Interferon therapy has become a treatment option for certain types of cancer, but it has not been used to treat cancers characterized by excessive production of blood cells. The FDA has now approved the PharmaEssentia interferon drug to treat polycythemia, a rare blood cancer that can develop into a series of serious cardiovascular problems.
The agency Friday night Regulatory nod The PharmaEssentia drug, ropeginterferon alfa, made it the first drug therapy approved for polycythemia vera. This is also the company’s first product approved by the FDA. PharmaEssentia Taiwan, which operates its US headquarters in Burlington, Massachusetts, will sell its drugs under the name of Besremi.
Polycythemia vera is caused by mutations in bone marrow stem cells, which are where blood cells are produced. The high red blood cell levels caused by this disease cause abnormally thick blood, which also means that blood clots are more likely to form, putting patients at risk of heart attack or stroke. Polycythemia vera can develop into other types of blood cancer.
According to the FDA, polycythemia vera affects approximately 6,200 Americans each year. Treatment options include bloodletting, that is, drawing blood to bring the red blood cell count close to normal. However, this procedure did not solve the problem of high white blood cell and platelet levels. Another treatment option is chemotherapy or other drugs that inhibit blood cell formation.But the National Rare Disease Organization Notes Some medications that work in this way can also increase the risk of leukemia.
PharmaEssentia drug is an engineered version of interferon alpha, which is a signal protein produced by the immune system that can regulate and activate the immune response. Interferon therapy has been developed and approved for certain viral infections and cancers. The way PharmaEssentia drugs treat polycythemia vera is not fully understood. It is believed that it works by attaching to interferon alpha receptors, which in turn initiate a signal cascade that reduces the production of bone marrow blood cells.
PharmaEssetia has designed Besremi to have a long-lasting effect on the body. The drug is given by subcutaneous injection once every two weeks. The FDA stated that if patients respond to treatment and can maintain normal red blood cell levels for at least one year, the frequency of dosing may be reduced to once every 4 weeks.
Besremi was evaluated in a seven-and-a-half-year open label study. The 51 patients in the study received PharmaEssentia for an average of five years. The main goal is to assess how many patients have achieved a complete hematological response-the red blood cell volume is less than 45% and has not been bleeding recently, the white blood cell and platelet counts are at normal levels, the spleen is normal in size and there is no thrombosis. The FDA stated that 61% of patients in the study achieved this response.
Side effects reported in clinical trials include elevated liver enzymes, low white blood cell and platelet levels, joint pain, fatigue, and upper respiratory tract infections. The FDA warns that more serious side effects may include urinary tract infections, depression, and stroke-like episodes. These risks are marked in a black box warning on the drug label. This warning reminds doctors and patients that, like other interferon alfa products, Besremi may cause or aggravate neuropsychiatric, autoimmune, ischemic and infectious diseases. These conditions can be life-threatening. The label recommends that clinicians closely monitor patients and stop treatment when symptoms of these diseases worsen.
The drug may also cause harm to the unborn baby. The FDA stated that before using the PharmaEssentia drug, women who may become pregnant should be tested for pregnancy.
Besremi was previously approved by the European Medicines Agency in 2019. It has also obtained regulatory approvals in Taiwan and South Korea. PharmaEssentia says The drug will be available in the United States in the next few weeks. The drug is still in phase 3 clinical trials for the treatment of essential thrombocythemia, a disease in which the body produces too many platelets. The company’s product line includes clinical-stage compounds for the treatment of breast and stomach cancers, liver infections, hepatitis D, and actinic keratosis of the skin.
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