Once-Daily Oral HIF-PH Inhibitor Activates
Physiologic Response to Manage Anemia
Akebia's Launch Strategy Developed to Drive
Toward a Potential New Oral Standard of Care
Company to Host Conference Call on
Thursday, March 28 at 8:00 AM ET
CAMBRIDGE, Mass., March 27,
2024 /PRNewswire/ -- Akebia Therapeutics®,
Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose
to better the lives of people impacted by kidney disease, today
announced that the U.S. Food and Drug Administration (FDA) has
approved Vafseo® (vadadustat) Tablets for the treatment of anemia
due to chronic kidney disease (CKD) in adults who have been
receiving dialysis for at least three months. Vafseo is a
once-daily oral hypoxia-inducible factor prolyl hydroxylase
(HIF-PH) inhibitor that activates the physiologic response to
hypoxia to stimulate endogenous production of erythropoietin to
manage anemia. Vafseo is now approved in 37 countries.
"With the approval of Vafseo in the U.S., we're proud to deliver
an alternative treatment option for the hundreds of thousands of
Americans on dialysis who are diagnosed with anemia due to CKD,"
said John P. Butler, Chief Executive
Officer of Akebia. "At Akebia we are committed to kidney patients,
a dedication that has driven our team to achieve this milestone. We
believe this commitment uniquely positions the company to execute a
successful launch designed to drive toward a potential new oral
standard of care for dialysis patients."
The approval of Vafseo for the treatment of anemia due to CKD in
adults who have been receiving dialysis for at least three months
is based on efficacy and safety data from the INNO2VATE
program and an assessment of post marketing safety data from
Japan where VAFSEO was launched in
August 2020. Results from the
INNO2VATE program were published in the New England
Journal of Medicine: (N Engl J Med 2021; 384:1601-1612); (N Engl J
Med 2021; 384:1589-1600). See the Important Safety Information
section below, including BOXED WARNING regarding increased risk of
death, myocardial infarction, stroke, venous thromboembolism and
thrombosis of vascular access.
Approximately 500,000 adult patients in the U.S. on dialysis
suffer from anemia due to CKD1, which may be associated
with many adverse clinical outcomes. The burden of managing
uncontrolled anemia in CKD patients can be substantial, both in
terms of healthcare costs and the impact on patients, healthcare
providers and caregivers. Today, most CKD patients are treated
for anemia with injectable erythropoiesis-stimulating agents mostly
administered at dialysis centers. "Patients receiving maintenance
dialysis would benefit from additional therapeutic options that can
effectively increase and maintain hemoglobin concentrations within
guideline-recommended target ranges," said Glenn M. Chertow, M.D., M.P.H., Professor of
Medicine, Division of Nephrology at Stanford
University and Co-Chair of the independent Executive
Steering Committee for PRO2TECT and
INNO2VATE, the global Phase 3 clinical development
programs for Vafseo.
Lori Hartwell, who has had kidney
disease since she was a young child, is the Founder and President
of the Renal Support Network. She expressed her support of this new
therapy for adults with anemia due to chronic kidney disease on
dialysis by stating, "Anemia is a debilitating condition that
significantly impacts our daily lives. It is promising to see the
introduction of innovative treatment options for people fighting
anemia."
Akebia intends to commercialize Vafseo in the U.S. with its
established commercial team that has deep renal experience and by
leveraging its relationship with CSL Vifor, an industry leader in
bringing innovative therapies to U.S. dialysis organizations. In
line with the approved label, Akebia will execute a launch strategy
to drive Vafseo toward the goal of becoming a new oral standard of
care for adult dialysis patients.
Mr. Butler added, "We are tremendously grateful for the
patients, physicians, investigators, and site coordinators who
participated in our clinical trials that led to this important
approval. This milestone is the culmination of years of
perseverance by Akebia employees and partners committed to
bettering the lives of people impacted by kidney disease."
