1. FDA approved ORLADEYO for pediatric HAE patients aged 2-12. 2. ORLADEYO is now the first oral prophylactic therapy for younger patients. 3. Clinical data show effective reduction in HAE attack rates. 4. A new child-friendly pellet formulation enhances administration ease. 5. BioCryst plans further global regulatory filings for ORLADEYO.
FAQ
Why Very Bullish?
FDA approval expands market access. Historical approvals by Biopharma firms typically boost stock prices.
How important is it?
The approval of ORLADEYO marks a significant advancement in treatment efficacy and marketability.
Why Long Term?
Increased adoption among pediatric patients may lead to sustained revenue growth over time.
BioCryst Pharmaceuticals Receives FDA Approval for ORLADEYO® Oral Pellets
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced a significant milestone in the treatment of hereditary angioedema (HAE) with the U.S. Food and Drug Administration's (FDA) approval of its New Drug Application (NDA) for ORLADEYO® (berotralstat) in an oral pellet formulation. This critical development provides the first and only targeted oral prophylactic therapy for pediatric patients aged 2 to <12 years suffering from HAE.
Understanding the Impact of HAE
Hereditary angioedema is a genetic condition that can manifest early in childhood, with approximately 40% of affected children experiencing their first attack by the age of 5. Symptoms of HAE can severely disrupt daily living and have a considerable psychosocial impact due to their unpredictable and potentially life-threatening nature. Prior to ORLADEYO's approval, treatment options for children under 12 were limited to intravenous or subcutaneous administration, which posed challenges for patients and their caregivers.
Key Features of ORLADEYO®
ORLADEYO's oral pellet formulation offers a child-friendly method of administration that allows for easier compliance and comfort. This new form can be taken directly with water or milk, or sprinkled over soft, non-acidic foods, making it a more accessible treatment option for children.
- First oral prophylactic therapy for HAE patients aged 2 and above.
- Supports individualized treatment plans for patients and families.
- Administered once daily, simplifying treatment regimens.
Clinical Trial Success and Safety Profile
The approval is based on promising interim data from the APeX-P clinical trial, which assessed ORLADEYO's long-term safety and efficacy in children aged 2 to <12 years. This trial is the largest to evaluate prophylactic therapies for HAE in this age group. Results published in the Annals of Allergy, Asthma & Immunology indicate that ORLADEYO was well-tolerated and showed early and sustained reductions in monthly attack rates.
The most commonly reported adverse event was nasopharyngitis, with no new safety concerns identified beyond those established in earlier adult trials.
Statements from Company Leaders
Jon Stonehouse, CEO of BioCryst, expressed his enthusiasm, stating, “This approval extends the benefits of oral prophylactic therapy to a vulnerable part of the HAE community, children ages 2 to <12, and we couldn’t be more excited to bring this treatment option to these kids and their caregivers.”
Anthony J. Castaldo, CEO of the U.S. Hereditary Angioedema Association (HAEA), added, “Today’s pediatric approval of ORLADEYO offers a welcome oral preventive choice for children living with HAE.”
Future Regulatory Efforts
BioCryst is proactive in expanding ORLADEYO's availability, with applications pending with the European Medicines Agency and the Japan Pharmaceutical and Medical Devices Agency. Further regulatory submissions are also planned in additional global territories, including Canada.
Patient and Caregiver Support
To support patients and healthcare providers, BioCryst offers Empower Patient Services, a comprehensive resource that aids in navigating insurance, affordability, and adherence concerns related to ORLADEYO.
For more information about ORLADEYO and its availability, visit www.ORLADEYO.com.
About ORLADEYO® (berotralstat)
ORLADEYO (berotralstat) is the first and only oral therapy designed specifically for the prophylaxis of hereditary angioedema attacks in adults and pediatric patients aged 2 years and older. Administered as a single daily dose, ORLADEYO works by inhibiting the activity of plasma kallikrein, thus preventing HAE attacks.
Important Safety Information
While ORLADEYO has been shown to be effective in preventing HAE attacks, it is not intended for treating acute episodes. Potential risks include QTc interval prolongation, especially at dosages exceeding 150 mg per day. Adverse reactions have been reported and include abdominal pain, vomiting, and diarrhea.