VIDEO from Medialink and Shire plc: Rare Diseases Must Be Top of the Health Agenda Feb 29, 2008
Case study: Fabry Disease (also known as Anderson-Fabry Disease) is a lysosomal storage disease with an estimated birth incidence of 1 in 117,000 ... Due to the varied nature of the symptoms and the slow progressive course of Fabry disease, there is an average delay between the onset of symptoms and diagnosis of 12 years ... Life expectancy is generally reduced by 20 years in men and 15 years in women with Fabry disease, compared with the general population. (PR Newswire)
Some question $48m aid package for drug maker Shire's Lexington expansion Feb 14, 2008
Shire's Human Genetic Therapies unit, which develops drugs to address rare medical disorders such as Fabry disease, is based in Cambridge, but needs additional office and laboratory space nearby to accommodate its rapid growth ... Shire's Human Genetic Therapies unit has two key drugs on the market to treat rare diseases: Elaprase for Hunter syndrome and Replagal for Fabry disease. (Boston Globe)
Genzyme 'bullish' on future growth Jan 9, 2008
Fabrazyme, a treatment for Fabry disease, increased 18 percent to $114 million. Genzyme's drugs for rare hereditary disorders treat illnesses caused by a deficiency of certain enzymes needed for critical bodily functions. (Boston Globe)
Beacon Hill leaders promise incentives to Shire Dec 21, 2007
Shire makes several drugs, including Elaprase for Hunter syndrome and Replagal for Fabry disease. As part of the expansion project, the company would move most of its Cambridge operations to Lexington. (Boston Globe)
Amicus Therapeutics to Present at the Lazard Capital Markets Fourth Annual Healthcare Conference Nov 21, 2007
Amicus has two product candidates in Phase II clinical trials, Amigal(TM) for the treatment of Fabry disease and Plicera(TM) for the treatment of Gaucher disease. The Company announced positive data from Phase I clinical trials of AT2220 for the treatment of Pompe disease in October 2007. (PR Newswire)
Profit soars tenfold at Genzyme Corp. Oct 25, 2007
The medicines include Fabrazyme for Fabry disease and Aldurazyme for a genetic condition known as MPS1. Each drug costs an average $200,000 a year per patient, except Myozyme, which costs about $300,000. (Boston Globe)
Shire deal might create 650 jobs Sep 20, 2007
Another of the company's drugs, Replagal, is not yet approved for use by the federal Food and Drug Administration for Fabry disease, a rare genetic disorder causing similar problems. Connie Paige can be reached at. (Boston Globe -- Business)
Genzyme to begin $150m expansion of Allston plant Sep 15, 2007
It produces several drugs to treat rare diseases: Cerezyme for Gaucher disease, Fabrazyme for Fabry disease, and Myozyme for Pompe disease. The plant is also used to package two other products, Aldurazyme, which treats MPS I disease, a genetic disorder; and Thyrogen, a thyroid cancer drug. (Boston Globe)
Combination Therapy Stops Loss Of Kidney Function In Rare Genetic Disease Jul 28, 2007
A combination of two types of blood pressure-lowering drugs--an angiotensin-converting enzyme inhibitor (ACEI) plus an angiotensin-receptor blocker (ARB), added to enzyme replacement therapy (ERT) with agalsidase-beta (Fabrazyme, Genzyme Corporation, Cambridge, MA)--is the first treatment shown to stop progressive loss of kidney function in patients with severe kidney involvement due to the rare genetic disorder Fabry disease, reports a study in the September Journal of the American Society of... (Science Daily)
Genzyme: Geared for Growth Jun 12, 2007
Other marketed ERT products include Fabrazyme, which was approved in the U.S. and Europe for the treatment of Fabry disease, a rare genetic disorder. Fabrazyme sales were $359 million in 2006, up 18% over 2005. (BusinessWeek)
Biotech firms seek space for manufacturing Mar 12, 2007
One of them treats Fabry disease, which causes circulatory problems, pain in the extremities, and kidney breakdown. Another treats children with Hunter syndrome, a rare and often fatal disorder that causes bulging features, growth problems, mental retardation, and serious organ damage. (Boston Globe)
Brandeis and Brigham and Women's Hospital license technology for Gaucher's to Amicus Therapeutics Feb 23, 2007
Amicus is currently conducting Phase 2 clinical trials for its lead compound, Amigal, for Fabry disease, has completed Phase 1 clinical trials of AT2101 for Gaucher disease and is conducting Phase 1 clinical trials of AT2220 for Pompe disease. . (EurekAlert! -- Business News)
Genzyme expects 2007 profit to rise on drug sales Jan 10, 2007
Fourth-quarter sales of Fabrazyme, the company's drug to treat Fabry disease, which causes blood fats to build up in blood vessels, rose 18 percent to $96 million. Sales of the company's biggest drug, Cerezyme, a treatment for Gaucher disease, which causes enlargement of the liver and spleen, rose 13 percent to $262 million. (ABC News -- Wire)
National drug plan could cost up to $$10-billion Sep 21, 2006
