From November 13th through the 15th, the American College of Allergy, Asthma & Immunology (ACAAI) held their 2020 Annual Scientific Meeting. While the coronavirus pandemic necessitated that the meeting be held online, several organizations still shared scientific and research data to shape the future of patient care. During the meeting, BioCryst Pharmaceuticals (“BioCryst”) presented three posters on the burden of injectable prophylactic treatment, and perceptions on said treatment, for patients with hereditary angioedema (HAE), their caregivers, and their physicians. The data was sourced from a cross-sectional study. You can view the poster presentations on the ACAAI Annual Scientific Meeting website under the presentation numbers #160, 161, and 162.

Prophylaxis Care

First off, what is prophylaxis therapy? According to RxList, prophylaxis therapy uses a prophylactic, or:

a medication or a treatment designed and used to prevent a disease from occurring. For example, prophylactic antibiotics may be used after a bout of rheumatic fever to prevent the subsequent development of Sydenham’s chorea.

In this case, prophylaxis therapy would be used to prevent HAE flares or attacks. However, as BioCryst discovers, there is still a significant treatment burden associated with current therapeutic options. 30 caregivers, 75 patients, and 109 doctors enrolled in the study. Findings included:

• Nearly half of the caregivers suggested that learning to properly administer HAE treatment was difficult. They needed to understand how to self-administer the therapy and grow comfortable with needle use.
• Approximately 90% of patients stated treatment could be difficult, but they were managing. However, 58% (44 patients) also said that they were tired of, or no longer wanted, injections. 86% (65 patients) would be open to another therapy that was administered differently.
• 103 doctors stated that new treatments are needed for patients with HAE. 63 patients and 26 caregivers from this study agreed.
• 21 caregivers noted that patients often experienced needle fatigue. Many caregivers also stated that a pill would offer more patient freedom and independence, and reduce the burden of care for caregivers.
• Around 76 doctors stated that the process to begin prophylaxis therapy was often stressful and scary. Additionally, doctors were found to underestimate how long it takes to prepare and administer prophylaxis treatment.

Ultimately, the study determined that there is an unmet need regarding new, more easily administered treatments for patients with HAE. BioCryst is attempting to fix this through the development of ORLADEYO (berotralstat), an orally-administered therapy for HAE.

Hereditary Angioedema (HAE)

Once you break down the name of this condition, it’s easy to understand what it is. Hereditary means genetic or inherited. Angio pertains to blood vessels, and edema is inflammation caused by excess fluid in body tissue. So hereditary angioedema refers to recurrent attacks or flares or severe swelling underneath the skin caused by chromosome 11 defects and mutations. Normally, we have a blood protein called C1-inhibitor that helps regulate processes like inflammation and coagulation. But gene mutations prevent C1-inhibitor from working. Instead, the body generates too many bradykinin peptides, causing inflammation. An estimated 1 in 50,000 people have HAE. Stress and injuries can cause flares, but sometimes these occur without prompting. While males and females are equally affected, hormonal fluctuations (such as those during the menstrual cycle) may trigger flares more frequently in females.

Symptoms of HAE include:

• Extreme swelling of the throat (larynx, esophagus, trachea)
  • Difficulty breathing
• Swelling of the intestines and stomach
  • Abdominal swelling and enlargement
  • Nausea and vomiting
  • Abdominal pain
• Swelling of the arms, legs, hands, fingers, and toes to 2x their normal size
• Eyelid, lip, and cheek inflammation