25 août 2009
SHIRE : Résultat de l'essai clinique de Phase III du Velaglucerase alfa pour la maladie de Gaucher de Type I
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Registered in Jersey, No. 99854, 22 Grenville Street, St Helier, Jersey JE4 8PX
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
Treatment Protocol Accepted and Rolling Submission of New Drug Application Initiated
Lexington, Massachusetts, US – August 3, 2009 – Shire plc (LSE: SHP, NASDAQ: SHPGY), 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. The Company also announced that the U.S. Food and Drug Administration (FDA) has accepted its treatment protocol for velaglucerase alfa and that Shire has begun its rolling submission of the New Drug Application (NDA) for velaglucerase alfa allowed under the Fast Track process.
“We are very pleased with the progress of the velaglucerase alfa program from both a clinical and regulatory perspective,” said Sylvie Grégoire, President of Shire Human Genetic Therapies. “This data are consistent with those previously reported from the Phase I/II and extension studies. We will continue to work diligently with the FDA and other regulatory agencies to make velaglucerase alfa available as soon as possible to help meet the needs of the Gaucher community.”
Shire’s velaglucerase alfa program is the largest and most comprehensive set of Phase III clinical trials conducted to date for Gaucher disease. Over 100 patients at 24 sites in 10 countries around the world have participated in the clinical studies.
Velaglucerase alfa is made using Shire’s proprietary technology, in a human cell line. The enzyme produced has the exact human amino acid sequence and carries a human glycosylation pattern.
Phase III Study Overview and Results
The first trial in the Phase III program to be completed was a multicenter, randomized, double-blind, two dose study of velaglucerase alfa in patients with Type 1 Gaucher disease.
The primary goal of this study was to evaluate the safety and efficacy of velaglucerase alfa in 25 patients with Type 1 Gaucher disease.
Patients aged two years and older who were treatment naïve were eligible to participate in the study if they presented with disease-related anemia and had at least one of the following clinical manifestations of Gaucher disease: thrombocytopenia, moderate splenomegaly or a readily palpable enlarged liver. Patients were randomized to receive velaglucerase alfa at either 45 U/ kg or 60 U/ kg for a duration of 12 months.
In the trial, the primary endpoint was reached with patients benefiting from a clinically important and statistically significant (p<0.0001) increase in mean hemoglobin concentration compared with baseline after receiving velaglucerase alfa at 60 U/kg IV every other week for 12 months. Statistically significant improvements compared with baselines were also observed in platelet and spleen sizes, and nominally significant improvements were observed in liver size at this dose. Results were clinically important as defined by standard criteria and consistent with the previously published Phase I/II data.
At the 45 U/kg IV dose, statistically significant improvements in hemoglobin, platelet count, and spleen volume were also demonstrated. The magnitude of changes in the 45U/kg dose was also clinically important, and a trend in liver volume reduction was observed. The 60U/kg dose performed numerically as well or better than 45U/kg across all measured clinical endpoints.
The specific data from this trial will be presented at a scientific meeting later this year.
Velaglucerase alfa was found to be generally well tolerated with no drug-related serious adverse events reported in the trial. No patients withdrew from the trial due to an adverse event.
Most of the drug-related adverse events were reported in association with velaglucerase alfa infusions, all of which were mild and resolved without sequelae.
“These findings are very encouraging. They illustrate important potential benefits that velaglucerase alfa may provide to patients who are affected by Type 1 Gaucher disease,” said Dr. Atul Mehta, Clinical Director of the Lysosomal Storage Disorders Unit, Royal Free Hospital, London. “Velaglucerase alfa appears to be an excellent choice for Type 1 Gaucher patients. The prospect of having another treatment option available to help patients achieve therapeutic goals is very important and is welcomed by both the physicians and patients.”
Regulatory Updates
With regard to ongoing interactions with the FDA, Shire provided the following important updates:
- The FDA has accepted Shire’s treatment protocol for velaglucerase alfa. The acceptance of the treatment protocol by the FDA will enable physicians to treat Gaucher patients with velaglucerase alfa prior to commercialization. Shire will initially provide velaglucerase alfa free of charge to patients who are enrolled in the protocol.
- Shire has begun a rolling submission of a New Drug Application (NDA) to the FDA for velaglucerase alfa to treat patients with Type 1 Gaucher disease. The submission was initiated on July 30, 2009, three weeks after Shire received Fast Track designation. Fast Track designation allows a company to file the sections of the NDA as they become available and enables the agency to commence its review on a rolling basis. The company expects to complete the NDA submission by the end of this quarter.
