CSL Behring

The FDA approved ANDEMBRY (garadacimab-gxii), the first and only prophylactic HAE treatment targeting factor XIIa, offering once-monthly dosing for patients 12 years and older.

Six pharmaceutical facilities received category awards for innovation, operations, and social impact at the 2025 ISPE Europe Annual Conference, marking the 20th anniversary of the prestigious FOYA program.

The Non-Cystic Fibrosis Bronchiectasis (NCFB) market is projected to grow from USD 1.7 billion in 2024 to USD 7.5 billion by 2035, representing a robust CAGR of 14.62%.

Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.

Pfizer has announced the cessation of development and commercialization of Beqvez, its FDA-approved gene therapy for hemophilia B, citing limited interest from patients and healthcare providers.

Data from the HOPE-B trial demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) provides long-term bleed protection for adults with hemophilia B.

2024 marked significant advances in hereditary angioedema (HAE) treatment, with Intellia's gene-editing therapy NTLA-2002 showing a remarkable 95% reduction in monthly attacks. The year also saw important developments in multiple therapeutic candidates, including donidalorsen and garadacimab, while established treatments like Takhzyro demonstrated continued efficacy in adolescent populations.

The European Commission and the MHRA have approved CSL's Andembry (garadacimab) for preventing hereditary angioedema (HAE) attacks in patients aged 12 and older.

ASH 2024 highlighted significant progress in hemophilia care since Hemlibra's approval, including gene therapies for hemophilia A and B, offering curative potential.

Several companies are actively involved in developing therapies for Hemophilia B, with Belief Biomed's drug candidates reaching Phase III clinical trials.