Rare disease drugmaker · based in Palo Alto, United States · founded 2015 · ticker BBIO · bridgebio.com
BridgeBio Pharma works on 6 rare diseases we track, including Achondroplasia, ATTR Amyloidosis (Transthyretin Amyloidosis) and 4 more. It has 10 active clinical trials, and 6 are looking for volunteers now. It also has 1 FDA-approved rare disease drug.
The FDA is due to decide on BBP-418 for Limb-girdle muscular dystrophy type 2I/R9 by November 27, 2026.
The FDA is due to decide on Infigratinib for Achondroplasia in children by February 4, 2027.
The FDA is due to decide on Encaleret for Autosomal dominant hypocalcemia type 1 (ADH1) by May 8, 2027.
BridgeBio Pharma develops therapies for genetic diseases using a subsidiary model to advance programs from discovery through commercialization. The company's lead product Attruby (acoramidis) was FDA-approved in November 2024 for ATTR cardiomyopathy, based on the placebo-controlled ATTRibute-CM trial in 611 adults.
BridgeBio Pharma pipeline
Each row lists the diseases BridgeBio Pharma is studying at that stage, from the ones closest to approval down to the earliest work.
These are BridgeBio Pharma trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
6 trials looking for volunteers now
Looking for volunteersPhase 3Recently updatedNCT07805356
Efficacy, Safety, and Tolerability of Encaleret in Participants With Chronic Hypoparathyroidism
The aim of the study is to evaluate the efficacy, safety, and tolerability of orally administered encaleret tablets in participants with chronichypoparathyroidism (HP).
Recent studies have shown that transthyretin amyloidosis (ATTR) can sometimes cause a type of heart failure where the pumping function of the heart is normal, also known as Heart Failure with Preserved Ejection Fraction (HFpEF) or diastolicheart failure. In this single center diagnostic study, we will evaluate for ATTR in patients with HFpEF in order to to determine how frequently this occurs and how we can predict which heart failure patients may have TTR amyloidosis. Our goal is to identify amyloidosis in heart failure patients earlier so that they can start treatment.
Looking for volunteersPhase 3Updated a few months agoNCT06563895
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
TestsAcoramidis, Placebo oral tablet
For Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies and Heart Diseases
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age.
Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloidpolyneuropathy or ATTR-PN.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.
This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.
Looking for volunteersPhase 2Updated a few months agoNCT07169279
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
TestsInfigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.
The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigationalAAV9-based gene therapy, in pediatric participants with Canavan disease.
This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile. This will encompass the significant majority of existing diagnosed patients based upon the established epidemiology of the disease.
Underway, not taking new volunteersUpdated a few months agoNCT07062848
An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.
TestsAI Toolkit for ATTR-CMDiagnosis
For Transthyretin (TTR) Amyloid Cardiomyopathy
This is a multi-center, observational study with the overall objective to examine the scale of under-diagnosis for transthyretin amyloid cardiomyopathy (ATTR-CM) across a broad range of diverse health systems in the US using a fully federated deployment of an artificial intelligence (AI) toolkit of algorithms that detect ATTR-CM on electrocardiography (ECG), point-of-care ultrasound (POCUS), and transthoracic echocardiography (TTE).
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Oral infigratinib for achondroplasia (Breakthrough Therapy designation) reported positive Phase 3 PROPEL 3 results in February 2026, and the full results published in the New England Journal of Medicine show children grew about 1.74cm more per year than on placebo in the main analysis, or 2.10cm per year as a simple average.
BridgeBio says it is the first achondroplasia drug to show a statistically significant improvement in body proportions against placebo. On October 6, 2026 the FDA accepted the infigratinib application for children with achondroplasia under priority review, with a decision date of February 4, 2027, and BridgeBio plans to file in Europe in the fourth quarter of 2026.
The FDA is also reviewing 2 more BridgeBio applications: BBP-418 for limb-girdle muscular dystrophy type 2I/R9, with a decision date of November 27, 2026, and encaleret for autosomal dominanthypocalcemia type 1 (ADH1), with a decision date of May 8, 2027.
Where BridgeBio Pharma trials take place
The trials above run at 186 study sites in 25 countries. Tap a country to see its states, cities and hospitals.
