Professor Wendy Erber is Professor of Pathology and Laboratory Medicine and a diagnostic haematologist at the University of Western Australia.
Category Archives: Uncategorized
NCCN Clinical Practice Guidelines in Oncology Update Recommends BESREMi® (ropeginterferon alfa-2b-njft) for the Treatment of Polycythemia Vera
Reflecting its strong clinical profile and broad label, NCCN Guidelines include BESREMi as an
option for PV regardless of treatment history, and for use in both low- and high-risk settings
March 2, 2022, Burlington, MA – PharmaEssentia USA Corporation, the U.S. 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 that the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines®) has been updated to include BESREMi® (ropeginterferon alfa-2b-njft) as a recommended therapeutic option for the treatment of adults with polycythemia vera (PV).
The NCCN is a highly renowned and respected not-for-profit alliance of leading cancer centers in the United States. Its treatment practice guidelines are widely respected and followed by the U.S. physician community, and serve to inform and facilitate coverage decisions with payers for oncology therapies. Consistent with the broad indication for BESREMi upon its U.S. regulatory approval in November 2021, the guidelines provide consideration for use of the treatment among both high-risk and low-risk adult patients, regardless of their treatment history.
“Importantly, the NCCN Guidelines update includes mention of BESREMi in multiple settings, and in particular, as the only systemic option for low-risk patients with PV, which signals a shift toward more proactive treatment earlier in the disease journey,” said Ruben Mesa, M.D., FACP, Executive Director of the UT Health San Antonio MD Anderson Cancer Center. “Now, treating physicians can leverage these expert guidelines to gain greater familiarity with BESREMi in the real world setting and understand its broad utility for patient care in a variety of treatment settings.”
“Our goal with BESREMi has been to offer a compelling therapeutic alternative to conventional treatment options that can enable physicians to gain durable control over the disease beyond the symptoms and help more patients reach their long-term health goals,” said Raymond
Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs. “The NCCN Guidelines update just three months following our approval illustrates the community’s recognition of the strong potential of BESREMi in PV care.”
CTI BioPharma Announces FDA Accelerated Approval of VONJO™ (pacritinib) for the Treatment of Adult Patients with Myelofibrosis and Thrombocytopenia
SEATTLE, Feb. 28, 2022 /PRNewswire/ — CTI BioPharma Corp. (Nasdaq: CTIC) today announced the U.S. Food and Drug Administration (FDA) has approved VONJO (pacritinib) for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. VONJO is a novel oral kinase inhibitor with specificity for JAK2 and IRAK1, without inhibiting JAK1. The recommended dosage of VONJO is 200 mg orally twice daily. VONJO is the first approved therapy that specifically addresses the needs of patients with cytopenic myelofibrosis.
“Today’s approval of VONJO establishes a new standard of care for myelofibrosis patients suffering from cytopenic myelofibrosis,” said John Mascarenhas, M.D., Associate Professor, Medicine, Hematology and Medical Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York. “Myelofibrosis with severe thrombocytopenia, defined as blood platelet counts below 50 × 109/L, has been shown to result in poor survival outcomes coupled with debilitating symptoms. Limited treatment options have rendered this disease as an area of urgent unmet medical need. I am pleased to see that a new, efficacious and safe treatment option is now available for these patients.”
“In the U.S., there are approximately 21,000 patients with myelofibrosis, two-thirds of which have cytopenias (thrombocytopenia or anemia), commonly resulting from the toxicity of other approved therapies. Severe thrombocytopenia, defined as a blood platelet count below 50 × 109/L, occurs in one-third of the overall myelofibrosis population, and has a particularly poor prognosis. With the approval of VONJO, we are excited to now be able to offer a new therapy that is specifically approved for patients with cytopenic myelofibrosis. We are fully funded for commercial launch, following our debt and royalty transactions with DRI, and we look forward to providing VONJO, the potential best-in-class therapy for cytopenic myelofibrosis patients, to patients within 10 days,” said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. “I would like to thank the patients, caregivers, clinical trial staff and investigators who made the VONJO clinical trials possible. I am also thankful to the CTI team for their hard work and dedication and their focus on the needs of patients.”
