BridgeBio Pharma Inc

The achondroplasia market across the seven major markets—the United States, Germany, France, Italy, Spain, the United Kingdom, and Japan—was valued at approximately USD 600 million in 2025 and is expected to grow at a compound annual growth rate of approximately 11% from 2026 to 2036, according to a new report from ResearchAndMarkets.com. The diagnosed prevalent population in these markets was estimated at approximately 28,500 cases in 2025, with the United States representing the largest patient population and commercial market at approximately 14,500 cases. The treatment landscape is shifting toward targeted therapies, with approved drugs VOXZOGO (vosoritide) and YUVIWEL (navepegritide; TransCon CNP) leading the way, while emerging pipeline candidates include infigratinib (BBP-831/BGJ398) from QED Therapeutics, a BridgeBio company, in collaboration with Novartis and Kyowa Kirin, RBM-007 (umedaptanib pegol) from RIBOMIC, and BMN 333 from BioMarin Pharmaceutical. Recent developments include RIBOMIC's Phase II summary and Phase III application in Japan, BridgeBio's positive Phase III PROPEL 3 results for infigratinib, and BioMarin's Phase I data for BMN 333. The report highlights opportunities in earlier genetic diagnosis, targeted FGFR3 therapies, and long-acting CNP analogs, while noting unmet needs such as limited treatment options across age groups and high treatment costs.

BridgeBio Pharma reported new cardiac magnetic resonance imaging data from the Phase 3 ATTRibute-CM study and its open-label extension, suggesting that some acoramidis-treated patients experienced improvements in cardiac structure and function. In a completer analysis, 54% of acoramidis-treated patients achieved a clinically meaningful improvement in left ventricular systolic function at Month 30, compared with 20% of placebo-treated patients, while a more conservative analysis showed improvement in 34% of acoramidis-treated patients versus 9% of placebo-treated patients. Acoramidis also reduced the estimated mean percentage of days lost to death or cardiovascular hospitalization to 7.5% from 11.7% with placebo through Month 30, translating to 38 additional days alive outside the hospital, with the difference reaching 65 days by Month 36. However, these exploratory analyses are not randomized and cannot establish disease reversal, and no head-to-head comparison with competing therapies was provided. BridgeBio has initiated ASCEND-ATTR, a Phase 3b/4 study to prospectively evaluate sustained improvement in cardiac structure, function, and amyloid burden.

BridgeBio Pharma has dosed the first participant in ASCEND-ATTR, a Phase 3b/4 study evaluating the long-term effects of acoramidis on cardiac structure, function, and amyloid burden in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The single-arm, open-label study will enroll approximately 150 participants and use annual cardiac MRI and echocardiography over 36 months, with the primary endpoint being improvement in left ventricular systolic function at month 36. This follows earlier Phase 3 ATTRibute-CM data suggesting acoramidis may reverse disease progression and restore heart health. Additional data from the ATTRibute-CM CMR substudy and its open-label extension will be presented at the ESC Congress 2026.

BridgeBio Pharma reported second-quarter revenues of $243.7 million, up 120% year over year and above the Zacks Consensus Estimate of $222.6 million, driven by Attruby U.S. product sales of $222.4 million, more than triple the $71.5 million from a year earlier. The company posted a net loss of 78 cents per share, wider than the expected 64-cent loss but improved from a 95-cent loss a year ago, as research and development expenses rose 34% to $149.4 million and selling, general and administrative expenses climbed 44% to $186.3 million. Management said Attruby's first-line share in ATTR-CM rose two to three percentage points in the quarter, while the pool of patients switching from Pfizer's Vyndaqel/Vyndamax has begun to normalize. BridgeBio ended June with $720.2 million in cash, cash equivalents and marketable securities, down from $940.2 million at the end of the prior quarter, but a $1 billion preferred equity financing that closed July 1 lifted the cash balance to about $1.7 billion. The company is preparing for three potential U.S. product launches over the next 12 months, including BBP-418 with an FDA decision date of November 27, 2026, encaleret under review for a May 8, 2027 decision, and infigratinib targeted for a potential early- to mid-2027 launch if approved.

