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New Study Demonstrates
Ropeginterferon Alfa-2b-njft
Is a Cost-Effective Treatment Option for a Broad Range of Patients with
Polycythemia Vera
2023-08-04
·
BioSpace
临床结果
上市批准
孤儿药
Analysis published in the Journal of Comparative Effectiveness Research shows
ropeginterferon alfa-2b-njft
provided a cost-effective benefit in quality-adjusted life years compared to a commonly used treatment pathway BURLINGTON, Mass.--(BUSINESS WIRE)-- PharmaEssentia USA Corporation, a subsidiary of
PharmaEssentia Corporation
(TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced the publication of a cost-effectiveness analysis of
ropeginterferon alfa-2b-njft
(marketed as
BESREMi
®) in the Journal of Comparative Effectiveness Research. The analysis, titled “Cost-Effectiveness of
Ropeginterferon Alfa-2b-njft
for the Treatment of
Polycythemia Vera
,” showed that
ropeginterferon
alfa-2b-njft
provided a cost-effective benefit for a broad range of patients with
polycythemia vera (PV)
versus first-line
hydroxyurea
followed by
ruxolitinib
. Cost effectiveness was demonstrated in a modeled population including both low- and high-risk patients receiving first- or second-line treatment with
ropeginterferon alfa-2b-njft
. “
PV
is a
chronic blood cancer
that requires lifelong therapy, which can prevent or delay negative clinical outcomes, but is also associated with additional costs to patients and payers. These new data demonstrate
ropeginterferon alfa-2b-njft
’s value not only to people living with
PV
, but also to the healthcare system at large,” said Dr. Aaron Gerds, MD, MS, Medical Director at
Case Comprehensive Cancer Center
Cancer
Center and lead study author. “This study shows
ropeginterferon alfa-2b-njft
is a cost-effective treatment option over the modeled lifetime and that earlier initiation of treatment of
PV
with effective therapy can translate to more favorable cost to benefit ratios.”
PV
is the most common
myeloproliferative neoplasm (MPN)
and a long-term, potentially life-threatening disease with limited approved treatment options. Patients with
PV
are at risk for disease progression to
myelofibrosis
(
post-PV MF
) or transformation to blast phase (MPN-BP) which is akin to
acute myeloid leukemia1
-3. Patients are also at an increased risk for
arterial and venous thromboembolic events (TEs)
which are associated with higher mortality, lower quality of life, and higher healthcare costs4-7. This new study used an economic model developed from the United States healthcare system perspective. Inputs were informed by the data from randomized clinical trials, including the PROUD-PV and CONTINUATION-PV studies, and from real-world sources. The model compared
ropeginterferon
alfa-2b-njft
used either as first- or second-line therapy versus an alternative treatment pathway of first-line
hydroxyurea
followed by
ruxolitinib
. To reflect the long-term consequences of treating PV, results were presented over a lifetime horizon. Findings from the study conclude
ropeginterferon
alfa-2b-njft
is a cost-effective treatment option for a broad range of patients with
PV
, including both low- and high-risk patients and patients with and without prior cytoreductive treatment with
hydroxyurea
. Of note: These data show that over the modeled lifetime, patients who receive
ropeginterferon alfa-2b-njft
have more years alive (0.4), higher quality-adjusted life years (QALYs) (0.4), and higher cost ($60,175) as compared to the alternative treatment pathway. Weighing the additional costs versus the additional QALY gains results in a cost per QALY of $141,783. This cost per QALY is less than a standard willingness to pay threshold of $150,000 per QALY8. In this study, treating patients at a younger age or those with low-risk disease led to more cost-effective results, suggesting that earlier initiation of treatment of
PV
with effective therapy can translate to more favorable cost to benefit ratios. The model was sensitive to treatment costs, the percentage of patients who discontinue
hydroxyurea
, the percentage of
ropeginterferon alfa-2b-njft
users who switch to monthly dosing, the percentage of
ropeginterferon alfa-2b-njft
users as 2nd line treatment, and the treatment response rates. “
Ropeginterferon
alfa-2b-njft
has demonstrated safety and efficacy in studies including both low- and high-risk patients and patients with and without prior cytoreductive treatment with
HU
. Recently, the National Comprehensive
Cancer
Network (NCCN) updated their treatment guidelines to include
ropeginterferon
as a preferred treatment regimen for both low- and high-risk PV patients9. As
ropeginterferon
continues to be more widely used in clinical practice, this study was imperative to demonstrate the cost-effectiveness of
ropeginterferon alfa-2b-njft
for a broad range of patients with
PV
,” said Raymond Urbanski, M.D., Ph.D., Senior Vice President and U.S. Head of Clinical Development and Medical Affairs at
PharmaEssentia
. “This analysis also underscores the importance of treating low-risk patients and patients early in their disease journey to help minimize more severe events and their corresponding costs to the healthcare system.” The full benefits of
ropeginterferon alfa-2b-njft
may not be fully captured in the model, and in particular, data on disutility of phlebotomy are lacking. Although some patients may tolerate regular phlebotomies, others can experience
iron deficiency
which can negatively impact quality of life. While the results from a scenario analysis incorporating a small decrement in utility had minimal impact on the cost-effectiveness results, due to the lack of data in this area, this estimate remains conservative. This study took a U.S. healthcare perspective, and the results may not generalize to other countries given the differences in healthcare resource use, costs and cost-effectiveness thresholds. Follow PharmaEssentia USA on Twitter and LinkedIn for news and updates. About
Polycythemia Vera (PV)
Polycythemia vera (PV)
is a
cancer
originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as
thrombosis
and
embolism
, and often transforms to
secondary myelofibrosis
or
leukemia
. While the molecular mechanism underlying
PV
is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.10 About
BESREMi
® (
ropeginterferon alfa-2b-njft
)
BESREMi
is an innovative monopegylated, long-acting interferon. With its unique pegylation technology,
BESREMi
has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients.