The accelerated approval is based on efficacy results from the pivotal Phase 3 PERSIST-2 study of VONJO in patients with myelofibrosis (platelet counts less than or equal to 100 × 109/L). Patients were randomized 1:1:1 to receive VONJO 200 mg twice daily (BID), VONJO 400 mg once daily (QD) or best available therapy (BAT). Prior JAK2 inhibitor therapy was permitted. In this study, in the cohort of patients with baseline platelet counts below 50 × 109/L who were treated with pacritinib 200 mg BD, 29% of patients had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy, which included ruxolitinib. As part of the accelerated approval, CTI is required to describe a clinical benefit in a confirmatory trial. To fulfil this post-approval requirement, CTI plans to complete the PACIFICA trial, with expected results in mid-2025.
The most common adverse reactions (≥20%) following VONJO 200 mg twice daily were diarrhea, thrombocytopenia, nausea, anemia and peripheral edema. The most frequent serious adverse reactions (≥3%) following VONJO 200 mg twice daily were anemia, thrombocytopenia, pneumonia, cardiac failure, disease progression, pyrexia and squamous cell carcinoma of skin.
CTI is committed to supporting patients with myelofibrosis and removing barriers to access. As part of that commitment, CTI has established CTI Access, a patient support program that provides reimbursement and financial assistance programs for eligible patients. For more information, visit www.CTIaccess.com.
Under the terms of the previously announced debt and royalty transaction with DRI Healthcare Trust, the FDA approval of VONJO triggers the acquisition by DRI of a tiered royalty on VONJO for US$60 million. The proceeds of the transactions will be used by CTI to fund the launch of VONJO. As of December 31, 2021, CTI had cash and cash equivalents of approximately $65 million.
Conference Call and Webcast
CTI will host a conference call and webcast to discuss this announcement tomorrow, March 1, at 8:00 a.m. ET. To access the live call by phone please dial (877) 735-2860 (domestic) or (602) 563-8791 (international); the conference ID is 8860186. A live audio webcast of the event may also be accessed through the “Investors” section of CTI’s website at www.ctibiopharma.com. A replay of the webcast will be available for 30 days following the event.
About VONJO (pacritinib)
Pacritinib is an oral kinase inhibitor with activity against wild type Janus Associated Kinase 2 (JAK2), mutant JAK2V617F form and FMS-like tyrosine kinase 3 (FLT3), which contribute to signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. Myelofibrosis is often associated with dysregulated JAK2 signaling. Pacritinib has higher inhibitory activity for JAK2 over other family members, JAK3 and TYK2. At clinically relevant concentrations, pacritinib does not inhibit JAK1. Pacritinib exhibits inhibitory activity against additional cellular kinases (such as CSF1R and IRAK1), the clinical relevance of which is unknown.
VONJO is indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Legislative and Policy Updates That Could Impact Your Healthcare
– Strong support for extending telehealth flexibilities
With uncertainty around when the Biden administration will end the Public Health Emergency declaration, members of Congress and stakeholders are seeking a temporary extension of the Medicare telehealth waivers implemented during the pandemic. To that end, Senators Catherine Cortez Masto (D-NV) and Todd Young (R-IN) have introduced the Telehealth Extension and Evaluation Act (S.3593) and Representative Lloyd Doggett (D-TX) has introduced the Telehealth Extension Act (H.R. 6202) that both include an extension of the telehealth waivers for two years. In addition, more than 300 health groups sent a letter to House and Senate leadership urging a temporary extension before taking up permanent comprehensive telehealth legislation.
– FY2022 annual spending bills under negotiation
Federal government spending is operating under a continuing resolution through March 11, five months after the start of fiscal year 2022. This will allow time for negotiations on the 12 annual spending bills. A deal was recently reached on the top-line spending amount for the bills as well as the allocations for the 12 bills. Negotiations center around both funding levels and policy issues to be addressed in the bills. In addition to the annual appropriations bills, the White House is expected to ask Congress for emergency supplemental funding to address the COVID pandemic as well as prepare for future pandemics.
– President to address a joint session of Congress on March 1
The President will make his State of the Union address on March 1 to lay out his priorities for the nation. The President’s FY2023 budget request is expected to be released in late March or early April. The delay in the release of the President’s budget will likely impact Congress’ timeline for receiving input and developing the FY2023 annual spending bills.
– Congressional focus mental health and substance use
Committees in the House and Senate have been holding hearings on mental health and substance use to inform the development of legislation to be released in a spring/summer timeframe. The COVID-19 pandemic has exacerbated the need for services and highlighted the workforce challenges and disjointed systems of care. In addition, there are a wide range of Substance Abuse and Mental Health Services Administration programs that expire in September.