BridgeBio Pharma has secured Priority Review status from the FDA for two of its rare disease drug candidates, BBP-418 and encaleret. BBP-418, a potential first therapy for limb-girdle muscular dystrophy type 2I/R9, has a PDUFA date of November 27, 2026, while encaleret for autosomal dominant hypocalcemia type 1 has a PDUFA date of May 8, 2027. The company also reported continued differentiated efficacy for acoramidis in ATTR-CM based on late-phase data and real-world findings. BridgeBio posted quarterly revenue of US$243.68 million in Q2 2026 with a smaller net loss than a year earlier, supporting its transition to a commercial-stage rare disease platform. The company completed a US$390 million follow-on equity offering in August 2026, highlighting financing risk as it funds multiple product launches.

BridgeBio Pharma shares have climbed 29% over the past three months, driven by strong sales of its Attruby heart medication and a promising late-stage drug pipeline. Attruby, approved by the FDA in late 2024 for transthyretin-mediated amyloid cardiomyopathy, saw second-quarter sales more than triple year over year to over $222 million from $71.5 million. Total revenue reached nearly $244 million, more than double the nearly $111 million reported a year earlier. The company has submitted New Drug Applications for all three late-stage programs, with BBP-418 receiving a PDUFA date of November 27. Analysts are largely bullish, with 16 of 17 rating the stock a buy, and Piper Sandler raised its price target while maintaining an overweight rating.

BridgeBio Pharma outlined plans for three upcoming drug launches, with the first PDUFA date for BBP-418 now set for November 27, 2026. CEO Neil Kumar said all three NDAs for LGMD2I, ADH1 and achondroplasia have been submitted, and the company expects NDA acceptance and potentially priority review for infigratinib in the fourth quarter of 2026 with approval following in mid-2027. The global Phase III RECLAIM-HP trial for encaleret has commenced and is expected to read out in the next 18 months. Second-quarter total revenues reached $243.7 million, including $222.4 million in Attruby net product revenue, while cash and investments stood at $720.2 million as of June 30, rising to approximately $1.7 billion after a $1 billion preferred equity investment closed on July 1.

BridgeBio Pharma announced the FDA has accepted its New Drug Application for encaleret for autosomal dominant hypocalcemia type 1, setting a target action date of May 8, 2027. If approved, encaleret would be the first therapy specifically indicated for ADH1, representing a potential blockbuster opportunity. The Phase 3 CALIBRATE trial met all primary and key secondary endpoints, normalizing disease markers without calcium and vitamin D supplements. BridgeBio is also enrolling a pediatric registrational study and plans to start a Phase 3 trial in chronic hypoparathyroidism later this summer, which could broaden the drug's use.

BridgeBio Pharma shares surged to a 52-week high of $93.42 on Thursday after a late-stage clinical failure by rival AstraZeneca boosted confidence in BridgeBio's sole marketed drug, Attruby. The stock closed 15% higher, adding roughly $2.3 billion in market value, as investors viewed AstraZeneca's phase III CARDIO-TTRansform study miss for Wainua in ATTR-CM as strengthening Attruby's competitive position. Attruby, approved in November 2024 for ATTR-CM, generated $362.4 million in U.S. sales in 2025 and nearly $181 million in the first quarter of 2026. BridgeBio estimates the diagnosed U.S. ATTR-CM patient population grew from fewer than 5,000 in 2019 to over 50,000 in 2025, with a global market opportunity exceeding $20 billion. Pfizer, which dominates the ATTR-CM market with its Vyndaqel family generating about $1.6 billion in first-quarter 2026 global sales, also saw its shares rise 1% on the news.

AstraZeneca shares fell nearly 8% in pre-market trading after the company announced that a phase III study of Wainua failed to meet its primary endpoint in patients with transthyretin-mediated amyloid cardiomyopathy. The CARDIO-TTRansform study did not show a statistically significant benefit for Wainua added to standard of care compared to placebo on the composite outcome of cardiovascular mortality and recurrent cardiovascular clinical events. Ionis Pharmaceuticals, which co-develops the drug, saw its stock slump 19%. Wainua is already approved for polyneuropathy of hereditary transthyretin-mediated amyloidosis, and success in the larger ATTR-CM market had been expected to significantly expand its commercial opportunity. Following the setback, shares of rivals Alnylam Pharmaceuticals and BridgeBio Pharma rose about 18% and 11%, respectively, as their ATTR-CM therapies Amvuttra and Attruby are already on the market.

