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FDA Approves New Preventive Treatment for Rare Hereditary Disease

FDA Approves New Preventive Treatment for Rare Hereditary Disease

The U.S. Food and Drug Administration (FDA) recently approved Takhzyro (lanadelumab-flyo) as the first preventive monoclonal antibody to treat hereditary angioedema (HAE) to help prevent attacks in patients 12 years and older. HAE is a rare and potentially life-threatening genetic disease that can cause unpredictable, debilitating episodes of painful swelling in the limbs, abdomen and airway.

HAE affects approximately 8,000 people (1 in 50,000) in the United States, according to the FDA. Symptoms of HAE typically begin in childhood and worsen following puberty. Some patients may have multiple painful attacks each month, while others can go months without an episode.

A low level or improper function of a protein called C1 inhibitor causes HAE. The low levels of C1 inhibitor lead to an imbalance of other messenger peptides and enzymes in the body, including bradykinin and kallikrein. This imbalance causes fluid from tiny blood vessels to leak into surrounding tissue, which can lead to sudden, painful swelling. Takhzyro is the first preventive agent that targets plasma kallikrein, an enzyme chronically uncontrolled in people with HAE. As a plasma kallikrein inhibitor, Takhzyro prevents swelling attacks from occurring.

Takhzyro offers several advantages over other approved injectable treatments, including less frequent dosing (every two to four weeks) and ease of administration (most patients can self-administer the injection in less than one minute.) Other injectable products approved for prevention of HAE attacks include Cinryze and Haegarda, but both require treatment twice weekly and take longer to administer. Cinryze presents additional challenges as it needs to be administered intravenously.

Efficacy & Safety

Drug manufacturer Shire's Phase 3 HELP study demonstrated Takhzyro is effective for long-term prevention of HAE attacks. In the HELP trial, patients with HAE treated with Takhzyro once every two or four weeks experienced 73% to 87% fewer swelling attacks compared to placebo. Additionally, patients treated with Takhzyro saw a similar reduction (74% to 87%) in the number of swelling attacks requiring acute treatment.

The most common side effects associated with Takhzyro include injection site reactions, upper respiratory infections, headache, rash, myalgia and dizziness. Prescribing information is available at https://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf

Market Availability & Costs

Due to the high price of plasma-derived and biologic therapies, HAE treatments can lead to significant costs — potentially reaching more than $1 million per patient annually for those with poorly controlled HAE.

Takhzyro costs $52,968 per month — more than $635,000 annually per patient. Market analysts predict peak sales could climb to $1.6 billion within six years, capturing up to 60% of HAE market share.

The FDA granted Takhzyro priority review and breakthrough therapy designation, which helps accelerate clinical investigation of drugs expected to greatly impact the treatment of a certain disease. Takhzyro also received orphan drug designation, which also provides incentives to streamline and expedite drug development for rare diseases.

Forward-Thinking Strategies to Manage Drug Costs

MedImpact’s Pharmacy and Therapeutics Committee conducted a full clinical review of Takhzyro and evaluated utilization management to optimally manage drug costs in July, including a Prior Authorization Guideline. Claims for Takhzyro began processing and adjudicating on Friday, Sept. 7.

To learn more about how MedImpact can help you manage blockbuster drugs, reduce costs and improve care, go to pbm.medimpact.com.

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