Gaucher disease is an autosomal recessive disease and the most prevalent Lysosomal Storage Disorder (LSD), with an incidence of about 1 in 20,000 live births. Despite the fact that Gaucher Disease consists of a phenotype, with varying degrees of severity, it has been sub-divided in three subtypes according to the presence or absence of neurological involvement. It is also the most common genetic disease affecting Ashkenazi Jewish people Eastern, Central and Northern European ancestry), with a carrier frequency of 1 in 10 (Dr. John Barranger and Dr. Ed Ginns 1989). This panethnic disease involves many organ systems, such as liver, spleen, lungs, brain, metabolism and bone marrow.
Gaucher Disease results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents. The disease course is quite variable, ranging from no outward symptoms to severe disability and death. Carrier status can be detected through blood or saliva to identify potential carriers of the Gaucher gene. Gaucher Disease can be diagnosed early through a blood test.
Worldwide the diagnosed population of Gaucher Disease patients is approximately 7,000.
Based on incidence, the estimated total world population is likely to be between 10,000 and 15,000 patients.
For further information please contact:
Investor Relations Cléa Rosenfeld (Rest of the World) +44 1256 894 160
Eric Rojas (North America) +1 617 551 9715
Media Jessica Mann (Rest of the World) +44 1256 894 280
Jessica Cotrone (North America) +1 617 613 4640
Notes to editors
SHIRE PLC
Shire’s strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI) diseases as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire’s in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.
For further information on Shire, please visit the Company’s website: www.shire.com.
"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT
OF 1995
Statements included herein that are not historical facts are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, the Company’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of research, development, approval, reimbursement, manufacturing and commercialization of the Company’s Specialty Pharmaceutical and Human Genetic Therapies products, as well as the ability to secure and integrate new products for commercialization and/or development; government regulation of the Company’s products; the
Company’s ability to manufacture its products in sufficient quantities to meet demand; the impact of competitive therapies on the Company’s products; the Company’s ability to register, maintain and enforce patents and other intellectual property rights relating to its products; the Company’s ability to obtain and maintain government and other third-party reimbursement for its products; and other risks and uncertainties detailed from time to time in the Company’s filings with the Securities and Exchange Commission.
Press Release
www.shire.com
Protalix : FDA approuve le "treatment protocol" pour le prGCD
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Subject: Protalix - press
release: U.S. Food and Drug Administration Approves Protalix's Treatment
Protocol for prGCD
CARMIEL, Israel--(BUSINESS WIRE)--Protalix BioTherapeutics, Inc.
(NYSE-Amex:PLX), announced today that the U.S. Food and Drug Administration
(FDA) has approved the Company’s treatment protocol for prGCD, the Company’s
proprietary plant-cell expressed recombinant form of glucocerebrosidase (GCD)
for the treatment of Gaucher disease. The treatment protocol allows physicians
and other care-providers to treat patients of Gaucher disease with prGCD in the
United States and additional countries world-wide while studies of prGCD
continue as part of the Company’s ongoing pivotal Phase III clinical trial.
Prior to accepting the protocol, the FDA reviewed available data from the
Company’s on-going Phase III clinical development programs.
The treatment protocol is a multicenter, open-label trial designed to
allow physicians and other care-providers to treat patients of Gaucher disease
with prGCD during the expected shortage of Cerezyme® and thereafter. Cerezyme®
is a mammalian cell expressed version of glucocerebrosidase and the only enzyme
replacement therapy currently approved for Gaucher disease. The treatment
protocol allows patients enrolled in the protocol to continue being treated
with prGCD until its anticipated marketing approval from the FDA. The Company
will provide the drug free of charge to patients enrolled in the protocol.
"We appreciate the guidance and vote of confidence provided by the
FDA in establishing a treatment protocol for prGCD and are working closely with
physicians and patient advocacy groups to allow Gaucher disease patients to
gain access to our drug," commented Dr. David Aviezer, the Company’s
President and Chief Executive Officer. "We expect to conclude our phase
III pivotal study next month and are looking forward to announcing top-line
results from this study in October. We anticipate filing an NDA with the FDA by
the end of this year.”
