In short
Berotralstat, also known as BCX7353 or Orladeyo®, is a drug being studied in clinical trials for the treatment of hereditary angioedema (HAE). These trials aim to evaluate its safety, effectiveness, and proper dosing in various patient populations, including children. The studies focus on using berotralstat as a preventive treatment to reduce the frequency and severity of HAE attacks.
At a glance
- Drug Name
- Berotralstat (BCX7353, Orladeyo®)
- Condition Treated
- Hereditary Angioedema (HAE)
- Administration
- Oral, once daily
- Age Groups Studied
- Children (2 to <12 years), Adults
- Primary Objectives
- Safety, Efficacy, Pharmacokinetics
- Study Durations
- Up to 144 weeks (pediatric), Up to 240 weeks (adult)
- Key Outcomes Measured
- Adverse events, HAE attack frequency and severity, Drug concentrations in blood
- Special Programs
- Expanded Access Program for eligible patients
What is Berotralstat?
Berotralstat, also known by its brand name Orladeyo® or research code BCX7353, is a medication designed to prevent attacks of hereditary angioedema (HAE). HAE is a rare genetic condition that causes sudden episodes of swelling in various parts of the body, including the hands, feet, face, and airway. These swelling attacks can be painful, disfiguring, and even life-threatening if they affect the throat.
What Does Berotralstat Treat?
Berotralstat is specifically used for the prophylaxis (prevention) of HAE attacks in both adults and children. This means it's taken regularly to reduce the frequency and severity of HAE episodes, rather than treating attacks when they occur. It's important to note that while berotralstat helps prevent attacks, patients may still need other medications to treat acute attacks if they do occur.
How is Berotralstat Administered?
Berotralstat is taken orally, which means it's a medication you can take by mouth. For adults, the typical dose is one 150mg capsule taken once daily. This makes it a convenient option for many patients, as it doesn't require injections or infusions like some other HAE treatments.
For children, the dosing is weight-based, meaning the amount of medication will depend on how much the child weighs. In some cases, berotralstat may be available as oral granules for children, which might be easier for younger patients to take.
Clinical Trials and Research
Berotralstat has been and continues to be studied in various clinical trials to ensure its safety and effectiveness. Here are some key points about the ongoing research:
- Pediatric Studies: A Phase 3 study is evaluating the safety and pharmacokinetics (how the drug moves through the body) of berotralstat in children aged 2 to less than 12 years old. This study aims to determine the appropriate weight-based dosing for children.
- Long-term Safety: Another study is looking at the long-term safety and tolerability of berotralstat in patients who have previously participated in berotralstat studies. This study will last up to 240 weeks (about 5 years) to gather data on the medication's effects over an extended period.
- Effectiveness Measures: Researchers are tracking various outcomes to assess how well berotralstat works, including the frequency and severity of HAE attacks.
Safety and Side Effects
As with any medication, it's important to understand the potential side effects of berotralstat. The clinical trials are closely monitoring for adverse events (side effects) and serious adverse events. Some of the safety measures being tracked include:
- The number and proportion of patients experiencing treatment-related side effects
- Any serious adverse events that occur
- Grade 3 or 4 side effects (which are considered severe)
- Any abnormal changes in blood chemistry related to the treatment
- The number of patients who stop taking the medication due to side effects
It's important to note that while these potential side effects are being monitored, it doesn't mean every patient will experience them. Your doctor will discuss the potential risks and benefits of berotralstat with you based on your individual health situation.
Access to Berotralstat
Berotralstat is available through different means depending on your location and circumstances:
- Commercial Availability: In some countries, berotralstat is already commercially available and can be prescribed by your doctor.
- Clinical Trials: If berotralstat is not yet approved in your country, you may be able to access it through participation in a clinical trial.
- Expanded Access Program: In the United States, there is an expanded access program that provides berotralstat to eligible patients with HAE who have an unmet medical need. This program is available until the medication becomes commercially available. If you're interested in this program, you should discuss it with your doctor, who can contact the program coordinators for more information.
Remember, the decision to use berotralstat should be made in consultation with your healthcare provider, who can assess your individual situation and determine if this medication is appropriate for you.