BioMarin Update for the Pompe Community

BioMarin Update for the Pompe Community

BioMarin greatly appreciates the many patients, families, and physicians who have participated in our Pompe program, as well as your ongoing support of the entire patient community. After much deliberation, BioMarin has decided to discontinue the clinical development of the BMN 701 (reveglucosidase alfa) Pompe program.

Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease

BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J.P. Morgan Annual Healthcare Conference

SAN RAFAEL, Calif., Jan. 11, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced interim results from INSPIRE, a Phase 2 trial for reveglucosidase alfa, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) being studied for the treatment of late-onset Pompe disease (LOPD).

Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease

Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that its investigational new drug (IND) application, submitted to U.S. Food and Drug Administration (FDA), is now effective which allows Amicus to begin site initiation and enrollment of a Phase 1/2 study of ATB200 in patients with Pompe disease.

Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease

Update from Genzyme on neoGAA Pompe clinical development program 11th February 2014

Genzyme is in the early stages of investigating a next generation enzyme replacement therapy (neoGAA) for Pompe disease. It differs from the original ERT, whereby carbohydrates are bonded onto the enzyme during the complex manufacturing process of neoGAA. These carbohydrates are thought to help the enzyme enter the muscles more efficiently. Though it’s still very early in the development process and there are many unknowns, the hope is that through this more efficient uptake into the muscle cell, neoGAA might improve on the effectiveness of the existing therapy.

Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease

Amicus Therapeutics Announces Positive Preliminary Results From Ongoing Phase 2 Chaperone-Enzyme Replacement Therapy (ERT) Co-Administration Study for Pompe Disease

CRANBURY, N.J., June 26, 2012 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), today announced positive preliminary results from an ongoing Phase 2 open-label drug-drug interaction study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) for Pompe disease.