Content about Gaucher's disease

October 2, 2012
Genzyme drug for Gaucher disease reduces enlarged spleen size in trial

An experimental drug for treating a rare genetic disease appears effective, according to a late-stage clinical trial.

CAMBRIDGE, Mass. — An experimental drug for treating a rare genetic disease appears effective, according to a late-stage clinical trial.

May 3, 2012
Orphan drugs to see growth through 2018, report finds

Drug makers could strike big with new treatments for rare diseases, according to a new report by a market research firm.

NEW YORK — Drug makers could strike big with new treatments for rare diseases, according to a new report by a market research firm.

May 2, 2012
FDA approves Pfizer, Protalix drug for Gaucher disease

The Food and Drug Administration has approved a new treatment for a rare genetic disorder that affects 10,000 people worldwide.

NEW YORK — The Food and Drug Administration has approved a new treatment for a rare genetic disorder that affects 10,000 people worldwide.

Pfizer and Protalix BioTherapeutics announced Wednesday the approval of Elelyso (taliglucerase alfa), an enzyme-replacement therapy for the long-term treatment of Type 1 Gaucher disease.

November 23, 2011
Shire seeks approval to make Gaucher disease drug at new plant

British drug maker Shire is looking to make a biotech drug for a rare genetic disorder at a new manufacturing plant in Massachusetts.

LEXINGTON, Mass. — British drug maker Shire is looking to make a biotech drug for a rare genetic disorder at a new manufacturing plant in Massachusetts.

The company said Tuesday that it had filed with the Food and Drug Administration and the European Medicines Agency for approval to produce the Gaucher disease drug Vpriv (velaglucerase alfa) at its Lexington, Mass., plant. The company expects to win approval to make the drug there early next year.

April 7, 2011
Pfizer, Zacharon enter collaboration

Pfizer is partnering with San Diego biotech company Zacharon Pharmaceuticals to develop treatments for rare genetic disorders Zacharon said Thursday.

SAN DIEGO — Pfizer is partnering with San Diego biotech company Zacharon Pharmaceuticals to develop treatments for rare genetic disorders Zacharon said Thursday.

Under the collaboration, which could garner Zacharon up to $210 million, the companies will discover and develop pharmaceutical drugs for treating lysosomal storage disorders, a class of illnesses that includes Fabry disease, Gaucher disease, Pompe disease and Tay-Sachs disease.

February 25, 2011
Protalix receives CRL for Gaucher disease treatment

The Food and Drug Administration has declined to approve a biologic treatment for a rare genetic disorder by Israeli drug maker Protalix Biotherapeutics, Protalix said Friday.

KARMIEL, Israel — The Food and Drug Administration has declined to approve a biologic treatment for a rare genetic disorder by Israeli drug maker Protalix Biotherapeutics, Protalix said Friday.

The company said the FDA issued a complete response letter for its application for taliglucerase alfa, a treatment for Gaucher disease. The FDA issues a CRL when it has finished reviewing a regulatory application, but questions remain that preclude final approval.

July 7, 2010
Genzyme, Hospira reportedly ink packaging deal

Generic drug maker Hospira will fill and package several drugs for Genzyme, according to published...

May 23, 2010
Genzyme to correct manufacturing quality violations at Mass. plant

Biotech company Genzyme will pay $175 million to the federal government as part of an...

February 25, 2010
FDA approves Shire's Type 1 Gaucher disease

The Food and Drug Administration has approved a new drug for treating a rare genetic...