Pfizer Inc.
Pfizer Inc. works on 63 rare diseases tracked on Trial Friend, including Achondroplasia, Acromegaly, Adrenocortical Carcinoma and 60 more, with 15 recruiting clinical trials and 47 FDA-approved rare disease drugs.
Pfizer has a broad rare disease portfolio spanning small molecules and biologics across hematology, neurology, and endocrinology. Key products include Vyndaqel (tafamidis) for transthyretin amyloid cardiomyopathy. Pfizer also pursues treatments for Duchenne muscular dystrophy, Gaucher disease, sickle cell disease, and other rare genetic conditions. The company discontinued its BEQVEZ gene therapy for hemophilia B in February 2025.
Focus areas at Pfizer Inc.
Within its broader pharmaceutical portfolio, Pfizer Inc. has active clinical trial programs and drug development efforts across 63 rare diseases, including Achondroplasia, Acromegaly, Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Aplastic Anemia, and 58 additional rare conditions. 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 all active and recruiting studies sponsored by Pfizer 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.
Pfizer Inc. is headquartered in New York, United States, founded in 1849, publicly traded under the ticker symbol PFE. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Pfizer Inc. Drug Pipeline
Pfizer Inc. has 15 active clinical trials across 5 development stages, with 15 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 Pfizer Inc.'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.
Pfizer Inc. Clinical Trials (15)
Active and recruiting clinical trials sponsored by Pfizer Inc., 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.
Pfizer Inc. FDA-Approved Drugs (47)
Medications developed or marketed by Pfizer Inc. 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 |
|---|---|---|---|
| SPIRONOLACTONE Aldosterone Antagonist [EPC] | Aldactone oral | Jan 21, 1960 | |
| SULFASALAZINE Aminosalicylate [EPC] | Azulfidine oral | Jun 20, 1950 | |
| SULFASALAZINE Aminosalicylate [EPC] | Azulfidine EN-tabs oral | Jun 20, 1950 | |
| PENICILLIN G BENZATHINE AND PENICILLIN G PROCAINE | BICILLIN C-R 900/300 intramuscular | May 18, 1953 | |
| PENICILLIN G BENZATHINE | BICILLIN L-A intramuscular | Jun 27, 1952 |
Pfizer Inc. Trial Locations
Pfizer Inc. clinical trials are running at 300 sites in 29 countries. 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.
Rare Disease Focus Areas (63)
Diseases targeted by Pfizer Inc.'s clinical trial and drug development programs
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...
Acromegaly is a rare hormonal disorder caused by excessive growth hormone production, typically from a pituitary adenoma, resulting in abnormal growth of hands, feet, and facial features. It also caus...
Adrenocortical carcinoma is a rare, aggressive cancer of the adrenal cortex, the outer layer of the adrenal glands that sit on top of each kidney. These glands produce essential hormones including cor...
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
Aplastic anemia is a rare bone marrow failure syndrome characterized by pancytopenia resulting from absent or severely reduced hematopoiesis. Approximately half are immune-mediated, while others resul...
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...
Patient Resources
Organizations and resources related to Pfizer Inc.'s rare disease focus areas
Frequently Asked Questions About Pfizer Inc.
Common questions about Pfizer Inc.'s rare disease programs, clinical trials, and treatments.