• Skip to primary navigation
  • Skip to main content
  • Skip to primary sidebar
  • Skip to footer

Kids v Cancer

A PEDIATRIC CANCER THINK TANK

DONATE
  • GIVE KIDS A CHANCE act
  • RACE for Children Act
  • Creating Hope Act Pediatric Vouchers
  • CLIMB THE HILL
    • What is Climb the Hill Day?
    • Climb the Hill Youth Advocacy Day Sign Up
    • Video and photo gallery
    • (video) Climb the Hill Youth Advocacy Day — Why Do We Do It?
  • Compassionate Use Navigator
    • Information for Families
    • Information for Physicians
  • Who We Are
    • About Us
    • Mission and Impact
    • Contact
  • News & publications
    • Kids v Cancer in the News
    • Kids v Cancer Publications

First Priority Review Voucher

February 14, 2014

FDA 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.

Link to FDA’s site

  • Creating Hope Act and Pediatric Priority Review Vouchers — Overview
  • Statutory text
  • FDA Guidance on Pediatric Vouchers
  • Pediatric Vouchers Issued and Sold, 2012-present
  • 2020 Testimony in Support of Making the Pediatric Voucher Program Permanent
  • Creating Hope Act News
President Obama before signing Creating Hope Act
  • Facebook
  • Twitter

DONATE SUBSCRIBE

Kids v Cancer · 3602 Newark Street NW · Washington D.C. 20016 · 646-361-3590 · contact

© 2023 Kids v Cancer All Rights Reserved a 501(c)(3) nonprofit organization EIN 27-1427784 Privacy Policy Terms of Use Sitemap