FDA Approves Treatment for Rare Blood Disease

Treatment is First FDA-Approved Option Patients Can Take Regardless of Previous Therapies

Today, the U.S. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. The excess cells thicken the blood, slowing blood flow and increasing the chance of blood clots.

“Over 7,000 rare diseases affect more than 30 million people in the United States. Polycythemia vera affects approximately 6,200 Americans each year,” said Ann Farrell, M.D., director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research. “This action highlights the FDA’s commitment to helping make new treatments available to patients with rare diseases.”

Besremi is the first FDA-approved medication for polycythemia vera that patients can take regardless of their treatment history, and the first interferon therapy specifically approved for polycythemia vera.

Treatment for polycythemia vera includes phlebotomies (a procedure that removes excess blood cells though a needle in a vein) as well as medicines to reduce the number of blood cells; Besremi is one of these medicines. Besremi is believed to work by attaching to certain receptors in the body, setting off a chain reaction that makes the bone marrow reduce blood cell production. Besremi is a long-acting drug that patients take by injection under the skin once every two weeks. If Besremi can reduce excess blood cells and maintain normal levels for at least one year, then dosing frequency may be reduced to once every four weeks.

The effectiveness and safety of Besremi were evaluated in a multicenter, single-arm trial that lasted 7.5 years. In this trial, 51 adults with polycythemia vera received Besremi for an average of about five years. Besremi’s effectiveness was assessed by looking at how many patients achieved complete hematological response, which meant that patients had a red blood cell volume of less than 45% without a recent phlebotomy, normal white cell counts and platelet counts, a normal spleen size, and no blood clots. Overall, 61% of patients had a complete hematological response. 

Besremi can cause liver enzyme elevations, low levels of white blood cells, low levels of platelets, joint pain, fatigue, itching, upper airway infection, muscle pain and flu-like illness. Side effects may also include urinary tract infection, depression and transient ischemic attacks (stroke-like attacks). 

Interferon alfa products like Besremi may cause or worsen neuropsychiatric, autoimmune, ischemic (not enough blood flow to a part of the body) and infectious diseases, which could lead to life-threatening or fatal complications. Patients who must not take Besremi include those who are allergic to the drug, those with a severe psychiatric disorder or a history of a severe psychiatric disorder, immunosuppressed transplant recipients, certain patients with autoimmune disease or a history of autoimmune disease, and patients with liver disease.

People who could be pregnant should be tested for pregnancy before using Besremi due to the risk of fetal harm. 

Besremi received orphan drug designation for this indication. Orphan drug designation provides incentives to assist and encourage drug development for rare diseases.

The FDA granted the approval of Besremi to PharmaEssentia Corporation.

Related Information

Polycythemia vera (POL-e-si-THEE-me-ah VAY-rah or VE-rah), or PV, is a rare blood complaint in which your body makes too numerous red blood cells.

The redundant red blood cells make your blood thicker than normal. As a result, blood clots can form more fluently. These clots can block blood inflow through your highways and modes, which can beget a heart attack or stroke.
Thicker blood also does not flow as snappily to your body as normal blood. Braked blood inflow prevents your organs from getting enough oxygen, which can beget serious problems, similar as angina (an-JI-nuh or AN-juh-nuh) and heart failure. (Angina is casket pain or discomfort.)

Overview
Red blood cells carry oxygen to all corridor of your body. They also remove carbon dioxide (a waste product) from your body’s cells and carry it to the lungs to be exhaled.

Red blood cells are made in your bone gist — a sponger-suchlike towel inside the bones. White blood cells and platelets ( PLATE-lets) also are made in your bone gist. White blood cells help fight infection. Platelets stick together to seal small cuts or breaks on blood vessel walls and stop bleeding.

Still, your bone gist makes too numerous red blood cells, If you have PV. It also can make too numerous white blood cells and platelets.

A mutation, or change, in the body’s JAK2 gene is the major cause of PV. This gene makes a protein that helps the body produce blood cells. What causes the change in the JAK2 gene is not known. PV generally is not inherited — that is, passed from parents to children through genes.

PV develops sluggishly and may not beget symptoms for times. The complaint frequently is plant during routine blood tests done for other reasons.

When signs and symptoms are present, they are the result of the thick blood that occurs with PV. This consistence slows the inflow of oxygen-rich blood to all corridor of your body. Without enough oxygen, numerous corridor of your body will not work typically.

For illustration, slower blood inflow deprives your arms, legs, lungs, and eyes of the oxygen they need. This can beget headaches, dizziness, itching, and vision problems, similar as blurred or double vision.

The FDA, an agency within theU.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of mortal and veterinary medicines, vaccines and other natural products for mortal use, and medical bias. The agency also is responsible for the safety and security of our nation’s food force, cosmetics, salutary supplements, products that give off electronic radiation, and for regulating tobacco products.

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