About Protalix BioTherapeutics
Protalix is a biopharmaceutical company. Its goal is to become a fully
integrated biopharmaceutical company focused on the development and
commercialization of proprietary recombinant therapeutic proteins to be
expressed through its proprietary plant cell based expression system.
Protalix’s ProCellEx(TM) presents a proprietary method for the expression of
recombinant proteins that Protalix believes will allow for the cost-effective,
industrial-scale production of recombinant therapeutic proteins in an
environment free of mammalian components and viruses. Protalix is conducting a
Phase III pivotal study for its lead product candidate, prGCD, to be used in
enzyme replacement therapy for Gaucher disease, a rare and serious lysosomal
storage disorder in humans with severe and debilitating symptoms. Protalix and
the U.S. Food and Drug Administration agreed on the final design of the pivotal
Phase III clinical trial through the FDA’s Special Protocol Assessment (SPA)
process. Protalix has completed enrollment for this study and is treating
patients in the study in North America, South America, Israel, Europe and South
Africa. The study is monitored by an independent Data Monitoring Committee,
including experts in the field, who monitor the on-going safety data, which has
recently held their last scheduled meeting before the end of the trial. No
serious adverse events have been reported in the study. Protalix is also
advancing additional recombinant biopharmaceutical drug development programs
22 août 2009
FDA : traitement SHIRE et PROTALIX
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Current Drug
Shortages
|
Drug Name |
Company |
|
Drug Name |
|
Cerezyme (imiglucerase)
Injection Updated 8/21/2009 |
Genzyme Corporation For inquiries contact
Genzyme Medical Information: medinfo@genzyme.com or 1-800-745-4447, option 2 |
Manufacturing delays |
Please see Dear Doctor
letter, Dear Patient letter, and Request Form also visit the Website: Genzyme
supply update for additional information. Please
see the following link Treatment Protocol of Velaglucerase Alfa (this is a
treatment protocol available under IND) Please see the following
link: Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase
(prGCD) in Patients With Gaucher Disease for information about prGCD, also
called Taliglucerase Alfa (this is a treatment protocol available under IND |
http://www.fda.gov/Drugs/DrugSafety/DrugShortages/ucm050792.htm#cerezyme
PROTALIX : Accés étendu à l'essai de Glucocerebrosidase (prGCD) Recombinant dans la maladie de Gaucher
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Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Expanded access is currently available for this treatment.
Verified by Protalix, August 2009
First Received: August 18, 2009 No Changes Posted
| Sponsored by: |
Protalix |
|---|---|
| Information provided by: | Protalix |
| ClinicalTrials.gov Identifier: | NCT00962260 |
Purpose
This is an open-label expanded access trial of prGCD in patients with Gaucher disease who require enzyme replacement therapy (ERT) and who have been treated with imiglucerase but for whom the dose has been reduced or discontinued due to shortage of the product.
| Condition | Intervention |
|---|---|
|
Gaucher Disease |
Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD) |
| Study Type: | Expanded Access |
| Official Title: | An Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement Therapy |
Resource links provided by NLM:
Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics: Gaucher's Disease
Drug Information available for: Alglucerase Imiglucerase
Further study details as provided by Protalix:
Intervention Details:Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
Intravenous infusion every two weeks at the dose level equal to each patient's previous imiglucerase dose before reduction or discontinuation due to shortage
Detailed Description:
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD), which have been mapped to chromosome 1 q21-q31, leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system. This accumulation leads to the visceral manifestations of hepatosplenomegaly, anemia and thrombocytopenia, as well as to the skeletal features and less frequently also to lung involvement.
prGCD is a plant cell expressed recombinant glucocerebrosidase enzyme for the treatment of Gaucher disease.
Expression of proteins in plant cell culture is highly efficient, does not require post-expression modification of the protein, and is not susceptible to contamination by agents such as viruses that are pathological to humans.
prGCD safety will be observed in this treatment protocol of patients with non-neuronopathic Gaucher disease who require enzyme replacement therapy. Eligible patients will receive intravenous (IV) infusions of prGCD every two weeks. The dose of prGCD will be equal to each patient's previous imiglucerase dose before reduction or discontinuation due to shortage. The infusions will be administered at the selected medical center.