BridgeBio Pharma announced new post-hoc analyses showing acoramidis is associated with early and sustained direct kidney-protective effects in patients with transthyretin amyloid cardiomyopathy. Treatment initiation led to a reversible eGFR dip of 8.5 mL/min/1.73 m² and a placebo-corrected 15.5% reduction in urinary albumin to creatinine ratio by Day 28, with no kidney-related adverse events. Through Month 30, acoramidis provided a sustained improved chronic eGFR slope of plus 2.47 mL/min/1.73 m² per year and a sustained UACR reduction of 13.7%. The pattern resembles that of drugs acting directly on the kidney, such as ACE inhibitors, ARBs, and SGLT2 inhibitors, and has not been observed with other approved ATTR-CM therapies. Participants with larger acute eGFR dips had a 58% lower risk of death or cardiovascular hospitalization in the first year, while eGFR dips in the placebo arm were associated with worse outcomes.

BridgeBio Pharma has secured up to $1 billion in convertible preferred equity from funds managed by Sixth Street and HealthCare Royalty, a business of KKR. Sixth Street provided $800 million as the lead investor, while HealthCare Royalty contributed $133.9 million at closing. The Series A Cumulative Convertible Participating Preferred Stock carries a 7% initial dividend and an initial conversion price of $137.79 per share, representing more than a 100% premium to BridgeBio's 30-day volume-weighted average price. The financing arrives as Attruby continues to grow into a multi-billion-dollar blockbuster drug and the company prepares for three additional potential blockbuster U.S. product launches over the next 12 months: BBP-418 for LGMD2I/R9, encaleret for ADH1, and infigratinib for achondroplasia.

Sixth Street Partners and KKR have reportedly agreed to provide $1 billion of preferred equity to biotechnology company BridgeBio Pharma. The financing, expected to be announced as soon as Wednesday, will help BridgeBio accelerate the launch of new drugs intended to treat genetic diseases, Bloomberg reported, citing people with knowledge of the matter. BridgeBio Pharma shares, down 1.4% in extended trading, are trading roughly 3% lower so far this year.

BridgeBio Pharma announced that positive results from the Phase 3 PROPEL 3 trial of oral infigratinib in children with achondroplasia were published in the New England Journal of Medicine and presented at the International Congress of Children's Bone Health. The study met its primary endpoint with a least-squares mean treatment difference in annualized height velocity of plus 1.74 centimeters per year versus placebo, and an observed mean improvement of plus 2.10 centimeters per year, the largest reported in any Phase 3 achondroplasia study. A key secondary endpoint of height Z-score change was also met, and a pre-specified exploratory analysis showed the first statistically significant improvement in body proportionality against placebo in children under eight years old. Additional data presented at the congress revealed a statistically significant improvement in arm span versus placebo of plus 0.37 standard deviations. Oral infigratinib was well tolerated with no discontinuations or serious adverse events related to study drug. BridgeBio intends to submit a New Drug Application to the FDA in the third quarter of 2026 and a Marketing Authorization Application to the EMA in the second half of 2026, with a U.S. launch anticipated in early to mid 2027.

Alnylam Pharmaceuticals' lead drug Amvuttra generated $889.9 million in global sales in the first quarter of 2026, accounting for 76% of the company's total revenues. The figure represents 187% year-over-year growth, driven by strong uptake in ATTR amyloidosis with cardiomyopathy patients in the United States and switches from Onpattro. Alnylam's rare disease portfolio, consisting of Givlaari and Oxlumo, contributed $125.7 million in net product revenues, a 15% increase from the prior year, as the company seeks to reduce its reliance on Amvuttra. The pipeline includes late-stage assets such as zilebesiran in partnership with Roche for hypertension and nucresiran in phase III for ATTR amyloidosis, while Amvuttra faces growing competition from Pfizer's Vyndaqel family, which posted $1.6 billion in global revenues, and BridgeBio's Attruby, which reached $180.6 million in sales. Alnylam shares have fallen 30.1% year-to-date, and the stock trades at a price-to-sales ratio of 8.97, below its five-year mean of 18.24.