Content about Inborn errors of metabolism

March 27, 2012
Hyperion buys rights to drug for rare genetic diseases

Hyperion Therapeutics has purchased rights to a drug for treating genetic metabolic disorders, the company said.

SOUTH SAN FRANCISCO, Calif. — Hyperion Therapeutics has purchased rights to a drug for treating genetic metabolic disorders, the company said.

Hyperion announced the purchase of Ravicti (glycerol phenylbutyrate) from Ucyclyd Pharma, a subsidiary of Medicis Pharmaceutical.

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.

October 28, 2010
GSK, Amicus to develop, commercialize Amigal

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.

 

 

August 29, 2010
BioMarin's Pompe disease treatment gets orphan-drug designation

BioMarin Pharmaceutical on Monday announced its investigational treatment for a lysosomal storage disorder has received...

August 19, 2010
BioMarin Pharmaceutical acquires ZyStor Therapeutics

BioMarin Pharmaceutical has acquired privately owned biotechnology company ZyStor Therapeutics for $22 million, BioMarin said....

May 24, 2010
FDA approves Pompe disease treatment

The Food and Drug Administration has approved a new treatment for a rare genetic disorder...

March 17, 2010
FDA approves Carbaglu

The Food and Drug Administration has approved a drug for treating a condition that causes...