The Senate Finance Committee announced five areas of focus for their legislation:
- strengthening the workforce
- increasing integration, coordination, and access to care
- ensuring parity between behavioral and physical health care
- furthering the use of telehealth
- improving access for children and young people
The Senate Health, Education, Labor, and Pensions Committee and the House Energy and Commerce Committee are also developing legislation.
– CMS maintains the previous administration’s position on co-pay accumulators in exchange plans
In January, CMS issued the proposed rule Notice of Benefit and Payment Parameters for 2023 for qualified health plans offered through federal and state Affordable Care Act insurance exchanges. CMS is proposing to strengthen plan offerings by reinstating standardized benefits plans with flat dollar co-pays for specialty medication and strong non-discrimination protections.
However, the rule does not include any reference to co-pay accumulator adjustment policies and therefore does not reverse the Trump administration’s decision to allow insurers and pharmacy benefit managers to include co-pay accumulator adjustment policies in their plans. PAN submitted comments requesting CMS reinstate the agency’s original rule requiring issuers to count all payments made by or on behalf of the beneficiary, including patient co-pay assistance, toward patients’ annual deductible and out-of-pocket limit.
– MedPAC discusses prescription drugs telehealth
The Medicare Payment Advisory Commission (MedPAC) held a meeting in January in which the commissioners reviewed the status of the Medicare Part D program in preparation for their report to Congress in March. One notable trend in Part D is that a small share of enrollees who reach the catastrophic phase drive overall spending. At future meetings, commissioners will review MedPAC’s previous recommendations to strengthen Medicare Part D.
Myelofibrosis market to see new approvals in 2022, driving market growth
Myelofibrosis is a rare hematological malignancy with limited therapeutic options and significant unmet clinical need. Current first line therapy options are two Janus kinase (JAK) inhibitors (JAKis), Incyte’s Jakavi/Jakafi (ruxolitinib) and Impact Biomedicine’s Inrebic (fedratinib). Jakafi has dominated the myelofibrosis market since its launch in 2011 and has seen lucrative financial returns, achieving blockbuster status. However, Jakafi is not a curative agent. Since patients often discontinue the treatment and experience exacerbated anemia, clinicians prescribe additional lines of therapy. Several pipeline JAKis have entered Phase III trials, seeking to expand the lines of therapy by targeting Jakafi-refractory patients, capitalizing on a currently underserved patient population.
Notably, topline data have recently been released from the randomized, double-blind, active control Phase III MOMENTUM study comparing the selective JAK1, JAK2 and ACVR1 inhibitor, Sierra Oncology’s momelotinib, versus the anti-anemia molecule danazol. In Jakafi-refractory patients, momelotinib achieved all prespecified primary and secondary endpoints. A total of 25% (n=130) of patients experienced a total symptom score (TSS) reduction of ≥50% on momelotinib, demonstrating clear superiority to danazol (TSS reduction ≥50% in 9% of patients). Momelotinib was also superior in splenic response rate (SRR) compared to danazol (23% versus 3%) and demonstrated promising safety data with 54% of patients experiencing grade 3 or higher toxicities, compared to 65% on danazol.
Prevalence of Frailty in Patients With MPNs Linked to Subtype
Frailty is a common feature among patients with myeloproliferative neoplasms (MPNs) and may be associated with MPN subtype, according to the results of a study recently published in the journal Leukemia Research.
“Due to MPN-related symptoms and decreased physiologic reserve, physical frailty may be prevalent in this population,” the study investigators explained in their report.
The findings came from an analysis of the prospective Ontario Registry of Chronic Hematologic Malignancies and Incident Cardiovascular Disease (ORCHID) study, which is ongoing. Patients in this analysis were enrolled within a year of diagnosis. Represented diagnoses included chronic phase chronic myeloid leukemia (CML), primary myelofibrosis (PMF), polycythemia vera (PV), and essential thrombocythemia (ET).
Evaluation of frailty prevalence and trends was the goal of the study, and frailty was assessed through a combination of patient self-reports, review of electronic medical records, and standardized physical measurements. Patients were grouped across 3 frailty categories: frail, prefrail, or not frail, based on a series of metrics.
The analysis included 156 patients, of whom 60 patients had CML, 51 had PV, 16 had PMF, and 29 had ET. The ratio of females to males was reportedly balanced across subtypes, although within the PMF subgroup, 75% of patients were female. The mean age for the overall population was 63 years.
