FDA won’t give its OK until Genzyme corrects deficiencies Nov 17, 2009
In perhaps the most significant new steps, Genzyme said it is transferring some finishing operations from Allston to outside contractors, at least temporarily, and flying more drums of Cerezyme - which treats Gaucher disease, another genetic disorder - to its state-of-the-art fill finishing plant in Waterford, Ireland. That plant already handles the finishing for most Cerezyme shipped to patients in Europe and the United States. (Boston Globe)
More contamination troubles for Genzyme Nov 14, 2009
Cerezyme treats Gaucher disease, which causes fatty substances to accumulate in the liver, spleen, and other organs ... Linda Rubenstein, a Gaucher disease patient in Carlisle, said she hadn t heard about the new contamination. (Boston Globe)
FTSE 100 Posts Biggest Weekly Drop Since March; Fresnillo, Kazakhmys Fall Oct 31, 2009
Shire Plc gained the most in three months in London trading after the U.K. drugmakers earnings beat analyst targets and the company affirmed prospects for its treatment for the rare genetic disorder Gaucher disease. which oversees the oldest U.K. investment fund, gained 0. (Bloomberg -- UK)
Shire Shares Gain in London on New Drug's Prospects Amid Genzyme Shortage Oct 31, 2009
Oct. 30 (Bloomberg) -- gained the most in six months in London trading after the U.K. drugmakers earnings beat analyst targets and the company affirmed prospects for its treatment for the rare genetic disorder Gaucher disease ... The drug, velaglucerase alfa, was rushed to the market a year earlier than planned after a manufacturing disruption in June prompted regulators to halt output of s competing Gaucher disease treatment, Cerezyme. (Bloomberg -- UK)
Inventive Approach May Improve Enzyme Replacement Therapy For Fabry Disease Oct 25, 2009
24, 2009) A new study uses a creative structure-based remodeling strategy to design a therapeutic protein that exhibits significant advantages over currently available treatments for a rare disease that often leads to cardiac and renal failure. The research, published by Cell Press on October 22nd in the American Journal of Human Genetics, describes a new and highly promising candidate for enzyme replacement therapy (ERT) for Fabry disease. (Science Daily)
Gene Behind Gaucher Disease a Player in Parkinson's Oct 23, 2009
WEDNESDAY, Oct. 21 (HealthDay News) -- An unprecedented worldwide study has clinched the case that the gene behind Gaucher disease, a rare neurological disorder, is also involved in Parkinson's disease ... Gaucher disease affects no more than one in 100,000 people in most populations, with an estimated 5,400 cases in the United States ... Gaucher disease develops in people who have two defective copies of a gene designated GBA. It codes for production of an enzyme that breaks down... (MEDLINEplus)
Study Conclusively Ties Rare Disease Gene To Parkinson's Oct 22, 2009
21, 2009) An international team led by a National Institutes of Health researcher has found that carriers of a rare, genetic condition called Gaucher disease face a risk of developing Parkinson's disease more than five times greater than the general public ... However, researchers say their work conclusively shows that mutations in the gene responsible for Gaucher disease are among the most significant risk factors found to date for Parkinson's disease ... "This analysis illustrates how studying... (Science Daily)
Genzyme begins Phase 3 clinical trial Oct 21, 2009
the Cambridge drug maker, said it has begun enrollment in the first of two global Phase 3 clinical trials of a potential new oral therapy for Gaucher disease, a rare genetic disorder. Genzyme currently markets Cerezyme, an injectable treatment for Gaucher disease, an enzyme deficiency in which fatty substances accumulate in the spleen, liver, lungs, bone marrow, and sometimes the brain ... In a statement yesterday, Genzyme senior vice president Dr. Geoff McDonough said: We remain very... (Boston Globe)
US stocks up as service industry improves Oct 6, 2009
The biopharmaceutical company said it won't pursue final-stage test 00001B37 ing of a drug for Gaucher disease after a study showed the treatment failed to provide ''clinically meaningful improvements. JPMorgan Chase & Co. downgraded the stock to ``neutral'' from ''overweight. (Sydney Morning Herald -- Business)
Genzyme expects shortages of two drugs to end by 2010 Sep 24, 2009