On the topic of expensive drugs for rare diseases, for instance, the task force says it will develop a framework based on on-going findings of a cost-shared research study of patients with Fabry disease to provide equitable access to proven drug therapies. On creating of a common list of approved drugs, the report recommends that the Common Drug Review, the national program that provides information to provincial drug plans, be expanded to include existing drugs that have new uses. (Globe and Mail)
FDA approves Hunter syndrome treatment likely to cost $300,000 per patient per year Jul 25, 2006
Cambridge has emerged as a capital for such ``ultra-orphan" drugs. Genzyme's success is built on Cerezyme, an enzyme replacement for a genetic disorder called Gaucher disease. It costs $200,000 or more annually for each patient. Cerezyme is used by about 4,500 patients worldwide and is expected to notch $1 billion in sales this year. Genzyme makes two related drugs for even rarer diseases, and sells a fourth developed by the California company . All are sold at similar prices, which vary by the... (Boston Globe)
Genzyme stock climbs on strong earnings Jul 13, 2006
Personal Tech Markets Your Money Technology Healthcare Columnists Latest news Message Boards. Genzyme stock leaps as profit tops forecast. (Boston Globe)
IPOs to Approach with Caution Jul 6, 2006
The company's lead product, a treatment for Fabry disease, a disorder in which the body cannot properly break down lipids, is in a midstage clinical trial, a process that will likely last several more years. Its proposed therapies for Gaucher disease and Pompe disease, both of which are also enzyme deficiencies, are less advanced in their development. (BusinessWeek)
Ottawa pact provides drugs to fight Fabry disease Jun 16, 2006
Federal Health Minister Tony Clement announced a $100-million agreement late yesterday that will provide two extraordinarily expensive drugs to those with Fabry disease, a rare disorder. Patients -- some for the first time -- will be able to access the drugs licensed to treat Fabry disease so long as they participate in a research protocol, part of a three-year agreement reached among provincial, federal and territorial governments and two drug companies ... An estimated 5,000 people worldwide,... (Globe and Mail)
The chosen ones Jun 4, 2006
One of his patients recently used PGD to test for Fabry disease, an inherited disorder in which the body cannot produce enough of an enzyme needed to clear cells in the body of fatty substances, especially in blood vessels and the kidney, heart, skin, and brain. "I had a family member die of the disease and I didn't want to go through that again," says the woman, who does not want to be identified. (Toronto Star -- Life)
Early stroke cause 'discovered' May 30, 2006
Scientists in Rostock found 4% of over 700 people aged 18 to 55 years who had a stroke also had Fabry disease ... Fabry disease is caused by a missing or faulty enzyme needed by the body to process oils, waxes, and fatty acids ... Researchers from the University of Rostock carried out genetic screening of over 700 adults suffering from unexplained stroke to see if they had Fabry disease. (BBC News -- Health)
Quaker BioVentures-backed company planning IPO May 18, 2006
Amicus' lead compound, Amigal, is in midstage testing for the treatment of Fabry disease, an inherited fat storage disorder caused by a deficiency of an enzyme involved in the body's biodegradation of lipids -- a type of fat. Quaker BioVentures participated in the company's $55 private stock placement last year by way of the Garden State Life Sciences Venture Fund, which is funded by the and managed by Quaker. (Philadelphia Business Journal, PA)
Provinces, Ottawa agree to drug therapy for rare illness May 13, 2006
Canadians with Fabry disease many of whom have no access to drug therapy will finally be able to obtain two staggeringly expensive medications under a new government deal ... "Federal and provincial governments and two drug developers are about to enter into a three-year funding agreement to support an independent post-market study of enzyme replacement therapies for Fabry disease," said a draft patient fact sheet from the federal and provincial governments dated May 11 and obtained by The Globe... (Globe and Mail)
Gift of life: Sister's kidney donation frees Stamford man from dialysis Apr 26, 2006
In 2004, he was diagnosed with Fabry disease, a genetically linked disorder that prevents lipids (a group of organic compounds) from being metabolized in the body. The ailment created a harmful buildup of lipids in his kidneys. (Greenwich Time)
Sales of PCs, iPods bolster Apple Apr 20, 2006
8 million, but sales of Fabrazyme, a treatment for the rare genetic disorder Fabry disease; Synvisc, for osteoarthritis of the knee; and Hectorol, for chronic kidney disease, were lower than expected. (Reuters). (Boston Globe -- Business)
Genzyme's Revenue Set to Climb Jan 10, 2006
The kidney disease drug Renagel saw a 12% increase in sales to $111 million, while the Fabry disease treatment Fabrazyme had a sales increase of 27% to $81 million. Sales of the thyroid drug Thyrogen were up 18% to $21 million. (TheStreet.com)
Medicare expands tier pricing system Jan 7, 2006
Posted 1/4/2006 1:12 AM Updated 1/4/2006 3:30 AM. In the private sector, some employers' insurance plans charge more for some so-called "lifestyle" drugs, such as impotence treatment Viagra or some allergy remedies. (USA Today -- Health)
