Related Articles

Genzyme Pompe Program Update December 2002



Q: What is the status of the Pompe program?

Genzyme has made strong progress within its Pompe disease program since its decision earlier this year to move forward with an internally developed product candidate. In November, we filed an IND with the FDA for the first of several planned clinical studies, marking a significant milestone in our Pompe program. We share with patients and their families a tremendous sense of urgency to begin clinical studies of Genzyme’s recombinant human acid alpha-Glucosidase (rhGAA), so that we can move toward the goal of making a treatment broadly available. Our discussions with regulatory authorities to date have been positive, and we are optimistic that they will support us in moving this process forward expeditiously. The Pompe program is currently Genzyme’s largest research and development initiative.

Q: When will new clinical trials begin?

Enrollment is expected to begin by the end of this year in the first of several planned clinical trials evaluating the use of rhGAA to treat infantile-onset Pompe disease. Patients up to three years old with the infantile-onset form of the disease will be included in this study. A second trial is expected to begin in the second quarter of 2003 and will focus on patients less than six months old with the most severe infantile-onset form of Pompe disease, in which infants generally die before the age of one year. The studies—to be conducted at medical centers in the United States, Europe, and Asia once we receive the necessary regulatory and IRB approvals—will focus on the effect of treatment on patient survival, as well as other factors such as respiratory function, cardiac status, motor development, and safety. Clinical studies for patients with late-onset Pompe disease are expected to begin once a sufficient supply of clinical trial material is available.

Q: Are you able to make product available for compassionate use?

Sufficient quantities of Genzyme rhGAA are currently available to conduct the two planned infantile studies. In addition, patients now receiving either transgenic rhGAA or rhGAA licensed from Synpac (North Carolina) Inc. are scheduled to transition to Genzyme rhGAA next year. Genzyme has held discussions with regulatory authorities regarding the current limitations on product supply and the urgent needs of Pompe patients. Until adequate product inventory exists, priority must be given to maintaining patients who are currently on therapy and to conducting the clinical studies necessary to secure product registrations worldwide. Only with this focus can treatment be made available as quickly as possible to patients who need it.

Q: How is product manufacturing going?

Genzyme has successfully initiated production of rhGAA in its development- and pilot-scale manufacturing facilities in Framingham, Massachusetts. In addition, Genzyme is now working to complete the installation of commercial-scale bioreactors at its Allston Landing manufacturing facility in Boston to provide capacity for rhGAA production in the longer term.

Q: When do you expect to seek product registration?

Genzyme is continuing its discussions with regulatory authorities in Europe regarding the content and timing of a marketing authorization application. These discussions have not yet been held with regulatory authorities in the United States or other countries

.

Q: Have there been program management changes?

A: Yes. John Crowley, senior vice president, Genzyme Therapeutics, and general manager of the Pompe program, is leaving the company to spend more time with his family, particularly his children, both of whom suffer from Pompe disease. Mr. Crowley came to Genzyme in 2001 following Genzyme’s acquisition of Novazyme Pharmaceuticals Inc., where he was president and chief executive officer. He will continue to work with Genzyme as a consultant. Frank Ollington, Ph.D., senior vice president, Genzyme Therapeutics, has assumed management responsibility for the Pompe program. Frank has held a number of important leadership positions in his 14 years with Genzyme. He directed the start-up of the Allston Landing manufacturing plant, served as president of the Genzyme Pharmaceuticals business unit and senior vice president of corporate operations, and chaired the steering committee overseeing the development of Aldurazyme® (laronidase), a product under development for the treatment of the genetic disorder MPS 1.

This document contains forward-looking statements that are subject to risks and uncertainties that could cause actual results to differ materially from those projected in these forward-looking statements including, among others, the risks and uncertainties described in reports filed by Genzyme Corporation with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including without limitation Exhibit 99.2 to Genzyme's 2001 Annual Report on Form 10-K, as amended. These statements speak only as of the date of this document. Genzyme® is a registered trademark of Genzyme Corporation. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. All rights reserved.




Go Back
   
 
 
 Upcoming Events/New Items  Links

United States Pompe Program Update, August 9, 2010

GENZYME: THE MAKING OF PROTEIN THERAPIES VIDEO

8TH ANNUAL UNITED POMPE FOUNDATION FUNDRAISER

Genzyme Invites the US Pompe Patient Community to a Town Hall Teleconference

UNITED STATES POMPE PROGRAM UPDATE, MAY 25, 2010

GENZYME RECEIVES FDA APPROVAL FOR LUMIZYME FOR POMPE DISEASE

UPF PORTLAND POMPE PATIENT MEETING 2010

US Pompe Program Update – December 3, 2009

POMPE PROGRAM UPDATE MAY 22, 2009

Pompe Program Update March 2, 2009

Genzyme Receives Complete Response Letter from FDA on Lumizyme Application

MYOZYME® (2000 L) SUPPLY UPDATE _US VERSION_JANUARY 2009

GUIDANCE TO THE POMPE COMMUNITY ON THE MANAGEMENT OF MYOZYME SUPPLY

GENZYME POMPE PROGRAM UPDATE NOVEMBER 17, 2008

MYOZYME PRODUCED AT THE 2000L BIOREACTOR SCALE TO RECEIVE ACCELERATED APPROVAL

Amicus Therapeutics Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease

Genzyme Provides Update on Myozyme® Manufacturing

Genzyme Study of Myozyme® for Late-Onset Pompe Patients Meets Co-Primary Efficacy Endpoints

Amicus Therapeutics Announces Positive Results From Two Phase 1 Clinical Studies of AT2220 for Pompe Disease

REHABILITATION MANAGEMENT OF POMPE DISEASE: GREGORY T. CARTER, MD, MS

JOINT LETTER FROM THE AMDA AND UPF

MYOZYME® TEMPORARY ACCESS PROGRAM (MTAP) UPDATES

MYOZYME® TEMPORARY ACCESS PROGRAM

THE MYOZYME MIRACLE

FDA APPROVES GENZYMES MYOZYME® FOR ALL PATIENTS WITH POMPE DISEASE

Expensive drug gives hope to rare disease sufferer

 

Video

GENZYME: THE MAKING OF PROTEIN THERAPIES

LUMIZYME HOME PAGE

HOME ADAPTIONS: SHARON KRUEGER

Canadian Association of Pompe

"WEAKLING WILLIE" BOOK

FLYING WITH A VENTILATOR IN THE USA

Association for Glycogen Storage Disease

JUAN MAGDARAOG BLOG

Myozyme Home Page

EXPRESSION OF HOPE

Amicus Therapeutics

UK Pompe Group

Helping Hands Loving Hearts

International Pompe Association IPA

Clinical Trial Information

Genzyme's Pompe Website

 
Pompe Disease | Clinical Trial Process | Donations | EP Magazine Article "Living With Pompe Disease"
Events | Patient Registry | Videos | Related Articles | Contact Us | Home
United Pompe 2010 All Rights Reserved.