Onyx Pharmaceuticals Inc.
) received a major setback as the US Food and Drug Administration
(FDA) did not grant accelerated approval designation to the
company's new drug application (NDA) for carfilzomib. Onyx Pharma
is seeking to get carfilzomib approved as a treatment for patients
with relapsed and refractory multiple myeloma.
The regulatory body has granted a standard review designation to
the candidate's NDA and set a target date of July 27, 2012.
The FDA in its letter to Onyx Pharma said that the application
and the study supporting the application are insufficient to review
the NDA on a priority basis. The regulatory body has also said that
since the NDA is based on a single-arm study, it is concerned
whether the benefit and risk of the candidate are appropriately
It was also mentioned in the letter that the FDA's advisory
committee prefers late-stage study results for granting accelerated
approval designation to a candidate's NDA, whereas, carfilzomib's
NDA was based on mid-stage trial (003-A1) results.
The phase II trial had demonstrated that patients, dosed with
the said candidate, experienced an overall response rate of 24.1%,
while the clinical benefit rate was higher at 34.2%. The duration
of response was 8.3 months, while the median overall survival was
15.5 months. The patients, who enrolled in the study, had already
received a median of five prior lines of therapy and their disease
was refractory to their last therapeutic regimen.
Additionally, the FDA had granted orphan drug designation to
carfilzomib for the treatment of multiple myeloma in 2008.
We note that Onyx Pharma is conducting a phase III study (FOCUS)
to support the European filing of carfilzomib as a treatment for
relapsed and refractory myeloma. Top-line data from the study is
expected in the first half of 2012.
We currently have a Neutral recommendation on Onyx Pharma. The
stock carries a Zacks #3 Rank (Hold rating) in the short-run. The
news of the FDA not granting carfilzomib's NDA accelerated approval
designation comes as a disappointment for Onyx Pharma, which is
heavily banking on the approval of the candidate as currently it
just has one marketed product, Nexavar (cancer), in its
We believe that any further delay in the approval of carfilzomib
as a treatment for refractory multiple myeloma would weigh heavily
on the stock.
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