More than half of the patients overall (57%) were considered to have prefrail status, one-fourth (25%) were categorized as frail, and 18% were deemed to be not frail. Across MPN subtypes, PMF showed the greatest association with frailty, while ET was the least associated with it. Age was also associated with frailty.
The researchers identified possible differences in features associated with frailty across MPN subtypes. For instance, PMF appeared to be linked to unintentional weight loss more often than PV and ET.
“Although these results cannot be directly compared to control populations without MPNs, this study suggests that patients with MPNs may have a higher burden of frailty compared to the general population,” the researchers reported.
Learn more
Higher Symptom Burden More Likely to Improve With MPN Treatment
By Vicki Moore, PhD
Higher Symptom Burden More Likely to Improve With MPN Treatment
Data from 2 clinical trials involving patients with myeloproliferative neoplasms (MPNs) revealed the importance of considering symptom burden in evaluating treatment efficacy and tolerability. The study results were published in The Lancet Haematology.
The analysis included data from the single-arm phase 2 MPN-RC 111 study (ClinicalTrials.gov Identifier: NCT01259817) and from the randomized, open-label phase 3 MPN-RC 112 study (ClinicalTrials.gov Identifier: NCT01258856). MPN-RC 111 evaluated clinical-hematologic response to pegylated interferon alfa-2a in patients who had hydroxyurea resistance or intolerance. MPN-RC 112 evaluated clinical-hematologic response to pegylated interferon alfa-2a in comparison with hydroxyurea in treatment-naïve patients who had high-risk essential thrombocythemia (ET) or polycythemia vera (PV).
This analysis made use of results from patient questionnaires including the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and the European Organisation for the Research and Treatment of Cancer Core Quality of Life Questionnaire. The objective of the analysis was to analyze symptom burden with respect to both the clinical-hematologic response at 12-months and the baseline symptom burden.
Symptom burden was categorized as either high (total symptom score ≥20) or low (total symptom score <20). The researchers considered a clinically significant improvement in symptom burden to be an improvement of 50% or more in MPN-SAF total symptom score over 12 months for patients who had a nonzero total symptom score at baseline.
There were 114 patients analyzed from the MPN-RC 111 study (64 with ET, 50 with PV) and 166 patients (79 with ET, 87 with PV) from the MPN-RC 112 study. Among patients from the MPN-RC 111 study, 32% of complete responders and 20% of partial responders reported clinically significant improvements in symptom burden at 12 months.
For patients from the MPN-RC 112 study, among those treated with pegylated interferon alfa-2a, 19% of complete responders and 18% of partial responders reported a clinically significant improvement in symptom burden at 12 months. Of those treated with hydroxyurea, this outcome was reported in 27% of complete responders and 22% of partial responders.
Overall, responders — complete or partial — more often experienced a clinically significant improvement than did nonresponders (22% and 5%, respectively; P =.0003). However, the study investigators noted that most responders did not report a clinically significant improvement.
Additionally, patients who had a high symptom burden at baseline showed improvements in symptom burden at both 3 and 12 months, whether treated with pegylated interferon alfa-2a or hydroxyurea, with mean symptom score changes of -10.2 for pegylated interferon alfa-2a and -6.8 for hydroxyurea. Patients with low baseline symptom burden, however, experienced worse symptom burden at these time points. Mean symptom score changes were 3.2 with pegylated interferon alfa-2a and 3.4 with hydroxyurea for these patients.
“In summary, clinicians, researchers, and regulatory agencies should consider symptom burden and quality of life when evaluating treatment efficacy in patients with essential thrombocythaemia and polycythaemia vera,” they concluded.
Disclosures: Some authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Sierra Oncology Announces Momelotinib Achieved Statistically Significant Benefit on Symptoms, Anemia and Splenic Size in the Pivotal MOMENTUM Study for Myelofibrosis
—New Drug Application submission planned for second quarter of 2022—
—Full data set to be presented at an upcoming medical meeting—
SAN MATEO, Calif.–(BUSINESS WIRE)– Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company dedicated to delivering targeted therapies for rare cancers, today announced positive topline data from the pivotal Phase 3 MOMENTUM study—a global, randomized, double-blind clinical trial evaluating momelotinib (MMB) in myelofibrosis patients who are symptomatic and anemic and previously treated with an approved JAK inhibitor. The trial met all of its primary and key secondary endpoints.
