A new therapy for polycythemia vera (PV), a rare and life-threatening blood cancer,1 could fundamentally change how physicians approach managing this chronic disease and improve the outlook for patients.
The rare nature of PV means the disease is often overlooked, with symptoms that can become more debilitating over time and progress to other deadly cancers.2 The disease begins with a mutation in stem cells in the bone marrow, resulting in the overproduction of red blood cells, white blood cells and platelets.1,3 This increase in blood cells may lead to serious complications, including blood clots, heart attack or stroke.2
“Currently, treatment of PV is guided predominantly by a watch and wait strategy, with the main goal of keeping the noticeable signs under control. However, the reality is that disease progression is inevitable, so improved approaches are critical to address clear unmet needs and improve the outlook for patients,” said John Mascarenhas, M.D., Director of the Adult Leukemia Program and Leader of Clinical Investigation within the Myeloproliferative Disorders Program at Mount Sinai.
The recent U.S. FDA approval of BESREMi® (ropeginterferon alfa-2b-njft) as the only interferon for the treatment of adults with PV enables physicians to move beyond managing the symptoms and complications and focus more on treating the totality of the disease earlier in the patient journey. BESREMi is a structurally innovative monopegylated interferon, which exhibits its cellular effects in PV within the bone marrow. The long-acting design of the treatment allows for dosing once every two weeks initially until hematologic parameters are stabilized, with favorable tolerability to help meet the individual needs of patients.
“With the availability of BESREMi, which has more than 7.5 years of clinical data and a well-established, long-term safety profile, we are better equipped to proactively control this life-long, progressive cancer,” added Dr. Mascarenhas. “This is the beginning of a shift toward a more holistic approach to PV care and will help more patients achieve their long-term health goals."
As with all interferons, certain precautions should be taken with BESREMi. The treatment was approved with a Boxed Warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders. Physicians should determine if BESREMi is the right treatment for their patient.
The U.S. FDA approval was based on efficacy data from the PEGINVERA clinical study program. The study showed that after 7.5 years of treatment with BESREMi, 61% of patients with PV experienced a complete hematological response (defined as hematocrit <45% without phlebotomy for at least 2 months since last phlebotomy, platelets ≤ 400 x 109/L, leukocytes ≤10 x 109/L, normal spleen size (longitudinal diameter ≤12 cm for females and ≤ 13 cm for males). Importantly, 80% of patients achieved a hematological response (based on objective laboratory parameters only, with the exclusion of normal spleen size and thrombosis). These parameters are the most commonly used metrics to make therapeutic decisions. In the pooled safety data from the PEGINVERA and PROUD/CONTINUATION-PV studies, the most common adverse reactions reported in > 40% of patients in the PEGINVERA study were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis and musculoskeletal pain. In the pooled safety population, 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%). The most common serious adverse reactions observed during the PEGINVERA study (> 4%) included urinary tract infection (8%), transient ischemic attack (6%) and depression (4%).
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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.
WARNINGS AND PRECAUTIONS
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%).
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
Please see accompanying full Prescribing Information, including Boxed Warning.
BESREMi is indicated for the treatment of adults with polycythemia vera