Lanadelumab

Indications

Lanadelumab is used for the routine prevention of hereditary angioedema (HAE) attacks. HAE is a rare genetic disorder that causes recurrent episodes of severe swelling in various parts of the body, such as the limbs, face, intestinal tract, and airway.

Usage Considerations

• Preventive Treatment: This medication is used as a long-term preventive measure to reduce the frequency and severity of HAE attacks.
• Age Range: It is approved for use in adults and pediatric patients 2 years of age and older.
• Not for Acute Attacks: Lanadelumab is not intended to treat an HAE attack once it has started. If an attack occurs, a separate rescue medication must be used as directed by a healthcare provider.

Mechanism of Action

Lanadelumab is a type of protein called a monoclonal antibody. It is designed to target and block the activity of an enzyme in the blood known as plasma kallikrein.

In individuals with hereditary angioedema (HAE), plasma kallikrein becomes overactive. This overactivity leads to the excessive release of a peptide called bradykinin. Bradykinin is responsible for making blood vessels leak fluid into nearby tissues, which results in the sudden and severe swelling attacks associated with the condition.

By inhibiting plasma kallikrein, lanadelumab helps to:

• Limit the production of bradykinin.
• Prevent the fluid leakage that causes swelling.
• Reduce the overall number of HAE attacks.

This medication is intended for long-term prevention. It works continuously in the system to keep protein levels regulated, rather than treating the symptoms of an active attack once it has begun.

Common Side Effects

The most frequently reported side effects of lanadelumab are usually mild and may include:

• Redness, pain, bruising, or swelling at the injection site
• Upper respiratory tract infections, such as the common cold or sore throat
• Headache
• Muscle aches
• Dizziness
• Diarrhea
• Skin rash

Serious Side Effects

In some cases, lanadelumab can cause severe allergic reactions. You should contact a healthcare provider immediately if you notice any of the following symptoms:

• Wheezing or difficulty breathing
• Chest tightness
• Swelling of the face, lips, tongue, or throat
• Severe rash or hives
• Fast heartbeat
• Feeling faint or lightheaded

Allergic Reactions

Serious hypersensitivity reactions, including anaphylaxis, have been reported with the use of lanadelumab. If signs of a severe allergic reaction occur—such as hives, swelling of the face or throat, difficulty breathing, or dizziness—medical attention is necessary.

Not for Acute Attacks

Lanadelumab is a preventive treatment and is not used to treat an acute hereditary angioedema (HAE) attack. In the event of an acute attack, a separate rescue medication should be used as directed by a healthcare provider.

Latex Sensitivity

The needle cap of the prefilled syringe may contain natural rubber latex. This may cause allergic reactions in individuals with a known sensitivity to latex.

Pregnancy and Breastfeeding

There is limited data regarding the safety of lanadelumab during pregnancy or while breastfeeding. The potential risks and benefits are typically discussed with a healthcare professional before starting or continuing treatment.

Pregnancy

It is currently unknown whether lanadelumab can cause harm to an unborn baby or increase the risk of miscarriage. Research in humans is insufficient to determine if there is a drug-associated risk of major birth defects.

Because lanadelumab is a monoclonal antibody, it is expected to cross the placenta as pregnancy progresses. The potential for effects on the fetus is likely to be greater during the second and third trimesters. If you are pregnant or planning to become pregnant, discuss the potential risks and benefits of this treatment with your healthcare provider.

Breastfeeding

It is not known if lanadelumab passes into human breast milk, how it affects milk production, or if it has any effect on a nursing infant. If you are breastfeeding or planning to breastfeed, consult your doctor to weigh the benefits of breastfeeding against the potential risks to the infant.

Lanadelumab is supplied as a sterile, preservative-free solution for injection under the skin (subcutaneous use). It is available in two primary delivery formats:

• Single-dose vials: Glass vials containing the liquid medication.
• Single-dose prefilled syringes: Ready-to-use syringes containing a fixed dose of the medication.

The solution is typically clear to slightly opalescent and colorless to slightly yellow. It is available in different strengths to accommodate different age groups, including adults, adolescents, and pediatric patients. Each vial or syringe is intended for a single use only and should be discarded after the dose is administered.

FDA Approval History

Lanadelumab was first approved by the U.S. Food and Drug Administration (FDA) in August 2018. It was initially indicated for the prevention of hereditary angioedema (HAE) attacks in patients 12 years of age and older. In February 2023, the FDA expanded the approved use to include pediatric patients aged 2 to less than 12 years.

Regulatory Designations

During its development and review, the FDA granted lanadelumab several special designations to facilitate its availability for a rare condition:

• Orphan Drug: This status is given to medications intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States.
• Breakthrough Therapy: This designation is intended to expedite the development and review of drugs for serious conditions where preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapies.
• Priority Review: This designation directs the FDA's attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.

Safety and Monitoring

There are currently no Boxed Warnings or Risk Evaluation and Mitigation Strategy (REMS) programs required by the FDA for lanadelumab. It is approved specifically for the prevention of HAE attacks and is not indicated for the treatment of acute attacks. The medication is administered via subcutaneous injection by a healthcare professional or through self-administration after proper training.

Insurance and Prior Authorization

Lanadelumab is classified as a specialty biologic medication. Most insurance providers require prior authorization, a process where your healthcare provider must submit documentation to justify the medical necessity of the drug before coverage is approved.

Specialty Pharmacy Services

Because it is a high-cost medication requiring specific handling, lanadelumab is usually dispensed through specialty pharmacies. These pharmacies coordinate with insurance companies to determine your specific co-pay or coinsurance amounts and arrange for the delivery of the medication.

Financial Assistance Programs

Several options may be available to help reduce the cost of treatment:

• Co-pay Assistance: Eligible patients with commercial insurance may qualify for co-pay cards that can significantly reduce out-of-pocket costs.
• Patient Assistance Programs: These programs may provide the medication at no cost to individuals who are uninsured or meet certain income requirements.
• Support Programs: Manufacturer-sponsored programs often provide dedicated case managers to help patients navigate insurance claims and identify financial resources.

Patients are encouraged to contact their insurance provider and the medication's manufacturer to understand the specific costs and support options available to them.