Genzyme’s Synvisc-ONE and Myozyme Could Both See Delays After Advisory Committees

A Dec. 9 advisory committee meeting for the osteoarthritis pain management candidate leaves two weeks for FDA turnaround by the product’s action date.

FDA would have two weeks to weigh an advisory committee's input on Genzyme's Synvisc-ONE if the agency were to approve the line extension of the osteoarthritis pain management product by its Dec. 23 action date.

Genzyme Executive VP Therapeutics, Biosurgery and Transplants David Meeker told investors during the company's Oct. 22 third quarter sales and earnings call that an FDA advisory committee will meet Dec. 9 to evaluate Synvisc-ONE (hylan G-F 20).

According the FDA's Web site, the panel scheduled to meet Dec. 9 is the Orthopedic and Rehabilitation Devices Advisory Committee.

"Based on our dialogue with the FDA thus far, we expect that they will acknowledge the statistical significance of our results, but ask questions about the clinical significance of those results. We feel we have a good case to make," Genzyme Biosurgery President Anne Merrifeld said.

Synvisc-ONE is a single treatment version of Synvisc, expected to provide up to six months of pain relief. The current version of Synvisc is delivered in three administrations at one-week intervals.

Editor's note: The original version of this story incorrectly reported that the product to receive an FDA advisory committee review on Dec. 9 was the bone marrow transplant preparation therapy Mozobil (plerixafor). Genzyme has not been informed of any such meeting for Mozobil, which has a Dec. 16 user fee date.

Myozyme also faces PDUFA crunch time

Another PDUFA date on Genzyme's near-term horizon which the firm is for the larger production scale of its Pompe's disease drug Myozyme (alglucosidase alfa).

Recommendations Oct. 21 from FDA's Endocrinologic and Metabolic Drugs Advisory Committee were largely for accelerated approval of the 2000 liter bioreactor scale product, with the condition that a verification study to demonstrate clinical benefit be conducted post-market.

FDA is likely to require a design of a post-approval study before they approve the 2000L production scale. The PDUFA date for the 2000L product is Nov. 29, and it seems as though the firm and FDA would need to develop the protocol before then.

Genzyme also expects a decision by the EMEA in the first half of 2009 for a 4000L product the firm has been developing for the last five years in Belgium.

Worldwide, Myozyme revenue increased 43 percent in the third quarter to $76.7 million, with U.S. sales constrained by the delay in approval for 2000L scale production.

Genzyme reported that third quarter revenue grew 21 percent year over year to $1.16 billion, driven by double digit growth in every Genzyme business unit.

Pending Mobozil Approval Could Mobilize Genzyme Hematology

Genzyme also awaits a Dec. 16 PDUFA date following a priority review for bone marrow transplant preparation therapy Mobozil (plerixafor).

Mobozil is designed to help 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 Executive VP Therapeutics, Biosurgery & Transplants David Meeker said Mozobil will provide advantage over the standard of care by adding reliability to the bone marrow transplant process.

"For a number of patients who have failed to mobilize in the setting of standard of care, this has been shown to allow them to mobilize quite reliably," Meeker said. He said that under current standard of care, chemotherapy results in the priming of bone marrow and the mobilization of rebounded cells, a process that "creates a lot of uncertainty in terms of the exact time when cells peak and can be harvested with reliability."

Genzyme conducted two Phase III studies of Mozobil which the firm says confirmed its potential to effectively and predictably prepare lymphoma and multiple myeloma patients for an autologous HSC transplant. Both studies successfully met primary and secondary endpoints (¹ (Also see "Genzyme Reports More Good News For Mozobil" - Pink Sheet, 2 Aug, 2007.)).

Mozobil is currently available in the U.S. under a compassionate use program, through which about 900 patients currently receive treatment. According to the firm, an additional 120 patients have been treated through programs in Europe and other countries. If approved in U.S. and Europe, patients would be converted to commercial supply of the drug.

European approval is expected in the first half of 2009.

"These are single-use patients," Meeker said during the call. "The important thing about the compassionate use experience is that it enables individual physicians and centers to treat patients who have limited options, and so that is a very important part of this."

Genzyme estimates the treatment would be used in the 55,000 stem cell transplants per year and projects peak annual sales of $400 million, although some analysts caution that improvements in cancer care will limit the transplant market (² (Also see "Genzyme Submits Mozobil In U.S. And Europe" - Pink Sheet, 17 Jun, 2008.)).

With the launch of Mozobil and adult acute myeloid leukemia candidate Clolar (clofarabine injection), Genzyme expects to add $1 billion in peak sales to its current $500 million hematology/oncology portfolio.

-Jamie Hammon ([email protected])