarin have reached an agreement on a Managed Access Agreement, which provides a basis for access for clinically suitable mucopolysaccharidosis type IVA (MPS IVA) patients to Vimizim® ? (elosulfase alfa) treatment for the next five years. The provision of access is subject to the completion of the ongoing NICE Highly Specialised Technologies process, where the final guidance is expected in mid-December.

The Morquio A community has been long awaiting this decision, which provides patients access to Vimizim, and we are relieved and elated to have resolution,? said Christine Lavery, Chief Executive of the MPS Society. ?With fewer than 80 people living with Morquio A syndrome in England, it is hard to comprehend what these patients and their families have been through over the last 18 months. Treatment to this community is so much more than just a therapy. It is hope for a future where their improved health allows them to reach their full potential.?

Morquio A syndrome is an ultra-rare, severely debilitating disease affecting an estimated 3,000 patients in the developed world. The most common features of the disease are progressive skeletal dysplasia, the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance, and breathing. 

This is fantastic news for all affected patients,? said Professor Chris Hendriksz of Salford Royal NHS Foundation Trust. ?Many of the patients who participated in the largest ever clinical trial for ultra rare enzyme replacement therapies are from England, and today’s announcement means they and all diagnosed patients in the country will now have a greater opportunity to access treatment. The decision by NICE also means many patients will have the possibility of access to treatment at home, which will put an end to long journeys to hospital to receive weekly treatment. The impact for patients will be significant. Previously, we have only been able to help manage symptoms, but this new treatment is now able to target the underlying cause of this devastating disorder.?

As Vimizim is the first and only treatment to address the underlying cause of Morquio A syndrome, we applaud the NICE’s decision to provide the patient community with access to this much-needed therapy,? said Jim Lennertz, Group Vice President and Regional Manager of Europe, the Middle East and Africa at BioMarin. ?As part of our commitment to the MPS community, we worked diligently with the NICE, advocates and patients to bring Vimizim to those who need it.?

The U.S. Food and Drug Administration (FDA) approved the Vimizim license application for the treatment of patients with Morquio A syndrome on February 14, 2014, and the European Commission approved it on April 28, 2014. The therapy is also approved in Australia, Canada, Brazil and Japan.