Eligibility
| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Bot |
Criteria
Inclusion Criteria:
- Males and females, 18 years or older
- Diagnosis of Gaucher disease treated historically with imiglucerase
- Able to provide written informed consent
Exclusion Criteria:
- Currently taking another experimental drug for any condition
- History of allergy to carrots
- Presence of anti-glucocerebrosidase (GCD) antibodies
- Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions
- Allergy to beta-lactam antibiotics
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00962260
Contacts
| Contact: Raul Chertkoff, MD | +972 (4) 988-9488 | raul@protalix.com |
Protalix
More Information
Publications:
| Responsible Party: | Protalix Ltd ( Einat Brill Almon, PhD ) |
| Study ID Numbers: | PB-06-004 |
| Study First Received: | August 18, 2009 |
| Last Updated: | August 18, 2009 |
| ClinicalTrials.gov Identifier: | NCT00962260 History of Changes |
| Health Authority: | United States: Food and Drug Administration; Israel: Ministry of Health |
Keywords provided by Protalix:
|
glucocerebrosidase enzyme replacement therapy Gaucher disease plant cell culture |
splenomegaly hepatomegaly anemia thrombocytopenia |
Study placed in the following topic categories:
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Anemia Sphingolipidosis Central Nervous System Diseases Brain Diseases Lymphatic Diseases Metabolism, Inborn Errors |
Thrombocytopenia Genetic Diseases, Inborn Splenomegaly Lipidoses Brain Diseases, Metabolic, Inborn Gaucher Disease Metabolic Disorder Hepatomegaly Lipid Metabolism Disorders Brain Diseases, Metabolic |
Additional relevant MeSH terms:
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Lymphatic Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |
Contact Help Desk
Lister Hill National Center for Biomedical Communications,
U.S. National Library of Medicine,
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Copyright,
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http://www.clinicaltrials.gov/ct2/show/NCT00962260?term=prgcd
19 août 2009
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
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Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Expanded access is currently available for this treatment.
Verified by Shire Human Genetic Therapies, Inc., August 2009
First Received: August 5, 2009 Last Updated: August 6, 2009 History of Changes
| Sponsored by: | Shire Human Genetic Therapies, Inc. |
|---|---|
| Information provided by: | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00954460 |
Purpose
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this treatment protocol is to observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase.
| Condition | Intervention |
|---|---|
| Gaucher Disease, Type 1 | Drug: velaglucerase alfa |
Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher DiseaseStudy Type:
Expanded Access Official Title:
Resource links provided by NLM:
Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics: Gaucher's Disease
Drug Information available for: Alglucerase Imiglucerase
Further study details as provided by Shire Human Genetic Therapies, Inc.:
Intervention Details:
Drug: velaglucerase alfa
up to 60 U/kg, every other week via intravenous infusion
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases of Gaucher disease and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Velaglucerase alfa (Gene-Activated™ human glucocerebrosidase;GA-GCB) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. Velaglucerase alfa contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This treatment protocol will observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase.
Patients currently being treated with ERT for their Gaucher disease will receive the same number of units of velaglucerase alfa per month as their imiglucerase dose for doses between 30-120 U/kg/month. For patients who experienced dose reductions in their imiglucerase treatment due to supply constraints the pre-reduction monthly dose may be used to determine the monthly dose of velaglucerase alfa.
Eligibility
| Ages Eligible for Study: | 3 Years and older |
| Genders Eligible for Study: | Both |
Criteria
Inclusion Criteria:
- The patient has a documented diagnosis of type 1 Gaucher disease
- The patient is > 2 years of age
- The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction to another ERT including imiglucerase
- Women of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study; and must have a negative result to a pregnancy test as required throughout their participation in the study. Male patients must use a medically acceptable method of birth control throughout their participation in the study and must report their partner's pregnancy.
