Alnylam Pharmaceuticals has secured FDA approval for its RNA interference treatment, Amvutra (vutrisiran), as a new therapy for transthyretin amyloid cardiomyopathy (ATTR-CM). This marks a significant milestone for the Massachusetts-based biotech firm, which aims to differentiate its approach from existing treatments.
The FDA has granted Amvutra approval for treating ATTR-CM in adults, with the goal of reducing cardiovascular death, hospitalizations, and urgent heart failure visits. This move expands Alnylam’s product portfolio, which already includes a treatment for polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR-PN).
Amvutra, an RNA interference drug, works by rapidly knocking down TTR production, thereby reducing the deposition of amyloid fibrils that cause permanent cardiovascular damage and premature death in patients with ATTR-CM. The FDA based its approval decision on data from Alnylam’s phase 3 Helios-B study, which demonstrated a significant reduction in all-cause mortality and recurrent cardiovascular events.
The potential patient pool for Amvuttra is substantial, with estimates suggesting around 9,000 U.S. patients in the first-line treatment setting and an additional 10,000 second-line patients who could switch from existing treatments. Jefferies analysts predict that Amvutra can bring in $250 million in cardiomyopathy sales this year, with a significant portion coming from tafamidis converts.
Alnylam’s commercial chief, Tolga Tanguler, believes that the approval of Amvuttra will be a game-changer for the field, as it introduces a new mechanism of action and expands diagnosis rates. The company plans to work closely with high-volume prescribers and healthcare systems during its launch, aiming to cast a wide net by reaching 100% of these key stakeholders.
With a pricing tag of around $450,000 per year before discounts, Amvuttra is expected to be a significant contributor to Alnylam’s growth story. The company expects broad coverage and out-of-pocket costs for the treatment, similar to those achieved with its existing hATTR-PN product.
Source: https://www.fiercepharma.com/pharma/alnylams-rna-interference-med-amvuttra-wins-key-approval-attr-cm-teeing-showdown-pfizer