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MPS VI: Plexcera Therapeutics’ FDA Orphan Drug Designation

January 10, 2015

Plexcera Therapeutics’ (Vero Beach, Florida) mission is to develop therapies for patients with genetic deficiencies. Plexcera Therapeutics is founded by Edward H. Schuchman, MPh, PhD, Professor of Genetics and Genomic Sciences at the Icahn School of Medicine at Mt. Sinai in New York.

On January 5, 2015, Plexcera Therapeutics receives an FDA Orphan Drug Designation (ODD) for Mucopolusaccharidosis (MPS) Type VI or Maroteaux-Lamy syndrome. MPS VI is an inherited Lysosomal Storage Disorder (LDS) that is a progressive condition causing organs and tissues to enlarge and become inflamed. MPS VI causes skeletal abnormalities and joint deformities. It is estimated to occur in 1/250,000 – 600,000 newborns.

Janssen Pharmaceuticals’ Elmiron (Pentosan Polysulfate Sodium (PPS)) is an FDA orphan designated drug (August 1985) that is approved in September 1996 for the treatment of Interstitial Cystitis, a painful bladder condition. PPS started to be studied for use in human tissue inflammation, including inflammation in Mucopolysaccharidosis (MPS) disorders. In January 2013, research is published in the peer-reviewed, open access journal PLoS One :

Schuchman, E.H., Ge, Y., Lai, A., Borisov, Y., Faillace, M., Eliyahu, E., … & Simonaro, C.M. (2013). Pentosan Polysulfate: A Novel Therapy for the Mucopolysaccharidoses. PLos ONE: 8(1): e54459.

The journal article describes the use of PPS for the treatment of MPS type VI rats and concludes that:

Based on these findings we conclude that PPS could be a simple and effective therapy for MPS that might provide significant clinical benefits alone and in combination with other therapies.

This journal article is followed by the publication in June 2014 in PloS ONE, another article that compares the once weekly subcutaneous injection of PPS versus daily, oral PPS in a rat model with MPS VI. The article concluded that “Once weekly subcutaneous administration of PPS in MPS VI rats led to equal or better therapeutic effects than daily oral administration”.

Plexcera Therapeutics FDA ODD Database Record

Generic Name: Pentosan polysulfate sodium
Trade Name: Lysosan (Tm)
Date Designated: 01-05-2015
Orphan Designation: Mucopolusaccharidosis (MPS) Type VI
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Plexcera Therapeutics, LLC 4445 North Highway A1A Suite 241 Vero Beach, FL 32963


Please Note: “Blowing Bubbles at Sunset” by JaneArt (Own work) [CC-BY-SA-3.0 or GFDL] | Wikimedia Commons.

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