- The patient is sufficiently cooperative to participate in this treatment plan as judged by the Investigator
If the patient is naïve or new to treatment, the patient has one or more of the following (in absence of the following criteria, please call the sponsor for treatment justification):
- Gaucher disease-related anemia
- Moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation
- Gaucher disease-related thrombocytopenia
- Gaucher disease-related palpable enlarged liver
Exclusion Criteria: None
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00954460
Contacts
| Contact: Dan Madden (One Path) | 1-866-888-0660 |
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
More Information
No publications provided
| Responsible Party: | Shire Human Genetic Therapies, Inc. ( Gabriel M. Cohn, M.D., MBA, FACMG, FACOG ) |
| Study ID Numbers: | HGT-GCB-058 |
| Study First Received: | August 5, 2009 |
| Last Updated: | August 6, 2009 |
| ClinicalTrials.gov Identifier: | NCT00954460 History of Changes |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Shire Human Genetic Therapies, Inc.:
| Enzyme Replacement Therapy Gaucher disease glucocerebrosidase beta-glucocerebrosidase Acid beta-glucocerebrosidase |
glucosylceramidase D-glucosyl-N-acylsphingosine glucohydrolase gene activation human |
Study placed in the following topic categories:
| Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Lymphatic Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Additional relevant MeSH terms:
| Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Lymphatic Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |
ClinicalTrials.gov processed this record on August 18, 2009
13 août 2009
UPDATE 3-FDA accélére l'acces des médicaments des sociétés Protalix et Shire pour la maladie de Gaucher
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Mon Jul 6, 2009 4:08pm EDT
* FDA asks Protalix to speed access to Gaucher drug
* FDA says Protalix drug meets expanded access criteria
* FDA action to offset likely shortages of Genzyme drug
* Protalix shares up 8 percent (Updates with comment from analyst, Protalix, Genzyme)
By Toni Clarke
BOSTON, July 6 (Reuters) - The U.S. 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 Genzyme Corp (GENZ.O: Quote, Profile, Research, Stock Buzz).
Biotechnology company Protalix BioTherapeutics Inc (PLX.A: Quote, Profile, Research, Stock Buzz) and Shire Plc (SHP.L: Quote, Profile, Research, Stock Buzz)(SHPGY.O: Quote, Profile, Research, Stock Buzz) said on Monday they have been approached by the FDA to expand access to their experimental drugs for the rare but serious disorder.
Patients with Gaucher are deficient in an enzyme that breaks down a certain type of fat molecule. Fatty cells accumulate in different parts of the body, including the spleen, liver and bone marrow.
The shares of Protalix, which is based in Israel, rose 8.2 percent to close at $4.88 on the American Stock Exchange. Genzyme's shares were up 30 cents, or 0.6 percent, at $55.05. Shire stock rose 1.8 percent to end at $41.03.
Genzyme, which makes Cerezyme, the world's leading treatment for Gaucher disease, recently announced it would shut down its plant in Boston after a virus halted production of Cerezyme and Fabrazyme, its treatment for Fabry disease, another rare condition.
Protalix said the FDA asked the company to consider submitting a treatment protocol that would allow use of its treatment prGCD under an expanded access program. Under this program, a treatment protocol may be submitted for a drug that has not yet been approved, but is in development for a serious disease for which no other therapy is available.
The company is discussing the parameters of a proposed treatment protocol that would allow an increased number of patients with Gaucher disease to have access to prGCD, which is in late-stage development.
Shire said it has already filed a treatment protocol for its drug velaglucerase alfa. Both Shire and Protalix said they would provide their drugs free of charge.
David Aviezer, the chief executive of Protalix, expects approval of the treatment protocol within the next 60 days. He said the company is on track to file an application for full approval of the drug by the end of this year.
Some analysts said the FDA's action gives a good indication of how the agency views the drug.
"We view the FDA's request as clearly positive for Protalix, as it indicates FDA's comfort with prGCD's safety and efficacy data, and increases the likelihood of ultimate prGCD approval," said Brian Abrahams, an analyst at Oppenheimer & Co.
Genzyme expects its Boston plant to be on line again in late July, with shortages of Cerezyme following the depletion of inventories expected to hit in August. Shortages of Fabrazyme will likely start to be seen in October.
Cerezyme, an enzyme replacement therapy, is Genzyme's biggest-selling product and, at $200,000 a year, is one of the world's most expensive drugs. Cerezyme and Fabrazyme represented about 37 percent of the company's 2008 revenue of $4.6 billion.
"The fact that the FDA has reached out to Protalix is not unexpected," said Geoff McDonough, senior vice president of the Genetic Diseases business at Genzyme. "They would be looking at all possible avenues."
McDonough said some patients on Cerezyme may choose to skip one or two doses rather than switch to another drug. Genzyme said supply will be conserved for infants and people with very severe disease.
"Conserving the equivalent of two doses can probably be done in a way that would not lead to long term impact on their health," McDonough said. "It is an individual treatment decision for patients and physicians."
Earlier Genzyme said the virus that affected the Boston plant, known as the Allston plant, is a strain known as Vesivirus 2117 that has not been shown to cause human infection, but is known to interfere with the growth of cells used to produce the drugs.
