Clolar (TM) accelerated approval by FDA as a drug for ALL - Acute Lymphocytic Leukemia and particularly for children 1 to 21 years - with relapsed ALL.

December 30, 2004: Source: Wall Street Journal and DOWnews

The FDA has authorized the company Genzyme official administering Clolar (TM) (clofarabine) in children aged 1 to 21 years with ALL - Acute Lymphoblastic Leukemia - after two first-line treatment with chemotherapy still have a relapse or not respond to chemotherapy. Already in January 2005 can Clolar (TM) (clofarabine) available in many hospitals around the world for ALL patients aged 1-21 years. This accelerated approval was obtained from an ALL just a phase II study in 49 patients. Here a short free translation of the results of this phase II study:

Clorar's consent is based on results from a preclinical phase II study in 49 heavily pretreated children with relapsed or repeated relapse of ALL - Acute Lymphoblastic Leukemia. Patients received 52mg/m2 Clorar intravenously for one to two hours per day and five days lang. This protocol was 28 days and then every two to six times repeated based on response to therapy and measured blood levels.
30% of participating patients responded to Clorar and 20% of all enrolled patients had a complete remission (CR) or a complete marrow remission in the absence of platelet recovery (CRp) - sorry do not know how to translate - and 10 % of all patients had a partial remission (PR). 7 patients were then eligible for a bone marrow - or stem cell transplant followed by treatment with Clorar. There is also Clorar study done in children with AML - Acute Leukemia Myolide, but these were the results are encouraging but not so spectacular that it also approved the FDA gave. However Phase III trials will have to follow course Clorar. Our comment is that in ALL often by other means a remission can be achieved and especially as a child, additional supplementation, but a defnitieve healing of ALL is not just achieved. Read stories from Yahoo and Robin Shifra among cancer patients experience stories and then under ALL.

- PRESS RELEASE: Clolar (TM) Wins FDA Approval for Most Common Pediatric Leukemia - CAMBRIDGE, Mass.., December 29 / PRNewswire-FirstCall / - Genzyme Corp.. (Nasdaq: GENZ) Announced Today That the U.S. Food and Drug Administration (FDA) HAS Granted marketing approval for Clolar (TM) (clofarabine) for the treatment of children with refractory or relapsed acute lymphoblastic leukemia (ALL). Clolar is the first new leukemia treatment approved specifically for children in More Than a Decade. Given the unmet need for new treatment options in this seriously ill patient population, Genzyme expect to make Clolar Commercially available as Quickly as Possible in January.

"FDA approval of Clolar is a welcome sign That the agency and industry are beginning to act on the needs of children with cancer very early in oncology drug development," said Susan Weiner, Ph.D., president of The Children's Cause for Cancer Advocacy . "Children resistant to current treatments badly need new therapeutic options, and hope fully this approval willing encouragement others in industry to measure the unmet medical needs or thesis very sick children." In 2005, an Estimated 3400 new cases of pediatric acute leukemia Will Be diagnosed in the United States, According To Cancer Metric. ALL is the must common form of pediatric leukemia and children who do not respond to initial therapy, or who relapse, have a very poor survival prognosis.

"We are very pleased thats the FDA has moved so assertively in making Children Suffering from cancer a priority in new drug development," said Duke Collier, executive vice president, Genzyme Corporation. "We are excited to offer young patiënten thesis and Their physicians Clolar through New Hope."

Clolar (TM) is Indicated for the Treatment of Pediatric Patients 1 to 21 years old with relapsed or refractory ALL after at least two prior regimens. This use is based on the induction of complete responses. Randomized trials demo Strating Increased survival or clinical benefit have nots Other Conducted leg.

Clolar HAS Received Orphan Drug designation for adult and pediatric acute lymphoblastic leukemia. Clolar compromise now have seven years of market exclusivity in pediatric ALL patients. Also Recently Granted The FDA six months of extended market exclusivity to Clolar under the Best Pharmaceuticals for Children Act.
Clolar was approved under the FDA's accelerated approval process, Which was Established for Serious or Life-Threatening Diseases Such As a product where clause ALL May providence meaningful therapeutic benefit to patient Existing treatments. Approvals Can Be Granted on thebasic or adequate and well-controlled clinical trials Establishing thats the product HAS an effect on a surrogate endpoint Reasonably Likely to predict clinical benefit. Post-marketing studies to verify clinical benefit are required under this mechanism. Genzyme is committed to continuous post-marketing evaluation of Clolar in pediatric ALL and pediatric HAS submitted a development plan That includes Clolar Further study or in combination with Existing therapies.

