Feraheme® Achieves Superiority on all Primary
Efficacy Endpoints
Conference Call to Be Held Today at
results from the second phase III study from its global registrational
program for Feraheme® (ferumoxytol) in patients with iron
deficiency anemia (IDA) regardless of the underlying cause. The study
being reported today, IDA-301, compared Feraheme treatment to
placebo and enrolled 808 patients at 136 sites in the US,
history of unsatisfactory response to, or could otherwise not tolerate,
oral iron therapy. Patients in this study had IDA associated with
various conditions including abnormal uterine bleeding, cancer,
gastrointestinal disorders or other causes. Feraheme demonstrated
superiority on all primary efficacy endpoints evaluated in this study.
The efficacy and safety of Feraheme in this study were comparable
to that reported earlier this year in the IDA-302 study, the phase III
IDA study comparing Feraheme to iron sucrose.
The IDA-301 study was a double-blind, placebo-controlled trial that
randomized patients 3:1 to receive a one gram IV course of Feraheme
or placebo, and it was powered to demonstrate superiority on efficacy.
In this study, 608 patients were treated with Feraheme and 200
received placebo, with the demographics and all baseline parameters well
balanced between the two treatment groups. The primary efficacy endpoint
of the study for US regulators is the proportion of subjects who
achieved a ≥ 2.0 g/dL increase in hemoglobin at any time from baseline
to week five; the primary efficacy endpoint of the study for EU
regulators is the mean change in hemoglobin from baseline to week five.
Patients enrolled in this study were eligible to enter an ongoing
extension study, IDA-303, to evaluate repeat dosing with Feraheme; the
extension study is fully enrolled with 634 patients.
In the IDA-301 study, Feraheme demonstrated robust efficacy,
achieving superiority on both primary efficacy endpoints. Patients
treated with Feraheme achieved a statistically significant mean
increase in hemoglobin at week five of 2.7 g/dL, compared to a mean
increase of only 0.1 g/dL in patients who received placebo; importantly,
these data are consistent with the 2.7 g/dL increase in hemoglobin
reported in the IDA-302 study. In addition, a ≥ 2.0 g/dL increase in
hemoglobin at any time from baseline to week five was achieved in a
statistically significantly greater proportion, 81.1%, of patients
treated with Feraheme in this study, compared with only 5.5% of
patients who received placebo; these data are also consistent with the
data from IDA-302, in which 84.0% of Feraheme-treated patients
achieved a ≥ 2.0 g/dL increase in hemoglobin. Further, a statistically
significant improvement in fatigue, as assessed by patient reported
outcome measures, was demonstrated at week five in Feraheme-treated
patients.
No new safety signals were observed with Feraheme and the types
of reported adverse events (AEs) were consistent with those seen in both
the previously reported IDA phase III study and the CKD phase III
studies, and those contained in the approved U.S. package insert for Feraheme.
Overall, AEs were reported in both study arms with AEs reported in 49.2%
of Feraheme-treated patients, compared to 43.0% of patients who
received placebo.
Patients in both groups experienced protocol-defined adverse events of
special interest, which included mild to severe hypotension or
hypersensitivity reactions ranging from fever alone to an anaphylactoid
reaction; 3.6% of Feraheme-treated patients experienced adverse
events of special interest compared to 1.0% of patients who received
placebo. Cardiovascular AEs were reported in 0.8% of Feraheme-treated
patients, all of which were considered unrelated to study drug by the
investigators, and none were reported in the placebo group. Serious
adverse events (SAEs) were reported at a comparable frequency in both Feraheme-treated
patients (2.6%) and patients who received placebo (3.0%); four of the
SAEs in Feraheme-treated patients (0.7%) were reported as related
to study drug by investigators. The percent of Feraheme-treated
patients that experienced an SAE in this study (2.6%) was lower than
that previously reported in the IDA-302 study (4.2%), and comparable to
the rate of SAEs in iron sucrose-treated patients (2.5%) in that study.
“We are very pleased that, consistent with the results from IDA-302, Feraheme
achieved all primary efficacy endpoints in this study and no new safety
signals were identified,” said
officer of AMAG. “With both phase III studies in our global
registrational program for Feraheme now complete, we will seek
approval for Feraheme for the treatment of a broader population
of patients with iron deficiency anemia and a history of an
unsatisfactory response to oral iron therapy. As demonstrated in this
study through patient reported outcomes, we believe that Feraheme
could provide an important clinical benefit with an improvement in the
quality of life for this patient population, and could be a valuable
therapeutic alternative to currently approved IV irons for the treatment
of their iron deficiency anemia.”
AMAG is planning to submit a supplemental new drug application for the
broad IDA indication to the
year-end 2012.
Conference Call and Webcast Access
at 8:00 a.m. E.T. to discuss the data from this study. To access the
conference call via telephone, dial 877-412-6083 from the
States or 702-495-1202 for international access. To access a telephone
replay of the call, dial 855-859-2056 from the United States or
404-537-3406 for international access. The telephone replay will be
available at approximately 11:00 a.m. E.T.
midnight
telephone replay is 96188567.
The call will be webcast live and archived through the Investors section
of the company’s website at www.amagpharma.com/investors on July
18, 2012 at 8:00 a.m. E.T.
About
manufactures and markets Feraheme® in
additional company information, please visit www.amagpharma.com.
About Feraheme
In
(IV) use is indicated for the treatment of iron deficiency anemia in
adult chronic kidney disease (CKD) patients. Feraheme received
marketing approval from the
30, 2009
thereafter. Ferumoxytol received marketing approval in
product information, please visit www.feraheme.com.
