What rare diseases does Savara Inc. focus on?

Savara Inc. has active programs across 2 rare diseases: Cystic Fibrosis, Pulmonary Alveolar Proteinosis. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Is Savara Inc. focused exclusively on rare diseases?

Yes. Savara Inc. is a rare disease specialist headquartered in Yardley, United States. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Cystic Fibrosis, Pulmonary Alveolar Proteinosis.

How can I find Savara Inc. clinical trials for rare diseases?

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Savara Inc., 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 Savara Inc. clinical trial?

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.

What FDA-approved rare disease drugs does Savara Inc. have?

None as of October 2026. Savara Inc. has no FDA-approved drug yet; its programs are still in trials. Most advanced: Molgramostim (Phase 3). Where to watch: the FDA calendar lists any Savara Inc. application once a decision date is public, and the trials list on this page shows what is recruiting now.

Savara Inc.

Rare disease drugmaker · based in Yardley, United States · ticker SVRA · savarapharma.com

Savara Inc. works on Cystic Fibrosis and Pulmonary Alveolar Proteinosis, 2 of the rare diseases we track. It has 2 active clinical trials, and 1 is looking for volunteers now.

Savara is a clinical-stage biopharmaceutical company in Yardley, Pennsylvania, focused on rare respiratory diseases. Its lead candidate, molgramostim inhalation solution (proposed name Molbreevi), is an inhaled GM-CSF for autoimmune pulmonary alveolar proteinosis, with an FDA decision due November 22, 2026, and is not yet approved anywhere.

Savara Inc. pipeline

Each row lists the diseases Savara Inc. is studying at that stage, from the ones closest to approval down to the earliest work.

Phase 3

Large studies, usually the last step before a company asks the FDA for approval.

Pulmonary Alveolar Proteinosis 2 trials recruiting

Savara Inc. clinical trials

These are Savara Inc. 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.

1 trial looking for volunteers now

Looking for volunteersPhase 3No updates in a whileNCT06431776

Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).

Tests Molgramostim

For Autoimmune Pulmonary Alveolar Proteinosis

The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are: The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP. This is an open-label study: all participants will receive treatment with molgramostim. Patients will: * Take molgramostim once daily via nebulizer every day for 12 months. * Visit the clinic approximately every 12 weeks for checkups and tests. * Keep a diary of any oxygen use.

Ages 6 Years - 18 Years1 location

View full study

1 trial underway, not taking new volunteers

Underway, not taking new volunteersPhase 3Recently updatedNCT04544293

Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Tests Molgramostim, Placebo, Molgramostim Open-label

For Autoimmune Pulmonary Alveolar Proteinosis

160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim (MOL) or placebo (PBO) for 48 weeks. Subjects completing the 48-week placebo-controlled period will receive open-label treatment with once daily inhaled molgramostim for 96 weeks.

Ages 18 Years+54 locations

View full study

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More about Savara Inc.

As a rare disease specialist, Savara Inc. has active clinical trial programs and drug development efforts across 2 rare diseases, including Cystic Fibrosis, Pulmonary Alveolar Proteinosis. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.

The clinical trials section below shows active and recruiting rare disease studies sponsored by Savara Inc., sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites.

The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.

Savara Inc. is headquartered in Yardley, United States, publicly traded under the ticker symbol SVRA. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.

Where Savara Inc. trials take place

The trials above run at 55 study sites in 18 countries. Tap a country to see its states, cities and hospitals.

United States

16▼

Japan

9▼

Germany

4▼

South Korea

4▼

Canada

3▼

Turkey (Türkiye)

3▼

Australia

2▼

Belgium

2▼

France

2▼

Portugal

2▼

Greece

1▼

Ireland

1▼

Rare diseases Savara Inc. works on

Each disease page lists every open trial, not only Savara Inc.'s, plus approved treatments.

Cystic Fibrosis

Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport.

How common: Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians

Pulmonary Alveolar Proteinosis

Pulmonary alveolar proteinosis (PAP) is a rare lung disease in which a soapy substance called surfactant builds up inside the air sacs because the cells meant to clear it have stopped working.

How common: Autoimmune PAP affects about 6 to 7 people per million (NORD and the PAP Foundation) and makes up 85-90% of cases, with hereditary, secondary and congenital forms each around 5%

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Savara Inc.'s own site is savarapharma.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Cystic Fibrosis Foundation
Cystic Fibrosis
NIH GARD - Cystic Fibrosis
Cystic Fibrosis
National Organization for Rare Disorders (NORD) - Pulmonary Alveolar Proteinosis
Pulmonary Alveolar Proteinosis
PAP Foundation - Frequently Asked Questions
Pulmonary Alveolar Proteinosis