Unlike Cerezyme, which is made using a transformed Chinese hamster ovary cell line, Protalix makes its drug using a plant cell culture, which if it works, could avoid risks from mammalian viruses. Protalix says it believes it can make a cheaper product that stays in the body longer than Cerezyme. (Reporting by Toni Clarke, editing by Gerald E. McCormick and Andre Grenon)
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http://www.reuters.com/article/rbssHealthcareNews/idUSN0624836120090706
Shire obtient un statut rapide pour le traitement de la maladie de Gaucher.
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FDAnews Drug Daily Bulletin
July 20, 2009 | Vol. 6 No. 139
Shire Gaucher’s Disease Treatment Gets Fast-Track Status
Shire’s investigational drug velaglucerase alfa for
Type I Gaucher’s disease has received fast-track designation from the
FDA. In anticipation of a shortage of the only approved treatment for
the disease, the FDA had asked Shire to submit its treatment protocol
for its experimental drug. Approval would allow physicians to treat
Gaucher’s disease patients with the drug on an early-access basis,
ahead of the regular market debut. Shire would provide the drug at no
cost at first so as many patients as possible could receive it quickly,
the company says in a statement.
Drug Industry Daily
http://www.fdanews.com/newsletter/article?articleId=118821&issueId=12842
Shire Traitement de la maladie de Gaucher Gets Fast-Track Status (Traduction sous toutes réserves)
Le médicament Velaglucerase alfa de la société SHIRE pour les malades de Gaucher de type I en cours d'investigation as reçu une appelation rapide de la FDA.En prévision d'un manque du seul traitement approuvé pour la maladie, la FDA avait demandé au Comté de soumettre son protocole de traitement pour son médicament expérimental.
L'approbation permettrait aux médecins de traiter les patients de maladie de Gaucher avec le médicament sur une base d'accès premier avant la commercialisaton régulier sur le marché. Le comté fournirait le médicament gratuitement d'abord afin que le plus patients possible pourraient le recevoir rapidement a dit la société dans une déclaration.
22 mai 2007
Genzyme annonce les résultats positifs de l'essai clinique d'un nouveau traitement oral
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Ghislaine SURREL
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Genzyme Announces Positive Initial Observations in Trial Evaluating Novel Oral Treatment for Gaucher Disease
Genzyme Corp. (Nasdaq: GENZ) announced today that it has completed enrollment in the ongoing Phase 2 trial of Genz-112638, a novel oral therapy being developed for the treatment of Gaucher disease. Based on positive results seen in the trial to date, Genzyme intends to meet with regulatory agencies in the coming weeks to discuss an expedited development strategy.
Initial observations from the first five patients suggest that Genz-112638 may produce a rapid and meaningful impact on important clinical endpoints including reductions in spleen and liver volume, and an increase in platelet counts and hemoglobin concentration. Safety observations from all patients enrolled to date suggest that the only drug-related adverse events seen in the trial have been mild and transient in nature, including one possibly related serious adverse event that is currently being investigated. These early findings will be presented today at Genzyme’s Analyst Day, and full trial results will be available in mid-2008.
If these early improvements continue and are observed in other patients enrolled in the trial, Genz-112638 may represent a promising novel approach to treating patients with Gaucher disease.
“We are very encouraged by our first observations from this trial,” said David Meeker MD, president of the Lysosomal Storage Disorder business. “Cerezyme has had a remarkable effect on the lives of patients with Gaucher Disease. We have set a very high bar for ourselves in trying to develop a convenient oral therapy that can provide a safe and effective choice for patients. We look forward to developing this molecule further and exploring the role it may play in the treatment of patients with Gaucher disease.”
The open-label trial has enrolled patients with Type 1 Gaucher disease at medical centers in Europe, Israel, North America and South America.
About Genz-112638
http://genzyme.com/corp/investors/GENZ%20PR-051607.asp
10 mai 2007
Découverte de la structure tridimensionnelle de l'enzyme impliquée dans la maladie de Gaucher
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Ghislaine SURREL
maladies-lysosomales-subscribe@yahoogroupes.fr
Un groupe interdisciplinaire de l'Institut Weizmann a identifié la structure tridimensionnelle d'une enzyme, la Glucocérébrosidase. Or, des mutations dans cette enzyme sont à l'origine de la maladie de Gaucher, maladie génétique qui touche essentiellement les Juifs ashkénazes. Un groupe interdisciplinaire de l'Institut Weizmann a identifié la structure tridimensionnelle d'une enzyme, la Glucocérébrosidase. Or, des mutations dans cette enzyme sont à l'origine de la maladie de Gaucher, maladie génétique qui touche essentiellement les Juifs ashkénazes. Cette étude, publiée récemment dans "EMBO Reports", pourrait être la base de nouvelles thérapies.
