Pfizer Inc.
Pfizer Inc. works on 64 rare diseases tracked on Trial Friend, including Achondroplasia, Acromegaly, Adrenocortical Carcinoma and 61 more, with 21 active clinical trials (12 recruiting) and 17 FDA-approved rare disease drugs.
Pfizer's rare disease medicines include Vyndamax (tafamidis) for transthyretin amyloid cardiomyopathy, Hympavzi (marstacimab) for hemophilia A and B, Somavert (pegvisomant) for acromegaly and Elelyso (taliglucerase alfa) for Gaucher disease. Its rare disease pipeline includes dazukibart for dermatomyositis in Phase 3. 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 64 rare diseases, including Achondroplasia, Acromegaly, Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Aplastic Anemia, and 59 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 active and recruiting rare disease 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 21 active clinical trials across 5 development stages, with 12 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 counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Pfizer Inc. Clinical Trials (21)
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.
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Pfizer Inc. FDA-Approved Drugs (17)
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 |
|---|---|---|---|
| AMLODIPINE AND ATORVASTATIN | Caduet oral | Jan 30, 2004 | |
| TRANEXAMIC ACID Antifibrinolytic Agent [EPC] | Cyklokapron intravenous | — | Dec 30, 1986 |
| DOXORUBICIN HYDROCHLORIDE | Doxorubicin Hydrochloride intravenous | Aug 7, 1974 | |
| TALIGLUCERASE ALFA Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC] | ELELYSO intravenous | May 1, 2012 | |
| SOMATROPIN | GENOTROPIN | Aug 24, 1995 |
Pfizer Inc. Trial Locations
Pfizer Inc. clinical trials are running at 427 sites in 21 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 (64)
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.