Pharming Group to Present 12 Studies on Rare Disease Treatments at 2025 ACAAI Meeting
核心洞察
Pharming Group will present 12 accepted abstracts at the 2025 ACAAI Annual Scientific Meeting in Orlando, showcasing new clinical and economic data for their rare disease portfolio.
Five presentations will focus on RUCONEST® for hereditary angioedema (搜索) treatment, including cost-effectiveness studies and indirect treatment comparisons with competing therapies.
Seven studies will highlight real-world effectiveness data for Joenja® (leniolisib) in treating APDS (搜索), including pediatric trial results and caregiver burden assessments.
Pharming Group N.V. announced that 12 abstracts have been accepted for presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting, taking place November 6-10 in Orlando, Florida. The presentations will showcase new clinical, economic, and real-world evidence for the company's rare disease portfolio.
RUCONEST® Data Highlights Treatment Economics and Efficacy
Five poster presentations will focus on RUCONEST® (recombinant C1 esterase inhibitor (搜索)), Pharming's treatment for hereditary angioedema (搜索) (HAE) attacks. Key studies include an indirect treatment comparison between RUCONEST® and sebetralstat for on-demand HAE therapy, presented by Dr. John Anderson from AllerVie Health (搜索) and the University of Alabama at Birmingham.
Dr. Jonathan A. Bernstein from Advanced Allergy Services will present cost-effectiveness data comparing RUCONEST® with sebetralstat for HAE attack treatment. Additional presentations will examine contemporary endpoint definitions in clinical data analysis and patient-reported outcome measures across HAE clinical trials.
RUCONEST® is delivered intravenously and is immediately bioavailable, enabling rapid intervention at HAE attack onset. At the recommended dose of 50 U/kg, the therapy has been shown to normalize C1INH activity levels by irreversibly binding to and deactivating key mediators such as coagulation factor FXII (搜索) and kallikrein, thereby halting bradykinin production and stopping attack progression.
Joenja® Real-World Evidence Expands APDS Understanding
Seven presentations will highlight new evidence on Joenja® (leniolisib), the first and only targeted treatment for activated phosphoinositide 3-kinase delta (搜索) (PI3Kδ) syndrome (APDS (搜索)). Dr. Niraj C. Patel from Duke University (搜索) will present data on leniolisib's effectiveness in reducing infections among APDS patients.
Studies will also examine healthcare utilization impacts, quality of life improvements in pediatric patients, and real-world adherence patterns. Dr. Joud Hajjar from Baylor College of Medicine will present caregiver perspectives on APDS (搜索) burden of illness, while other presentations will detail patient experiences through the APPEAL study and characterization data from the APDS-CHOIR Clinical Outcomes Registry.
Leniolisib is an oral small molecule PI3Kδ inhibitor approved in the U.S., U.K., Australia, and Israel for APDS (搜索) treatment in patients 12 years and older. Results from a randomized, placebo-controlled Phase III clinical trial demonstrated statistically significant improvement in coprimary endpoints, reflecting favorable impact on immune dysregulation and deficiency characteristic of APDS patients.
Addressing Critical Unmet Needs in Rare Diseases
HAE is a rare genetic disorder affecting approximately 1 in 10,000 to 1 in 50,000 people worldwide, caused by C1 esterase inhibitor protein deficiency. The condition leads to spontaneous and recurrent swelling episodes affecting skin, airways, and internal organs, with throat, nose, or tongue edema being particularly dangerous and potentially life-threatening.
APDS (搜索), first characterized in 2013, affects approximately 1 to 2 people per million worldwide. The condition is caused by variants in PIK3CD or PIK3R1 genes, leading to PI3Kδ pathway hyperactivity that causes immune cell dysfunction. APDS patients frequently experience severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy, with a median 7-year diagnostic delay contributing to progressive damage including permanent lung damage and lymphoma risk.
"We are proud that our scientific contributions in both hereditary angioedema (搜索) (HAE) and activated phosphoinositide 3-kinase delta (搜索) (PI3Kδ) syndrome (APDS (搜索)) have been recognized with a significant number of abstract acceptances at this year's ACAAI," said Anurag Relan, Chief Medical Officer of Pharming. "The work underscores the rigor of our research and our commitment to advancing patient care."
The ePosters will be accessible online and on demand to registered attendees beginning November 6, 2025, at 08:00 EST on ACAAI's website, in addition to exhibit hall displays at scheduled presentation times.