BESREMi
has orphan drug designation for the treatment of
polycythemia vera (PV)
in adults in the United States. The product was approved by the
European Medicines Agency (EMA)
in 2019, in the United States in 2021, and has recently received approval in Taiwan and South Korea. The drug candidate was invented by
PharmaEssentia
and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by
EMA
in January 2018.
PharmaEssentia
retains full global intellectual property rights for the product in all indications. Indication
BESREMi
is indicated for the treatment of adults with
polycythemia vera
. Important Safety Information WARNING: RISK OF SERIOUS DISORDERS Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy. CONTRAINDICATIONS Existence of, or history of severe
psychiatric disorders
, particularly severe
depression
,
suicidal ideation
, or suicide attempt Hypersensitivity to interferons including
interferon alfa-2b
or any of the inactive ingredients of
BESREMi
. Moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment History or presence of active serious or untreated
autoimmune disease
Immunosuppressed transplant recipients WARNINGS AND PRECAUTIONS
Depression
and
Suicide
: Life-threatening or fatal neuropsychiatric reactions have occurred in patients receiving
interferon alfa-2b
products, including
BESREMi
. These reactions may occur in patients with and without previous
psychiatric illness
. Other central nervous system effects, including
suicidal ideation
,
attempted suicide
,
aggression
,
bipolar disorder
,
mania
and
confusion
have been observed with other
interferon alfa products
. Closely monitor patients for any symptoms of
psychiatric disorders
and consider psychiatric consultation and treatment if such symptoms emerge. If psychiatric symptoms worsen, it is recommended to discontinue
BESREMi
therapy. Endocrine Toxicity: These toxicities may include worsening
hypothyroidism
and
hyperthyroidism
. Do not use
BESREMi
in patients with active serious or untreated
endocrine disorders
associated with
autoimmune disease
. Evaluate thyroid function in patients who develop symptoms suggestive of
thyroid disease
during
BESREMi
therapy. Discontinue
BESREMi
in patients who develop
endocrine disorders
that cannot be adequately managed during treatment with
BESREMi
. Cardiovascular Toxicity: Toxicities may include
cardiomyopathy
,
myocardial infarction
,
atrial fibrillation
and
coronary artery ischemia
. Patients with a history of
cardiovascular disorders
should be closely monitored for cardiovascular toxicity during
BESREMi
therapy. Avoid use of
BESREMi
in patients with severe or
unstable cardiovascular disease
, (e.g.,
uncontrolled hypertension
,
congestive heart failure
(≥ NYHA class 2), serious
cardiac arrhythmia
,
significant coronary artery stenosis
,
unstable angina
) or
recent stroke
or
myocardial infarction
. Decreased Peripheral Blood Counts: These toxicities may include
thrombocytopenia
(increasing the risk of
bleeding
),
anemia
, and
leukopenia
(increasing the risk of
infection
). Monitor complete blood counts at baseline, during titration and every 3-6 months during the maintenance phase. Monitor patients for signs and symptoms of
infection
or
bleeding
.
Hypersensitivity Reactions
: Toxicities may include serious,
acute hypersensitivity reactions
(e.g.,
urticaria
,
angioedema
, bronchoconstriction,
anaphylaxis
). If such reactions occur, discontinue
BESREMi
and institute appropriate medical therapy immediately. Transient rashes may not necessitate interruption of treatment.
