's Kyprolis has passed multiple clinical trials, but the failure
today will overshadow the rest.
That's because the only thing that really matters to cancer
patients is survival. Does the drug help the patient live
Living longer without the disease progressing (progression
free survival, or PFS) or responding to the drug (response rate)
are helpful measures of a drug's potential, but in the end,
survival is all that matters.
That goes double in Europe.
Kyprolis has been on the market in the U.S. for over two years
based on phase 2 data showing 23% of multiple myeloma patients
responded to Kyprolis after failing at least two prior
treatments. Earlier this month, Amgen announced results from a
trial dubbed ASPIRE, that showed Kyprolis in combination
's Revlimid and a corticosteroid helped patients live 8.7
months longer without their disease worsening (
) compared to Revlimid and the corticosteroid alone.
The latest study, dubbed FOCUS, measured the more important
overall survival. Unfortunately Kyprolis was no better than the
control arm where patients got low-dose dexamethasone or
equivalent corticosteroids, plus optional cyclophosphamide.
Does that mean Kyprolis doesn't work?
The patients in FOCUS had failed a median of five treatments
before entering the study. What FOCUS tells us is that Kyprolis
isn't any better than the current standard of care for patients
that have exhausted most of their other options.
The ASPIRE PFS data should be enough to change Kyprolis'
accelerated approval into a full approval; Onyx Pharmaceuticals,
the former owner of Kyprolis before Amgen bought the company,
designed ASPIRE in conjunction with the FDA to do just that. The
data even look good enough to justify changing the label so
Kyprolis is approved for treating patients in combination with
Revlimid that have failed as few as one prior treatment.
Currently Kyprolis is approved for patients that have failed two
prior treatments including a drug in the same class as
In Europe it's a different story
European regulators are likely to want to see overall
survival. FOCUS was clearly designed with European regulators in
mind. The name of the trial (Car
mib for Advan
ed Refractory M
ltiple Myeloma European
tudy) even contains "European" in the name -- finding FOCUS as an
acronym is a little harder.
ASPIRE, another trial with a wonky acronym (C
rfilzomib, Lenalidomide, and Dexametha
one versus Lenalidomide and Dexamethasone for the treatment of
elapsed Multiple My
loma), might be enough to gain approval in Europe -- it sounds
like Amgen is going to give it a try -- but without overall
survival data, it's going to be a tough sell.
Even if ASPIRE is good enough to gain approval in Europe,
where there are actually more multiple myeloma patients than in
the U.S., the lack of overall survival data may make it hard to
gain reimbursement by national health organizations in Europe.
The UK's National Institute for Health and Care Excellence (
), for instance, takes efficacy and pricing into account when
determining whether it'll pay for a drug. And with two brand-name
treatments in the regimen -- Kyprolis and Revlimid -- the
combination isn't going to be cheap.
We don't have the full ASPIRE data -- perhaps at the American
Society of Hematology (
) meeting in December -- but Amgen hinted that the overall
survival data "showed a trend in favor" of the combination
containing Kyprolis but that the difference wasn't statistically
significant. Since this was only an interim look, it's possible
that, as more patients progress, the difference might become
statistically significant, and then Amgen could reapply for
regulatory and/or reimbursement approval if Kyprolis ends up
getting turned down without the survival data.
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Why Amgen, Inc.'s Kyprolis FOCUS Failure Is a Big
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