Genzyme started rationing Cerezyme, used for Gaucher disease, and Fabrazyme, for Fabry disease, after discovering the virus. Cerezyme generated $1. (Boston Globe)
Blood-brain Barrier As Therapy Delivery System Sep 22, 2009
We have taken advantage of that finding to delivery gene therapy, but we also can use this knowledge to better understand how the diseases impact other cell types such as neurons," she added. Lysosomal storage diseases are individually quite rare, but as a group they affect approximately 1 in 8,000 live births. The diseases are caused by deficiencies in enzymes that break down larger molecules. Without these enzymes, the large molecules accumulate inside cells and cause cell damage and... (Science Daily)
Genzyme struggles to recover from virus Sep 21, 2009
The rationing program continues for Cerezyme, which treats Gaucher disease, and Fabrazyme, a treatment for Fabry disease ... Kline is one of about 5,700 people worldwide affected by Gaucher disease ... Because it takes so long for Gaucher disease symptoms to become obvious, Kline said he has not experienced swelling of organs or shortness of breath. (Boston Globe)
Genzyme Factory Woes May Give Shire $50 Million Windfall on Drug Shortage Sep 11, 2009
1 billion market for Gaucher disease treatments by 2012 ... Gaucher disease is an inherited disorder caused by low levels of an enzyme responsible for breaking down a fatty substance that can build up in the spleen, liver and brain, according to the. (Bloomberg)
Analyst Picks and Pans: Fannie Mae, Freddie Mac, Genzyme Sep 1, 2009
Shares of Genzyme Corp. fell in premarket trade on Aug. 31 after Robert W. Baird analyst Christopher J. Raymond said more potential manufacturing delays could cut into sales of Cerezyme, a drug that treats Gaucher disease, an enzyme disorder that can result in liver and neurological problems. In addition to downgrading the shares, Raymond cut his price target to $58 from $64. (BusinessWeek)
Shire Completes Submission of NDA for Velaglucerase Alfa for Type 1 Gaucher Disease and Reports Positive Results for Remaining Two Phase III Trials Sep 1, 2009
CAMBRIDGE, Massachusetts, September 1 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: ), the global specialty biopharmaceutical company, today reported that it has completed its submission of a New Drug Application (NDA) for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease, with the U.S. Food and Drug Administration (FDA) ... "The submission of the NDA for velaglucerase alfa, earlier than previously announced, is an important... (PR Newswire)
Inspectors find major flaw at Genzyme plant Sep 1, 2009
The shutdown of the 13-year-old plant caused a shortage of Cerezyme, a drug to treat Gaucher disease, and of Fabrazyme, for Fabry disease ... Shire PLC and Protalix BioTherapeutics Inc. are both developing rival drugs for Gaucher disease. (Boston Globe)
Cerezyme option to get fast-track review at FDA Aug 26, 2009
NEW YORK - Protalix BioTherapeutics Inc. said yesterday that US regulators will review its experimental treatment for Gaucher disease on a fast-track basis and it aims to have data from a late-stage trial of the drug by October ... In patients with the rare Gaucher disease, organs such as the spleen and liver become enlarged due to the lack of an enzyme needed to break down fats. (Boston Globe)
Let the race begin Aug 21, 2009
Its Cerezyme treatment for the ultra-rare Gaucher disease, which can cost $200,000 or more annually for each patient, is in short supply due to manufacturing problems at the company s Allston Landing plant. So regulators will permit doctors to prescribe two other yet-to-be approved treatments for patients who would otherwise be stuck rationing Cerezyme until Genzyme can prove it has beaten the virus found in a bioreactor two months ago. (Boston Globe)
2d Cerezyme rival available in trials Aug 18, 2009
For the second time in two weeks, federal regulators have moved to counter the ongoing supply shortage of Cerezyme, Genzyme Corp. s treatment for Gaucher disease, by letting doctors prescribe a rival drug before it has won approval for commercial use ... Earlier this month, the FDA approved a similar plan for velaglucerase alfa, another rival Gaucher disease treatment that is in clinical trials ... Both moves were prompted by the supply shortage of Cerezyme, the market-leading drug for Gaucher... (Boston Globe)
FDA seeks contingency details from Genzyme Aug 13, 2009