Pancreatitis
:
Pancreatitis
has occurred in 2.2% of patients receiving
BESREMi
. Symptoms may include
nausea
,
vomiting
,
upper abdominal pain
, bloating, and
fever
. Patients may experience elevated
lipase
,
amylase
, white blood cell count, or altered renal/hepatic function. Interrupt
BESREMi
treatment in patients with possible
pancreatitis
and evaluate promptly. Consider discontinuation of
BESREMi
in patients with confirmed
pancreatitis
. Colitis: Fatal and serious ulcerative or hemorrhagic/ischemic colitis have occurred in patients receiving
interferon alfa products
, some cases starting as early as 12 weeks after start of treatment. Symptoms may include
abdominal pain
,
bloody diarrhea
, and
fever
. Discontinue
BESREMi
in patients who develop these signs or symptoms.
Colitis
may resolve within 1 to 3 weeks of stopping treatment. Pulmonary Toxicity: Pulmonary toxicity may manifest as
dyspnea
, pulmonary infiltrates,
pneumonia
,
bronchiolitis obliterans
,
interstitial pneumonitis
,
pulmonary hypertension
, and
sarcoidosis
. Some events have resulted in
respiratory failure
or death. Discontinue
BESREMi
in patients who develop pulmonary infiltrates or
pulmonary function impairment
. Ophthalmologic Toxicity: These toxicities may include severe
eye disorders
such as
retinopathy
,
retinal hemorrhage
,
retinal exudates
,
retinal detachment
and
retinal artery or vein occlusion
which may result in
blindness
. During
BESREMi
therapy, 23% of patients were identified with an
eye disorder
. Eyes disorders ≥5% included
cataract
(6%) and
dry eye
(5%). Advise patients to have eye examinations before and during
BESREMi
therapy, specifically in those patients with a
retinopathy-associated disease
such as
diabetes mellitus
or
hypertension
. Evaluate eye symptoms promptly. Discontinue
BESREMi
in patients who develop new or worsening
eye disorders
.
Hyperlipidemia
: Elevated triglycerides may result in
pancreatitis
. Monitor serum triglycerides before
BESREMi
treatment and intermittently during therapy and manage when elevated. Consider discontinuation of
BESREMi
in patients with persistently, markedly elevated triglycerides. Hepatotoxicity: These toxicities may include increases in serum alanine aminotransferase (ALT),
aspartate aminotransferase (AST)
,
gamma-glutamyl transferase (GGT)
and bilirubin. Liver enzyme elevations have also been reported in patients after long-term
BESREMi
therapy. Monitor liver enzymes and hepatic function at baseline and during
BESREMi
treatment. Discontinue
BESREMi
in patients who develop evidence of hepatic decompensation (characterized by
jaundice
,
ascites
,
hepatic encephalopathy
,
hepatorenal syndrome
or
variceal hemorrhage
) during treatment. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Avoid use of
BESREMi
in patients with eGFR <30 mL/min. Discontinue
BESREMi
if severe
renal impairment
develops during treatment. Dental and Periodontal Toxicity: These toxicities may include
dental and periodontal disorders
, which may lead to loss of teeth. In addition, dry mouth could have a damaging effect on teeth and mucous membranes of the mouth during long-term treatment with
BESREMi
. Patients should have good oral hygiene and regular dental examinations. Dermatologic Toxicity: These toxicities have included
skin rash
,
pruritus
,
alopecia
,
erythema
,
psoriasis
, xeroderma,
dermatitis acneiform
,
hyperkeratosis
, and
hyperhidrosis
. Consider discontinuation of
BESREMi
if clinically significant dermatologic toxicity occurs. Driving and Operating Machinery: BESREMi may impact the ability to drive and use machinery. Patients should not drive or use heavy machinery until they know how
BESREMi
affects their abilities. Patients who experience
dizziness
,
somnolence
or
hallucination
during BESREMi therapy should avoid driving or using machinery. Embryo-Fetal Toxicity: Based on the mechanism of action,
BESREMi
can cause fetal harm when administered to a pregnant woman. Pregnancy testing is recommended in females of reproductive potential prior to treatment with
BESREMi
. Advise females of reproductive potential to use an effective method of contraception during treatment with
BESREMi
and for at least 8 weeks after the final dose. ADVERSE REACTIONS The most common adverse reactions reported in > 40% of patients in the PEGINVERA study (n=51) were
influenza-like illness
,
arthralgia
,
fatigue
,
pruritis
,
nasopharyngitis
, and
musculoskeletal pain
. In the pooled safety population (n=178), the most common adverse reactions greater than 10%, were liver enzyme elevations (20%),
leukopenia
(20%),
thrombocytopenia
(19%),
arthralgia
(13%),
fatigue
(12%),
myalgia
(11%), and
influenza-like illness
(11%). DRUG INTERACTIONS Patients on
BESREMi
who are receiving concomitant drugs which are CYP450 substrates with a narrow therapeutic index should be monitored to inform the need for dosage modification for these concomitant drugs. Avoid use with myelosuppressive agents and monitor patients receiving the combination for effects of excessive
