Category Archives: Treatment

pharma& Receives FDA Approval for Pegasys® Prior Approval Supplement, Adding Loba biotech for API Production to Ensure Stable Long-term Supply in the United States

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Vienna, Austria, August 19, 2026 – pharmaand GmbH (pharma&) today announced that the U.S. Food and Drug Administration (FDA) has approved a Prior Approval Supplement (PAS) for Pegasys® (peginterferon alfa-2a), for the registration of Loba biotech GmbH, a wholly owned manufacturing subsidiary of pharma&, to produce the active pharmaceutical ingredient (API) peginterferon alfa-2a. This approval reinforces the long-term stability of the U.S. supply, ensuring continuity of care for eligible patients.

“We are delighted that the FDA has granted PAS approval so peginterferon alfa-2a can be produced at Loba biotech GmbH – this follows approvals from other regulatory agencies across the globe,” said Elmar Zagler, Co-Founder and Managing Director, pharma&. “Since acquiring peginterferon alfa-2a in 2021, pharma& has committed to maintaining access to the medicine for eligible patients, and this approval marks a significant step in supporting continuous supply in the U.S. and underscores our ongoing commitment to patients.”

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FDA Approves PharmaEssentia’s BESREMi® (ropeginterferon alfa-2b-njft) for Adults with Essential Thrombocythemia, A Rare Blood Cancer

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August 31, 2026

BURLINGTON, Mass, August 31, 2026 – PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446) today announced the U.S. Food and Drug Administration (FDA) has approved BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythemia (ET), making it the first new FDA-approved treatment for ET in nearly three decades. BESREMi has been approved for adults with ET, regardless of their genotype or disease status, including newly diagnosed patients who are naive to cytoreductive therapy. The ET approval expands the BESREMi product label, which has been commercially available for the treatment of adults with polycythemia vera (PV).

This approval marks a major milestone for people living with ET, a rare chronic blood cancer characterized by an overproduction of platelets. Patients with ET often require years of ongoing disease management and remain at risk for serious complications, such as heart attacks, strokes and pulmonary embolism.1 The approval of BESREMi provides a new ET treatment option and offers the potential for durable disease control, addressing a longstanding unmet need in ET care.

BESREMi is a long-acting interferon-based therapy designed to provide sustained disease management for ET. It offers a differentiated treatment option compared with current therapies that primarily focus on controlling platelet counts and symptoms, with limited impact on the underlying disease biology.2 Using novel monopegylation technology and an extended half-life, BESREMi is designed to target disease-driving cells in the bone marrow while reducing elevated platelet counts and disease burden.

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Takeda Receives U.S. FDA Approval of MIMRYLO™ (rusfertide), Marking a Potential Shift in the Treatment Paradigm for Polycythemia Vera

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August 28, 2026

  • MIMRYLO, a First-in-Class Medicine with a Unique Mechanism of Action, is Approved for the Treatment of Erythrocytosis in Adults with Polycythemia Vera (PV)
  • MIMRYLO Has Been Shown to Maintain Hematocrit Control, the Primary Treatment Goal in PV, as Well as Reduce Phlebotomy Burden and Improve Fatigue
  • Approval Supported by Phase 3 VERIFY Results Showing 76.9% of Patients Achieved Clinical Response During Weeks 20-32

OSAKA, Japan and CAMBRIDGE, Massachusetts, August 28, 2026 – Takeda (TSE:4502/NYSE:TAK) announced U.S. Food and Drug Administration (FDA) approval of the New Drug Application (NDA)* for MIMRYLO™ (rusfertide) for the treatment of erythrocytosis in adults with polycythemia vera (PV), a blood cancer.

