Pompe disease is a rare and often fatal muscle disease caused by an inherited deficiency of the enzyme acid alpha-glucosidase, which is responsible for breaking down glycogen within cells. Pompe disease ranges from a rapidly fatal infantile-onset form with severe cardiac involvement to a more slowly progressive late-onset form primarily affecting skeletal muscle. There is currently no therapeutic treatment available for the disease, which affects an estimated 5,000-10,000 people worldwide.
Angiochem announced today a global collaboration with GlaxoSmithKline (GSK) to discover, develop and commercialize treatments for lysosomal storage diseases.
Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement. An enzyme replacement treatment pioneered at Duke University has saved many lives, but some children with Pompe disease produce an immune reaction that blocks the benefits of the life-saving enzyme treatment.
BioMarin Pharmaceutical Inc. announced today that the Investigational New Drug (IND) application for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia is active.
BioMarin Pharmaceutical Inc. announced today that the Committee for Medicinal Products for Human Use of the European Medicines Agency has recommended approval for the company's manufacturing facility expansion in Novato, CA.
BioMarin Pharmaceutical Inc. today announced the initiation of a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA.
PerkinElmer, Inc., a global leader focused on improving the health and safety of people and the environment, announced today that its newborn screening laboratory service, PerkinElmer Genetics, has launched a new panel to screen for six Lysosomal Storage Disorders (LSDs) as an addition to its current newborn testing and diagnostics portfolio.
BioMarin Pharmaceutical Inc. today announced an update on the GALNS Phase 1/2 extension study (MOR-100) in which patients have continued treatment on an ongoing basis.
BioMarin Pharmaceutical Inc. today announced the initiation of a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with advanced hematological malignancies.
BioMarin Pharmaceutical Inc. announced today that it has entered into a definitive agreement to acquire a bulk biologics manufacturing plant from Pfizer, located in Shanbally, Cork, Ireland.
BioMarin Pharmaceutical Inc. announced today preliminary results from ADAPT (A Diversified Approach for PKU Treatment) in an abstract (Abstract #91, Mental Health Screening in Phenylketoniuria Clinic) presented by Barbara Burton, MD at the 2011 annual American College of Medical Genetics conference in Vancouver, Canada.
Amicus Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease.
Amicus Therapeutics today announced that additional positive data from the ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease will be presented at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada, February 16-18th, 2011.
BioMarin Pharmaceutical announced today that it has initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase, intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA, also called Morquio A Syndrome.
Genzyme Corporation today announced that it has completed the sale of its diagnostic products business to Sekisui Chemical Co., Ltd. for $265 million in cash.
Genzyme Corp. today announced that it will build an additional manufacturing plant in Geel, Belgium, to support the long-term growth of Myozyme® and Lumizyme® for Pompe disease.
BioMarin Pharmaceutical Inc. announced today that it has initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase in development for the treatment of Pompe disease.
The 2010 Mid-Atlantic Bio today completed another successful conference hosting 728 registrants from 22 states and 4 countries, with more than 400 companies and 50 qualified investors participating. Dedicated to promoting the biotechnology industry in the Mid-Atlantic region, the conference is hosted collectively by the region's most influential bioscience and investor associations, the Mid-Atlantic Venture Association (MAVA), the Tech Council of Maryland/MdBio (MdBio) and the Virginia Biotechnology Association (VaBIO).
BioMarin Pharmaceutical Inc. announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease. An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is expected to start in the first quarter of 2011.
BioMarin Pharmaceutical Inc. announced today that it has acquired ZyStor Therapeutics, Inc. (ZyStor), a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders. ZyStor's lead product candidate is ZC-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease.
Genzyme Corp. today reported that second-quarter revenue was $1.08 billion, compared with $1.23 billion in the same period last year, reflecting limited shipments of Cerezyme® (imiglucerase for injection) and Fabrazyme® (agalsidase beta) due to product supply constraints in 2010. The company expects to be able to increase shipments of Cerezyme and Fabrazyme during the second half of the year.