Content about Fabry disease

May 3, 2012

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.

November 23, 2011

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.

September 15, 2011

GlaxoSmithKline and Amicus Therapeutics have started a late-stage clinical trial for a drug to treat a rare genetic disorder, the companies said.

CRANBURY, N.J. — GlaxoSmithKline and Amicus Therapeutics have started a late-stage clinical trial for a drug to treat a rare genetic disorder, the companies said.

GSK and Amicus announced the start of a second phase-3 trial of Amigal (migalastat hydrochloride) for Fabry disease, a rare lysosomal storage disorder that affects between 5,000 and 10,000 people worldwide. The market for Fabry disease has long been dominated by Genzyme, a subsidiary of French drug maker Sanofi that makes the biologic drug Fabrazyme (agalsidase beta).

April 7, 2011

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.

March 7, 2011

The board of directors of Genzyme unanimously recommended that shareholders accept French drug maker Sanofi-Aventis’ $74-per-share buyout offer, Genzyme said Monday.

CAMBRIDGE, Mass. — The board of directors of Genzyme unanimously recommended that shareholders accept French drug maker Sanofi-Aventis’ $74-per-share buyout offer, Genzyme said Monday.

Genzyme recently accepted Sanofi’s $20.1 billion offer for the Cambridge, Mass.-based biotech company. Sanofi had sought to buy the company since last July, when it offered $18.5 billion, or $69 per share.

February 16, 2011

Amicus Therapeutics received good results from its mid-stage trial of a drug for a rare, genetic disorder, the drug maker said Wednesday.

CRANBURY, N.J. — Amicus Therapeutics received good results from its mid-stage trial of a drug for a rare, genetic disorder, the drug maker said Wednesday.

December 13, 2010

Biotech company Genzyme still is saying “no thanks” to French drug maker Sanofi-Aventis’ tender offer of $18.5 billion, or $69 per share, to acquire it, saying the offer “substantially undervalues” the company.

CAMBRIDGE, Mass. — Biotech company Genzyme still is saying “no thanks” to French drug maker Sanofi-Aventis’ tender offer of $18.5 billion, or $69 per share, to acquire it, saying the offer “substantially undervalues” the company.

The offer has been extended until Jan. 11, Genzyme said.

Sanofi has sought to buy Genzyme since August. Genzyme, based in Cambridge, Mass., specializes in treatments for rare genetic disorders, such as Fabry disease and Gaucher disease.

October 28, 2010

British drug maker GlaxoSmithKline will work with U.S.-based Amicus Therapeutics to develop a drug for...

CRANBURY, N.J. British drug maker GlaxoSmithKline will work with U.S.-based Amicus Therapeutics to develop a drug for a rare genetic disease.

 

The two companies announced a deal to develop and commercialize Amigal (migalastat hydrochloride), a treatment for Fabry disease. Under the deal, GSK will pay Amicus $30 million upfront, as well as milestone payments of up to $170 million and royalties on future sales.

 

 

October 7, 2010

The board of directors of Genzyme again has rejected a buyout offer by French drug...

CAMBRIDGE, Mass. The board of directors of Genzyme again has rejected a buyout offer by French drug maker Sanofi-Aventis, Genzyme said Thursday.

The biotech company’s board unanimously turned down the hostile offer Sanofi made Monday to acquire Genzyme for $18.5 billion, or $69 per share, saying it was “opportunistic” and undervalued the company.

 

August 25, 2010

Biotech drug maker Genzyme’s takeover by French drug maker Sanofi-Aventis may have run into trouble,...

NEW YORK Biotech drug maker Genzyme’s takeover by French drug maker Sanofi-Aventis may have run into trouble, according to published reports.

Bloomberg reported Wednesday that Sanofi was unwilling to raise its offer of $70 per share for Genzyme, a Cambridge, Mass.-based company that has carved a niche for itself by manufacturing treatments for such rare genetic disorders as Fabry disease and Pompe disease but lately has been dogged by manufacturing problems.

July 7, 2010

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

February 4, 2010

GlaxoSmithKline has identified more than 5,500 rare diseases it could potentially target as part of...