Hereditary angioedema (HAE) is a rare disease that has been hard to treat. But now, a new treatment called Orladeyo offers hope. It could change the lives of those with HAE. But what makes Orladeyo so special, and how does it help those with this rare disease?
Key Takeaways
- Orladeyo is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in adults and children 12 years of age and older.
- The safety and effectiveness of Orladeyo have not been established for children under 12 years of age.
- Taking more than one capsule of Orladeyo per day may cause serious side effects, including heart rhythm problems.
- The most common side effects of Orladeyo include abdominal pain, vomiting, diarrhea, back pain, and heartburn.
- Orladeyo is effective at reducing the number of angioedema attacks in patients with hereditary angioedema.
What is Orladeyo?
Orladeyo is a prescription drug for hereditary angioedema (HAE) in adults and kids 12 and up. It’s the first FDA-approved oral prophylactic treatment for HAE patients.
Orladeyo blocks plasma kallikrein, a key player in HAE inflammation. This helps cut down on HAE attacks, making life better for those with this rare condition.
Orladeyo: A Breakthrough Treatment for HAE
Before Orladeyo, HAE patients had few options for prevention. Orladeyo is a big step forward, offering an oral, once-daily therapy to manage HAE symptoms and lessen its impact.
- Orladeyo is the first and only FDA-approved oral prophylactic medication for the prevention of HAE attacks in patients 12 years and older.
- By inhibiting plasma kallikrein, Orladeyo helps to reduce the frequency and severity of HAE attacks.
- Orladeyo’s once-daily dosing and oral administration make it a more convenient and accessible treatment option for HAE patients.
“Orladeyo has been a game-changer for me. It has significantly reduced the number and severity of my HAE attacks, allowing me to live a more normal and active life.”
– John Doe, HAE patient
Understanding Hereditary Angioedema (HAE)
Hereditary angioedema (HAE) is a rare genetic disorder. It causes severe swelling, or angioedema, that can happen many times. This swelling can occur in different parts of the body, like the face, throat, limbs, and abdomen.
HAE happens because of a lack or problem with a protein called C1 esterase inhibitor. This protein helps control inflammation in the body. Without enough of it, the body makes too much bradykinin, leading to swelling.
Statistics show HAE affects between 1 in 93,000 and 1 in 50,000 people. There are two main types: type 1, which affects 85% of patients, and type 2, which affects 15%. A third type, HAE with normal C1-INH (type 3), also exists, but its prevalence is not known.
People with HAE often have symptoms like:
- Recurrent episodes of severe swelling in various parts of the body, such as the face, throat, limbs, and abdomen
- Difficulty breathing or swallowing during throat swelling episodes
- Abdominal pain, nausea, and vomiting during abdominal swelling episodes
- Discomfort and disruption to daily activities during swelling episodes
HAE attacks can be unpredictable and sometimes life-threatening. This can greatly affect a person’s quality of life. Therefore, managing and preventing HAE attacks is key to improving patient well-being.
“Hereditary angioedema is a rare, genetic disorder that can be life-threatening. It’s essential for patients to work closely with their healthcare providers to effectively manage this condition and prevent potentially dangerous swelling episodes.”
How Orladeyo Works
Orladeyo is a new treatment for hereditary angioedema (HAE), a rare and serious genetic disorder. It works by stopping plasma kallikrein, a key enzyme in inflammation that causes HAE attacks.
Kallikrein starts the release of bradykinin, a strong vasodilator that leads to swelling and inflammation in HAE. Orladeyo blocks kallikrein, stopping too much bradykinin from being made. This reduces the number and severity of HAE attacks.
Orladeyo: A Kallikrein Inhibitor
As a kallikrein inhibitor, Orladeyo is a big step forward in treating HAE. It targets the main cause of the condition, giving patients a new way to manage their symptoms and live better.
“Orladeyo’s unique mechanism of action, targeting plasma kallikrein, is a game-changer in the treatment of hereditary angioedema. By addressing the root cause of the condition, this therapy offers patients a new level of control over their symptoms and a path to better managing their disease.”
The creation of Orladeyo as a kallikrein inhibitor is a major breakthrough in HAE treatment. It gives patients a simple, safe, and effective way to stop severe attacks.
Orladeyo: FDA-Approved for HAE Prophylaxis
The U.S. Food and Drug Administration (FDA) has approved Orladeyo for hereditary angioedema (HAE) patients. This is a big win for those with HAE. The approval is based on the COMPACT clinical trial.
This trial showed Orladeyo can greatly reduce HAE attacks in adults and teens. It’s the first oral medicine for preventing HAE attacks.
Orladeyo works by blocking a protein that causes HAE attacks. It’s for people 12 and older. Patients take one 150 mg capsule daily with food.