MIMRYLO is a first-in-class hepcidin mimetic designed to regulate iron distribution in the body and red blood cell overproduction to control hematocrit levels, which is the ratio of red blood cells to the total amount of blood in the body. Maintaining controlled hematocrit levels below 45% is the primary treatment goal in PV.1

“For patients living with PV, uncontrolled hematocrit can have serious consequences, including an elevated risk of life-threatening thrombotic events,” said Andrew T. Kuykendall, M.D., VERIFY lead investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center. “Current treatments, such as phlebotomy, leave a significant gap for too many patients and can pose challenges to daily life and routines. The approval of MIMRYLO offers clinicians and patients a novel, first-in-class therapy that targets erythrocytosis, which drives excess red blood cell production in PV. The strength and consistency of the VERIFY data give me real confidence in MIMRYLO’s potential to advance how we treat PV in everyday practice and to maintain hematocrit control.”

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Ipsen completes acquisition of Kartos Therapeutics, strengthening late-stage Oncology pipeline

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Ipsen (Euronext: IPN; ADR: IPSEY) announced today it has completed the acquisition of Kartos Therapeutics, a clinical-stage biopharmaceutical company adding late-stage MDM2 inhibitor navtemadlin in Phase III clinical development in myelofibrosis.

About navtemadlin
Navtemadlin is an investigational oral MDM2 inhibitor being developed as an add-on therapy to ruxolitinib for patients with myelofibrosis who have a suboptimal response to ruxolitinib. The Phase III POIESIS study is evaluating whether the addition of navtemadlin could improve clinical outcomes compared with ruxolitinib alone in this patient population. Early clinical data demonstrate navtemadlin has the potential to transform suboptimal responses to standard of care ruxolitinib into clinically meaningful responses in patients with intermediate and high risk TP53wt myelofibrosis, to provide both enhanced clinical outcomes and potential disease-modifying benefit.

About myelofibrosis
Myelofibrosis is a myeloproliferative neoplasm, frequently linked to alterations in the JAK/STAT pathway, in which patients develop bone marrow fibrosis due to the abnormal proliferation of hematopoietic stem cells and secretion of fibrogenic cytokines. As marrow function declines, blood production shifts to other organs, most often the spleen, leading to splenomegaly. Myelofibrosis is characterized by bone marrow failure, fibrosis, splenomegaly and a high symptom burden that can significantly affect quality of life, including fatigue, night sweats and other progressive symptoms. It also carries a risk of transformation to acute myeloid leukemia. The median age at diagnosis is approximately 67–69 years and the condition affects around 1.5 per 100,000 people in the U.S. and Europe. Approximately 75–89% of patients are intermediate- or high-risk at diagnosis and more than 95% are TP53wt. Ruxolitinib, a JAK inhibitor, is the first-line standard of care; however, it is estimated that a significant proportion of patients have an initial suboptimal response and approximately 50%-75% discontinue treatment after three years. Median overall survival is typically one to two years after treatment discontinuation, underscoring the need for new strategies that can increase the number of patients that can achieve optimal clinical outcomes.

About Ipsen
We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries.

Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com.

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SVR With Pacritinib Is Linked With OS Benefit in Myelofibrosis With Thrombocytopenia

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May 26, 2025

Author(s): Ashling Wahner

Fact checked by: Gina Mauro

The achievement of spleen volume response (SVR) of at least 10% (SVR10) at week 12 with pacritinib (Vonjo) was positively associated with overall survival (OS) in patients with myelofibrosis and thrombocytopenia, although more stringent SVR thresholds were not significantly associated with OS, according to findings from a post hoc landmark analysis of the phase 3 PERSIST-2 trial (NCT02055781).1

The investigators assessed OS among SVR responders vs nonresponders using 4 different SVR thresholds: at least 35% (SVR35), at least 20% (SVR20), SVR10, and greater than 0% (SVR0). The data, which were published in the European Journal of Haematology, showed that the greatest separation of OS curves between responders and nonresponders was observed among patients who received pacritinib (n = 89) at 200 mg twice daily and achieved SVR10 vs those who did not respond at that threshold (HR, 0.00; 95% CI, 0.00-0.14; P < .01). No deaths were observed among the responders, whereas 5 nonresponders died.

“We found that a significant association was not observed with more stringent SVR or total symptom score [TSS] thresholds, likely because the number of responders was lower, and thus a larger number of observations would be needed to detect statistical significance at such thresholds,” lead study author Helen Ajufo, MD, MS, of the Department of Medicine, Leukemia Service, at Memorial Sloan Kettering Cancer Center, in New York, New York, and coinvestigators explained in the paper.

