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

Posted on by

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.”

Read more

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

Posted on by

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.

Read more

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

Posted on by

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.”

Read more

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

Posted on by

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.

Read more

QIAGEN and Incyte Announce Precision Medicine Collaboration to Develop Companion Diagnostics for Patients With Mutant CALR-expressing Myeloproliferative Neoplasms (MPNs)

Posted on by

VENLO, Netherlands & WILMINGTON, Del.–(BUSINESS WIRE)–Jun. 15, 2025– QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) and Incyte (Nasdaq: INCY) today announced a new global collaboration to develop a novel diagnostic panel to support Incyte’s extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), a group of rare blood cancers, including Incyte’s monoclonal antibody INCA033989, targeting mutant calreticulin (mutCALR), which is being developed in myelofibrosis (MF) and essential thrombocythemia (ET).

Click here for the full article

Positive Late-Breaking Data for Incyte’s First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025

Posted on by

WILMINGTON, Del.–(BUSINESS WIRE)–Jun. 15, 2025– Incyte (Nasdaq:INCY) today announced the first clinical data from two studies evaluating the safety, tolerability and efficacy of INCA033989, a novel, first in class, Incyte-discovered, targeted monoclonal antibody in patients with mutant calreticulin (mutCALR)-expressing myeloproliferative neoplasms (MPNs). These data – featured today in the Late-Breaking Oral Session (#LB4002) at the European Hematology Association 2025 (EHA2025) Congress in Milan, Italy – focus on the dose escalation portion of the studies in patients with high risk essential thrombocythemia (ET) who are resistant/intolerant to prior cytoreductive therapy.

The studies evaluated the safety and efficacy of INCA033989 in patients with ET as measured by hematologic response and reduction in mutCALR variant allele frequency (VAF).

Click here for the full article

Interferon Improves Myelofibrosis-Free Survival in AYA Patients with ET and PV

Posted on by

By Melissa Badamo – Last Updated: May 27, 2025

While cytoreductive drugs did not reduce thrombosis risk in adolescent and young adult (AYA) patients with essential thrombocythemia (ET) and polycythemia vera (PV), interferon “significantly improved” myelofibrosis-free survival (MFS) compared with other treatments, according to a study published in Leukemia.

The retrospective study explored the long-term complications and impact of cytoreductive drugs on patient outcomes, thrombotic risk, and progression to secondary myelofibrosis (sMF) in 348 patients diagnosed with ET (n=278) or PV (n=70) before the age of 25 years. The primary end points were MFS, thrombosis-free survival (TFS), and overall survival. Secondary end points included identification of risk factors associated with thrombotic events and progression to sMF.

In the ET cohort, 147 (53%) patients had JAK2 mutations, 43 (16%) had CALR mutations, 3 (1%) had MPL mutations, and 85 (30%) were triple negative (TN). All patients with PV had JAK2 mutations.

A total of 237 (68%) patients were treated with a cytoreductive drug, including 185 patients with ET (66.5%) and 52 patients with PV (74.3%). Patients received one line of therapy (n=97; 41%), two lines of therapy (n=82; 35%), or three or more lines of therapy (n=58; 24%). The most prescribed first-line treatments were hydroxycarbamide (n=126; 53%), interferon (n=55; 23%), anagrelide (n=52; 22%), and alternative drugs (n=4; 2%). The median follow-up was 8.5 years.

Thrombotic Risk

Forty-four patients presented 57 thrombotic events, with a risk of 1.9 per 100 patient-years. The 10- and 20-year probability of TFS was 86.8% and 78.8% for the entire cohort, 86.9% and 80.0% for patients with ET, and 84.4% and 76.3% for patients with PV.

In a multivariate analysis, elevated white blood cell count (>11 × 109/L; hazard ratio [HR], 2.7; P=0.012) and the absence of splenomegaly at diagnosis (HR, 5.7; P=0.026) were associated with increased risk for thrombosis.

Choice of first treatment did not correlate with differences in TFS. The 10- and 20-year TFS were 83.9% and 79.9% for interferon, 81.4% and 70.2% for hydroxycarbamide, and 91.6% and 76.3% for anagrelide (P=0.281)

Read more

How Essential Thrombocythemia and Polycythemia Vera Affect Blood Flow

Posted on by

May 27, 2025

Author(s): Dr. Tiziano Barbui

Fact checked by: Spencer Feldman

Excess blood cell production in two myeloproliferative neoplasms (MPNs) — essential thrombocythemia, or ET, and polycythemia vera, also known as PV — can impair circulation, according to Dr. Tiziano Barbui.

Barbui is a professor of hematology and founder of the department of hematology at Bergamo Hospital. He is currently the scientific director of clinical research foundation at Papa Giovanni XXIII Hospital, Bergamo, Italy.

In a video interview with CURE, Barbui explained that both diseases begin in the bone marrow and are marked by abnormal increases in blood cell counts. In polycythemia vera, red blood cells are overproduced, increasing blood viscosity and making it harder for blood to flow through small vessels. In essential thrombocythemia, the problem lies with excess platelet production.

Barbui emphasized that treatment is essential not only to relieve symptoms but also to prevent more serious complications linked to impaired blood circulation in both MPNs.

Read more

 

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

Posted on by

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.

Read more

Efficacy of JAK1/2 inhibition in murine myeloproliferative neoplasms is not mediated by targeting oncogenic signaling

Posted on by

May 24, 2025

Sivahari Prasad Gorantla, Michael Rassner, Kirstyn Anne Crossley,…Robert Zeiser & Justus Duyster

Abstract

Ruxolitinib is a potent JAK1/JAK2 inhibitor, approved for the treatment of primary myelofibrosis (PMF) patients based on the concept of inhibition of oncogenic signaling. However, the effect of ruxolitinib on JAK2-V617F allelic burden is modest, suggesting that inhibition of JAK2-V617F signaling-driven clone expansion is not the main mechanism of action. We evaluate whether ruxolitinib mainly blocks the proliferation of the malignant clone or exerts its effects also by targeting non-malignant cells. Therefore, we develop two JAK2-V617F-driven myeloproliferative neoplasm (MPN) mouse models harboring ruxolitinib resistance mutations. Mice carrying ruxolitinib-resistant JAK2-V617F-driven MPN respond to ruxolitinib treatment similar to mice with ruxolitinib-sensitive JAK2-V617F MPN with respect to reduction of spleen size, leukocyte count and pro-inflammatory cytokines in the serum. Ruxolitinib reduces pro-inflammatory cytokines in both stromal cells and non-malignant hematopoietic cells. Using a rigorous ruxolitinib resistance mutation approach, we can prove that ruxolitinib acts independent of oncogenic JAK2-V617F signaling and reduces the main features of MPN disease such as spleen size and leukocyte counts. Our findings characterize the mechanism of action for ruxolitinib in MPN.

Read more