Forward Looking Statements
This press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995 and
other federal securities laws. Any statements contained herein which do
not describe historical facts, including but not limited to: the
preliminary nature of the data being reported in this press release, the
fact that no new Feraheme safety signals were identified,
our expectation to seek approval for Feraheme for treatment of
the broader population of patients with iron deficiency anemia and a
history of an unsatisfactory response to oral iron therapy, our belief
that Feraheme could be a valuable therapeutic alternative to
currently approved IV irons for the treatment of their iron deficiency
anemia, and our intent to submit a supplemental new drug application for
the broad IDA indication to the
year-end 2012.
Such risks and uncertainties include: (1) uncertainties regarding our
and Takeda’s ability to successfully compete in the intravenous iron
replacement market both in the U.S. and outside the U.S., (2)
uncertainties regarding our ability to successfully and timely complete
our clinical development programs and obtain regulatory approval for Feraheme in
the broader IDA indication both in the U.S. and in territories outside
of the U.S., including the European Union, (3) the fact that significant
safety or drug interaction problems could arise with respect to Feraheme,
(4) the risk that final data from the clinical trial described herein
could differ materially from or be subject to differing interpretations
by regulatory authorities than set forth in this press release, (5)
uncertainties regarding our ability to manufacture Feraheme,
(6) uncertainties relating to our patents and proprietary rights, and
(7) other risks identified in our Securities and Exchange
Commission filings, including our Quarterly Report on Form 10-Q for the
three months ended March 31, 2012. We caution you not to place undue
reliance on any forward-looking statements, which speak only as of the
date they are made.
We disclaim any obligation to publicly update or revise any such
statements to reflect any change in expectations or in events,
conditions or circumstances on which any such statements may be based,
or that may affect the likelihood that actual results will differ from
those set forth in the forward-looking statements.
The important safety information below is based on
prescribing information.
Important Safety Information About Feraheme
Indication and contraindications
Feraheme is indicated for the treatment of iron deficiency anemia in
adult patients with chronic kidney disease. Feraheme is contraindicated
in patients with known hypersensitivity to Feraheme or any of its
components.
Warnings and precautions
Serious hypersensitivity reactions, including anaphylactic-type
reactions, some of which have been life-threatening and fatal, have been
reported in patients receiving Feraheme. Observe patients for
signs and symptoms of hypersensitivity during and after Feraheme
administration for at least 30 minutes and until clinically stable
following completion of each administration. Only administer the
drug when personnel and therapies are immediately available for the
treatment of anaphylaxis and other hypersensitivity reactions.
Anaphylactic type reactions, presenting with cardiac/cardiorespiratory
arrest, clinically significant hypotension, syncope, and
unresponsiveness have been reported in the post-marketing experience.
In clinical studies, serious hypersensitivity reactions were reported in
0.2% (3/1,726) of subjects receiving Feraheme. Other adverse reactions
potentially associated with hypersensitivity (e.g., pruritus, rash,
urticaria or wheezing) were reported in 3.7% (63/1,726) of subjects.
Severe adverse reactions of clinically significant hypotension have
been reported in the post-marketing experience. In clinical studies,
hypotension was reported in 1.9% (33/1,726) of subjects, including three
patients with serious hypotensive reactions. Monitor for signs and
symptoms of hypotension following each Feraheme injection. Excessive
therapy with parenteral iron can lead to excess storage of iron with the
possibility of iatrogenic hemosiderosis. Patients should be regularly
monitored for hematologic response during parenteral iron therapy,
noting that lab assays may overestimate serum iron and transferrin bound
iron values in the 24 hours following administration of Feraheme. As a
superparamagnetic iron oxide, Feraheme may transiently affect magnetic
resonance diagnostic imaging studies for up to 3 months following the
last Feraheme dose. Feraheme will not affect X-ray, CT, PET, SPECT,
ultrasound, or nuclear imaging.
Adverse reactions
In clinical trials, the most commonly occurring adverse reactions in
Feraheme treated patients versus oral iron treated patients reported in
≥ 2% of chronic kidney disease patients were diarrhea (4.0% vs. 8.2%),
nausea (3.1% vs. 7.5%), dizziness (2.6% vs. 1.8%), hypotension (2.5% vs.
0.4%), constipation (2.1% vs. 5.7%) and peripheral edema (2.0% vs.
3.2%). In clinical trials, adverse reactions leading to treatment
discontinuation and occurring in 2 or more Feraheme treated patients
included hypotension, infusion site swelling, increased serum ferritin
level, chest pain, diarrhea, dizziness, ecchymosis, pruritus, chronic
renal failure, and urticaria.
Post-marketing safety experience
The following adverse reactions have been identified during
post-approval use of Feraheme. Because these reactions are reported
voluntarily from a population of uncertain size, it is not always
possible to reliably estimate their frequency or establish a causal
relationship to drug exposure.
The following serious adverse reactions have been reported from the
post-marketing spontaneous reports with Feraheme: life-threatening
anaphylactic-type reactions, cardiac/cardiorespiratory arrest,
clinically significant hypotension, syncope, unresponsiveness, loss of
consciousness, tachycardia/rhythm abnormalities, angioedema, ischemic
myocardial events, congestive heart failure, pulse absent, and cyanosis.
These adverse reactions have occurred up to 30 minutes after the
administration of Feraheme injection. Reactions have occurred following
the first dose or subsequent doses of Feraheme.
For full prescribing information, please visit www.feraheme.com.
Source:
AMAG Pharmaceuticals, Inc.
Amy Sullivan, 617-498-3303