Conference Call
Akebia will host a conference
call on Thursday, March 28 at
8:00 a.m. Eastern Time to discuss the
approval and planned next steps for launch. To access the call,
please register by clicking on this Registration Link
(https://register.vevent.com/register/BI8d947513ebdd4e32bf2fbc68d973108f),
and you will be provided with dial in details. To avoid delays and
ensure timely connection, we encourage dialing into the conference
call 15 minutes ahead of the scheduled start time.
A live webcast of the conference call will be available via the
"Investors" section of Akebia's website at: https://ir.akebia.com/.
An online archive of the webcast can be accessed via the Investors
section of Akebia's website at https://ir.akebia.com approximately
two hours after the event.
About Akebia Therapeutics
Akebia Therapeutics, Inc. is
a fully integrated biopharmaceutical company with the purpose to
better the lives of people impacted by kidney disease. Akebia was
founded in 2007 and is headquartered in Cambridge, Massachusetts. For more
information, please visit our website at www.akebia.com, which does
not form a part of this release.
About Anemia due to Chronic Kidney Disease (CKD)
Anemia is a condition in which a person lacks enough healthy red
blood cells to carry adequate oxygen to the body's tissues. It
commonly occurs in people with CKD because their kidneys do not
produce enough erythropoietin, a hormone that helps regulate
production of red blood cells. Anemia due to CKD can have a
profound impact on a person's quality of life2 as it can
cause fatigue, dizziness, shortness of breath and cognitive
dysfunction. Left untreated, anemia leads to deterioration in
health and is associated with increased mortality3 in
people with CKD.
About Vafseo® (vadadustat)
Tablets
Vafseo® (vadadustat) tablets is a
once-daily oral hypoxia-inducible factor prolyl hydroxylase
inhibitor that activates the physiologic response to hypoxia to
stimulate endogenous production of erythropoietin, increasing
hemoglobin and red blood cell production to manage anemia. Vafseo
is approved for use in 37 countries.
INDICATION
VAFSEO is indicated for the treatment of anemia due to chronic
kidney disease (CKD) in adults who have been receiving dialysis for
at least three months.
Limitations of Use
- VAFSEO has not been shown to improve quality of life, fatigue,
or patient well-being.
- VAFSEO is not indicated for use:
- As a substitute for red blood cell transfusions in patients who
require immediate correction of anemia.
- In patients with anemia due to CKD not on dialysis.
IMPORTANT SAFETY INFORMATION about VAFSEO (vadadustat)
tablets
WARNING: INCREASED
RISK OF DEATH, MYOCARDIAL INFARCTION, STROKE, VENOUS
THROMBOEMBOLISM, and THROMBOSIS OF VASCULAR ACCESS.
See full
prescribing information for complete boxed warning.
|
VAFSEO increases the
risk of thrombotic vascular events, including major adverse
cardiovascular events (MACE).
Targeting a hemoglobin
level greater than 11 g/dL is expected to further increase the risk
of death and arterial and venous thrombotic events, as occurs with
erythropoietin stimulating agents (ESAs), which also increase
erythropoietin levels.
No trial has
identified a hemoglobin target level, dose of VAFSEO, or dosing
strategy that does not increase these risks.
Use the lowest
dose of VAFSEO sufficient to reduce the need for red blood cell
transfusions.
|
CONTRAINDICATIONS
- Known hypersensitivity to VAFSEO or any of its components
- Uncontrolled hypertension
WARNINGS AND PRECAUTIONS
- Increased Risk of Death, Myocardial Infarction, Stroke,
Venous Thromboembolism, and Thrombosis of Vascular Access
A
rise in hemoglobin (Hb) levels greater than 1 g/dL over 2 weeks can
increase these risks. Avoid use in patients with a history of
myocardial infarction, cerebrovascular event, or acute coronary
syndrome within the 3 month prior to starting VAFSEO. Targeting a
Hb level of greater than 11g/dL is expected to further increase the
risk of death and arterial and venous thrombotic events, as occurs
with ESAs, which also increase erythropoietin levels. No specific
Hb target level, dose of VASFEO, or dosing strategy has been
identified to avoid these risks. Use the lowest effective dose and
adhere to dosing and Hb monitoring recommendations to avoid
excessive erythropoiesis.
