Genzyme Corporation (NASDAQ: GENZ) today provided an update on the impact of Cerezyme® (imiglucerase for injection) supply constraints on patient treatment regimens and on the company’s 2009 financial results. The company’s Fabrazyme® (agalsidase beta) supply constraint period, Fabrazyme dose conservation guidelines for patients, and Fabrazyme financial guidance are unchanged.
Genzyme has been working to determine what portion of the work-in-process Cerezyme material that was unfinished when the company temporarily shut down its Allston facility may be finished and released. Since the announcement of its second-quarter results on July 22, Genzyme has made the decision to discard approximately 80 percent of remaining work-in-process material. This decision was made to minimize risk to the Allston plant and ensure that new supply will be available by the end of this year.
Manufacturing in Allston has resumed, and Genzyme has implemented additional viral monitoring procedures. Half of the bioreactors at the plant are currently up and running and all are expected to be operational this month. Genzyme expects to begin releasing new material from these production runs in November and December.
“Reaching a decision on the work-in-process material has been difficult for us as we balance the medical benefit of Cerezyme for patients with minimizing the risk to our newly cleaned Allston plant,” said Henri Termeer, CEO of Genzyme. “In the end, we could not take the risk of processing material that has any possibility of re-contaminating the plant and setting back our ability to supply Cerezyme to patients. Now that we have resumed production, we are focused on the road to recovery.”
Since the contamination occurred during the second quarter and the company decided to write off some of the work-in-process material prior to filing its second-quarter 10-Q report with the Securities and Exchange Commission, second-quarter financial information was adjusted accordingly.
Genzyme’s decision to discard the majority of the work-in-process material has resulted in a second-quarter write-off of approximately $8.4 million in addition to the $14.2 million write-off previously reported. As a result, the company’s second-quarter GAAP net income is now $187.6 million or $0.68 per diluted share, compared with $192.2 million or $0.70 per diluted share reported on July 22. Genzyme’s second-quarter non-GAAP net income is now $226.6 million or $0.82 per diluted share, compared with $232.5 million or $0.85 per diluted share previously reported.
The company continues to evaluate whether to finish the remaining work-in-process material. If Genzyme does not finish and release any of the remaining material, the company would incur an additional write-off of approximately $2.7 million. In addition, Genzyme is currently in discussions with regulatory authorities regarding the release of two lots of Cerezyme that were finished before the plant was shut down. If these finished goods are not released, Genzyme will incur an additional write-off of $3.1 million.
As a result of the decision regarding the work-in-process material, Genzyme now expects its 2009 Cerezyme revenue, total revenue and GAAP/non-GAAP earnings per share to be at the low end of the guidance ranges provided on July 22, which were: Cerezyme revenue of $750 million – $1 billion; total revenue of $4.6 billion – $5 billion; GAAP EPS of $1.74 – $2.29 per diluted share; and non-GAAP EPS of $2.35 – $2.90 per diluted share. Non-GAAP EPS excludes the impact of stock compensation expenses and acquisition-related items. This guidance depends on the release of the two remaining finished Cerezyme lots but no work-in-process material.
Shipments of Fabrazyme were not put on hold following the shutdown of the Allston plant. Dose conservation guidelines for Fabrazyme are in place worldwide to preserve supply during the anticipated 6-8 week period of constraint. Product shipments are expected to resume in November and December. Genzyme previously adjusted its Fabrazyme revenue guidance for this year to $510 million – $520 million from $560 million – $570 million.
Patient Access to Treatment
Following the shutdown of the Allston plant, Genzyme initially communicated voluntary Cerezyme treatment guidelines in conjunction with stakeholders from the physician and patient communities intended to preserve supply for the most vulnerable patients. At that time, the company had projected a supply shortfall that would end in October. As a result of the recent decision regarding the work-in-process material, Genzyme now projects a larger supply shortfall, lasting through the end of the year. Cerezyme supply is expected to begin to normalize when newly produced product is released in late November and December.
In the United States, Genzyme last week implemented a dose conservation program to try to ensure that the most vulnerable patients continue to receive Cerezyme. The company is now shipping Cerezyme only to two patient populations: patients with Gaucher disease type 1 who are 18 years of age or younger, and patients with Gaucher disease types 2 and 3.
As part of U.S. dose conservation, Genzyme has also created an emergency access program, through which physicians may apply to receive Cerezyme for patients who are in life-threatening situations. Applications will be reviewed using criteria formulated in consultation with stakeholders from the physician and patient communities, and decisions will be made by a Genzyme medical committee with guidance from an independent group of physicians and patient representatives. Patient access via this program will be determined by available inventories going forward. Genzyme expects the U.S. dose conservation measures to remain in place until supply begins to normalize at the end of this year. This dose conservation program depends on the release of the two remaining finished Cerezyme lots but no work-in-process material.
Outside of the United States, Genzyme is currently in discussions with regulatory authorities, physicians, and patient organizations to determine how to manage the supply of Cerezyme, and the company will begin shipping according to the revised inventory levels this week.
“These actions are intended to preserve inventory for the most vulnerable patients and to ensure global equity in this extremely challenging time for patients and physicians,” said Geoff McDonough, M.D., Genzyme’s senior vice president and general manager of lysosomal storage diseases. ”We are very focused on restoring normal production at Allston as quickly as possible. In addition, we will move to rapidly provide access to our oral small-molecule therapy, Genz-112638, during this period.”
The company is currently recruiting patients for two global, multi-center, phase 3 trials of Genz-112638, a potential new oral therapy for Gaucher disease type 1. The first, a randomized, double-blind, placebo-controlled study, will include untreated Gaucher disease patients. It is expected to enroll 36 patients who will be treated for 9 months. The second trial will be a randomized conversion study involving patients who have previously received Cerezyme, with an anticipated enrollment of more than 96 patients and a 9-month treatment period. On July 31, Genzyme submitted to the FDA a treatment IND to potentially provide patients with early access to Genz-112638.
Construction continues on an additional manufacturing facility for Cerezyme and Fabrazyme in Framingham, Mass. The plant is expected to be mechanically complete by the end of this year, and engineering and process validation runs will take place next year. Regulatory approval is expected in mid-2011. This plant, which will include four 2000 L bioreactors, will provide substantial additional capacity both to create ample backup inventory and to support the growth of the two products. Genzyme has already hired approximately 100 people to complement existing staff associated with the start-up of this facility.