An independent real-world study of 2,684 ATTR-CM patients from U.S. electronic health records found that acoramidis was associated with an 18% lower risk of death, hospitalization, atrial fibrillation, and diuretic intensification compared to tafamidis. Acoramidis showed 27% lower risk of cardiovascular and heart failure hospitalizations. These findings replicate earlier comparative effectiveness analyses and support acoramidis as a preferred stabilizer treatment option.

BridgeBio announced positive Phase 3 CALIBRATE trial results for encaleret in autosomal dominant hypocalcemia type 1 (ADH1), showing 91% of participants achieved normal PTH levels compared to 0% on standard care, with 100% reporting symptom improvements versus 46% on conventional therapy. The FDA accepted the NDA with Priority Review (PDUFA date May 8, 2027), and the company initiated the Phase 3 RECLAIM-HP trial for chronic hypoparathyroidism.

BridgeBio Pharma announced FDA acceptance of its regulatory filing for infigratinib, an oral drug to treat achondroplasia in children, with priority review and a decision expected by February 4, 2027. The drug demonstrated 2.1 cm/year greater height growth than placebo in Phase III trials. If approved, it would be the first oral therapy for achondroplasia, differentiating it from injectable competitors. BridgeBio also has two other near-term regulatory catalysts (BBP-418 and encaleret) that could diversify its revenue beyond its current marketed product Attruby.

BioMarin Pharmaceutical announced it is discontinuing development of Voxzogo for Noonan syndrome due to study feasibility and treatment landscape concerns, though not due to safety or efficacy issues. Despite this setback, Voxzogo generated $472 million in first-half 2026 sales (up 9% YoY) and is on track to achieve blockbuster status with expected 2026 sales of $1.00-$1.05 billion. The drug faces increasing competition from Ascendis Pharma's Yuviwel and BridgeBio's infigratinib, though BioMarin secured a royalty-bearing settlement with Ascendis.

BioMarin settled a patent dispute with Ascendis Pharma over the achondroplasia drug Yuviwel, converting a competitive threat into a royalty stream. Under the agreement, BioMarin will receive 20% royalties on U.S. Yuviwel sales and 18% in the EU, Brazil, and South Korea through May 2030. While this monetizes competition, BioMarin still faces market pressure from Yuviwel and potential future competitors like BridgeBio's infigratinib.

BridgeBio Pharma has entered into a voluntary agreement with the U.S. government to expand Medicaid access to its marketed medicine Attruby and lower drug costs for American patients. The agreement includes expanded state Medicaid access via the GENEROUS Model and maintains the company's ForgingBridges patient assistance program. BridgeBio expects not to be subject to future pricing mandates under this arrangement.

BridgeBio Pharma's stock has surged 29% in three months, driven by strong performance of its FDA-approved heart medication Attruby, which saw sales more than triple year-over-year to $222 million in Q2. With 16 out of 17 analysts recommending a buy and three promising drugs in late-stage development under FDA review, Wall Street believes the stock has further upside potential despite the company remaining unprofitable.

The transthyretin amyloidosis treatment market is projected to grow from USD 8.95 billion in 2025 to USD 35.10 billion by 2035 at a 14.64% CAGR. Growth is driven by increased ATTR-CM diagnosis rates, FDA approvals of gene-silencing therapies like vutrisiran and acoramidis, improved diagnostic imaging, and expanding pipeline therapies. North America dominates with 46% market share, while Asia Pacific is the fastest-growing region.

Alnylam Pharmaceuticals has gained strong analyst support with 21 of 29 analysts rating it as buy or strong buy, with an average price target of $436 (45% upside). The company is transitioning from a clinical biotech to a profitable commercial powerhouse, with Q1 product revenue surging 121% year-over-year to $1.04 billion, driven by its ATTR franchise. The company's pipeline is expanding into mainstream markets like cardiomyopathy and hypertension, supported by a unique RNAi delivery platform that creates a competitive moat. However, the stock trades at a high valuation of 75x trailing earnings, with risks from competitive pricing pressure.

BridgeBio Pharma announced a $1 billion preferred equity investment led by Sixth Street ($800M) and HealthCare Royalty ($133.9M). The Series A Cumulative Convertible Preferred Stock features a 7% dividend and conversion price of $137.79 per share (100%+ premium to 30-day VWAP). The capital strengthens BridgeBio's balance sheet as it prepares for potential launches of three blockbuster drugs: BBP-418 for LGMD2I/R9, encaleret for ADH1, and infigratinib for achondroplasia, while Attruby continues to grow as a multi-billion-dollar drug.

The cardiovascular disease market is experiencing significant growth with U.S. heart disease costs projected to quadruple by 2050. The global AI cardiology market is expected to reach $14 billion by 2034, while interventional cardiology devices market will grow from $31.12 billion to $42.39 billion by 2031. Five major companies are advancing cardiac solutions: VentriPoint Diagnostics is expanding globally with regulatory submissions in China and new distribution partnerships; BridgeBio Pharma's acoramidis showed 44.7% reduction in all-cause mortality; Bristol Myers Squibb's Camzyos met primary endpoints in adolescent trials; Arrowhead Pharmaceuticals' plozasiran achieved 83% median triglyceride reduction; and Amgen's Repatha reduced cardiovascular event risk by 31% in primary prevention patients.

BridgeBio announced long-term efficacy data from the ATTRibute-CM trial showing acoramidis (Attruby) achieved a 44.7% reduction in all-cause mortality and 49.3% reduction in cardiovascular mortality at Month 54 compared to placebo. The drug demonstrated sustained clinical benefits including mitigation of NT-proBNP progression and maintained quality of life scores, with a favorable safety profile.