Competition breeds innovation, and that’s good news for ATTR-CM patients who could have three different drugs to choose from: Vyndaqel (Pfizer), Attruby/Beyonttra (BridgeBio), and Amvuttra (Alnylam).
- ATTR-CM affects more than 120k Americans with 5-7k new cases each year, but underdiagnosis means there could be even more.
- The disease occurs when misfolded proteins build up in the heart and stiffen the heart muscle, leading to heart failure.
- With diagnostics and awareness improving, analysts now estimate the ATTR-CM treatment could become a $15-20B market.
The reigning ATTR-CM drug is Pfizer’s Vyndaqel (tafamidis), which entered the scene unchallenged in 2019, and although it was first, new research has suggested it might not be the best.
- In the study, patients on Vyndaqel had a survival probability of only 54.1% after 65 months.
- Those who received Vyndaqel within six months of ATTR-CM diagnosis had even worse results (49.6% at 65 months).
- Even so, Pfizer raked in $5.4B in Vyndaqel sales last year, speaking to the drug’s advantage as a lone contender with a $268k list price per year.
Entering the race as a fresh challenger, BridgeBio’s Attruby (acoramidis) hit the U.S. market in December of 2024 and is rapidly gaining momentum thanks to its broader label advantage and similar mechanism to Vyndaqel.
- Attruby already has 1,000 unique prescriptions since launch, leading analysts to predict it will easily surpass its current $86M sales prediction for 2025.
- Though gaining prescription popularity, Attruby is barely cheaper ($244k per year) and works the same way as Vyndaqel, so time will tell if it leads to better outcomes.
Appearing as a wild card pick, Alnylam’s Amvuttra (vutrisiran) is currently waiting for the March 23rd verdict of an ATTR-CM label expansion filed last October following its impressive HELIOS-B trial results.
- As a monotherapy, Amvuttra reduced CV death risk by 33%, while pairing it with Vyndaqel led to a 41% reduction (so it might help instead of hinder Pfizer).
- Alnylam’s Amvuttra already treats ATTR related nerve damage and is a member of the siRNA drug class, setting it apart from Pfizer and BridgeBio’s ATTR options.
The Takeaway
It’s pretty remarkable that just six years ago we had no pharmaceutical options for treating ATTR-CM and now we have two (with a potential third coming). While that brings hope for patients suffering from this rare disease, providers will likely have to weigh the drugs’ exorbitant costs with their long-term benefits.
