ALXN2220 for ATTR amyloidosis with cardiomyopathy granted fast track designation
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Key takeaways:
- ALXN2220, a novel investigational depleter monoclonal antibody, was granted fast track designation by the FDA.
- It is designed for the treatment of transthyretin amyloidosis with cardiomyopathy.
AstraZeneca announced its rare disease unit, Alexion, has been granted fast track designation by the FDA for ALXN2220 for the treatment of transthyretin amyloidosis with cardiomyopathy.
The fast track designation is granted to therapies with the potential to fill unmet needs of patients with serious medical conditions. It includes the ability for the developer to have more frequent interactions with the agency, and potentially in the future, accelerated priority review and accelerated approval.
ALXN2220, a novel investigational depleter monoclonal antibody, is designed to remove amyloid deposits, according to a press release issued by the company.
The fast track designation was granted based on the results of a phase 1b trial published in The New England Journal of Medicine in 2023.
In 40 patients with wild-type or variant transthyretin amyloidosis cardiomyopathy (ATTR-CM) and chronic HF, IV ALXN2220 improved cardiac tracer uptake and extracellular volume — surrogates of cardiac amyloid load — with no apparent drug-related adverse events over 12 months.
ALXN2220 is currently being evaluated in the phase 3 DepleTTR-CM trial design to assess its efficacy as an add-on treatment on top of standard of care for patients with ATTR-CM, according to the release. The primary endpoint is a composite of all-cause mortality and cumulative frequency of CV clinical events.
“Given the progressive and disabling nature of ATTR-CM, there is an urgent need for additional treatment options,” Gianluca Pirozzi, senior vice president and head of development, regulatory and safety at Alexion, AstraZeneca Rare Disease, said in the release. “With a novel depleter mechanism designed to remove existing amyloid deposits and restore organ function, we believe that ALXN2220 offers the potential to transform the course of this devastating disease.”
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