MPS IVA

14 Sep 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Saskatoon Siblings Permalink

For Immediate Release – Saskatoon Siblings Await Funding For Life-Saving Treatment

Sticky Post By On 14 September, 2015

FOR IMMEDIATE RELEASE

SASKATCHEWAN SIBLINGS AWAIT FUNDING FOR LIFE-SAVING TREATMENT

Children Diagnosed With Ultra-Rare Condition; Treatment Already Being Funded In Ontario and Quebec

(Sept. 14, 2015) The Saskatchewan Ministry of Health is currently considering an application for exceptional funding of a life-sustaining treatment required by three siblings from Saskatoon. 8 year-old Sara Amir, along with her siblings Khadija, 10 and Muhammad, 12 have been diagnosed with Morquio Syndrome, and all three require the life-sustaining treatment immediately in order to halt further progression of this devastating disease. The Saskatchewan Ministry of Health received their application to begin treatment with Vimizim, an enzyme replacement therapy approved by Health Canada in July 2014, seven months ago. A review of the application has just been initiated and a decision is expected in the coming weeks.

The children suffer from a rare enzyme deficiency called MPS IVA (also known as Morquio A Syndrome). Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG). These GAGs build up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, impaired mobility, shortened stature, and premature death.

While there is no known cure for Morquio Syndrome, a treatment does exist. Vimizim is an Enzyme-Replacement Therapy (ERT) 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 Ontario and Quebec. Recently, the National Institute for Health and Care Excellence (NICE) recommended reimbursement for all patients suffering from Morquio Syndrome throughout the UK.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, is urging Health Minister Dustin Duncan and Premier Brad Wall to expedite their decision so the children can get the immediate help they need. Since the application was submitted in March, McFadyen has met with Minister Duncan twice to present all of the expert opinion and Canadian and International data that exists regarding the treatment. “Minister Duncan has been privy to all opinions and guidance from the best medical minds across the globe. All of them have recommended this treatment for these kids and I’m confident that a positive decision will be returned in a speedy fashion.”

Though hopeful of a positive outcome for the family, McFadyen can’t help but feel frustrated by the length of time it’s taken to render a decision. “The Minister has had this file for over 7 months, and the initial request for therapy was submitted 19 months ago. Saskatchewan already pays for every other available treatment for MPS Diseases – MPS I, MPS II, MPS VI. This treatment does the exact same thing – saves lives. With the UK and the US already ensuring access to this treatment, there is ample evidence available to help them with their decision. We’ve given them everything they need – it’s time to take action and save these kids.”

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, couldn’t agree more. “The Canadian Expert Panel and the International Guidelines for treating Morquio A Syndrome both call for treatment to begin as soon as possible to stave off the devastating effects of this disease. With every day that passes, the chance that irreversible symptoms will appear increases. I am hopeful that the Saskatchewan government won’t allow bureaucracy to have a negative impact of the lives of these children and am therefore confident that a positive decision will be returned soon.”

Myrah and McFadyen both note how impactful the treatment has been for patients already receiving therapy in Canada and in most other developed countries throughout the world. Myrah states, “We are seeing kids improve dramatically because of this treatment – kids’ internal organs reducing back down to a normal size, rates of growth increasing, heart function improved, walking distance increased and the use of mobility aids reduced. Until recently, only supportive care that treats the symptoms of the disease was available to patients, including medication, multiple surgeries, and ongoing occupational and physical therapy. By delaying access to the first and only pharmaceutical treatment option available, governments are leaving patients dependent on supportive therapies that do not address the underlying cause of this severely debilitating disorder. We know this works and we know it changes lives.”

McFadyen adds “We have a video of a 17 year old girl prior to starting treatment and she can’t walk more than 10 steps without having to stop. She’s in agony and it’s heartbreaking to see. After 12 weeks on therapy, she’s happy and walking long distances normally. I’m hopeful we can ensure the same opportunity is given to the Akhter children. It’s the role of government to protect and ensure fair and equitable access to Health Care for all Canadians –regardless of whether they are suffering from a rare disease or not – and we’re calling on this government to take action and save the lives of these children now. They can’t afford to wait.”

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For more information about this topic, or to schedule an interview to discuss, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com.

 

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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09 Feb 2014
Sticky Post By Posted in Advocacy, MPS IVA Permalink

Morquio A Syndrome (MPS IVA) Advocacy

Sticky Post By On 9 February, 2014

morquiotitleHi Everyone,

I’m excited to announce that The Isaac Foundation will be working with families throughout Canada to help ensure provincial funding for Enzyme Replacement Therapy (ERT) for MPS IVA patients is forthcoming once the treatment gets approval from Health Canada.

Morquio Syndrome is a rare disease that is part of the MPS umbrella of lysosomal storage disorders.  The disease affects major organ systems in the body and leads to devastating symptoms for sufferers.  These symptoms included bone and joint disease, heart and airway disease, a shortened stature, and premature death.

We have been fortunate to have had success advocating for families seeking support and treatment options for other forms of MPS in numerous provinces over the past 8 years.  Currently, there are no treatment options for sufferers of MPS IVA.  However, recent clinical trials for Biomarin’s Vimizim have produced very promising results, and Health Canada is expected to approve the treatment for use in Canada in the very near future – most likely before the summer of 2014.   A decision by the FDA in the United States is imminent, and an approval in the US could mean that patients can access the treatment via the Federal Government’s Special Access Program.  However, approval through the SAP is always contingent on provincial funding being made available.

We are excited to help advocate with families as they seek treatment options for themselves or their affected children, and will work with the same passion and resolve that we have given all of our other advocacy efforts.

If you or your family is interested in working with The Isaac Foundation to help bring ERT to your province for yourself or your children, please don’t hesitate to contact Andrew McFadyen at HERE or via telephone at 613.328.9136.

For more information about Morquio A Syndrome please click HERE.