Transthyretin amyloidosis (ATTR) is a protein conformational disorder caused by the build-up of transthyretin, the protein carrying thyroxine and retinol in the bloodstream. While cardiomyopathy is a characteristic of patients with wild-type ATTR (ATTR-wt), some patients with hereditary ATTR (ATTR-h) also experience cardiomyopathy symptoms along with their polyneuropathy. ATTR-h patients have multiple treatment options that their prescribers can use to manage the disorder and delay progression. However, few medicines are approved for ATTR-wt. GlobalData estimates that vutrisiran’s entrance into the ATTR-wt market will change the treatment paradigm, with the newly approved medicine likely to steal the majority of the patient share from competitors.
ATTR-wt patients account for the majority of ATTR patients, as ATTR-h is still considered a rare disease, but only tafamidis 61mg and, more recently, acoramidis were approved as treatment options for ATTR-wt. European key opinion leaders (KOLs) with cardiomyopathy patients told GlobalData that they are waiting for vutrisiran’s launch for ATTR-wt, as only ATTR-h patients can access it at the moment.
Vutrisiran was approved by the European Commission for ATTR patients with cardiomyopathy (ATTR-CM) on 11 June, under the name Amvuttra, and it is administered every three months through subcutaneous injection. The HELIOS-B Phase III trial demonstrated a decrease in all-cause mortality and cardiovascular symptoms, including health failure and functional capacity, proving higher benefits in ATTR-CM compared to the ones observed with the standard of care tafamidis.
Thus, treatment paradigms for ATTR-CM patients are likely to change, as many more therapies in clinical trials are expected to be tailored to this patient population, for instance, monoclonal antibodies coramitug and ALXN2220. This is an unprecedented development in the ATTR space while patient numbers continue to increase. Cost of treatment remains the biggest unmet need, especially considering the average patient age, which may limit access to therapy and healthcare authorities’ reimbursement.
Finally, vutrisiran’s launch in the ATTR-CM market will be an opportunity for its manufacturer, Alnylam, to potentially increase sales, as ATTR-wt patient numbers continue growing.
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By GlobalData