United States
95▼
Italy
9▼
United Kingdom
9▼
Australia
7▼
Spain
7▼
Canada
6▼
Netherlands
6▼
France
5▼
Germany
5▼
Brazil
4▼
South Korea
4▼
Japan
3▼
Rare diseases BridgeBio Pharma works on
Each disease page lists every open trial, not only BridgeBio Pharma's, plus approved treatments.
Achondroplasia is the most common form of short-limbed dwarfism, caused by a mutation in the FGFR3 gene that disrupts the conversion of cartilage to bone in the growth plates.
How common: Approximately 1 in 25,000 live births; estimated 25,000 to 30,000 people in the United States
ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs.
How common: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S
Hypoparathyroidism is an endocrine disorder caused by insufficient production of parathyroid hormone (PTH), resulting in chronically low calcium and elevated phosphorus levels in the blood.
How common: Approximately 22 per 100,000 for postsurgical forms; estimated 70,000 to 77,000 people living with chronichypoparathyroidism in the United States
Limb-Girdle Muscular Dystrophy (LGMD) refers to a genetically heterogeneous group of muscular dystrophies characterized by progressive weakness of the hip and shoulder girdle muscles.
How common: Approximately 1 per 14,500 to 1 per 123,000 people depending on geographic region; exact prevalence varies by subtype
Wild-type ATTRcardiacamyloidosis is an age-related disease where normal transthyretin protein spontaneously misfolds and accumulates in the heart, causing progressive heart failure.
How common: Estimated to affect up to 13% of males aged 85 and older; substantially underdiagnosed
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. BridgeBio Pharma's own site is bridgebio.com, and our help paying for treatment page lists drugmaker programs and charity funds.
1 shared disease, including ATTR Amyloidosis (Transthyretin Amyloidosis)
Questions about BridgeBio Pharma
BridgeBio Pharma develops therapies for genetic diseases using a subsidiary model to advance programs from discovery through commercialization. The company's lead product Attruby (acoramidis) was FDA-approved in November 2024 for ATTR cardiomyopathy, based on the placebo-controlled ATTRibute-CM trial in 611 adults. Oral infigratinib for achondroplasia (Breakthrough Therapy designation) reported positive Phase 3 PROPEL 3 results in February 2026, and the full results published in the New England Journal of Medicine show children grew about 1.74cm more per year than on placebo in the main analysis, or 2.10cm per year as a simple average. BridgeBio says it is the first achondroplasia drug to show a statistically significant improvement in body proportions against placebo. On October 6, 2026 the FDA accepted the infigratinib application for children with achondroplasia under priority review, with a decision date of February 4, 2027, and BridgeBio plans to file in Europe in the fourth quarter of 2026. The FDA is also reviewing 2 more BridgeBio applications: BBP-418 for limb-girdle muscular dystrophy type 2I/R9, with a decision date of November 27, 2026, and encaleret for autosomal dominanthypocalcemia type 1 (ADH1), with a decision date of May 8, 2027.
BridgeBio Pharma has active programs across 6 rare diseases: Achondroplasia, ATTR Amyloidosis (Transthyretin Amyloidosis), Canavan Disease, Hypoparathyroidism, Limb-Girdle Muscular Dystrophy, Wild-Type ATTRCardiacAmyloidosis. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. BridgeBio Pharma is a rare disease specialist headquartered in Palo Alto, United States, founded in 2015. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Achondroplasia, ATTR Amyloidosis (Transthyretin Amyloidosis), Canavan Disease among others.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by BridgeBio Pharma, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
BridgeBio Pharma has 1 FDA-approved drug with a rare disease use that we track, as of November 2026.
Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.
Yes, for 1 of its FDA-approved rare disease medicine that we track, each checked on BridgeBio Pharma's own website.
Attruby: ForgingBridges, 1-888-552-7434. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked November 9, 2026.
Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.
Eligibility is decided by each program, so call the number listed before a prescription is filled.
BridgeBio Pharma was founded in 2015 and is headquartered in Palo Alto, United States. The company has been active for 11 years. It trades publicly under the ticker symbol BBIO. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.