Sanofi / Genzyme
Sanofi / Genzyme works on 54 rare diseases tracked on Trial Friend, including Alpha-1 Antitrypsin Deficiency, Alport Syndrome, Amyotrophic Lateral Sclerosis and 51 more, with 58 active clinical trials (33 recruiting) and 17 FDA-approved rare disease drugs.
Sanofi-Genzyme is the specialty care business unit of Sanofi with over 35 years of experience pioneering treatments for rare genetic diseases, particularly lysosomal storage disorders including Gaucher, Fabry, and Pompe diseases. The division operates across rare diseases, rare blood disorders, and immunology therapeutic areas.
Focus areas at Sanofi / Genzyme
Within its broader pharmaceutical portfolio, Sanofi / Genzyme has active clinical trial programs and drug development efforts across 54 rare diseases, including Alpha-1 Antitrypsin Deficiency, Alport Syndrome, Amyotrophic Lateral Sclerosis, Aplastic Anemia, Becker Muscular Dystrophy, and 49 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 Sanofi / Genzyme, 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.
Sanofi / Genzyme is headquartered in Paris, France, founded in 1981, publicly traded under the ticker symbol SNYNF. 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.
Sanofi / Genzyme Drug Pipeline
Sanofi / Genzyme has 58 active clinical trials across 5 development stages, with 33 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.
Sanofi / Genzyme Clinical Trials (58)
Active and recruiting clinical trials sponsored by Sanofi / Genzyme, 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.
Sanofi / Genzyme FDA-Approved Drugs (17)
Medications developed or marketed by Sanofi / Genzyme 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 |
|---|---|---|---|
| TERIFLUNOMIDE Pyrimidine Synthesis Inhibitor [EPC] | Aubagio oral | Sep 12, 2012 | |
| VANDETANIB Kinase Inhibitor [EPC] | CAPRELSA oral | Apr 6, 2011 | |
| ELIGLUSTAT Glucosylceramide Synthase Inhibitor [EPC] | Cerdelga oral | Aug 19, 2014 | |
| IMIGLUCERASE Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC] | Cerezyme intravenous | May 23, 1994 | |
| DUPILUMAB Interleukin-4 Receptor alpha Antagonist [EPC] | Dupixent subcutaneous | Mar 28, 2017 |
Sanofi / Genzyme Trial Locations
Sanofi / Genzyme clinical trials are running at 2,725 sites in 66 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 (54)
Diseases targeted by Sanofi / Genzyme's clinical trial and drug development programs
Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective protein alpha-1 antitrypsin. Without adequate protection, neutro...
Alport Syndrome is a genetic disorder that causes progressive damage to the kidneys, ears, and eyes due to defects in a type of collagen that provides structure and flexibility to tissues. The conditi...
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...
Becker Muscular Dystrophy (BMD) is an X-linked inherited muscular dystrophy caused by mutations in the dystrophin gene that produce a partially functional dystrophin protein. BMD is milder than Duchen...
Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230. The condition leads to formation of large fluid-fille...
Patient Resources
Organizations and resources related to Sanofi / Genzyme's rare disease focus areas
Frequently Asked Questions About Sanofi / Genzyme
Common questions about Sanofi / Genzyme's rare disease programs, clinical trials, and treatments.