SVR0 and SVR20 also conferred improved OS, although these more stringent response thresholds were less prognostic of the pacritinib survival benefit.

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Real-World Data Shows Favorable Efficacy and Safety With Busulfan for MPNs

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Andrea S. Blevins Primeau, PhD, MBA
Publish Date

Busulfan treatment of myeloproliferative neoplasms (MPNs) demonstrated high response rates and infrequent adverse events (AEs), according to a study of real-world data from hospitals in the United Kingdom.

“Given the lack of prospective studies on the use of busulphan, our study contributes valuable real-world data on the safety and efficacy of busulphan which clinicians should find useful in managing this challenging cohort,” the researchers wrote in their report.

In the retrospective study, researchers analyzed data from 115 patients with MPNs from 13 hospitals. The median age of the cohort was 78 years and 44% of patients were male. The majority of patients had a diagnosis of essential thrombocythemia (ET) at 67%, followed by 24% with polycythemia vera (PV), 5% with MPN not otherwise specified, and 4% with myelofibrosis. JAK2 and CALR mutations were present among 62% and 13% of patients, respectively.

There were 16% of patients with a history of malignancy, including 8% of nonmelanoma skin cancers, and 8% with cancers that included those of the breast, prostate, lung, low-grade lymphoma, and melanoma.

One previous line of therapy (LOT) had been received by 13% of patients, 63% had 2 LOTs, 19% had 3 LOTs, and 5% had 4 LOTs. The most common previous cytoreductive therapy was hydroxycarbamide (78%), followed by anagrelide (16%), pegylated interferon (8%), P32 (3%), and ruxolitinib (2%).

The dosing regimens of busulfan included repeated single doses (31%) with a median dose of 38 mg, 1- to 4-week courses (30%) with a median dose 3.5 mg, and continuous therapy lasting more than 4 weeks (35%) with a median dose of 2 mg.

Our study contributes valuable real-world data on the safety and efficacy of busulphan which clinicians should find useful in managing this challenging cohort.

The median time from busulfan initiation to last follow-up or death was 23 months. There were 14% of patients who were alive with acceptable blood count control without any other cytoreductive therapy.

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Interferon Lowers Myelofibrosis Risk in Young Patients With Polycythemia Vera, Essential Thrombocytopenia

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May 15, 2025

Author(s): Alexandra Gerlach, Associate Editor

Early treatment with interferon reduced secondary myelofibrosis (sMF) risk in adolescent and young adult (AYA) patients with polycythemia vera (PV) or essential thrombocytopenia (ET), according to study data published in Leukemia.1

Red blood cells | Image Credit: © fotogurmespb - stock.adobe.com

MF is an incurable, rare hematologic malignancy characterized by the overproduction of red blood cells. This causes bone marrow scarring, which leads to severe anemia—a key factor impacting overall survival (OS) for patients with MF. It can develop as a primary disease or secondary to ET and PV, as well as progress to acute myeloid leukemia in some cases. Although MF is primarily diagnosed in older adults, 20% of cases are in individuals under the age of 40.1,2

AYA patients with ET or PV face unique clinical challenges due to their longer life expectancy because of a lack of data on long-term outcomes and treatment strategies. Existing guidelines are specific to older populations, despite the unique clinical considerations in younger patients.1

“These patients are expected to live for several decades, and the accumulation of additional thrombotic risk factors (age, cardiovascular conditions, additional mutations) may progressively heighten this risk,” the authors wrote. “This highlights a substantial and emerging concern regarding thrombotic events in this younger demographic.”1

The study evaluates complication rates, including thrombosis and progression to sMF, and the impact of interferon (IFN) on outcomes in patients diagnosed before the age of 25. The primary outcomes of the study were thrombosis-free survival (TFS), myelofibrosis-free survival (MFS), and OS in the entire cohort and for ET and PV patients separately, and the impact of treatment on these outcomes. Secondary outcomes were identification of risk factors associated with thrombotic events and sMF.1