“These data are extremely exciting and everything we had hoped to see from the trial,” said Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology. “To achieve statistically significant and clinically important efficacy across all prespecified primary and key secondary endpoints while maintaining platelet counts in such a difficult to treat patient population is remarkable, and a confirmation of the anemia response we identified in the comprehensive review of our previous Phase 3 studies.”
Topline data announced based on 195 patients (MMB n = 130; DAN n = 65) include:
- Primary Endpoint of Total Symptom Score (TSS) of >50%: 25% in the MMB arm vs. 9% in the control arm (p=0.0095)
- Secondary Endpoint of Transfusion Independence (TI): 31% in the MMB arm vs. 20% in the control arm (one-sided p=0.0064; non-inferiority)
- Secondary Endpoint of Splenic Response Rate (SRR) >35%: 23% in the MMB arm vs. 3% in the control arm (p=0.0006)
- The rate of Grade 3 or worse adverse events in the randomized treatment period was 54% in the MMB arm and 65% in the control arm. Serious treatment emergent adverse events were 35% in the MMB arm and 40% in the control arm.
- Mean baseline characteristics for all patients were TSS of 27, Hemoglobin (Hgb) of 8 g/dL and platelet count of 145 x 10 9/L
- The full data set will be presented at an upcoming medical meeting
“As a clinician, I am thrilled to see data that confirm the potential of momelotinib as a treatment option for myelofibrosis patients who are anemic or at risk of becoming anemic,” said Ruben Mesa, MD, FACP, Executive Director of the Mays Cancer Center, home to UT Health San Antonio, MD Anderson Cancer Center, and co-Principal Investigator of the study. “Anemia of myelofibrosis is strongly correlated with reduced quality of life and a decrease in overall survival. Half of all myelofibrosis patients present with anemia at diagnosis and virtually all become anemic over time. With currently approved therapies being myelosuppressive, it’s wonderful to know that we may soon have such an effective treatment option for these patients.”
Barbara Klencke, MD, Chief Medical Officer of Sierra Oncology, stated, “We are committed to working tirelessly to bring momelotinib to patients as quickly as possible. We would like to thank the patients and investigators who participated in this study and look forward to presenting the full data set at an upcoming medical meeting.”
Conference Call & Webcast
In connection with this announcement, Sierra will host a conference call and webcast on Tuesday, January 25, 2022, at 8:00 am ET. The call may be accessed by calling (833) 927-1758 (Toll-free in North America) or +1 (929) 526-1599 (International Dial-in) and entering the Conference ID number: 007608. The call will be webcast live and will be accessible through the Investor section of the Company’s website at www.SierraOncology.com. An archived replay of the webcast will be made available at the same location.About Momelotinib
Momelotinib is a potent, selective and orally bioavailable JAK1, JAK2 and ACVR1 / ALK2 inhibitor under investigation for the treatment of myelofibrosis in symptomatic, anemic patients previously treated with an approved JAK inhibitor. More than 1,200 subjects have received momelotinib since clinical studies began in 2009, including approximately 1,000 patients treated for myelofibrosis, several of whom remain on treatment for over 11 years. Momelotinib is the first and only JAK inhibitor to demonstrate positive data for all key hallmarks of the disease—symptoms, splenic response and anemia.About Myelofibrosis
Myelofibrosis is a rare blood cancer that results from dysregulated JAK-STAT signaling and is characterized by constitutional symptoms, splenomegaly (enlarged spleen) and progressive anemia. From prior studies with momelotinib, we know approximately half of myelofibrosis patients are moderately to severely anemic when eligible for JAK inhibitor treatment. Furthermore, currently approved JAK inhibitors only address symptoms and splenomegaly and are myelosuppressive. This can lead to worsening anemia, resulting in dose reductions that potentially reduce treatment effect.About the Pivotal MOMENTUM Clinical Trial
MOMENTUM is a global, randomized, double-blind Phase 3 clinical trial of momelotinib versus danazol in patients with myelofibrosis who were symptomatic and anemic, and had been previously treated with an FDA-approved JAK inhibitor. The study was designed to evaluate the safety and efficacy of momelotinib for the treatment and reduction of the key hallmarks of disease: symptoms, blood transfusions (due to anemia) and splenomegaly (enlarged spleen).The primary endpoint of the study is Total Symptom Score (TSS) reduction of >50% over the 28 days immediately prior to the end of Week 24 compared to baseline TSS, using the Myelofibrosis Symptom Assessment Form (MFSAF). Secondary endpoints included Transfusion Independence (TI) rate for >12 weeks immediately prior to the end of Week 24 with Hgb levels ≥ 8 g/dL, and Splenic Response Rate (SRR) based on splenic volume reduction of >35% at Week 24. The study enrolled 195 patients based on a planned 180 patients across 21 countries.
