FDA Greenlights BridgeBio's Attruby, Challenging Pfizer's Dominance in Deadly Heart Disease Market

FDA Approves BridgeBio's Attruby for Deadly Heart Condition, Setting Stage for Major Competition with Pfizer

BridgeBio Pharma Inc. has reached a major milestone: the FDA has approved Attruby (acoramidis) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and fatal form of heart disease. ATTR-CM affects the heart muscle by causing amyloid deposits, which progressively impair cardiac function, leading to high mortality. The approval of Attruby is a big moment for patients who need better treatment options, and it puts BridgeBio in direct competition with Pfizer's already well-established drugs, Vyndaqel and Vyndamax, in the ATTR-CM market.

Attruby is an oral medication that works by stabilizing Transthyretin (TTR), a protein involved in transporting thyroid hormones and vitamin A throughout the body. With near-complete stabilization of TTR, Attruby is designed to halt the damage caused by misfolded proteins, a hallmark of ATTR-CM. This makes the drug a potentially more effective treatment compared to its main competitors.

The FDA decision follows positive results from the ATTRibute-CM Phase 3 trial, where Attruby showed significant benefits, including a 42% reduction in all-cause mortality and cardiovascular-related hospitalizations over 30 months. The drug also demonstrated a 50% reduction in cardiovascular-related hospitalizations and an 81% patient survival rate compared to 74% in the placebo group. With these strong results, BridgeBio aims to position Attruby as a superior alternative in the rapidly expanding ATTR-CM market, which was valued at $5.2 billion in 2023 and is expected to grow to $9.4 billion by 2031.

Key Takeaways

1. Attruby vs. Pfizer's Tafamidis: Attruby is priced at $18,759 for a 28-day supply, slightly undercutting Pfizer's Vyndaqel/Vyndamax at $20,600. This pricing strategy is expected to make Attruby an attractive option for patients and healthcare systems.

2. Market Potential: The ATTR-CM market is growing, with over 300,000 patients affected worldwide. Advances in diagnosis and increasing awareness are driving the potential for expanded treatment uptake.

3. Competitive Edge: Attruby provides greater efficacy with a 42% reduction in mortality and hospitalizations compared to Pfizer's 25%. Its ability to achieve 96% TTR stabilization also surpasses Pfizer's 50% stabilization rate.

4. Support Programs: BridgeBio has launched the ForgingBridges™ patient support program to provide insurance guidance and financial assistance, ensuring better access for patients in need of treatment.

Deep Analysis

The FDA approval of Attruby introduces an important player into the ATTR-CM landscape, which has so far been dominated by Pfizer's tafamidis drugs, known commercially as Vyndaqel and Vyndamax. These two drugs reported $3.3 billion in sales last year, indicating the significant demand for treatments addressing ATTR-CM. BridgeBio's Attruby, with a pricing strategy slightly below its rival, is aiming to carve out a share of this expanding market.

The competitive landscape in ATTR-CM is heating up beyond just Pfizer. Alnylam Pharmaceuticals is expecting FDA approval for its ATTR-CM therapy next year, while Intellia Therapeutics and Ionis Pharmaceuticals are also developing promising alternatives, including gene-editing solutions. These emerging treatments may push the boundaries of efficacy and offer different mechanisms of action, which could change the treatment paradigm for ATTR-CM in the coming years.

One of BridgeBio's significant advantages is Attruby's rapid clinical effect. Data from the Phase 3 trial showed a substantial reduction in mortality and cardiovascular hospitalizations just 30 months into treatment. Mayo Clinic's Dr. Martha Grogan highlighted the early and rapid benefits, which she believes could be crucial for patients who are facing the life-threatening challenges of ATTR-CM. Attruby's near-complete TTR stabilization of 96% is also seen as a major point of differentiation, giving it an edge over Pfizer's 50% stabilization rate.

However, BridgeBio also faces challenges. Despite having a potential efficacy advantage, BridgeBio is a smaller company with fewer resources compared to Pfizer's vast sales force and established market presence. BridgeBio will need to meet or exceed ambitious sales targets, with analysts expecting it to achieve $165 million in U.S. sales by 2025.

To support patient access, BridgeBio has rolled out the ForgingBridges™ program. This initiative aims to ease the financial burden on patients and assist with insurance navigation—a smart move to remove barriers to treatment adoption and foster long-term patient loyalty. BridgeBio CEO Neil Kumar remains optimistic, despite acknowledging skepticism, and believes Attruby's rapid action and strong efficacy data will lead to success in treating this debilitating disease.

Did You Know?

• ATTR-CM is often misdiagnosed as other forms of heart failure. Recent advances in testing, such as non-invasive imaging and the use of biomarkers, have significantly improved the accuracy and speed of diagnosis, helping more patients receive the correct treatment.

• Transthyretin amyloidosis can also affect other organs besides the heart, including nerves, leading to a broader condition called ATTR-PN (polyneuropathy).

• The ATTR-CM treatment market is one of the fastest-growing sectors in cardiovascular diseases, with a projected annual growth rate of over 8% from 2023 to 2031, largely driven by innovations like gene-editing therapies and precision medicine.

BridgeBio's entry with Attruby has already started to shake up the ATTR-CM landscape, and as awareness grows and diagnosis improves, more patients stand to benefit from these life-saving treatments. For now, Attruby's clinical results and competitive pricing provide hope for ATTR-CM patients who previously had limited options.