morquio syndrome

03 Nov 2015
Sticky Post By Posted in Advocacy, Media, Saskatoon Siblings Permalink

LIFE-SAVING TREATMENT APPROVED FOR SASKATOON SIBLINGS

Sticky Post By On 3 November, 2015

Ministry of Health Reverses Funding Decision; Family Elated That “Hope Has Returned”

FOR IMMEDIATE RELEASE:  The Minister of Health in Saskatchewan has approved funding for a life-saving treatment required by three siblings from Saskatoon.  8 year-old Sara Amir, along with her siblings Khadija, 10 and Muhammad, 12, suffer from MPS IVA (Morquio Syndrome) and require the life-saving treatment immediately in order to halt further progression of their devastating disease. After initially denying funding for the children, the Minister of Health ordered a review of the files and asked a panel of experts to provide feedback to the Ministry regarding the children and the treatment required.

Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. This waste builds up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, was thrilled with the news. “I applaud the Minister for his leadership during this review, and thank him for the open and transparent way with which he conducted this review. This is life-saving for these children, and life-changing for their family. This is incredible news.”

Jamie Myrah, Executive Director of The Canadian MPS Society, also thanked the NDP Health Critic, Danielle Chartier, for her work bringing this issue to the legislature and for the advocacy she provided the family during what was a very emotional period.

McFadyen adds, “The Saskatchewan government has taken a leadership role on this and have come in line with the Canadian Expert Opinion and the International Treatment Guidelines for Morquio Syndrome. They have also mirrored the recent recommendations from the UK. Most importantly, they’ve given hope back to this family – hope for a new life and the chance at fighting back against this relentless disease. For that, I am incredibly thankful to Minister Duncan and his team.”

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For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.

Backgrounder

While not a cure for Morquio Syndrome, the necessary Enzyme-Replacement Therapy (ERT) is designed to provide patients with a synthetic version of the enzyme they are lacking by infusing small doses into the patient’s bloodstream on a weekly basis. The treatment slows down or halts the progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. It was approved by Health Canada in July 2014, and has been reimbursed for use by patients in Saskatchewan, Ontario, and Quebec. Recently, the National Institute for Health and Care Excellence (NICE) recommended reimbursement for all 88 patients suffering from Morquio Syndrome throughout the UK.

14 Feb 2014
Sticky Post By Posted in MPS IVA Permalink

FDA Approves Vimizim for Treatment of MPS IVA

Sticky Post By On 14 February, 2014

morquiotitleFDA NEWS RELEASE

For Immediate Release: Feb. 14, 2014
Media Inquiries: Andrea Fischer, 301-796-0393, andrea.fischer@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

FDA approves Vimizim to treat rare congenital enzyme disorder
First drug to receive Rare Pediatric Disease Priority Review Voucher

The U.S. Food and Drug Administration today approved Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome). Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. Absence of this enzyme leads to problems with bone development, growth and mobility. There are approximately 800 patients with Morquio A syndrome in the United States.

Vimizim was granted priority review. An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment. Vimizim is also the first drug to receive the Rare Pediatric Disease Priority Review Voucher – a provision that aims to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

“This approval and rare pediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Andrew E. Mulberg, M.D., deputy director, Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research (CDER). “Prior to today’s approval, patients with this rare disease have had no approved drug treatment options.”

The safety and effectiveness of Vimizim were established in a clinical trial involving 176 participants with Morquio A syndrome, ranging in age from 5 to 57 years. Participants treated with Vimizim showed greater improvement in a 6-minute walk test than participants treated with placebo. On average, patients treated with Vimizim in the trial walked 22.5 meters farther in 6 minutes compared to the patients who received placebo.

The most common side effects in patients treated with Vimizim during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue. The safety and effectiveness of Vimizim have not been established in pediatric patients less than 5 years of age. Vimizim is being approved with a boxed warning to include the risk of anaphylaxis. During clinical trials, life-threatening anaphylactic reactions occurred in some patients during Vimizim infusions.

Vimizim is marketed by Novato, Calif.-based BioMarin Pharmaceutical Inc.

For more information:

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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