The study included a total of 348 patients, of whom 278 were diagnosed with ET and 70 with PV, with a median age at diagnosis of 20 years. The incidence of thrombotic events was about 1.9 per 100 patient-years, with elevated white blood cell count (≥ 11 × 109/L) and absence of splenomegaly at diagnosis identified as significant risk factors. However, cytoreductive therapy did not reduce thrombotic risk. The incidence of sMF was 0.7 per 100 patient-years, with CALR mutations and a history of thrombosis associated with higher risk.1

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Seiter Explores Time of Initiation and Alternate Dosing of Ruxolitinib in Myelofibrosis

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By Targeted Oncology Staff
Fact checked by Jonah Feldman

During a live event, Karen Seiter, MD, discussed the role of the JAK/STAT pathway in myeloproliferative neoplasms and the use of ruxolitinib to treat myelofibrosis.

Karen Seiter, MD

Karen Seiter, MD

Professor of Medicine
New York Medical College
Director of  the Adult Leukemia Service
Westchester Medical Center
Valhalla, NY

CASE SUMMARY

  • A 68-year-old woman presented to her physician with symptoms of mild fatigue.
  • Spleen was palpable 6 to 7 cm below the left costal margin
  • Past medical history: no known comorbidities
  • Next-generation sequencing testing: JAK2 V617F mutation
  • Karyotype: 46,XX
  • Bone marrow biopsy: megakaryocyte proliferation and atypia with evidence of reticulin fibrosis
  • Blood smear: leukoerythroblastosis
  • Diagnosis: primary myelofibrosis

Risk

  • Dynamic International Prognostic Scoring System: intermediate-1
  • Mutation-Enhanced International Prognostic Scoring System for Transplantation-Age Patients: intermediate risk

Laboratory Values

  • Red blood cell count: 3.40 ×106/μL
  • Hemoglobin level: 9.7 g/dL
  • Hematocrit: 32.3%
  • Mean corpuscular volume: 94 μm3
  • White blood cell count: 23,000/μL
  • Platelet count: 450,000/μL
  • Peripheral blood blasts: <1%

Peers & Perspectives in Oncology: What disease states are currently classified as myeloproliferative neoplasms (MPNs)?

Seiter: The latest classification is the 2022 World Health Organization [WHO] classification.1,2 As a clinician, when we have myeloproliferative diseases, we think of MPNs [as meaning the patient] has too much of something: The WBC count is too high but with maturing myeloid and not with a lot of blasts, or the hemoglobin [level] is too high, or the [platelet count is] too high. That would be potentially chronic myelocytic leukemia [CML], polycythemia vera [PV], essential thrombocytopenia [ET], and for primary myelofibrosis, the most common presentation would be somebody with either anemia, with abdominal distension or early satiety from splenomegaly. The WBC count could be high; it could be low; platelets could be high, or they could be low.

When we’re working up patients with MPN, the first step is always to make sure that they don’t have CML, because CML can look like myelofibrosis because CML can present with a high platelet count. The first step is always to [test for] the Philadelphia chromosome and perform BCR::ABL1 testing to make sure it’s not CML.

Most of these patients are going to have a bone marrow [biopsy] done. There’s always the debate about, if you have somebody with a hemoglobin level of 20 g/dL, where it looks pretty clear that they have PV, do you need a bone marrow [biopsy] in order to formally diagnose the patient? You do because that’s part of the diagnostic criteria. But sometimes, if you have a much older patient with frailty, you have to consider the goals of care. Sometimes, there may be cases of patients who don’t get the bone marrow biopsy done. The only time it would be reasonable is if the hemoglobin level is high; for anything else, there are so many other things that [it] could be that it’s important to do the bone marrow biopsy for a diagnosis.

Mastocytosis used to be [listed among the] MPNs, but [the WHO] made it its own classification. MPNs with eosinophilia are a separate category. Now we have the myelodysplastic syndrome [MDS]/MPN overlap; that’s a separate category. Then, we have MDS, secondary myeloid neoplasms, and acute myeloid leukemia.

