Core Viewpoint - CSL has received FDA approval for ANDEMBRY®, a novel treatment for hereditary angioedema (HAE), marking a significant advancement in the management of this rare genetic disorder [1][3][11] Group 1: Product Overview - ANDEMBRY is the first monoclonal antibody developed entirely by CSL, targeting factor XIIa to prevent HAE attacks in patients aged 12 and older [1][3][11] - The treatment offers once-monthly subcutaneous self-injection, providing a convenient administration method [1][3] - ANDEMBRY has shown a median reduction of more than 99% in HAE attacks compared to placebo, with a least squares mean reduction of 89.2% [6][7] Group 2: Clinical Trial Data - The approval is based on data from the pivotal Phase 3 VANGUARD trial, which demonstrated the efficacy and safety of ANDEMBRY [3][12] - In the pivotal trial, 62% of patients treated with ANDEMBRY remained attack-free throughout the treatment period [7] - The most common adverse reactions reported were nasopharyngitis and abdominal pain, with injection-site reactions occurring in 14% of patients [4][7] Group 3: Market Impact and Availability - ANDEMBRY's approval expands CSL's HAE franchise and reinforces the company's commitment to innovation in the HAE community [6][8] - The product is set to launch commercially immediately, with availability expected before the end of June 2025 [9] - ANDEMBRY has also received approvals in multiple regions, including Australia, the UK, the EU, Japan, Switzerland, and the UAE [8]

RESEARCH TRIANGLE PARK, N.C., June 13, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the National Institute of Drug and Food Surveillance (INVIMA) in Colombia has granted approval for oral, once-daily ORLADEYO® (berotralstat) for the prophylaxis of hereditary angioedema (HAE) attacks in adults and pediatric patients 12 years of age or older. “Today’s announcement marks another important step toward bringing ORLADEYO to people living with HAE who are in need of n ...

Core Insights - BioCryst Pharmaceuticals announced new data on ORLADEYO (berotralstat) for hereditary angioedema (HAE) treatment to be presented at the EAACI meeting in Glasgow from June 13 to 16, 2025 [1][2][3] Group 1: Presentation Details - Four abstracts will be presented at the EAACI meeting, including a pooled analysis of the APeX-2 and APeX-J trials focusing on the impact of berotralstat on quality of life for HAE patients [1] - A focus group study reported positive perceptions from patients following berotralstat treatment [2] - Interim results from the APeX-P study indicated that berotralstat use reduced the number of HAE attacks requiring treatment in pediatric patients [3] Group 2: Product Information - ORLADEYO (berotralstat) is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [4][5] - The recommended dosage is one capsule per day, with caution advised for dosages higher than 150 mg due to potential QT prolongation [6][7] - The safety and effectiveness of ORLADEYO in pediatric patients under 12 years have not been established, and there is limited data on its use during pregnancy [9][10] Group 3: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases, utilizing structure-guided drug design for developing therapeutics [11]

New data shared at the 14th C1-inhibitor Deficiency & Angioedema Workshop supports berotralstat use at all stages of lifeRESEARCH TRIANGLE PARK, N.C., May 30, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data which highlights the reduction in the percentage of days with hereditary angioedema (HAE) symptoms among young children initiating berotralstat in the APeX-P trial, and the broad safety and efficacy outcomes observed across all age groups of patients taking ...

Core Insights - BioCryst Pharmaceuticals announced significant real-world evidence demonstrating the effectiveness of ORLADEYO (berotralstat) in reducing hereditary angioedema (HAE) attack rates in adolescents and individuals with severe HAE over an 18-month follow-up period [1][3][7] Group 1: Study Findings - The study presented at the ISPOR 2025 conference showed that patients experienced significantly lower HAE attack rates while on ORLADEYO, with a reduction from a baseline of 7.78-8.23 attacks/month to 1.24-1.90 attacks/month [10] - Specifically, patients had 6.25 fewer attacks/month at 12 months and 6.43 fewer attacks/month at 18 months after starting ORLADEYO [10] - Adolescents aged 12-17 years also showed significant reductions, with 1.56 fewer attacks/month at 12 months and 1.85 fewer attacks/month at 18 months [10] Group 2: Patient Population - The findings were based on a retrospective pre-post study involving 56 U.S. patients with C1-inhibitor deficiency and 99 U.S. adolescents with HAE [5][6] - The results indicate that ORLADEYO is effective across different age groups and severity levels of HAE, providing confidence to both physicians and patients [3][7] Group 3: Product Information - ORLADEYO is the first and only oral therapy specifically designed for the prophylaxis of HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [8][9] - The therapy is administered as a once-daily capsule, which works by decreasing the activity of plasma kallikrein to prevent HAE attacks [8]

Pharvaris (PHVS) FY 2025 Conference February 12, 2025 09:20 AM ET Company Participants Wim Souverijns - CCOPeng Lu - Chief Medical Officer Conference Call Participants Jeff Jones - Managing Director - Senior Analyst Jeff Jones Alright. Good morning, everyone, and welcome back to the Oppenheimer Healthcare Conference. I'm I'm Jeff Jones, one of the biotechnology analysts here on the team, and I'm joined by the team from Farvaris. Farvaris is a late clinical stage company developing, the novel oral bradykinin ...