The disclosure on July 31 that inspectors will revisit the Allston plant was followed on Aug. 3 by news that the FDA had given doctors approval to prescribe a rival drug to Cerezyme, the Genzyme treatment for Gaucher disease, though the competing drug developed by Shire PLC has not been approved for commercial use. Those developments sent Genzyme s shares down more than 10 percent, though they have recovered much of that loss. (Boston Globe)
Genzyme to discard drug’s raw materials Aug 11, 2009
The move will prolong the disruption in supplies of Cerezyme for thousands of Gaucher disease patients across the country who depend on the drug. The company has issued conservation guidelines to ration doses for less vulnerable patients and preserve full doses for patients under 18 and those suffering from the most severe versions of Gaucher disease, a rare enzyme deficiency condition ... Genzyme s efforts to resume drug shipments are being watched closely by many of the roughly 5,700 people... (Boston Globe)
Lack of treatment options boosts drug Aug 4, 2009
plc is reporting that the will allow the company to distribute its treatment for Gaucher disease before FDA approval, to blunt the effect of a shortage of the only drug on the market, Genzyme s Cerezyme ... Shire officials say they are conducting the largest and most comprehensive set of Phase III clinical trials conducted to date for Gaucher disease ... The first trial in the Phase III program to be completed was a multicenter, randomized, double-blind, two dose study of velaglucerase alfa in... (Philadelphia Business Journal, PA)
Genzyme rival gets positive test results Aug 4, 2009
British drug maker Shire PLC stepped up the pressure on Cambridge biotechnology company yesterday, reporting positive test results for a competitor to Genzyme s drug for Gaucher disease and disclosing that US regulators will let doctors prescribe the rival drug before it is approved for commercial use ... Shire s competing drug, velaglucerase alfa, could be administered to some Gaucher disease patients in a matter of weeks, following Food and Drug Administration approval of a company plan for... (Boston Globe)
Shire Reports Positive Results From First of Three Phase III Trials of velaglucerase alfa for Type 1 Gaucher Disease and Provides Important Updates on Interactions With FDA Aug 3, 2009
LEXINGTON, Massachusetts, August 3 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: ), the global specialty biopharmaceutical company, today reported positive results from the first of three Phase III studies of velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease ... Shire's velaglucerase alfa program is the largest and most comprehensive set of Phase III clinical trials conducted to date for Gaucher disease ... The first trial in... (PR Newswire)
Genzyme says FDA will reinspect Allston plant Aug 1, 2009
The plant produces the drugs Cerezyme to treat Gaucher disease and Fabrazyme to treat Fabry disease, both enzyme deficiency disorders. We are reinspecting as part of our normal procedures, FDA spokeswoman Karen Riley said in a statement yesterday. (Boston Globe)
Shire Receives Fast Track Designation for velaglucerase alfa for Gaucher Disease Jul 16, 2009
LEXINGTON, Massachusetts, July 16 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: ), the global specialty biopharmaceutical company, announces it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Type I Gaucher disease ... On July 6th, Shire announced that it filed a treatment protocol for velaglucerase alfa at the request of the FDA, which if accepted would allow... (PR Newswire)
Genzyme patients not negatively affected by production halt, they say Jul 10, 2009
The plant manufactures Cerezyme, a drug for Gaucher disease, and Fabrazyme, which treats Fabry disease. Both are genetic enzyme disorders that allow wastes to build up in the body, swelling organs and causing other problems. (Boston Globe)
FDA moves to speed access to Gaucher drugs Jul 7, 2009
The Food and Drug Administration is moving to speed up access to experimental drugs for Gaucher disease to help offset likely shortages of Cerezyme, the top-selling drug made by ... Cambridge-based Genzyme, which makes Cerezyme, the world s leading treatment for Gaucher disease, recently said it would shut down its plant in Allston after a virus halted production of Cerezyme and Fabrazyme, its treatment for Fabry disease, another rare condition. (Boston Globe)
