What rare diseases does BridgeBio Pharma focus on?

BridgeBio Pharma has active programs across 4 rare diseases: Achondroplasia, ATTR Amyloidosis (Transthyretin Amyloidosis), Hypoparathyroidism, Peripartum Cardiomyopathy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Is BridgeBio Pharma focused exclusively on rare diseases?

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), Hypoparathyroidism among others.

How can I find BridgeBio Pharma clinical trials for rare diseases?

This page shows all active and recruiting clinical 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.

How do I enroll in a BridgeBio Pharma clinical trial?

To explore enrollment, find a recruiting trial 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.

What FDA-approved rare disease drugs does BridgeBio Pharma have?

The FDA-Approved Drugs section on this page lists all BridgeBio Pharma drugs with FDA approval that are relevant to rare diseases, including brand names, generic names, approval dates, and the rare conditions each drug treats. This data comes from the FDA's openFDA database and is updated automatically.

When was BridgeBio Pharma founded and where is it based?

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.

BridgeBio Pharma

BridgeBio Pharma works on 4 rare diseases tracked on Trial Friend, including Achondroplasia, ATTR Amyloidosis (Transthyretin Amyloidosis), Hypoparathyroidism and 1 more, with 1 recruiting clinical trial and 1 FDA-approved rare disease drug.

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, where it demonstrated superiority over tafamidis in the ATTRibute-CM trial. Oral infigratinib for achondroplasia (Breakthrough Therapy designation) reported positive Phase 3 PROPEL 3 results in February 2026, meeting its primary endpoint with a 2.1 cm/year improvement in annualized height velocity over placebo and showing the first statistically significant improvement in body proportionality versus placebo; NDA and MAA submissions are planned in H2 2026. The pipeline also includes encaleret for autosomal dominant hypocalcemia and other genetic disease programs across its subsidiary network.

Type

Rare Disease Specialist

Ticker

BBIO

Headquarters

Palo Alto, United States

Founded

2015

Website

bridgebio.com

Active Rare Disease Trials

Approved Rare Disease Drugs

Rare Diseases in Portfolio

Years Active

FDA decision ahead

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 Encaleret for Autosomal dominant hypocalcemia type 1 (ADH1) by May 8, 2027.

See all upcoming rare disease FDA decisions →

BridgeBio Pharma Drug Pipeline

BridgeBio Pharma has 1 active clinical trial across 1 development stage, with 1 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline includes all of BridgeBio Pharma's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.

Understand BridgeBio Pharma's pipeline

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Other1 trial

Transthyretin (TTR) Amyloid Cardiomyopathy

Recruiting

BridgeBio Pharma Clinical Trials (1)

Active and recruiting clinical trials sponsored by BridgeBio Pharma, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

Ask about BridgeBio Pharma's trials

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ACTIVE NOT RECRUITINGRecently updatedNCT07062848

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.

Intervention: AI Toolkit for ATTR-CM Diagnosis

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).

Ages 50 Years - 95 Years12 locations

View full study

BridgeBio Pharma FDA-Approved Drugs (1)

Medications developed or marketed by BridgeBio Pharma that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.

Drug Name	Brand Name	Rare Diseases	Approval Date
ACORAMIDIS HYDROCHLORIDE	Attruby oral	ATTR Amyloidosis (Transthyretin Amyloidosis)	Nov 22, 2024

BridgeBio Pharma Trial Locations

BridgeBio Pharma clinical trials are running at 12 sites in 1 country. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States

12▼

Rare Disease Focus Areas (4)

Diseases targeted by BridgeBio Pharma's clinical trial and drug development programs

AchondroplasiaConnective Tissue & Musculoskeletal

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. This results in dispropo...

Prevalence: Approximately 1 in 25,000 live births; estimated 25,000 to 30,000 people in the United States

ATTR Amyloidosis (Transthyretin Amyloidosis)Metabolic & Lysosomal

ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs. It can be inherited (hereditary ATTR) or dev...

Prevalence: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S.

HypoparathyroidismEndocrine & Hormonal

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. The most c...

Prevalence: Approximately 22 per 100,000 for postsurgical forms; estimated 70,000 to 77,000 people living with chronic hypoparathyroidism in the United States

Peripartum CardiomyopathyCardiovascular

Peripartum cardiomyopathy is a rare form of heart failure that develops during the last month of pregnancy or within 5 months after delivery in women with no prior history of heart disease. The heart ...

Prevalence: About 1 in 1,000 to 1 in 4,000 pregnancies in the U.S.; significantly more common in women of African descent