Takeda has received U.S. FDA approval for MIMRYLO to treat erythrocytosis in adults with polycythemia vera (PV). MIMRYLO is a first-in-class hepcidin mimetic developed to regulate iron distribution. It also targets excess red blood cell production and helps control hematocrit levels.
Keeping the hematocrit level below 45% is still the main aim in the management of PV patients. Nevertheless, there are still some patients who continue having uncontrolled hematocrit levels.
“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.”
Polycythemia vera impacts around 90,000 individuals in the country of America. The disease leads to the overproduction of red blood cells and hematocrit level in the body. Thus, an increase in the blood viscosity increases the chances of the occurrence of a thrombotic event such as stroke and pulmonary embolism.
Hematocrit regulation can help in minimizing the risks involved, as well as alleviate some symptoms. Symptoms that could be experienced include fatigue, itching, inability to concentrate, and night sweats. Standard management methods of the condition currently include phlebotomy & cytoreductive therapy; however, 78% of patients continue to have uncontrolled hematocrit.
Patients with uncontrolled hematocrit also face a higher cardiovascular risk. Their risk of cardiovascular death or major cardiovascular events can increase fourfold.
“People living with PV often experience complex and invisible symptoms, from extreme fatigue to the emotional strain of living with a chronic blood cancer,” said Kapila Viges, Chief Executive Officer, MPN Research Foundation. “At the same time, we know that every patient’s experience with PV is different, underscoring the need to continue to listen closely to the community to understand what matters most. There remains a need for treatments that better address these daily challenges. This meaningful approval reflects important progress and brings forward a new treatment option in a disease where patients have long needed innovation and more choices. We are encouraged by MIMRYLO’s potential to help patients meet their treatment goals.”
MIMRYLO Shows Efficacy in Phase 3 VERIFY Trial
FDA approval was based on the results of the global Phase 3 VERIFY trial. In this trial, 293 patients with PV were analyzed using MIMRYLO along with regular medical treatments. MIMRYLO proved to be effective by meeting all endpoints and showing a good safety profile. Patients who used MIMRYLO showed better response rates than the ones in the placebo group. MIMRYLO helped control hemoglobin levels, reduced the need for phlebotomy, and alleviated fatigue.
“The approval of MIMRYLO underscores the strength of Takeda’s late-stage pipeline and our focus on developing genuinely differentiated therapies for patients who are urgently waiting for new options,” said Julie Kim, President and Chief Executive Officer, Takeda. “We are at an important inflection point as we prepare to deliver three new medicines, which have the potential to drive our future growth and are a reflection of our commitment to advancing innovation that doesn’t just add to the treatment landscape, but reshapes it. We are grateful to the patients, care partners, advocates and investigators who helped make this approval possible.”
Takeda will continue its research of MIMRYLO via the VERIFY extension. The company will also collaborate with authorities outside of the U.S. Adverse effects that were the most prevalent were injection site reactions and anemia.
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News Source: Businesswire.com
