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January
15th 2003. Earlier in the week, the Food and Drug
Administration’s (FDA) Endocrinologic and Metabolic Drugs
Advisory Committee (EMDAC) met to review Genzyme’s (NASDAQ: GENZ)
Fabrazyme and Transkaryotic Therapeutics’ (NASDAQ: TKTX)
Replagal to treat Fabry disease. Based upon the committee’s
recommendation, we conclude Genzyme will be the sole winner to
market Fabrazyme under the Orphan Drug Exclusivity Act in the U.S.
As we mentioned in last week’s update, Genzyme’s Phase III
study shows positive results for surrogate endpoints such as
clearance of globotriaosylceramide deposits from the vascular
endothelial cells of the kidney. Whether these data will be
adequate for U.S. approval given the lack of clinical efficacy in
the renal function data remains an open question. But the EMDAC
ruled the Phase III data
demonstrating that Fabrazyme sufficiently clears accumulated
globotriaoslyceramide from the blood vessels of the kidney is
important for such clearance and is an appropriate surrogate
marker for purposes of accelerated approval. The committee voted
14-1 in favor of Fabrazyme, and recommended that Genzyme complete
an ongoing placebo-controlled, phase IV study as currently
designed.
Fabrazyme’s
Prescription Drug User Fee Act (PDUFA) date occurs on April 24,
2003 so the FDA must decide if Fabrazyme receives marketing
approval. The FDA has not asked its committee to vote on whether
to approve either Replagal or Fabrazyme, but the recommendations
of the EMDAC reduce the chances that the FDA might have concerns
about Fabrazyme’s therapeutic benefits.
For
Transkaryotic, the EMDAC voted unanimously 15-1 against Replagal,
because the company has failed to provide enough evidence of the
therapeutic benefits for patients with Fabry disease. In a second
decision a vote of 8-7, the panel indicated that the methods by
which the company attempted to measure benefit were suboptimal. In
spite of Transkaryotic’s plans to press forward the Replagal
application, Genzyme is now almost certain to gain orphan drug
exclusivity for Fabrazyme in the U.S.
With
EMDAC’s opinion it’s likely the FDA will significantly delay
Replagal approval. The 8-7 split regarding trial methodology
leaves open the possibility that future results are needed from
appropriately designed trials, or even re-analysis of current
data.
More good news for
Genzyme and Biomarin as the FDA advisory panel unanimously
said Aldurazyme for treating MPS 1, a rare and often fatal genetic
disease was effective for improving patients' lung function and
working capacity.
Aldurazyme should receive FDA
marketing approval by January 28, 2003.
With two drugs expected to be on
the U.S. market, Genzyme’s revenues will grow.
Genentech (NYSE: DNA) opened the
seasonings by matching expected earnings with strong sales of
Rituxin to treat lymphoma and Herceptin to treat breast cancer.
The company
reported earnings of 24 cents per share, in line with the
consensus earnings estimate. Total revenues rose 29.7% year over
year to $778.3 million.
We anticipate the
company will continue its path of continued growth once the FDA
has given marketing approval to Xolair for asthma and Raptiva for
psoriasis. Genentech is hoping Xolair will be approved by the
middle of 2003 and its psoriasis drug Raptiva toward the end of
the year. Genentech will co-promote Xolair and Raptiva with Xoma
(NASDAQ: XOMA) and Novartis AG (NYSE:
NVS), respectively. We re-iterate that peer-reviewed
publications report Xolair’s ability to treat asthma is
impressive, thus the likelihood of FDA approval is highly
probable. While the odds
of marketing approval looks promising for Raptiva, Biogen’s
(NASDAQ: BGEN)
Amevive appears it will reach the market first.
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