Advise patients to seek immediate medical attention if they develop
signs or symptoms of myocardial infarction, stroke, venous
thromboembolism, or thrombosis of vascular access. Evaluate and
manage promptly if these occur.
- Hepatotoxicity
Hepatocellular injury attributed to
VAFSEO was reported in less than 1% of patients, including one
severe case with jaundice. All events were asymptomatic and
resolved after discontinuation of VAFSEO. The time to onset was
generally within the first 3 months of treatment. Elevated serum
ALT, AST, and bilirubin levels were observed in 1.8%, 1.8%, and
0.3% of CKD patients treated with VAFSEO, respectively. Measure
ALT, AST, and bilirubin before treatment and monthly for the first
6 months, then as clinically indicated. Discontinue
VAFSEO if ALT or AST is persistently elevated or accompanied by
elevated bilirubin. Not recommended in patients with cirrhosis or
active, acute liver disease.
- Hypertension
Worsening of hypertension was reported
in 14% (9.4 per 100 person-years [PY]) of patients receiving VAFSEO
and 17% (11.8 per 100 PY) of patients receiving darbepoetin alfa.
Serious worsening of hypertension was reported in 2.7% (1.7 per 100
PY) of patients receiving VAFSEO and 3% (1.8 per 100 PY) of
patients receiving darbepoetin alfa. Cases of hypertensive crisis
including hypertensive encephalopathy and seizures have also been
reported in patients receiving VAFSEO. Monitor blood pressure.
Adjust anti-hypertensive therapy as needed.
- Seizures
Seizures occurred in 1.6% (1.0 per 100 PY) of patients who received
VAFSEO and 1.6% (1.0 per 100 PY) of patients who received
darbepoetin alfa. Following initiation of VAFSEO, monitor patients
closely for premonitory neurologic symptoms. Monitor for new-onset
seizures, premonitory symptoms, or change in seizure
frequency.
- Gastrointestinal Erosion
Gastric or esophageal erosions occurred in 6.4% (4.0 per 100 PY) of
patients receiving VAFSEO and 5.3% (3.3 per 100 PY) of darbepoetin
alfa-treated patients. Serious gastrointestinal (GI) erosions,
including GI bleeding and the need for red blood cell transfusions
were reported in 3.4% (2.1 per 100 PY) and 3.3% (2.0 per 100 PY) of
those receiving VAFSEO and darbepoetin alfa, respectively. Consider
the risk of GI erosion in high-risk patients, including those with
a history of GI erosion, peptic ulcer disease, and tobacco or
alcohol use.
Advise patients of the signs and symptoms of erosions and GI
bleeding and urge them to seek prompt medical care if present.
- Serious Adverse Reactions in Patients with Anemia Due to
Chronic Kidney Disease and Not on Dialysis
The safety of
VAFSEO has not been established for the treatment of anemia due to
CKD in adults not on dialysis and its use is not recommended in
this setting. In large clinical trials in adults with anemia of CKD
who were not on dialysis, an increased risk of mortality, stroke,
myocardial infarction, serious acute kidney injury, serious hepatic
injury, and serious GI erosions was observed in patients treated
with VAFSEO compared to darbepoetin alfa.
- Malignancy
VAFSEO has not been studied and is not
recommended in patients with active malignancies. Malignancies were
observed in 2.2% (1.3 per 100 PY) of patients treated with VAFSEO
and 3.0% (1.8 per 100 PY) of patients treated with darbepoetin
alfa. No evidence of increased carcinogenicity was observed in
animal studies.
ADVERSE REACTIONS
- The most common adverse reactions (occurring at ≥ 10%) were
hypertension and diarrhea.
DRUG INTERACTIONS
- Iron supplements and iron-containing phosphate binders:
Administer VAFSEO at least 1 hour before products containing
iron.
- Non-iron-containing phosphate binders: Administer VAFSEO
at least 1 hour before or 2 hours after non-iron-containing
phosphate binders.
