Mozobil Receives Priority Review with FDA Action Expected by Year
End; Synvisc-ONE FDA Action also Expected by Year End
Genzyme Corporation (Nasdaq: GENZ) 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.