Genzyme drugs could be rationed for longer Jun 26, 2009
Cerezyme treats Gaucher disease, while Fabrazyme treats Fabry disease ... In general, for Cerezyme, doctors on both continents are being asked to give priority to children, adolescents, and adults with active Gaucher disease progression, while other adult patients will get half their normal doses every two weeks or their normal doses every four weeks until regular shipments can be resumed this fall ... Carrie Ostrea, 40, a Texas mother of a 10-month-old daughter with Gaucher disease, said she... (Boston Globe)
After a virus invades, Genzyme scrubs down Jun 25, 2009
Cerezyme treats Gaucher disease, while Fabrazyme treats Fabry disease. Both conditions cause waste to build up in the body, swelling organs. (Boston Globe)
Genzyme's story riveting, sad Jun 21, 2009
Linda Rubenstein takes the Genzyme drug Cerezyme to treat Gaucher disease. (Josh Reynolds for The Boston Globe) June 21, 2009. (Boston Globe)
Genzyme puts the squeeze on developing countries Jun 21, 2009
Should drugs for rare diseases, such as Gaucher disease, be considered essential medicines, making governments responsible for their provision. Latin American court cases have increasingly enforced government provision of essential medicines, regardless of price. (Boston Globe -- Editorial)
Genzyme sees outside vendor as source of plant contamination Jun 20, 2009
The conditions, Gaucher disease and Fabry disease, cause waste to build up in parts of the body, swelling organs and causing a litany of problems. The treatments, which are given intravenously about every two weeks, are extremely expensive - about $200,000 a year per patient. (Boston Globe)
Framingham Genzyme operations OK despite Allston closing Jun 19, 2009
Cerezyme treats Gaucher disease, an enzyme disorder that can result in liver and neurological problems. At its 51 New York Ave. facility in Framingham, Genzyme produces smaller batches of Fabrazyme and another drug, Myozyme. (Framingham TAB, MA)
How Cerezyme worked for Costa Rican girl Jun 18, 2009
When the results came back, they came with an answer: Tania had Gaucher disease ... Gaucher disease is so rare that a country like Costa Rica might have only a handful of cases; fewer than 10,000 of the world's 6 billion people are known to have it ... And with the only effective treatment for Gaucher disease, Genzyme never needed to lower the price, even as production efficiencies raised profit margins on the drug to as much as 90 percent. (Boston Globe)
Genzyme plight leaves patients uneasy Jun 18, 2009
Linda Rubenstein, who takes a Genzyme drug to treat Gaucher disease, said she would not have been able to have her son, Zach, (left) without the medication ... The drug, Cerezyme, combats Hannah s severe form of Gaucher disease, a rare genetic disorder that caused her liver and spleen to swell ... Gaucher disease causes fatty substances to accumulate in the spleen, liver, lungs, bone marrow, and - in the worse cases - the brain. (Boston Globe)
Genzyme Shuts Down Facility After Detecting Virus Jun 17, 2009
Cerezyme treats Gaucher disease, an enzyme disorder that can result in liver and neurological problems. Genzyme said it has confirmed that the virus was the cause of two previous declines in cell productivity at the facility located in the Boston neighborhood of Allston and another one in Geel, Belgium. (ABC News -- Wire)
Virus shuts Genzyme plant,holds up drugs for 8,000 Jun 17, 2009
Cerezyme is used to treat Gaucher disease, an enzyme deficiency in which fatty substances accumulate in the spleen, liver, lungs, bone marrow, and - sometimes - the brain. It can cause bruising, enlarged organs, and lung and kidney ailments. (Boston Globe)
Expert urges testing for genetic disorders May 29, 2009
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Hemophilia Center on the move May 1, 2009
Operating on a budget of about $18 million, the Hemophilia Center provides a variety of clinic-based and home-based services for individuals with bleeding disorders, such as hemophilia, Von Willebrand disease and Gaucher disease. It also provides advocacy, educational programs and research. (Buffalo Business First, NY)
Myozyme sales boost Genzyme profit Feb 12, 2009
The drugs are some of the world's costliest and include Cerezyme for Gaucher disease and Aldurazyme for a condition called MPS1. Both cost about $200,000 a year. (Boston Globe)