Cette maladie, décrite pour la première fois en 1882, par un médecin français, le Dr Philippe GAUCHER, se caractérise par une hypertrophie de la rate et du foie et par leur dysfonctionnement ; il arrive même, dans certains cas rares, que le cerveau soit aussi atteint. Dans les années 20, fut découvert que la maladie était causée par l'accumulation excessive d'un lipide, le Glucosylcéramide. Dans les années 60, on a découvert que cet excès était dû à une déficience de la Glucocérébrosidase qui a pour fonction de décomposer ce lipide et d'en réguler la quantité. Puis, dans les années 80, on a réussi à isoler le gène responsable de la fabrication de cette enzyme : les chercheurs ont découvert que des mutations du gène perturbent le fonctionnement de l'enzyme, ce qui provoque la maladie de Gaucher.Au début des années 90, la société américaine GENZYME a entrepris la production de cette enzyme, d'abord à partir de placenta, puis en utilisant le génie génétique. A l'heure actuelle, des milliers de personnes atteintes de la maladie de Gaucher sont traitées par des injections de cette enzyme, méthode appelée thérapie par enzyme de remplacement, dont le coût annuel est de l'ordre de 100.000 à 300.000 dollars par patient. Il est évidemment nécessaire de trouver des alternatives plus abordables, comme celles qui pourraient résulter de l'étude récente des Départements de Chimie Biologique, de Biologie Structurale et de Neurobiologie de l'Institut Weizmann.
La première étape vers la caractérisation de la structure tridimensionnelle d'une enzyme est d'élaborer ses cristaux. Dans le cas de la Glucocérébrosidase impliquée dans la maladie de Gaucher, la cristallisation représentait un véritable défi, et les chercheurs ont réussi à le relever en découpant des parties de certaines molécules de sucre à la surface de l'enzyme. Ils ont ensuite eu recours à la cristallographie aux rayons X. Les résultats ont été obtenus à l'ESRF "European Synchrotron Radiation Facility" (l'installation européenne de rayonnement synchrotron) à Grenoble.
Quant au développement de nouvelles thérapies pour la maladie de Gaucher, en premier lieu, l'information structurale peut aider à concevoir une enzyme plus efficace qui pourrait améliorer l'actuelle thérapie par enzyme de remplacement. On obtiendrait alors des traitements permettant d'attendre le développement d'une thérapie génique adéquate. Un autre type de thérapie pouvant résulter des travaux de l'Institut Weizmann serait la possibilité de concevoir de molécules qui complèteraient l'enzyme endommagée dans le corps du patient, rétablissant ainsi son fonctionnement normal.
La société Yeda Research & Development, qui a pour tâche la commercialisation des recherches de l'Institut Weizmann, a fait une demande de brevet pour les applications médicales de ces résultats.
Cette information est un extrait du BE Israël numéro 13 du 25/06/2003 rédigé par l'Ambassade de France en Israël. Les Bulletins Electroniques (BE) sont un service ADIT et sont accessibles gratuitement sur www.bulletins-electroniques.com rArticle écrit le 2003-06-25
05 juin 2006
EMEA panel recommends goat-milk therapy
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Liens utiles à la fin des catégories.
Ghislaine SURREL
maladies-lysosomales-subscribe@yahoogroupes.fr
Reversing an earlier rejection, an EMEA panel of experts has recommended approval for GTC Biotherapeutics' ATryn, an anti-clotting agent that is produced in the milk of genetically engineered goats. If the EMEA gives its official blessing, this would be the first new therapy to be approved using transgenic animals. This is big news for the fledgling transgenics field, boosting the hopes of other players in the area, such as the Netherlands' Pharming Group. PharmAthene is also developing a therapy in goat's milk. GTC added a gene to goats that allows them to produce the human protein antithrombin in their milk. Genzyme represented GTC in the European presentation. The EMEA had originally rejected the application on ATryn because only a handful of people had been tested. GTC shares almost doubled on the news.
- here's the AP report on ATryn
ALSO: Denmark's TopoTarget won an EMEA recommendation in favor of Savene. Release
