[January 07, 2013]

Research and Markets: BioMarin's GALNS - Enzyme replacement therapy for Morquio Syndrome A

Research and Markets (http://www.researchandmarkets.com/research/k78r8l/biomarins_galns) has announced the addition of the "BioMarin's GALNS - Enzyme replacement therapy for Morquio Syndrome A" report to their offering.

BioMarin has built a portfolio of commercially successful products for rare orphan diseases. The company's latest effort is GALNS, an enzyme replacement therapy in phase III testing for MPS IVA, a rare genetic disease. BioMarin modeled its GALNS program after Aldurazyme and Naglazyme, which BioMarin successfully developed in the last 10 years. In phase I/II testing, MPS IVA patients taking GALNS improved on several measures of clinical activity.

Howeve, the ongoing phase III study will provide the best evidence yet of clinical efficacy and safety, with implications for approvability as well as marketability. In this report, we discuss the rationale for GALNS in MPS IVA, results from BioMarin's completed phase I/II trial, and the potential outcomes in phase III with respect to approval and commercialization.

Key Topics Covered:

INTRODUCTION AND BACKGROUND

1. Morquio Syndrome A (MPS IVA)

- Clinical picture

- Epidemiology

- Current treatments

2. BioMarin's GALNS enzyme replacement therapy for MPS IVA

- Phase I/II

- Phase III

CLINICAL AND REGULATORY DISCUSSION

1. ERT is a proven treatment for lysosomal storage diseases

2. Preclinical studies with GALNS

3. Phase I/II provide preliminary evidence of activity

4. Phase III design

5. What is known about safety

6. Do neutralizing antibodies matter

7. Phase III changes in design add some uncertainty

8. How GALNS should be viewed by regulators

MARKET ISSUES

1. Enzyme replacement therapy has become a profitable niche market

2. Market opportunity depends on epidemiology and patient access

3. GALNS pricing will be driven by phase III results

4. GALNS revenue projections

SUMMARY OPINION

For more information visit http://www.researchandmarkets.com/research/k78r8l/biomarins_galns

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