Berinert

Indications for Berinert

Berinert is a C1 esterase inhibitor (human) used to treat sudden episodes of swelling caused by hereditary angioedema (HAE). This rare genetic condition occurs when the body does not produce enough of a specific protein, leading to fluid buildup and inflammation in various tissues.

Types of Attacks Treated

This medication is specifically approved to treat acute attacks affecting the following areas:

• Abdomen: Attacks in the stomach area can cause intense pain, nausea, and vomiting.
• Face: Swelling can occur in the lips, eyes, or other facial features.
• Larynx: Swelling in the throat (laryngeal attacks) is a medical emergency because it can block the airway and interfere with breathing.

Patient Population

Berinert is used for both adults and pediatric patients. It is intended for the treatment of active symptoms rather than for the routine prevention of attacks. Because throat swelling can be life-threatening, patients are typically advised to seek immediate medical attention even after using the medication for a laryngeal attack.

Restoring Protein Levels

Hereditary angioedema (HAE) is a genetic condition caused by a deficiency or dysfunction of a protein called C1 esterase inhibitor. This protein plays a critical role in regulating several systems in the body that control inflammation and fluid balance.

Blocking the Cause of Swelling

When C1 esterase inhibitor levels are too low, certain enzymes in the blood become overactive. This leads to the excessive production of a peptide called bradykinin. Bradykinin causes blood vessels to dilate and leak fluid into the surrounding tissues, which results in the severe swelling, pain, and inflammation seen during an HAE attack.

Berinert is a concentrate of human C1 esterase inhibitor. It works by:

• Replacing the missing protein in the bloodstream.
• Inhibiting the enzymes responsible for producing bradykinin.
• Regulating the body's inflammatory response to stop the progression of an attack.

By restoring the balance of these proteins, Berinert helps reduce the fluid leakage from blood vessels and eases the symptoms of an acute attack in the abdomen, face, or throat.

Common Side Effects

The most frequently reported side effects of Berinert include:

• Nausea and vomiting
• Abdominal pain or diarrhea
• Headache
• Muscle spasms
• Cough, sore throat, or stuffy nose
• Altered sense of taste
• Pain, redness, or swelling at the injection site

Serious Side Effects

While less common, Berinert can cause severe reactions that require immediate medical attention:

• Allergic reactions: Symptoms may include hives, rash, chest tightness, wheezing, or swelling of the face, lips, and tongue.
• Blood clots: There is a risk of blood clots forming in the lungs, brain, or limbs. Seek help for sudden shortness of breath, chest pain, swelling or pain in one leg, or sudden weakness.
• Infection risk: Because Berinert is made from human blood, there is a very small risk of transmitting infectious agents, such as viruses.

Hypersensitivity

Severe hypersensitivity reactions, including anaphylaxis, are possible. Signs of a reaction may include hives, generalized itching, chest tightness, wheezing, and low blood pressure. If a serious allergic reaction occurs, the infusion must be stopped immediately and appropriate treatment started.

Blood Clots

Serious blood clotting events (thromboembolic events) have been observed in patients using C1 esterase inhibitor products at recommended doses and during off-label use at high doses. Monitoring is recommended for patients with known risk factors for blood clots, such as:

• A history of thrombosis or underlying cardiovascular disease.
• The use of indwelling central venous catheters.
• The use of oral contraceptives or certain androgen medications.
• Prolonged periods of immobility.

Laryngeal Attacks

Treatment of laryngeal (throat) attacks requires careful monitoring. Because throat swelling can lead to sudden airway obstruction, patients must seek immediate medical care at an emergency facility after self-administering the medication for a throat attack.

Infectious Agents

Berinert is derived from human plasma. Although the manufacturing process includes steps to screen donors and remove or inactivate viruses, there remains a potential risk of transmitting infectious agents. This includes viruses and, theoretically, the agent that causes Creutzfeldt-Jakob disease.

Pregnancy

It is currently unknown whether Berinert can cause harm to an unborn baby or affect a person's ability to become pregnant. Clinical studies in pregnant women have not been conducted, and animal reproduction studies are also unavailable. Because of this lack of data, Berinert should be used during pregnancy only if a healthcare provider determines it is clearly needed.

Breastfeeding

It is not known if Berinert passes into human breast milk or if it could affect a nursing infant. If you are breastfeeding or planning to breastfeed, you should discuss the potential risks and benefits with your healthcare provider before using this medication to determine the best course of action for you and your baby.

Dosage Forms

Berinert is supplied as a sterile, lyophilized (freeze-dried) powder for reconstitution. It is intended for intravenous administration only and is not for subcutaneous use.

Available Strengths

Berinert is available in a single-use vial containing 500 International Units (IU) of C1 esterase inhibitor (human). It is typically provided in a kit that includes the following components:

• One vial of Berinert powder
• One vial of Sterile Water for Injection (diluent)
• A Mix2Vial transfer device
• Administration supplies, such as a filter and syringe

The powder must be reconstituted with the provided diluent before it is injected. Because it does not contain preservatives, the medication should be used immediately after mixing.

FDA Approval Status

Berinert is a C1 esterase inhibitor (human) that is FDA-approved for the treatment of acute abdominal, facial, or laryngeal attacks of hereditary angioedema (HAE). It was initially approved by the FDA in 2009 for use in adults and adolescents, and its indication was later expanded to include pediatric patients of all ages.

Regulatory Requirements

There are currently no Boxed Warnings or Risk Evaluation and Mitigation Strategy (REMS) requirements for Berinert. However, the FDA monitors the product for safety concerns common to plasma-derived therapies and C1 esterase inhibitors, including:

• Hypersensitivity: Severe allergic reactions, including anaphylaxis, have been reported.
• Thromboembolic Events: There is a risk of blood clots associated with the administration of C1 esterase inhibitor products, particularly at high doses or in patients with existing risk factors.
• Infectious Agents: Because Berinert is made from human blood, it carries a theoretical risk of transmitting infectious agents, such as viruses, despite screening and inactivation steps during manufacturing.

Factors Affecting Price

Berinert is a specialty biologic medication, and its cost is influenced by several variables:

• Weight-Based Dosing: The standard dose is 20 International Units (IU) per kilogram of body weight. Patients with higher body weights require more medication, which increases the cost per treatment.
• Insurance and Prior Authorization: Most insurance plans require prior authorization before they will cover Berinert. The specific out-of-pocket cost depends on the individual's pharmacy or medical benefit structure, including deductibles and coinsurance.
• Site of Care: Costs may differ depending on whether the medication is self-administered at home or administered by a healthcare professional in a clinical setting, which may incur additional service fees.

Financial Support Options

To help manage the cost of treatment for hereditary angioedema, financial assistance may be available:

• Manufacturer Support: The manufacturer often provides patient support services that can help navigate insurance approvals and identify financial aid options.
• Copay Assistance: Patients with commercial insurance may qualify for copay cards to lower their out-of-pocket expenses.
• Patient Assistance Programs: There may be programs available for patients who are uninsured or underinsured to help them access the medication at a lower cost.