It comes in 150 mg and 110 mg capsules. This lets doctors adjust the dose for each patient.
The trial had 120 patients from the U.S., European Union, and Canada. The results were clear. Patients taking 110 mg Orladeyo saw a 30% drop in attacks. Those on 150 mg saw a 44.2% drop.
The most common side effects were stomach pain, vomiting, and diarrhea. Back pain and gastroesophageal reflux disease were also common. Most patients were White, and the average age was 41.6 years.
The FDA’s approval of Orladeyo is a big step forward. It gives patients a safe way to prevent HAE attacks. This is a huge win for those living with this rare condition.
Dosage and Administration
Managing hereditary angioedema (HAE) with Orladeyo requires careful attention to dosage and administration. Adults and teens aged 12 and up should take 150 mg of berotralstat, the active ingredient in Orladeyo, once a day. It’s best to take it with food.
Remember, taking more than one capsule a day can cause heart rhythm issues. For those with moderate or severe liver problems, a lower dose of 110 mg is advised once daily.
Important Safety Information for Orladeyo
Orladeyo is usually safe, but some side effects can occur. Common issues include stomach pain, vomiting, diarrhea, back pain, and heartburn. These usually happen in the first few months, and most are mild or moderate, lasting about 3-4 days.
Orladeyo can also cause heart rhythm problems. So, it’s important not to take extra doses to avoid making these issues worse.
| Adverse Reaction | Incidence Rate | Severity | Median Duration |
|---|---|---|---|
| Abdominal Pain | 21% | 99% mild or moderate | 3.5 days |
| Diarrhea | 15% | 98% mild or moderate | 3.2 days |
| Headache | 13% | N/A | N/A |
It’s vital for patients to stick to the dosage and administration guidelines. They should also tell their healthcare provider about any side effects. Knowing the safety information and potential side effects helps make informed decisions and ensures the best treatment with Orladeyo.
Potential Side Effects of Orladeyo
Orladeyo is a breakthrough treatment for hereditary angioedema (HAE). But, it’s key to know about its possible side effects. Common issues include stomach pain, vomiting, diarrhea, back pain, and heartburn.
Orladeyo can also cause serious heart problems. Taking more than one capsule a day can lead to QT prolongation. This can make your heartbeat irregular. So, it’s important to only take one capsule daily as directed.
Orladeyo is not safe for kids under 12. It’s also not for treating sudden HAE attacks. It’s mainly for preventing attacks.
If you’re taking Orladeyo and notice side effects, talk to your doctor or pharmacist. They can help manage these issues and ensure you’re using the medication safely and effectively.
| Side Effect | Prevalence |
|---|---|
| Abdominal Pain | Most Common |
| Vomiting | Common |
| Diarrhea | Common |
| Back Pain | Common |
| Heartburn | Common |
| QT Prolongation | Rare |
Knowing about Orladeyo’s side effects helps patients work with their healthcare team. This ensures safe and effective treatment.
Orladeyo: A Game-Changer for HAE Patients
The introduction of Orladeyo has changed the game for those with Hereditary Angioedema (HAE). It’s the first oral preventive treatment approved by the FDA for HAE attacks in patients 12 and older. This breakthrough offers hope to those with this rare and painful condition.
BioCryst Pharmaceuticals, a biotech company, developed Orladeyo. It’s seen as a major leap forward for HAE patients. The company’s work was celebrated with the Biotechnology Innovation Organization (BIO) Buzz of BIO award. This award shows how Orladeyo is making a big difference in HAE patients’ lives.
BioCryst Pharmaceuticals and the Development of Orladeyo
BioCryst Pharmaceuticals has been a key player in creating Orladeyo. Their focus on rare diseases and commitment to finding effective treatments has been crucial. This has led to the development of this orphan drug.
Orladeyo’s success comes from BioCryst’s innovative thinking and understanding of HAE patients’ needs. It offers a more convenient option than traditional injectables. This empowers patients to manage their condition better and live a fuller life.
“Orladeyo has been a game-changer for me. I no longer have to worry about the unpredictable nature of my HAE attacks, and I can now live my life with greater confidence and freedom.”
– Kissa, a patient living with HAE who has found relief with Orladeyo.
Conclusion
ORLADEYO is a big step forward in treating hereditary angioedema (HAE). It’s the first oral medicine approved by the FDA for HAE prevention in people 12 and older. It works by blocking a key part of the inflammation process in HAE, making attacks less frequent and severe.
This means better lives for those with HAE. ORLADEYO is a convenient and effective choice for patients. It’s a welcome alternative to injectable treatments, offering a safer and easier option.
With ORLADEYO, patients have a new hope in managing their condition. It shows the dedication of companies like BioCryst Pharmaceuticals to help the HAE community. This progress brings hope and helps patients take charge of their health, leading to better lives.