myelosuppression
. Avoid use with narcotics, hypnotics or sedatives and monitor patients receiving the combination for effects of excessive CNS toxicity. USE IN SPECIFIC POPULATIONS Pregnancy: Based on mechanism of action and the role of
interferon alfa
in pregnancy and fetal development,
BESREMi
may cause fetal harm and should be assumed to have abortifacient potential when administered to a pregnant woman. There are adverse effects on maternal and fetal outcomes associated with
polycythemia vera
in pregnancy. Advise pregnant women of the potential risk to a fetus. Lactation: There are no data on the presence of
BESREMi
in human or animal milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children from BESREMi, advise women not to breastfeed during treatment and for 8 weeks after the final dose. Females of Reproductive Potential:
BESREMi
may cause embryo-fetal harm when administered to a pregnant woman. Pregnancy testing prior to
BESREMi
treatment is recommended for females of reproductive potential. Advise female patients of reproductive potential to use effective contraception during treatment with
BESREMi
and for at least 8 weeks after the final dose. Pediatric Use: Safety and effectiveness in pediatric patients have not been established. Geriatric Use: In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function and of concomitant disease or other therapy. Please see accompanying full Prescribing Information, including Boxed Warning. About
PharmaEssentia
PharmaEssentia
(TPEx: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles,
PharmaEssentia
aims to deliver effective new biologics for challenging diseases in the areas of hematology and oncology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today
PharmaEssentia
is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website, LinkedIn or Twitter. Forward Looking Statement This press release may contain forward-looking statements, including statements about a cost-effectiveness analysis involving
BESREMi
and the expected impact of
BESREMi
on healthcare outcomes. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and similar legislation and regulations under Taiwanese law. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include changes in healthcare policies and regulations, the availability and acceptance of
BESREMi
in the market, changes in reimbursement that may impact the acceptance and adoption of
BESREMi
, and competition from other products. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. © 2023
PharmaEssentia Corporation
. All rights reserved. BESREMi and
PharmaEssentia
are registered trademarks of
PharmaEssentia Corporation
, and the
PharmaEssentia
logo is a trademark of
PharmaEssentia Corporation
. 1 McKinnell Z, Karel D, Tuerff D, Sh Abrahim M, Nassereddine S.
Acute Myeloid Leukemia
Following
Myeloproliferative Neoplasms
: A Review of What We Know, What We Do Not Know, and Emerging Treatment Strategies. J Hematol, 11(6), 197-209 (2022). 2 Masarova L, Bose P, Daver N et al. Patients with post-
essential thrombocythemia
and
post polycythemia vera
differ from patients with
primary myelofibrosis
. Leuk Res, 59, 110-116 (2017). 3 Finazzi G, Caruso V, Marchioli R et al.
Acute leukemia
in
polycythemia vera
: an analysis of 1638 patients enrolled in a prospective observational study. Blood, 105(7), 2664-2670 (2005). 4 Griesshammer M, Kiladjian JJ, Besses C. Thromboembolic events in
polycythemia vera
. Ann Hematol, 98(5), 1071-1082 (2019). 5 Tefferi A, Rumi E, Finazzi G et al. Survival and prognosis among 1545 patients with contemporary
polycythemia vera
: an international study.
Leukemia
, 27(9), 1874-1881 (2013). 6 Marchioli R, Finazzi G, Landolfi R et al. Vascular and neoplastic risk in a large cohort of patients with
polycythemia vera
. J Clin Oncol, 23(10), 2224-2232 (2005). 7 Parasuraman SV, Shi N, Paranagama DC, Bonafede M. Health Care Costs and Thromboembolic Events in
Hydroxyurea
-Treated Patients with
Polycythemia Vera
. J Manag Care Spec Pharm, 24(1), 47-55 (2018). 8 2020–2023 Value Assessment Framework. Institute for Clinical and Economic Review (2020), 9 Barbui et al. NEJM Evidence. 2023; Gisslinger et al.
Lancet
. 2020; Kiadjian et al.
Leukemia
. 2022; Kiladjian et al. ASH. 2022. 10 Cerquozzi S, Tefferi A. Blast Transformation and Fibrotic Progression in
Polycythemia Vera
and
Essential Thrombocythemia
: A Literature Review of Incidence and Risk Factors. Blood Cancer J (2015) 5, e366; doi:10.1038/bcj.2015.95.
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机构
药华医药股份有限公司
The Case Comprehensive Cancer Center
The European Medicines Agency
[+2]
适应症
出血
甲状腺功能亢进症
腹痛
[+93]
靶点
Lipase
amylase
sialin
[+1]
药物
Ropeginterferon alfa-2b-NJFT
羟基脲
磷酸芦可替尼
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