FDA Places Clinical Hold on Intellia’s Gene Therapy Trials Following Patient Hospitalization

The U.S. Food and Drug Administration placed a clinical hold on two Intellia Therapeutics gene therapy clinical trials after a patient was hospitalized with grade 4 liver transaminase elevations, meeting the trials’ protocol-defined pausing criteria.

The paused phase 3 MAGNITUDE and MAGNITUDE-2 trials are evaluating nex-z, an investigational CRISPR-based therapy for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN). More than 650 patients with ATTR-CM and 47 with ATTR-PN are enrolled, and the company estimates that 450 have received the gene therapy.

The patient is receiving medical care, and Intellia is reviewing potential risk mitigation strategies and engaging with regulatory authorities.