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Lysosomal Storage Disorders: Valentine’s Day FDA Approval

February 15, 2014

This is the fifth Blog Post in a series that will examine Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post reviews yesterday’s FDA approval of Biomarin Pharmaceutical’s orphan drug Vimizim (Elosulfase Alfa) for the treatment of LSD Mucopolysaccharidosis Type IV A (Morquio A syndrome). The late FDA approval on Valentine’s Day, ends the week on a high note, after the Lysosomal Disease Network’s 10th Annual WORLDSymposium. Vimizim is an Enzyme Replacement Therapy (ERT) for Morquio A Syndrome.

Many were tracking the FDA website to see the outcome of the 02/14/14 scheduled decision on Chelsea Therapeutics’ orphan drug Northera (Droxidopa), when late on Valentine Day’s, the FDA approves Biomarin’s orphan drug. Chelsea Therapeutics releases an announcement that the 02/14/14 FDA PDUFA Date has been extended to the next business day, 02/18/14, due to severe weather conditions. The FDA’s February 14th approval comes 2 weeks before the scheduled PDUFA Date :


Generic    Name Trade       Name Sponsor Indication 2014       PDUFA  Date
Droxidopa * Northera Chelsea   Therapeutics Symptomatic     Neurogenic Orthostatic Hypotension For Patients With Primary Autonomic     Failure 02/14
Elosulfase Alfa ** Vimizim BioMarin   Pharmaceutical Mucopolysaccharidosis     Type IV A (Morquio A Syndrome) 02/28

* Resubmission    ** FDA Priority Review

Vimizim is the first:

•   FDA approved treatment for Morquio A Syndrome

•   Drug to receive the Rare Pediatric Disease Priority Review Voucher.

The purpose of the FDA’s Rare Pediatric Disease Priority Review Voucher Program is to “encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.” FDASIA (FDA Safety and Innovation Act) added Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C).  This provision defines the criteria for the FDA awarding of priority review vouchers to sponsors of rare pediatric disease product applications. Section 529 can be found here (reference Section 908 of FDASIA, which amends the FD&C Act by adding Section 529). The FDA  is to publish guidelines about the Rare Pediatric Disease Priority Review Voucher Program in the future.

Morquio A Syndrome is an inherited LSD that is caused by the deficiency of an enzyme, N-acetylgalactosamine-6-sulfate sulfatase or GALNS. Vimizim is to replace the missing GALNS enzyme. The deficiency in GALNS causes problems with bone development, mobility, and growth. There are about 3,000 patients with Morquio A Syndrome in the developed world.

Vimizim’s Regulatory History

•   FDA grants Orphan Drug Designation – May 2009

•   First drug to receive a voucher under FDA’s Rare Pediatric Disease Priority Review voucher program (FDASIA)

•   FDA grants Priority Review Designation – May 2013

•   Marketing application submitted to ANVISA in Brazil – July 2013 

•   French National Agency for Medicines & Health Products Safety (ANSM) grants “Autorisation Temporaire d’Utilisation de cohorte (ATU cohort)”, or Temporary Authorization for Use –  November 2013 

•   FDA Advisory Committee recommends approval – November 2013

•   FDA approval – February 2014.

Per the February 14th Biomarin Pharmaceutical Press Release, both Vimizim shipments to the distribution channels and promotion will start immediately in the United States. Marketing applications for Vimizim has been submitted in the EU, Australia, Canada, and Mexico.

Please Note: “First Pigment Analysis (5893349712)” by NASA ICE (First Pigment Analysis  Uploaded by russavia) [CC-BY-2.0] | Wikimedia Commons.

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