ORLADEYO Transforming Hereditary Angioedema Treatment Outcomes

Understanding the Burden of Rare Diseases

Rare diseases represent a significant challenge for both patients and healthcare systems worldwide. Research demonstrates that the collective burden of rare diseases is comparable to that of major conditions like cancer and heart failure.[i] Despite affecting smaller patient populations individually, these conditions create substantial healthcare demands and impose considerable economic strain on medical resources ORLADEYO.

Fortunately, modern advances in diagnostic capabilities and healthcare delivery systems are transforming the landscape for rare disease management. These innovations help alleviate disease burden, prevent unnecessary medical interventions, optimize health resource allocation, and guide clinicians toward selecting the most effective treatment options. Such improvements are essential for addressing the ongoing challenges facing healthcare systems globally.[ii]

What is Hereditary Angioedema (HAE)?

The Disease and Its Impact

Hereditary angioedema (HAE) exemplifies how targeted treatment can dramatically minimize disease burden in rare conditions. This genetic disorder is characterized by recurrent swelling attacks that affect approximately 1 in 50,000 people in the United States.[iii] These unpredictable episodes can significantly impact patients’ daily lives and overall wellbeing.

Attack Frequency and Consequences

HAE patients experience attacks an average of two to three times per month.[iv] These episodes are far from minor inconveniences—they cause:

• Severe pain and physical discomfort
• Reduced functioning in daily activities
• Diminished quality of life
• Potential life-threatening complications

The severity of HAE is underscored by hospitalization data: patients hospitalized with HAE remain in the hospital for more than five days per visit on average.[v] This extended hospitalization period highlights both the serious nature of the condition and its significant impact on healthcare resources.

ORLADEYO: A Breakthrough in HAE Prevention

The First Oral Prophylactic Option

Preventing HAE attacks is critical to improving patient outcomes, and ORLADEYO® (berotralstat) represents a major advancement in this effort. Approved by the FDA in 2020, ORLADEYO is the only FDA-approved oral, once-daily preventative (prophylactic) treatment option for HAE.

What ORLADEYO Is Approved For

ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema in adults and pediatric patients 12 years and older.

Important limitations: ORLADEYO is not approved for treating acute HAE attacks, and additional doses are not recommended due to the potential for QT prolongation. Patients should always consult their healthcare provider about appropriate treatment options.

Clinical Trial Evidence and Real-World Results

Proven Safety and Efficacy

Clinical trials have demonstrated that ORLADEYO is both safe and effective. Beyond reducing HAE attack frequency, studies have documented significant improvements in quality of life for patients taking the medication.[vi],[vii]

Real-World Performance Data

Extensive real-world studies have validated ORLADEYO’s clinical and economic benefits outside controlled trial settings. A comprehensive real-world study using specialty pharmacy data in the United States revealed remarkable outcomes:

• Patients experienced substantial and sustained reductions in HAE attack rates
• Benefits persisted over 18 months of treatment
• Improvements occurred regardless of baseline attack frequency[iv],[viii],[ix],[x]

This consistency across different patient populations demonstrates ORLADEYO’s robust effectiveness in diverse real-world scenarios.

Healthcare Cost Reduction and Resource Utilization

Significant Decreases in Healthcare Visits

A recent retrospective study examining a two-year treatment period revealed ORLADEYO’s impressive impact on healthcare resource utilization:[xi]

All-Cause Medical Visits:

• 34% decrease in all-cause hospitalizations
• 14% decrease in all-cause outpatient or emergency department visits

HAE Attack-Related Medical Visits:

• 60% decrease in HAE-related hospitalizations
• 50% decrease in HAE-related outpatient or emergency department visits
• 32% decrease in on-demand medication use for treating attacks in clinical settings

Economic Implications

These reductions represent substantial cost savings for the healthcare system while simultaneously improving patient experiences and outcomes. Fewer hospitalizations mean less disruption to patients’ lives and reduced strain on hospital resources.

Expert Perspectives on HAE Management

The Value of Long-Term Prophylactic Treatment

Dr. Sandra Christiansen, professor of medicine and director of translational research at the US HAEA Angioedema Center at the University of California, San Diego, emphasizes the transformative potential of prophylactic treatments:

“We are fortunate to have long-term prophylactic treatment (LTP) options for HAE which carry the potential to normalize patients’ lives. Unfortunately, patients may face barriers with approval for coverage of these transformational therapies.”

Dr. Christiansen stresses the importance of considering the totality of costs to the healthcare system, including those from sub-optimally controlled disease, when making coverage decisions. “Evidence from this study supports adopting this holistic view of HAE care,” she explains. “Oral LTP treatment use was shown to achieve a significant reduction in the utilization of healthcare resources. This outcome is welcome news for all—improved HAE control translates into a reduction in disease burden for patients and cost for payors.”

Commitment to Patient Access

Sandra Nestler-Parr, senior vice president of value and patient access at BioCryst (ORLADEYO’s manufacturer), underscores the company’s dedication: “We are committed to providing the broadest benefit possible to patients with HAE. By generating real-world evidence, we are able to show that our effective oral LTP option can not only meet the needs of patients but benefits the healthcare system as well.”

Important Safety Information

Indication

ORLADEYO® (berotralstat) is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

Key Safety Considerations

QT Prolongation: An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

Common Adverse Reactions (≥10% and higher than placebo):

• Abdominal pain
• Vomiting
• Diarrhea
• Back pain
• Gastroesophageal reflux disease

Dosage Modifications

A reduced dosage of 110 mg taken orally once daily with food is recommended in:

• Patients with moderate or severe hepatic impairment (Child-Pugh B or C)
• Patients taking chronically administered P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (e.g., cyclosporine)

Drug Interactions

P-gp inducers (e.g., rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. Appropriate monitoring and dose titration is recommended for concomitant medications with a narrow therapeutic index.

Special Populations

The safety and effectiveness of ORLADEYO in pediatric patients under 12 years have not been established. There are insufficient data regarding ORLADEYO use in pregnancy or breastfeeding.

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