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Fedratinib Shows Promise in Rare Myeloproliferative Neoplasms

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By Andrew Kuykendall, MD

Andrew Kuykendall, MD, an assistant member at the H. Lee Moffitt Cancer Center of University of South Florida in Tampa, FL, discusses data from a phase 2 trial (NCT05177211) which evaluated fedratinib (Inrebic), a JAK2-selective kinase inhibitor, in patients with atypical chronic myeloid leukemia (aCML), chronic neutrophilic leukemia (CNL), myelodysplastic syndrome (MDS)/myeloproliferative neoplasm (MPN)-unclassifiable, and MDS/MPN-ring sideroblasts and thrombocytosis per the 2016 WHO classification.

According to findings from this multi-institutional, investigator-initiated study presented at the 2024 American Society of Hematology (ASH) Annual Meeting, fedratinib demonstrated promising efficacy and a manageable safety profile in this patient population.

At the time of data cutoff, 24 patients were enrolled and 19 patients were evaluable. A total of 53% of patients achieved a response at 24 weeks, including 50% symptom responses and 37.5% spleen responses. Of those treated for ≥24 weeks, spleen volume decreased by an average of 32%, and 85% showed symptomatic improvement. Responses were enriched in patients with CSF3R mutations (83%) and JAK-STAT pathway mutations (70%). Further, the median overall survival (OS) was estimated at 19.7 months.

“This was a very high-risk population, as these diagnoses are associated with poor prognosis. Despite that, we observed spleen responses in approximately 35% of patients, symptom responses in 50%, and some durable responses, with the median time on treatment nearing a year. These outcomes were particularly exciting given the molecular features of this patient population, which typically predict lower response rates,” Kuykendall shares.

Safety findings showed that fedratinib was well tolerated, The majority of adverse events (AEs) were grades 1 or 2, and the most commonly seen AEs were diarrhea (37.5%), constipation (37.5%), and anemia (29%). A single grade 4 neutropenia event resolved with dose adjustments.

“Regarding the safety profile, it was consistent with prior studies of fedratinib. There were some manageable low-grade gastrointestinal [adverse events]. We implemented a more proactive approach to mitigate these, including the use of antiemetics during the first 8 weeks. Overall, the [adverse event] profile remained manageable, and the efficacy outcomes likely exceeded our initial expectations,” adds Kuykendall.

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Ruxolitinib Discontinuation at Conditioning Does Not Increase GVHD Risk in Myelofibrosis

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May 7, 2025

Author(s): Alexandra Gerlach, Associate Editor

Stopping ruxolitinib (Jakafi; Incyte Corp) prior to the conditioning regimen may not influence graft-versus-host disease (GVHD) risk more than in patients with myelofibrosis (MF), according to data published in Cancer Immunology, Immunotherapy.1

MF is an incurable, rare hematologic malignancy that is part of a group of diseases called myeloproliferative neoplasms, which are characterized by the overproduction of red blood cells. This causes bone marrow scarring, leading to severe anemia—a key factor impacting overall survival for patients with MF. Other symptoms include fatigue, enlarged spleen, night sweats, bone pain, and weight loss. MF can develop as a primary disease or secondary to essential thrombocythemia and polycythemia vera. In some cases, it can progress to acute myeloid leukemia.2

Ruxolitinib is the standard of care for first-line treatment of patients with MF that was originally approved by the FDA in 2011 for the treatment of intermediate- and high-risk MF. It was also the first approved treatment for patients 12 years of age and older with steroid-refractory acute GVHD in 2019. Ruxolitinib is a Janus kinase (JAK) 1/2 inhibitor that targets the JAK/STAT pathway, a critical cellular signaling pathway that helps cells respond to cytokines. Identification of mutations within the JAK/STAT pathway paved the way for the development of agents such as ruxolitinib, fedratinib (Inrebic; Bristol Myers Squibb), momelotinib (Ojjaara; GSK), and pacritinib (Vonjo; CTI BioPharma).2,3

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