-- Findings from two health economics analyses of
Acthar Gel provide further insight into its cost-effectiveness as a
treatment option for advanced symptomatic sarcoidosis,1
and its impact on clinical, economic, and real-world outcomes in
patients with nephrotic syndrome2 --
DUBLIN, Oct. 26,
2023 /PRNewswire/ -- Mallinckrodt plc (OTCMKTS: MNKTQ), a global
specialty pharmaceutical company, recently shared findings from
Mallinckrodt's latest health economics
outcomes research on Acthar® Gel (repository
corticotropin injection) for patients with advanced symptomatic
sarcoidosis and nephrotic syndrome (NS)1,2 at the
Academy of Managed Care Pharmacy (AMCP) Nexus 2023 in Orlando, FL October
16-19, 2023.
A full list of accepted abstracts presented at AMCP Nexus
2023 can be found here.
Acthar is a naturally sourced complex mixture of
adrenocorticotropic hormone analogs and other pituitary peptides.
Acthar Gel is approved by the U.S. Food and Drug Administration
(FDA) for the treatment of several autoimmune disorders and medical
conditions known to cause inflammation.3
Please see indications and Important Safety Information
below.
In a poster (D20) titled "Cost-Effectiveness of Acthar Gel
versus Standard of Care for the Treatment of Advanced Symptomatic
Sarcoidosis," results were presented from a cost-effectiveness
analysis of Acthar Gel and the standard of care (SoC) treatments
over two and three years in patients with advanced symptomatic
sarcoidosis in the U.S.1 In this analysis, a
probabilistic cohort-level state-transition approach was used.
Patients were monitored at the end of a three-month cycle for a
partial or complete response. Clinical parameters and health
utility data were sourced from the PULSAR trial (NCT03320070) and
healthcare utilization, costs, and disutilities were sourced from
the published literature.1
- From the payer perspective – including treatment and direct
medical costs – the findings of this analysis showed that Acthar
Gel versus SoC resulted in an incremental cost-effectiveness ratio
(ICER) of $134,796 per
quality-adjusted life-year (QALY) and $39,179 per QALY over two and three years,
respectively.
- From a societal perspective – including treatment, direct
medical and indirect (caregiving, productivity loss, work-related
training) costs – Acthar versus SoC resulted in an ICER of
$117,622 per QALY over two years and
$21,967 per QALY over three
years.
Model inputs used in the analysis were derived or extrapolated
from the PULSAR clinical trial or published literature, which may
not reflect real-world experience or may result in under- or
over-estimation of results. The simplified care paradigm used for
the model may not capture the complexity of sarcoidosis. Clinical
response was based on composite sarcoidosis treatment scores, which
might result in variation in cost-effectiveness estimates.
The full data manuscript from this study was published in
ClinicoEconomics and Outcomes Research on October 17, 2023.
Additionally, a poster (N1) titled "Nephrotic Syndrome: Patient
Characteristics, Treatment Patterns, and Related Outcomes After
Treatment with Acthar Gel or Comparable Standard of Care in a Large
Administrative Claims Database" detailed results from a
retrospective, observational cohort study characterizing patients
with NS who initiated Acthar or other therapies used after
corticosteroids or calcineurin inhibitors.2 In this
study, treatment for NS with Acthar for proteinuria and other
treatments (azathioprine, chlorambucil, cyclophosphamide,
mycophenolate mofetil, or rituximab) was analyzed via a large
commercial claims database (Symphony Health). Patients had a
confirmed diagnosis for NS, were 18 years of age or older, and had
12 months of continuous enrollment pre- and
post-index.2
- In this analysis, after treatment with Acthar Gel, there was a
significant reduction in the proportion of patients (n=315) taking
corticosteroids (66%; n=209 vs. 51%; n=162, p<0.001), patients
on extended use (≥60 days) of corticosteroids (37%; n=117 to 25%;
n=80, p<0.001), and the average daily patient dose of
corticosteroids (32.1 ± 21.3 to 21.7 ± 21.1, p=0.001) compared to
baseline.
- The Acthar Gel cohort had an increase in patients on dialysis
(6%; n=20 to 14%; n=45, p<0.001), but no change was reported in
renal transplants (10%; n=30 to 10%; n=32, p=0.774) or transplant
complications (6%; n=18 to 6%; n=19, p=1.000). The comparator
cohort of patients taking other therapies (n=6,812) had fewer
patients on dialysis (16%; n=1,105 to 12%; n=823, p<0.001), but
an increase in renal transplants (19%; n=1,315 to 22%; n=1,475,
p<0.001) and transplant complications (8%; n=547 to 10%; n=681,
p<0.001).
