FDA puts ATTR-CM therapy candidate coramitug on fast track

The U.S. Food and Drug Administration (FDA) has granted fast track designation to coramitug, an experimental treatment in late-stage clinical testing for transthyretin amyloid cardiomyopathy (ATTR-CM).

The FDA gives this designation to investigational medicines that have the potential to meaningfully improve care for serious health conditions, with the aim of speeding the development of sorely needed new treatments.

The designation gives the therapy’s developer, Novo Nordisk, access to perks such as more frequent communication with the FDA during the development process, and, if certain criteria are met, rolling review and priority review, which provide faster routes to approval. Novo Nordisk is developing coramitug after acquiring rights to the therapy from its original developer, Prothena, in 2021.

“We are encouraged by Novo Nordisk’s receipt of fast track designation for coramitug, which underscores their continued commitment to advancing coramitug for patients living with ATTR-CM,” Gene Kinney, PhD, Prothena’s president and CEO, said in a company press release.

The therapy previously received orphan drug designation for ATTR-CM in both the U.S. and the European Union, a status also meant to accelerate clinical development and regulatory review.

Recommended Reading

April 10, 2026 Columns by Jade River

The long journey to an amyloidosis diagnosis can be full of setbacks

Phase 3 trial continues

Coramitug is being tested in a global, placebo-controlled Phase 3 clinical trial, CLEOPATTRA (NCT07207811), that aims to enroll more than 1,200 adults with ATTR-CM at nearly 300 centers worldwide. The Novo Nordisk-sponsored study is expected to conclude in 2029.

“We are pleased that the FDA has granted fast track designation for coramitug, a recognition that reflects both the seriousness of ATTR-CM and the significant unmet need that remains for people living with this life-threatening disease, despite standard of care,” said Michelle Lim-Watson, PhD, Novo Nordisk’s associate portfolio vice president, cardiovascular disease and rare diseases, U.S. research and development.

The company believes the CLEOPATRA trial “could mark a meaningful step forward in how we treat ATTR-CM in the future,” Lim-Watson said.

ATTR-CM is a heart disease marked by toxic clumps of a misfolded transthyretin protein that accumulate in the heart, damaging it.

Coramitug (previously called NNC6019 or PRX004) is an antibody-based medicine designed to bind to abnormal transthyretin clumps, marking them for clearance without affecting healthy versions of the transthyretin protein.

One-year data from a Phase 2 clinical trial (NCT05442047) showed that monthly infusions of the therapy led to significant reductions in blood NT-proBNP levels, an established marker of heart damage, in adults with ATTR-CM. No significant differences relative to a placebo were seen in the six-minute walk test, which assesses exercise capacity and was the other main goal of the study.

CLEOPATTRA is designed to further assess coramitug’s safety and efficacy in patients with ATTR-CM and heart failure. The study is open to patients with mutations in the gene encoding transthyretin (hereditary ATTR-CM) and to those without these mutations (wild-type ATTR-CM).

Participants will be randomly assigned to receive infusions of either coramitug or a placebo, in addition to their usual heart medications, for up to four years. The trial’s main goal is to see if coramitug is superior to the placebo at reducing the risk of a composite outcome that includes hospitalization due to cardiovascular problems, urgent heart failure visits, and/or death due to cardiovascular disease.

Secondary goals include group differences in terms of measures of disease burden, the six-minute walk test, blood NT-proBNP levels, and time to first heart-related outcomes or death.