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MPS: In The News

January 28, 2015



This Blog Post will review recent developments for the rare disease Mucopolysaccharidosis (MPS).

I – Shire’s FDA Fast Track Designation For MPS II (Hunter Syndrome)

Shire announces January 26th that the company’s investigational product, SHP609 (Idursulfase-IT), is granted FDA Fast Track designation for the neurocognitive decline associated with MPS II (Hunter Syndrome). The product receives FDA Orphan Drug Designation (ODD) in September 2009. SHP609 is for direct administration into the cerebrospinal fluid via an intrathecal drug delivery device (IDDD) and is being developed for use with Shire’s drug Elaprase (Idursulfase), which is FDA approved for Hunter Syndrome. Elaprase is intravenously administered (ERT) and doesn’t cross the blood-brain barrier in any relevant amounts.

Hunter Syndrome is a progressive, rare, and severe Lysosomal Storage Disorder (LSD), caused by inadequate activity of the enzyme that breaks down complex sugars – Iduronate-2-Sulfatase (IDS).

A Shire Phase II/III pivotal trial (HGT-HIT-094 or AIM-IT) using a monthly administration of Idursulfase IT in pediatric patients is currently enrolling patients. According to Dr. Philip J. Vickers, Head of Shire R&D:

“This is not only the first treatment being investigated to address the significant unmet need of slowing the cognitive decline in MPS II patients, but also the furthest an intrathecal program for enzyme replacement has ever progressed.”                   

FDA ODD Database Record For Idursulfase-IT

Generic Name: idursulfase IT
Trade Name: n/a
Date Designated: 09-03-2009
Orphan Designation: For treatment of neurocognitive symptoms associated with Hunter Syndrome
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Shire Human Genetic Therapies 300 Shire Way Lexington, MA


II – Synageva BioPharma’s FDA Fast Track Designation For MPS IIIB (Sanfilippo B Syndrome)

Synageva BioPharma announces January 26th that the FDA has granted investigational product, SBC-103, Fast Track Designation for MPS IIIB (Sanfilippo B Syndrome). The company also announces that it has begun dosing patients with SBC-103 for a Phase I/II clinical trial. SBC-103 receives FDA ODD in April 2013.

Per the January 26th Press Release:

“MPS IIIB is a rare disease caused by a decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfate (HS) in the brain and other organs. The accumulation of abnormal HS in multiple cells and tissues leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death. SBC-103 is a recombinant form of natural human NAGLU designed to replace the missing (or deficient) NAGLU enzyme.”

FDA ODD Database Record For Synageva BioPharma

Generic Name: Recombinant human alpha-N-acetylglucosaminidase
Trade Name: n/a
Date Designated: 04-15-2013
Orphan Designation: Treatment of mucopolysaccharidosis IIIB (Sanfilippo B syndrome)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Synageva BioPharma Corp. 33 Hayden Avenue Lexington, MA


III – Vote By FDA’s Advisory Committee On Heritable Disorders In Newborns/Children For MPS I (Hurler Syndrome)

At the upcoming February 12 – 13 meeting of the FDA’s Advisory Committee On Heritable Disorders In Newborns And Children (DACHDNC), there will be a vote to recommend or not recommend to the Secretary, the addition of MPS I to the newborn screening’s Recommended Uniform Screening Panel (RUSP).

The meeting is open to the public and one can either attend in-person or can view the meeting via webinar. Further information about the Agenda and registration can be found on the Advisory Committee’s website.


The chart below shows FDA ODDs for MPS since January 2014 in FDA ODD chronological date order:

Row Num Generic Name Sponsor Company FDA ODD Date Indication
1 Recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase Abeona Therapeutics 04.29.14 MPS III A (Sanfilippo Syndrome Type A)
2 Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase Abeona Therapeutics 04.30.14 MPS III B (Sanfilippo Syndrome Type B)
3 Chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 BioMarin Pharmaceutical 11.25.14 MPS III B (Sanfilippo Syndrome Type B)
4 Ataluren (Translarna) PTC Therapeutics 12.10.14 MPS I (Hurler Syndrome)
5 Lysosan ™ (Pentosan polysulfate sodium) Plexcera Therapeutics 01.05.15 MPS VI


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

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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