"We are pleased to have shared our latest health economics
research on Acthar Gel. These data further our understanding of
treatment cost-effectiveness, clinical impact, and real-world
outcomes for patients with conditions like symptomatic sarcoidosis
and nephrotic syndrome,"1,2 said George Wan, Ph.D., M.P.H., Vice President,
Evidence Generation and Data Sciences, Mallinckrodt. "Acthar has a robust history of
clinical evidence and experience, and we are committed to providing
the medical community with the most up to date research to support
its availability, use, and potential to improve outcomes for
autoimmune and chronic inflammatory conditions across a range of
FDA-approved indications."
Data collected in retrospective analyses and health economics
models may have errors or omissions. Outcomes may be influenced by
therapies not evaluated in the study and the clinical/health
economics outcomes may not be solely attributable to Acthar.
These studies were funded by Mallinckrodt Pharmaceuticals.
ABOUT PROTEINURIA IN NEPHROTIC SYNDROME (NS)
NS is a
collection of symptoms that occur when the blood vessels in the
kidney begin to leak excess protein in the urine, a condition
called proteinuria.4 A variety of diseases and
underlying disorders damage the kidneys and cause proteinuria in
people with NS.4 These etiologies can include glomerular
diseases such as: idiopathic membranous nephropathy, focal
segmental glomerulosclerosis, minimal change disease,
membranoproliferative glomerulonephritis, lupus nephritis, and IgA
nephropathy.4,5,6,7,8,9 In these and other related
disorders, the glomeruli, or small blood vessels that work as the
kidney's filtering system, are damaged.4
Proteinuria is one of the most important adverse prognostic
factors for progression to end stage renal failure in patients with
glomerular disease. One of the goals of treating NS includes
reducing or eliminating proteinuria.10
ABOUT SYMPTOMATIC SARCOIDOSIS
Sarcoidosis is a
challenging and rare multisystem disease.11 In some
cases, the symptoms may come and go throughout a
lifetime.11 This is referred to as symptomatic
sarcoidosis.11 In people with sarcoidosis, the immune
system overreacts, forming clumps of cells called granulomas that
result in inflammation to the body's tissues.12 The
disease can impact any organ, but it most often impacts the lungs,
lymph nodes, eyes, and skin.13 Nearly 90 percent of
people with sarcoidosis will suffer lung problems. Concomitant
involvement of organs outside of the lungs is common, occurring in
more than half of all sarcoidosis cases, according to one
study.14
INDICATIONS
Acthar Gel is indicated for:
- Inducing a diuresis or a remission of proteinuria in nephrotic
syndrome without uremia of the idiopathic type or that due to lupus
erythematosus
- Monotherapy for the treatment of infantile spasms in infants
and children under 2 years of age
- Treatment of acute exacerbations of multiple sclerosis in
adults. Controlled clinical trials have shown Acthar to be
effective in speeding the resolution of acute exacerbations of
multiple sclerosis. However, there is no evidence that it affects
the ultimate outcome or natural history of the disease
- Severe acute and chronic allergic and inflammatory processes
involving the eye and its adnexa such as: keratitis, iritis,
iridocyclitis, diffuse posterior uveitis and choroiditis, optic
neuritis, chorioretinitis, anterior segment inflammation
- Symptomatic sarcoidosis
- Treatment during an exacerbation or as maintenance therapy in
selected cases of systemic lupus erythematosus
- Treatment during an exacerbation or as maintenance therapy in
selected cases of dermatomyositis (polymyositis)
- Adjunctive therapy for short-term administration (to tide the
patient over an acute episode or exacerbation) in: psoriatic
arthritis; rheumatoid arthritis, including juvenile rheumatoid
arthritis (selected cases may require low-dose maintenance
therapy); ankylosing spondylitis
IMPORTANT SAFETY INFORMATION
Contraindications
Acthar is contraindicated:
- For intravenous administration
- In infants under 2 years of age who have suspected congenital
infections
- With concomitant administration of live or live attenuated
vaccines in patients receiving immunosuppressive doses of
Acthar
- In patients with scleroderma, osteoporosis, systemic fungal
infections, ocular herpes simplex, recent surgery, history of the
presence of a peptic ulcer, congestive heart failure, uncontrolled
hypertension, primary adrenocortical insufficiency, adrenocortical
hyperfunction, or sensitivity to proteins of porcine origin
Warnings and Precautions
