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FDA Approves Rare Blood Disease Injection For Adults
Two individual studies found that after 7.5 years of treatment with Besremi 61% of patients with the rare blood disease polycythemia vera saw a complete hematological response.
FDA recently approved PharmaEssentia’s injection to treat adults with polycythemia vera, a rare blood disease that causes overproduction of red blood cells.
The monopegylated, long-acting interferon Besremi is the first FDA-approved medication for polycythemia vera that patients can take regardless of their treatment history. The drug is injected once every two weeks.
Individuals with polycythemia vera generally have AK2V617F mutation, increasing the risk for serious health problems, including blood clots, stroke, heart attack, myelofibrosis, and some cancers.
“The FDA approval of Besremi for people with polycythemia vera represents the next step in advancing patient care as it provides a critical addition to managing not only symptom burden and near-term complications, but also treating the cancer early, which may help reduce the risk of disease progression over time,” Srdan Verstovsek, MD, PhD, director of the Hanns A. Pielenz clinical research center for myeloproliferative neoplasms in the department of leukemia at the University of Texas MD Anderson Cancer Center, said in a PharmaEssentia press release.
More than 7,000 rare diseases affect over 30 million people in the US.
Polycythemia vera is a cancer originating from a disease-initiating stem cell in the bone marrow, resulting in a chronic increase of red blood cells, white blood cells, and platelets. Polycythemia vera affects nearly 6,200 Americans each year.
Current treatment regimens include phlebotomies and medicines to reduce the number of blood cells. Besremi works by attaching to certain receptors of the body and setting off a chain reaction that makes the bone marrow reduce blood cell production, an FDA spokesperson explained.
FDA approved Besremi based on safety from the PEGINVERA and PROUD/ CONTINUATION-PV studies, as well as efficacy data from the PEGINVERA clinical study program. Specifically, researchers looked at how many patients achieved a complete hematological response.
Overall, the trial found that after 7.5 years of treatment with Besremi, 61 percent of patients with polycythemia vera experienced a complete hematological response.
But the FDA highlighted that Besremi can cause liver enzyme elevations, low levels of white blood cells, platelets, joint pain, fatigue, itching, upper airway infection, muscle pain, and flu-like illness.