Danazol was selected as the treatment comparator given its use to ameliorate anemia in patients with myelofibrosis, as recommended by National Comprehensive Cancer Network (NCCN) and European Society of Medical Oncology (ESMO) guidelines. Patients were randomized 2:1 (MMB n = 130 and DAN n = 65) to receive either momelotinib or danazol. After 24 weeks of treatment, patients on danazol were allowed to crossover to receive momelotinib. Early cross-over to momelotinib was available for confirmed symptomatic splenic progression.
About Sierra Oncology
Sierra Oncology is a late-stage biopharmaceutical company on a mission to deliver targeted therapies that treat rare forms of cancer. We harness our deep scientific expertise to identify compounds that target the root cause of disease to advance targeted therapies with assets on the leading edge of cancer biology. Our team takes an evidence-based approach to understand the limitations of current treatments and explore new ways to change the cancer treatment paradigm. Together we are transforming promise into patient impact.For more information, visit www.SierraOncology.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Sierra Oncology’s expectations regarding the regulatory timeline, presentation of the results of the MOMENTUM trial, commercialization and future success of momelotinib, the company’s potential opportunity in myelofibrosis, the company’s ability to identify compounds and statements by the company’s Chief Medical Officer and the Executive Director of the Mays Cancer Center. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements. Such forward-looking statements are subject to risks and uncertainties, including, among others, the factors described under the heading “Risk Factors” set forth in Sierra Oncology’s filings with the Securities and Exchange Commission from time to time. Sierra Oncology undertakes no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.View source version on businesswire.com: https://www.businesswire.com/
news/home/20220125005390/en/
PharmaEssentia’s BESREMi® (ropeginterferon alfa-2b-njft) Now Available for the Treatment of People with Polycythemia Vera in the United States
PHARMAESSENTIA’S BESREMi® (ROPEGINTERFERON ALFA-2B-NJFT) NOW AVAILABLE FOR THE TREATMENT OF PEOPLE WITH POLYCYTHEMIA VERA IN THE UNITED STATES
BESREMi® is the only therapy indicated for adults with PV regardless of treatment history
PharmaEssentia introduces holistic services program to support prescribed patients
December 6, 2021, Burlington, MA – 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 that BESREMi® (ropeginterferon alfa-2b-njft) is now commercially available in the U.S. to eligible patients with polycythemia vera (PV). BESREMi was approved by the FDA in November as the only interferon for adults with polycythemia vera. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.
“Today marks the beginning of a new chapter in the treatment of PV. Our team is delivering on our goal to bring an innovative solution that may help more people manage not only the symptoms of PV, but target the disease itself to gain durable control with potential to reduce progression over time,” said Meredith Manning, U.S. General Manager. “We look forward to working closely with U.S. providers to raise awareness of this therapy and help advance treatment goals.”
PharmaEssentia SOURCE Now Available to Support People with PV in the U.S.
With the commercial availability of BESREMi, PharmaEssentia is also launching a comprehensive patient support program, which can be found at www.pharmaessentiaSOURCE.com.
U.S. FDA Approves BESREMi® (ropeginterferon alfa-ab-njft) as the Only Interferon for Adults With Polycythemia Vera
U.S. FDA APPROVES BESREMi® (ROPEGINTERFERON ALFA-2B-NJFT) AS THE ONLY INTERFERON FOR ADULTS WITH POLYCYTHEMIA VERA
With deep, durable control demonstrated by over 7.5 years of clinical data, BESREMi® can be used at any point in the PV journey to support treatment goals
Milestone represents PharmaEssentia’s first approved indication in the United States
November 12, 2021, Burlington, MA – 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 that the U.S. Food and Drug Administration (FDA) has approved BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with polycythemia vera (PV).
BESREMi is an innovative monopegylated, long-acting interferon, which exhibits its cellular effects in polycythemia vera in the bone marrow. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic, and infections disorders. PharmaEssentia is preparing to make BESREMi available in the coming weeks in the U.S.