MozobilTreatment for Bone Marrow Transplantation
Update: Mozobil Now FDA Approved - December 15, 2008
FDA Priority Review for Mozobil NDA
Genzyme Announces FDA Priority Review for Mozobil New Drug Application
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep 23, 2008 - Genzyme Corporation today provided updates on three therapies with regulatory actions anticipated by year end. The company reported that it expects FDA to act on its biologic license application (BLA) for alglucosidase alfa (Myozyme) produced at the 2000L bioreactor scale by November 29, 2008. Genzyme also announced that its new drug application (NDA) for Mozobil (plerixafor) has been granted priority review and an action date of December 16, 2008. For Synvisc-ONE, the company has received an action date of December 23, 2008.
Alglucosidase Alfa (Myozyme)
Alglucosidase alfa is under review for the treatment of late-onset patients with Pompe disease, a severe, progressively debilitating and life-threatening inherited disorder. The FDA's Endocrinologic and Metabolic Drugs Advisory Committee will meet on October 21, 2008, to discuss the company's BLA, as required by the FDA Amendments Act for all new drug and biologic license applications.
Genzyme currently has U.S. approval for Myozyme manufactured at the 160L bioreactor scale. FDA approval of 2000L-scale production is needed to provide broader access to treatment for adult patients in the United States. The agency views alglucosidase alfa manufactured at the 2000L scale as a separate product because of differences in its carbohydrate structure and has required Genzyme to submit a separate BLA.
The first portion of the October 21 advisory committee meeting will focus on proprietary manufacturing data and will be closed to the public. The second portion of the advisory meeting will be open to the public and will focus on clinical data from Genzyme's Late-Onset Treatment Study (LOTS), which will be the basis for the approval of the proposed labeled indication for the new scale product. This study, which met its co-primary efficacy endpoints, evaluated the safety and efficacy of Myozyme in adult patients with Pompe disease.
FDA and Genzyme briefing documents for the advisory committee, along with the agency's questions for the panel, are expected to be posted on the FDA's web site at least two business days before the meeting. Genzyme anticipates that the FDA review process will culminate in the availability of two commercial versions of alglucosidase alfa in the United States: one produced at the 160L scale and the other produced at the 2000L scale.
"We are confident that the strong clinical data from the LOTS trial will support U.S. approval of alglucosidase alfa manufactured at the 2000L scale," said Genzyme Senior Vice President Alison Lawton. "We are working cooperatively with the FDA to move this process forward expeditiously so that we can provide broader access to treatment as soon as possible."
Mozobil is a product candidate designed to mobilize hematopoetic stem cells (HSC) from the bone marrow into the bloodstream where they can be collected, making it more likely for patients with certain types of cancers to receive a successful transplant. Genzyme conducted two phase 3 studies that confirmed the potential of Mozobil to effectively and predictably prepare lymphoma and multiple myeloma patients for an autologous HSC transplant. Both studies successfully met primary and secondary endpoints.
More than 900 patients have received Mozobil through a compassionate use program in the United States. An additional 120 patients have been treated through similar compassionate use programs which have recently begun in European and other international countries. Genzyme is preparing to launch Mozobil in the United States during the first quarter of next year upon approval.
Synvisc-ONE, the next generation of Synvisc (hylan G-F 20), is designed to simplify osteoarthritis pain management and thereby expand the benefits of viscosupplementation to a broader number of patients. The FDA is expected to convene an advisory committee meeting in December to discuss Genzyme's marketing application for Synvisc-ONE. As with the alglucosidase alfa panel in October, this meeting is expected to focus on the clinical data Genzyme submitted to support the approval and labeling of the product. Genzyme expects to be able to provide further details of this advisory committee meeting once they are confirmed with the FDA and published in the Federal Register.
One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 10,000 employees in locations spanning the globe and 2007 revenues of $3.8 billion. In 2007, Genzyme was chosen to receive the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.
With many established products and services helping patients in nearly 90 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease, and other areas of unmet medical need.
Genzyme's press releases and other company information are available at www.genzyme.com and by calling Genzyme's investor information line at 1-800-905-4369 within the United States or 1-678-999-4572 outside the United States.
This press release contains regulatory updates that contain forward-looking statements. These forward looking statements include: Genzyme's expectations for approval of Mozobil and alglucosidase alfa produced at the 2000L-scale capacity, and the timing thereof; and its expectations regarding FDA action on its marketing application for Synvisc-ONE, and the timing thereof. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecasted. These risks and uncertainties include, among others, that FDA and advisory committee actions are delayed or are unfavorable, as well as the risks and uncertainties described in Genzyme's SEC reports filed under the Securities Exchange Act of 1934, including the factors discussed under the caption "Risk Factors" in Genzyme's 2008 Quarterly Report on Form 10-Q for the quarter ended June 30, 2008. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of today's date and Genzyme undertakes no obligation to update or revise the statements.
Genzyme and Myozyme, are registered trademarks, and Mozobil and Synvisc-ONE are unregistered trademarks, of Genzyme or its subsidiaries. All rights reserved.
Lori Gorski, 617-768-9344
Patrick Flanigan, 617-768-6563
Posted: September 2008
- FDA Approves Genzyme's Mozobil - December 16, 2008
- Genzyme Files Applications for Approval of Mozobil in the United States and Europe - June 17, 2008