Recommendation Provides Patient Access to Treatment for Ultra-Rare Condition With Reimbursement
SAN RAFAEL, Calif., Nov. 23, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that the 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, (United Kingdom). “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.
“As Vimizim is the first and only treatment to address the underlying cause of Morquio A syndrome, we applaud the Institute’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 Institute, 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.
About Vimizim
Vimizim® (elosulfase alfa) is a treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). Vimizim is the first approved enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome—a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Vimizim is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of GAGs. Morquio A syndrome is a rare, severely debilitating and progressive disease that previously had no approved, standard-of-care treatment other than supportive care.
SAN RAFAEL, Calif., Nov. 23, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that the 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, (United Kingdom). “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.
“As Vimizim is the first and only treatment to address the underlying cause of Morquio A syndrome, we applaud the Institute’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 Institute, 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.
About Vimizim
Vimizim® (elosulfase alfa) is a treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). Vimizim is the first approved enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome—a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Vimizim is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of GAGs. Morquio A syndrome is a rare, severely debilitating and progressive disease that previously had no approved, standard-of-care treatment other than supportive care.
