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Lysosomal Storage Disorders: Orphan Drugs And Mucopolysaccharidosis

February 3, 2014

Lysosomal Storage Disorders (LSDs) are attracting a lot of interest from investors, scientists, researchers, patients and their families, with the approach of next week’s Lysosomal Disease Network’s 10th Annual WORLDSymposium. The symposium is scheduled for February 10 – 14, 2014, in San Diego, California.

This is the first Blog Post in a series over the next week that will examine LSDs in the rare disease and orphan drug space. This Blog Post reviews orphan drugs for Mucopolysaccharidosis (MPS), part of the LSD group.

LSDs are a group of rare inherited metabolic disorders that are usually progressive, resulting in a patient’s debilitating health. LSDs are made up of over 40 disorders, where each disorder is caused by a deficiency of a specific enzyme that is responsible for the metabolism of lipids, glycoproteins, or mucopolysaccharides. Several distinct clinical types and numerous subtypes of MPS have been identified  based on the name of the enzyme deficiency :

MPS I H Hurler a-L-Iduronidase
MPS I S Scheie a-L-Iduronidase
MPS I H-S Hurler-Scheie a-L-Iduronidase
MPS II Hunter Iduronate sulfatase
MPS III A Sanfilippo A Heparan N-sulfatase
MPS III B Sanfilippo B a-N-Acetylglucosaminidase
MPS III C Sanfilippo C Acetyl CoA: a-glycosaminide acetyltransferase
MPS III D Sanfilippo D N-Acetylglucosamine 6-sulfatase
MPS IV A Morquio A Galactose 6-sulfatase
MPS IV B Morquio B B-Galactosidase
MPS VI Maroteaux-Lamy(arylsulfatase B) N-Acetylgalactosamine 4-sulfatase
MPS VII Sly B-Glucuronidase
MPS IX Hyaluronidase.

FDA ODD Approvals For MPS

Since the enactment of the US Orphan Drug Act in 1983, only 3 orphan drugs receive Orphan Drug Designation (ODD) for MPS:

Row Num

Generic/Trade  Names

Sponsor Company

FDA Approval   Date

Indication

1

                                                                      Laronidase/Aldurazyme BioMarin Pharmaceutical 04.30.03 MPS I

2

     N-acetylgalactosamine-4-sulfatase,   recombinant human/Naglazyme BioMarin Pharmaceutical 05.31.05 MPS VI

3

     Idursulfase/Elaprase Shire 07.24.06 MPS II

.

2013 FDA ODD For MPS

The FDA grants 4 ODDs for MPS in 2013:

Row Num

Generic Name

Sponsor Company

FDA ODD Date

Indication

1

recombinant human Naglu-   insulin-like growth factor II Shire 03.05.13 MPS III B

2

recombinant human   alpha-N-acetylglucosaminidase Synageva BioPharma 04.15.13 MPS III B

3

adeno associated viral vector   serotype rh.10 carrying the human SGSH and SUMF1 cDNAs Lysogene 05.06.13 MPS III A

4

HIRMAb-IDS ArmaGen Technologies 05.15.13 MPS II

.

Upcoming FDA PDUFA Dates For MPS

This month there is a FDA PDUFA Date for MPS:

Row Num

Generic/Trade Names

Sponsor Company

FDA PDUFA Date

Indication

1

Elosulfase   Alfa/Vimizim BioMarin Pharmaceutical 02.28.13 MPS IV A

.

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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