- BCRP substrates: Monitor for signs of substrate adverse
reactions and consider dose reduction.
- Statins: Monitor for statin-related adverse reactions.
Limit the daily dose of simvastatin (20 mg) and rosuvastatin (5
mg).
USE IN SPECIFIC POPULATIONS
- Pregnancy: May cause fetal harm.
- Lactation: Breastfeeding not recommended until two days
after the final dose.
- Hepatic Impairment: Not recommended for use in patients
with cirrhosis or active, acute liver disease.
Please note that this information is not comprehensive.
Please click here for the Full Prescribing Information, including
BOXED WARNING and Medication Guide.
Forward-Looking Statements
Statements in this press release regarding Akebia Therapeutics,
Inc.'s ("Akebia's") strategy, plans, prospects, expectations,
beliefs, intentions and goals are forward-looking statements within
the meaning of the U.S. Private Securities Litigation Reform Act of
1995, as amended, and include, but are not limited to, statements
regarding: Akebia's ability to execute a successful commercial
launch of Vafseo; and Akebia's plans with respect to
commercializing Vafseo, including Vafseo's potential to become a
new oral standard of care; and timing of commercial availability of
Vafseo. The terms "intend," "believe," "plan," "goal," "potential,"
"anticipate, "estimate," "expect," "future," "will," "continue,"
derivatives of these words, and similar references are intended to
identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Actual
results, performance or experience may differ materially from those
expressed or implied by any forward-looking statement as a result
of various risks, uncertainties and other factors, including, but
not limited to, risks associated with: whether Vafseo will be
commercially available when expected; the potential demand and
market potential and acceptance of, as well as coverage and
reimbursement related to, Auryxia® and Vafseo, including estimates
regarding the potential market opportunity; the competitive
landscape for Auryxia and Vafseo, including potential generic
entrants; the ability of Akebia to attract and retain qualified
personnel; Akebia's ability to implement cost avoidance measures
and reduce operating expenses; decisions made by health
authorities, such as the FDA, with respect to regulatory filings;
the potential therapeutic benefits, safety profile, and
effectiveness of Vafseo; the results of preclinical and clinical
research; the direct or indirect impact of the COVID-19 pandemic on
the markets and communities in which Akebia and its partners,
collaborators, vendors and customers operate; manufacturing, supply
chain and quality matters and any recalls, write-downs, impairments
or other related consequences or potential consequences; and early
termination of any of Akebia's collaborations. Other risks and
uncertainties include those identified under the heading "Risk
Factors" in Akebia's Annual Report on Form 10-K for the fiscal year
ended December 31, 2023, and other
filings that Akebia may make with the U.S. Securities and Exchange
Commission in the future. These forward-looking statements (except
as otherwise noted) speak only as of the date of this press
release, and, except as required by law, Akebia does not undertake,
and specifically disclaims, any obligation to update any
forward-looking statements contained in this press release.
Akebia Therapeutics®, Auryxia® and Vafseo® are registered
trademarks of Akebia Therapeutics, Inc. and its affiliates.
Sources:
1United States Renal Data System. 2022 USRDS Annual Data
Report: Epidemiology of kidney disease in the United States. National Institutes of
Health, National Institute of Diabetes and Digestive and Kidney
Diseases, Bethesda, MD, 2022;
Dopps.org
2Eriksson D, et al. BMC Nephrol. 2016;17:97; Finkelstein
FO, et al. Clin J Am Soc Nephrol. 2009;4:33-38; Farag YM, et al.
Clin Nephrol. 2011;75:524-533
3 Portolés J, et al. BMC Nephrol. 2013;14:2. 3. NICE.
Clinical Guideline: Anaemia Management in Chronic Kidney Disease:
Partial Update 2015. 4. Silverberg DS, et al. Clin Lab Haematol.
2001;23:1-6. 5. Herzog CA, et al. J Card Fail. 2004;10:467-472
Akebia Therapeutics Contact
Mercedes Carrasco
mcarrasco@akebia.com
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