- The adverse effects of Acthar are related primarily to its
steroidogenic effects
- Acthar may increase susceptibility to new infection or
reactivation of latent infections
- Suppression of the hypothalamic-pituitary-adrenal (HPA) axis
may occur following prolonged therapy with the potential for
adrenal insufficiency after withdrawal of the medication. Adrenal
insufficiency may be minimized by tapering of the dose when
discontinuing treatment. During recovery of the adrenal gland
patients should be protected from the stress (e.g., trauma or
surgery) by the use of corticosteroids. Monitor patients for
effects of HPA axis suppression after stopping treatment
- Cushing's syndrome may occur during therapy but generally
resolves after therapy is stopped. Monitor patients for signs and
symptoms
- Acthar can cause elevation of blood pressure, salt and water
retention, and hypokalemia. Monitor blood pressure and sodium and
potassium levels
- Acthar often acts by masking symptoms of other
diseases/disorders. Monitor patients carefully during and for a
period following discontinuation of therapy
- Acthar can cause gastrointestinal (GI) bleeding and gastric
ulcer. There is also an increased risk for perforation in patients
with certain GI disorders. Monitor for signs of perforation and
bleeding
- Acthar may be associated with central nervous system effects
ranging from euphoria, insomnia, irritability, mood swings,
personality changes, and severe depression to psychosis. Existing
conditions may be aggravated
- Patients with comorbid disease may have that disease worsened.
Caution should be used when prescribing Acthar in patients with
diabetes and myasthenia gravis
- Prolonged use of Acthar may produce cataracts, glaucoma, and
secondary ocular infections. Monitor for signs and symptoms
- Acthar is immunogenic and prolonged administration of Acthar
may increase the risk of hypersensitivity reactions. Cases of
anaphylaxis have been reported in the postmarketing setting.
Neutralizing antibodies with chronic administration may lead to
loss of endogenous ACTH and Acthar activity
- There may be an enhanced effect in patients with hypothyroidism
and in those with cirrhosis of the liver
- Long-term use may have negative effects on growth and physical
development in children. Monitor pediatric patients
- Decrease in bone density may occur. Bone density should be
monitored in patients on long-term therapy
Adverse Reactions
- Commonly reported postmarketing adverse reactions for Acthar
include injection site reaction, asthenic conditions (including
fatigue, malaise, asthenia, and lethargy), fluid retention
(including peripheral swelling), insomnia, headache, and blood
glucose increased
- The most common adverse reactions for the treatment of
infantile spasms (IS) are increased risk of infections,
convulsions, hypertension, irritability, and pyrexia. Some patients
with IS progress to other forms of seizures; IS sometimes masks
theses seizures, which may become visible once the clinical spasms
from IS resolve
Pregnancy
- Acthar may cause fetal harm when administered to a pregnant
woman
Please see full Prescribing Information for additional
Important Safety Information.
ABOUT MALLINCKRODT
Mallinckrodt is a global business consisting of
multiple wholly owned subsidiaries that develop, manufacture,
market and distribute specialty pharmaceutical products and
therapies. The company's Specialty Brands reportable segments areas
of focus include autoimmune and rare diseases in specialty areas
like neurology, rheumatology, hepatology, nephrology, pulmonology,
ophthalmology, and oncology; immunotherapy and neonatal respiratory
critical care therapies; analgesics; cultured skin substitutes and
gastrointestinal products. Its Specialty Generics reportable
segment includes specialty generic drugs and active pharmaceutical
ingredients. To learn more about Mallinckrodt, visit www.mallinckrodt.com.
Mallinckrodt uses its website as a channel of distribution
of important company information, such as press releases, investor
presentations and other financial information. It also uses its
website to expedite public access to time-critical information
regarding the company in advance of or in lieu of distributing a
press release or a filing with the U.S. Securities and
Exchange Commission (SEC) disclosing the same information.
Therefore, investors should look to the Investor Relations page of
the website for important and time-critical information. Visitors
to the website can also register to receive automatic e-mail and
other notifications alerting them when new information is made
available on the Investor Relations page of the website.
CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING
STATEMENTS
This release contains forward-looking statements,
including with regard to Acthar® Gel, its potential to
improve health and treatment outcomes, and its potential impact on
patients. The statements are based on assumptions about many
important factors, including the following, which could cause
actual results to differ materially from those in the
forward-looking statements: the impact of Mallinckrodt's pending Chapter 11 cases;
satisfaction of, and compliance with, regulatory and other
requirements; actions of regulatory bodies and other governmental
authorities; changes in laws and regulations; issues with product
quality, manufacturing or supply, or patient safety issues or
adverse side effects or adverse reactions associated with Acthar;
and other risks identified and described in more detail in the
"Risk Factors" section of Mallinckrodt's most recent Annual Report on Form
10-K and other filings with the SEC, all of which are available on
its website. The forward-looking statements made herein speak only
as of the date hereof and Mallinckrodt
does not assume any obligation to update or revise any
forward-looking statement, whether as a result of new information,
future events and developments or otherwise, except as required by
law.
CONTACT
Media Inquiries
Green Room Communications
954-816-6003
mediainquires@grcomms.com
Investor Relations
Daniel J.
Speciale
Senior Vice President, Finance and Chief Financial Officer,
Specialty Generics
314-654-3638
daniel.speciale@mnk.com
Derek Belz
Vice President, Investor Relations
314-654-3950
derek.belz@mnk.com
Mallinckrodt, the "M" brand mark and
the Mallinckrodt Pharmaceuticals logo are trademarks of
a Mallinckrodt company. Other brands are trademarks of
a Mallinckrodt company or their respective owners.
©2023 Mallinckrodt. US-2300804
10/23
References
1 Bindra J, et al. Cost-Effectiveness of Acthar Gel
for the Treatment of Advanced Symptomatic Sarcoidosis. Poster to
be presented at the Academy of Managed Care Pharmacy (AMCP) 2023
Nexus. October 2023.
2 Hayes K, et al. Nephrotic Syndrome: Patient
Characteristics, Treatment Patterns, and Related Outcomes after
treatment with Acthar Gel or comparable standard of care in a large
administrative claims database. Encore Poster to be presented at
the Academy of Managed Care Pharmacy (AMCP) 2023 Nexus.
October 2023.
3 Acthar® Gel (repository corticotropin
injection) [prescribing information]. Mallinckrodt ARD LLC.
4 National Institute of Health. National Institute
of Diabetes and Digestive and Kidney Diseases. Nephrotic Syndrome
in Adults. 2020. Available at:
https://www.niddk.nih.gov/health-information/kidney-disease/nephrotic-syndrome-adults.
Accessed October 2023.
5 NephCure Kidney International. Focal Segmentation
Glomerulosclerosis (FSGS). Available at
https://nephcure.org/livingwithkidneydisease/ns-and-other-glomerular-diseases/understanding-fsgs/.
Accessed October 2023.
6 NephCure Kidney International. Nephrotic Syndrome.
Available at
https://nephcure.org/livingwithkidneydisease/understanding-glomerular-disease/understanding-nephrotic-syndrome/.
Accessed October 2023.
7 NIDDK. IgA Nephropathy. Available at:
https://www.niddk.nih.gov/health-information/kidney-disease/iga-nephropathy/.
Accessed October 2023.
8 Satpathy HK. IgA Nephropathy. In: Ferri FF, ed.
Ferri's Clinical Advisor 2013. 1st ed. St
Louis: Mosby; 2012;
570-571.
9 Meija-Vilet JM, Pankh SV. Overview of the Current
Approach to Glomerular Disease Classification. In: H. Trachtman et
al. (eds.), Glomerulonephritis: Spring Nature Switzerland
2019; Chapter 5; 60-82.
10 Troyanov et al. J Am Soc Nephrol.
2005;16(4):1061–1068.
11 Sarcoidosis. American College of Chest Physicians.
Available at: http://chestnet.org/sarcoid. Accessed October 2023.
12 Sarcoidosis Overview. 2019. Cleveland Clinic.
Available at:
https://my.clevelandclinic.org/health/diseaes/11863-sarcoidosis-overview.
Accessed October 2023.
13 Judson MA, Boan AD, Lackland DT. The Clinical Course
of Sarcoidosis: Presentation, Diagnosis, and Treatment in a Large
White and Black Cohort in the United
States. Sarcoidosis Vasc Diffuse Lung Dis. 2012.
29(2);119-127.
14 Chopra I, et al. Repository Corticotropin
Injection in Patients with Advanced Symptomatic Sarcoidosis:
Retrospective Analysis of Medical Records. Ther Adv Res Dis.
2019(13);1-11. DOI: 10.1177/1753466619888127.
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