FDA Approves Jivi for Pediatric Hemophilia A Patients Aged 7-12 Years

The US FDA has approved Jivi, a recombinant DNA-derived, extended half-life factor VIII concentrate, for use in pediatric patients 7 years of age and older with hemophilia A. The approval is based on data from the Alfa-PROTECT and PROTECT Kids studies, demonstrating Jivi's safety and efficacy in children aged 7 to under 12 years with severe hemophilia A. Jivi is approved for routine prophylaxis, on-demand treatment, and perioperative management of bleeding in previously treated adult and pediatric patients 7 years of age and older.

Metagenomi, Inc. (Nasdaq: MGX) has announced positive results from a nonhuman primate (NHP) study on their hemophilia A therapy, demonstrating sustained factor VIII (FVIII) activity for approximately 19 months with a favorable safety profile. The company's AI-driven metagenomics platform is expected to guide the future of their MGX-001 program [1].

The study, published in Nature Communications, showed that FVIII activity remained at therapeutically relevant levels, and no serious treatment-related safety issues were observed, aside from one unrelated incident [1]. The company reported a strong financial position with $226 million available as of March 31, 2025, sufficient to fund operations into 2027 [1].

Metagenomi also published findings on novel gene editing technologies, which represent advancements in targeted therapeutic applications. The company's CEO, Brian C. Thomas, highlighted the significance of their AI-driven technology, which allows for the development of novel, potentially curative genome editing therapeutics [1].

However, the study also noted that one animal died prematurely from an unrelated cause, raising concerns about the long-term safety of the treatment [1]. Additionally, collaboration revenue significantly decreased from $11,159 in Q1 2024 to $4,127 in Q1 2025, indicating potential challenges in partnerships or commercialization efforts [1].

Despite these challenges, Metagenomi remains well-capitalized, with a net loss of $25,039 for the quarter ended March 31, 2025, compared to a loss of $25,148 in the previous year, highlighting ongoing financial difficulties despite a high cash balance [1].

References:

[1] https://www.nasdaq.com/articles/metagenomi-reports-positive-nhp-study-results-hemophilia-program-and-quarterly-financials