Label Expansion to Include All Iron Deficiency Anemia Patients Who
Cannot Take Oral Iron
submitted a supplemental new drug application (sNDA) to the
States Food and Drug Administration
Injection for Intravenous (IV) use. The sNDA requests
expand the indication for ferumoxytol beyond the current indication for
the treatment of iron deficiency anemia (IDA) in adult patients with
chronic kidney disease (CKD) to all adult patients with IDA who have
failed or could not tolerate oral iron treatment. The application
includes data from two well-controlled phase III clinical trials of more
than 1,400 patients.
“This submission marks an important corporate milestone and represents
the culmination of several years of work here at AMAG. The regulatory
approval of Feraheme for a broader IDA patient population would expand
our market opportunity in the existing U.S. IV iron market, beyond our
current CKD indication,” said
executive officer of AMAG. “We believe that Feraheme, if approved for
this indication, could provide a new treatment option for patients
suffering from iron deficiency anemia who cannot tolerate or do not
respond to oral iron therapy.”
The sNDA submission is based on data from a global phase III program
that evaluated the use of ferumoxytol in a broad range of adult IDA
patients, all of whom had failed or could not tolerate oral iron
treatment. More than 1,400 patients were enrolled in the two phase III
clinical trials, IDA-301 (placebo comparator) and IDA-302 (active
comparator). Both studies achieved their primary efficacy endpoints,
with meaningful improvements in hemoglobin from baseline to the 35-day
endpoint of the studies. Adverse events and serious adverse events
associated with IV iron therapy, including hypersensitivity reactions,
were reported in both studies. No new safety signals, outside of those
described in the current Feraheme® (ferumoxytol) label, were observed
with ferumoxytol treatment in these studies. These clinical trials also
included patient-reported outcomes data as pre-specified secondary and
exploratory endpoints. These outcomes endpoints, including quantitative
measures of patients’ fatigue and measures of quality of life, captured
the negative pre-treatment impact anemia has on these patients’ lives –
and the significant improvement in these scores following a one gram
course of therapy with ferumoxytol.
About Iron Deficiency Anemia
More than 4 million Americans have IDA; 1.6 million of whom are
estimated to have CKD, while the other 2.4 million suffer from anemia
due to other causes.1 For these patients with anemia due to
other causes, the underlying diseases or issues causing IDA include
abnormal uterine bleeding, gastrointestinal disorders, inflammatory
diseases and chemotherapy-induced anemia. Many IDA patients fail
treatment with oral iron due to intolerability or side effects.2
manufactures and markets Feraheme® in
driving organic growth of its lead product, AMAG intends to expand its
portfolio with additional commercial-stage specialty pharmaceuticals.
The company is seeking complementary products that leverage the
company’s commercial footprint and focus on hematology and oncology
centers and hospital infusion centers. For additional company
information, please visit www.amagpharma.com.
About Feraheme (ferumoxytol)/Rienso
indicated for the treatment of iron deficiency anemia in adult CKD
patients. Feraheme received marketing approval from the
thereafter. Ferumoxytol received marketing approval in
marketed by Takeda as Rienso®. For additional product information,
please visit www.feraheme.com.
The important safety information below is based on
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
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.
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.
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 statements
regarding: the expected timing for regulatory review of the submission
and outcome of the supplemental new drug application for the broader IDA
indication and the availability of treatment options for patients; the
company’s intent to drive organic growth of Feraheme; and the company’s
plans to seek complementary commercial products to add to its portfolio
are forward-looking statements which involve risks and uncertainties
that could cause actual results to differ materially from those
discussed in such forward-looking statements.
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 US and outside the US, including the EU,
(2) uncertainties regarding our ability to successfully and timely
complete our clinical development programs and obtain regulatory
approval for Feraheme/Rienso in the broader IDA indication
both in the US and outside of the US, including the EU, (3) the
possibility that significant safety or drug interaction problems could
arise with respect to Feraheme/Rienso, (4) uncertainties
regarding the manufacture of Feraheme/Rienso, (5)
uncertainties relating to our patents and proprietary rights, both in
the US and outside of the US, (6) the risk of an Abbreviated New Drug
Application (ANDA) filing following the FDA’s recently published draft
bioequivalence recommendation for ferumoxytol, and (7) other risks
identified in our Securities and Exchange Commission filings, including
our Quarterly Report on Form 10-Q for the quarter ended September 30,
2012 and subsequent filings with the
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.
Rienso is a registered trademark of
1 U.S. Census; U.S. Renal Data System, USRDS 2010 Annual
Data Report: Atlas of Chronic Kidney Disease and End-Stage Renal Disease
Institute of Diabetes and Digestive
2010: 41-42; Fishbane, S. et al. Iron Indices in CKD in the NHANES
1998-2004. Clin J Am Soc Nephrol. 2009 January; 4(1): 57–61.
2 Barton, James et al. Intravenous iron dextran therapy in
patients with iron deficiency and normal renal function who failed to
respond to or did not tolerate oral iron supplementation. Am J
Medicine. 2000; 109: 27-32.
AMAG Pharmaceuticals, Inc.
Amy Sullivan, 617-498-3303