"With this approval, we are now willing to expand our commercial operations, include the creation of Genzyme's first oncology sales force for Clolar," Stated Mark Enyedy, senior vice president and general manager of Genzyme Oncology. "We have Worked diligence Gently to make Clolar available to physicians and patients, and are eager to Embark on the product launch. This is the first step in our comprehensive development plan for Clolar, Which We Are Also studying for adult leukemia and solid tumor cancers. "

Clinical Trial Results
Clolar's approval was based on results from a pivotal Phase II trial involv 49 Heavily pretreated children with relapsed or refractory ALL. Patients Received Clolar or 52mg/m2 intravenously for one to two hours a day for Five Consecutive Days. This process was repeated for two to six cycles everytime 28 days depends on response to treatment and blood count recovery.
In the study, a 30 percent response rate was Achieved, with 20 percent of ALL patients Achieving a complete remission (CR) or a complete marrow remission in the absence of platelet recovery (CRp) and 10 percent of ALL patients Achieving a partial response ( PR). Seven patients (14 percent) went on to receive bone marrow or stem cell transplants following treatment with Clolar.
Additional Results of 12 Patients Were Recently presented at the American Society of Hematology 46th Annual Meeting and Exposition in San Diego and supported a 30 percent response rate in this patient population Same.
"The children who participated in the study Clolar had failed an average of three prior treatment regimens and had very poor chances of survival," said lead investigator Sima Jeha Clolar, MD, Director, Developmental Therapeutics, Division of Leukemia / Lymphoma, St. Jude Children's Research Hospital. "As a Pediatric Oncologist, I am excited to have a new well-tolerated and effective treatment option for thesis patiënten with highly-resistant leukemia." Also Clolar HAS bone studied in children with refractory or relapsed acute myeloid leukemia (AML). A Phase II study to date HAS Shown a 26 percent response rate in 42 children with this leukemia, with one CRp and 10 partial remission (PR). In Addition, 34 percent, or 12 patients Treated with clofarabine went to transplant. On December 1, 2004, the Oncologic Drugs Advisory Committee of the FDA advised Additional conducting studies in this group before recommending an AML approval. Currently plans are underway to Clolar Further support in this setting.

Clolar Should Be Administered under the supervision of a qualified physician Experienced in the use of antineoplastic therapy. Suppression of bone marrow function, Which is Usually reversible and dose dependent, and Should Be Anticipated May Increase the risk of infection, include severe sepsis. Administration of Clolar results in a rapid reduction in peripheral leukemia cells. Patients Should Be Evaluated and monitored for signs and symptoms or tumor lysis syndrome and cytokine release That Could developement Writing systemic inflammatory response syndrome (SIRS) / capillary leak syndrome, and organ dysfunction. Assessment of respiratory status and blood pressure monitoring with Clolar consistently infusion is recommended. Clolar Should Be Immediately discontinued in the event of Clinically significant signs or symptoms or SIRS or capillary leak syndrome and Measures' appropriate supportive care initiated. The Most common adverse effects after treatment Clolar, Regard less or causality, Were gastrointestinal tract symptoms, include vomit, nausea, and diarrhea, hematologic effects, include anemia, leucopenia, thrombocytopenia, neutropenia, and febrile neutropenia, and infection. Careful hematologic monitoring constantly therapy is important.
Hepatic and renal function Should Be assessed prior to treatment with Clolar and consistently, as the liver is a target organ for Clolar toxicity and the kidney are the predominant mode of excretion Clolar. Cardiac disorders included tachycardia, pericardial Effusion, and Systolic left ventricular dysfunction in up to 35% or patients. However, the presence or synthesis disorders in patient prior to Clolar administration and / or previous therapy or competitor illness in patient receiving Clolar makes the etiology of disorders Unclear thesis. For more information about Clolar, please call 1-800-RX or Clolarhttp://www.CLOLAR.com visit.

About Genzyme Genzyme Corporation is a global biotechnology company dedicated to making a major positive impact on the lives of people with serious diseases. The company's broad product portfolio is focused on rare genetic disorders, renal disease, osteoarthritis, cancer and immune-mediated diseases, and includes an industry-leading array of diagnostic products and services and sophisticated biomaterials. Genzyme's commitment to innovation continues today with expanded research, writing novel approaches to treat cancer, heart disease, and Other areas of unmet medical need. More than 7,000 Genzyme employees in offices around the globe serve patients in over 80 Countries.

This press release contains forward-looking statements include statements about: the timing of the commercial availability of Clolar, the number of Patients diagnosed with pediatric leukemia, the expansion of commercial operations in support of Clolar, and plans to conduct clinical trials with Additional Clolar . These statements are subject to risks and uncertainties That Could cause actual results to differ materially from projected Those in theses forward-looking statements. These risks and uncertainties include, among others: the ability of Genzyme's contract manufacturer and logistics agent to make Clolar Commercially available at the time predicted, the accuracy or third party estimates of the number of pediatric oncology patients; Genzyme's ability to recruit and hire qualified sales , marketing, and Other personnel in support or Clolar; actual timing and content of submissions to and Decisions made by the FDA and institutional review boards or entities conducting clinical trials for Clolar, as well as the ability to recruit patiënten thesis to Participate in trials; and the risks and uncertainties described in reports filed by Genzyme with the Securities and Exchange Commission. Please see the disclosure under the heading "Factors Affects Future Operating Results" in the Management's Discussion and Analysis of Financial Condition and Results of Operations section of Genzyme's Quarterly Report on Form 10-Q for the quarter ended September 1930, 2004 for a more complete discussion and Other risks or thesis. Genzyme Cautions Substantial investors not to place reliance on the forward-looking statements contained in this press release. These statements speak only as of the date of this press release, and Genzyme takes under no obligation 'to Revise or update the statements.
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