Core Viewpoint - BioCryst Pharmaceuticals has received FDA approval for ORLADEYO, the first and only targeted oral prophylactic therapy for pediatric patients aged 2 to less than 12 years with hereditary angioedema (HAE) [1][4]. Group 1: Product Information - ORLADEYO is an oral pellet formulation designed for once-daily administration, providing a child-friendly method of treatment [1][6]. - The approval is based on positive interim data from the APeX-P clinical trial, which demonstrated early and sustained reductions in monthly attack rates for pediatric patients [5][7]. - ORLADEYO has been prescribed to over 3,500 patients in the U.S. since its initial launch for older patients [5]. Group 2: Market Context - Approximately 40% of children with HAE experience their first attack by age 5, highlighting the need for effective treatment options for younger patients [2]. - Prior to this approval, treatment options for children under 12 were limited to intravenous or subcutaneous methods, which can be burdensome [2][9]. - The new oral pellet formulation aims to alleviate the treatment burden and improve the quality of life for children with HAE and their caregivers [7][9]. Group 3: Regulatory and Future Plans - BioCryst has filed applications for ORLADEYO oral pellets with the European Medicines Agency and the Japan Pharmaceutical and Medical Devices Agency, with additional regulatory filings planned in other territories, including Canada [7][8]. - The company is committed to providing comprehensive support through its Empower Patient Services program, which assists patients and healthcare providers with various needs related to ORLADEYO [10].

Core Insights - Pharvaris is a biopharmaceutical company focused on developing oral treatments for hereditary angioedema (HAE) using a bradykinin B2 antagonist approach, addressing both on-demand and preventive needs for patients [1][6] Stock Performance - As of December 3, 2025, Pharvaris (PHVS) stock was priced at $29.37, reflecting a 21.75% increase from the previous session, with fluctuations between $23.80 and $29.68 on that day [4] - The stock has experienced a yearly high of $29.67 and a low of $11.51, indicating its volatile nature [4] Market Analysis - Morgan Stanley set a price target of $41 for PHVS, suggesting a potential increase of approximately 39.62% based on the company's promising late-stage pipeline and anticipated phase 3 data by the end of 2025 [2][6] - Pharvaris has a market capitalization of approximately $1.91 billion, indicating a substantial presence in the biopharmaceutical sector [5] Clinical Development - The company has received a buy rating with a target price of $40, driven by strong phase 2 results and regulatory precedents [3][6] - The mechanism of Deucrictibant, a key component of their treatment, has been validated, with an estimated probability of success for the upcoming phase 3 readout exceeding 90% [3]

Core Viewpoint - BioCryst Pharmaceuticals has received approval from INVIMA in Colombia for ORLADEYO (berotralstat) as a prophylactic treatment for hereditary angioedema (HAE) in patients aged 12 and older, marking a significant advancement in treatment options for HAE in the pan-Latin America region [1][2]. Group 1: Product Information - ORLADEYO is the first and only oral therapy specifically designed to prevent HAE attacks in adults and pediatric patients aged 12 years and older, administered as one capsule daily [4]. - The drug functions by inhibiting plasma kallikrein, which is involved in the pathophysiology of HAE [4]. - ORLADEYO has already received regulatory approvals in several Latin American countries, including Chile, Argentina, Brazil, Mexico, and Peru [3]. Group 2: Commercial Strategy - BioCryst has an exclusive collaboration with Pint Pharma GmbH for the registration and promotion of ORLADEYO in the pan-Latin America region, with Pint responsible for obtaining marketing authorizations and commercialization [3]. - Following the approval from INVIMA, BioCryst is working with Pint Pharma to ensure the availability of ORLADEYO to patients in Colombia [2]. Group 3: Safety and Usage Information - ORLADEYO is indicated for the prophylaxis of HAE attacks but is not approved for the treatment of acute HAE attacks [5][6]. - The recommended dosage is 150 mg once daily, with a reduced dosage of 110 mg for patients with moderate or severe hepatic impairment [7]. - Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease, occurring in 10% or more of patients [7].