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.
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.
The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered Wednesday, June 16, at the University of Florida.
The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered today (Wednesday, June 16) at the University of Florida.
The U.S. Food and Drug Administration approved Lumizyme (alglucosidase alfa) for patients ages 8 years and older with late-onset (non-infantile) Pompe disease, a rare genetic disorder.
Genzyme Corporation announced today that the FDA has granted U.S. marketing approval for Lumizyme™ (alglucosidase alfa), produced at the 4000 liter (L) bioreactor scale at its manufacturing facility in Geel, Belgium. Lumizyme is the first treatment approved in the United States specifically to treat patients with late-onset Pompe disease.
Genzyme Corp. has signed a consent decree agreeing to correct manufacturing quality violations at its Allston, Mass., manufacturing facility and will turn over to the federal government $175 million in unlawful profits from the sale of products that were made at the plant, the U.S. Food and Drug Administration announced today.
Genzyme Corp. today announced that its Board of Directors has voted to pursue several actions to increase shareholder value. The company will initiate a $2 billion stock buyback, under which $1 billion of stock will be repurchased in the near term and financed with debt. The additional $1 billion of stock will be repurchased during the next 12 months.
Genzyme Corp. today announced that the results from its Late-Onset Treatment Study have been published in today's New England Journal of Medicine. The study was undertaken to evaluate the safety and efficacy of Myozyme (alglucosidase alfa) in older children and adults with Pompe disease.
Enobia Pharma Inc. unveiled findings from the first hypophosphatasia self-reported patient survey intended to evaluate the burden of illness associated with HPP at the 2010 American College of Medical Genetics Annual Clinical Genetics Meeting.
Pathway Genomics Inc., a U.S. based genetic testing company, announced the immediate availability of its personal Genetic Health Report. This customized and innovative personal genetic health report contains information on more than 70 health conditions, including pharmacogenetics (prescription medication response), propensity for complex disease, and carrier status (pre-pregnancy health).
Guidepoint Global, LLC, a leading primary research firm, today announced the launch of their Genetic Disorder Tracker, a monthly longitudinal data release that captures commercial trends, on a worldwide basis, associated with the treatment of eight rare genetic disorders: Gaucher's disease, Fabry's disease, PKU, MPS I (Hurler Syndrome), MPS II (Hunter Syndrome), MPS VI (Maroteaux-Lamy Syndrome), Niemann-Pick disease and Pompe disease.
Genzyme Corp., a diversified, global biotechnology company, today announced fourth-quarter and full year 2009 financial results and provided 2010 guidance that reflects growth across its businesses and a focus on strengthening core areas of the company.
Repairing birth defects in the womb. Inserting a tiny laser into the mother's uterus to seal off an abnormal blood flow and save fetal twins. Advancing the science that may allow doctors to deliver cells or DNA to treat sickle cell anemia and other genetic diseases before birth.
Amicus Therapeutics announced today additional positive preliminary data from its ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease at the Lysosomal Disease Network WORLD Symposium in Miami, Florida.
Hypertrophic Cardiomyopathy (HCM) is a genetic disease that causes the heart muscle to become abnormally large. It often goes undetected and, as a result, HCM has the dubious distinction of being the leading cause of sudden death in children, young adults and athletes.
As scientists work to find new treatments for Pompe disease - the devastating genetic "villain" that drives the efforts of the main characters in the new film "Extraordinary Measures" - University of Florida researchers are hopeful that gene therapy will help patients in the late stages of the disease breathe on their own.
Genzyme Corporation announced today that it has received a June 17, 2010 PDUFA date from the FDA for its Biologics License Application for Lumizyme™ (alglucosidase alfa) seeking approval for the therapy produced at the 4000 L scale.
Amicus Therapeutics today outlined the Company's three key strategic priorities and presented a corporate outlook for 2010 at the 28th Annual J.P. Morgan Healthcare Conference.