Roche Holding AG / Genentech
Roche Holding AG / Genentech works on 63 rare diseases tracked on Trial Friend, including Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Angelman Syndrome and 60 more, with 71 recruiting clinical trials and 25 FDA-approved rare disease drugs.
Roche, through its subsidiary Genentech, develops therapies across oncology, immunology, neurology, and rare diseases. The company applies its expertise in biotechnology and precision medicine to address rare genetic and complex diseases with limited treatment options.
Focus areas at Roche Holding AG / Genentech
Within its broader pharmaceutical portfolio, Roche Holding AG / Genentech has active clinical trial programs and drug development efforts across 63 rare diseases, including Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Angelman Syndrome, Atypical Hemolytic Uremic Syndrome, Autoimmune Hepatitis, 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 Roche Holding AG / Genentech, 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.
Roche Holding AG / Genentech is headquartered in Basel, Switzerland, founded in 1896, publicly traded under the ticker symbol RHHBY. 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.
Roche Holding AG / Genentech Drug Pipeline
Roche Holding AG / Genentech has 71 active clinical trials across 5 development stages, with 71 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 Roche Holding AG / Genentech'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.
Roche Holding AG / Genentech Clinical Trials (71)
Active and recruiting clinical trials sponsored by Roche Holding AG / Genentech, 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.
Roche Holding AG / Genentech FDA-Approved Drugs (25)
Medications developed or marketed by Roche Holding AG / Genentech 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 |
|---|---|---|---|
| TOCILIZUMAB Interleukin-6 Receptor Antagonist [EPC] | ACTEMRA intravenous, subcutaneous | Jan 8, 2010 | |
| ALTEPLASE | Activase | Nov 13, 1987 | |
| ALECTINIB HYDROCHLORIDE | ALECENSA oral | Dec 11, 2015 | |
| BEVACIZUMAB Vascular Endothelial Growth Factor Inhibitor [EPC] | Avastin intravenous | Feb 26, 2004 | |
| ALTEPLASE | Cathflo Activase intravenous | Nov 13, 1987 |
Roche Holding AG / Genentech Trial Locations
Roche Holding AG / Genentech clinical trials are running at 2,638 sites in 60 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 Roche Holding AG / Genentech's clinical trial and drug development programs
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...
Angelman Syndrome is a rare neurological disorder caused by loss of function of the UBE3A gene on the maternal chromosome 15. People with this condition typically develop normal until 6-12 months of a...
Atypical Hemolytic Uremic Syndrome (aHUS) is a rare kidney disease caused by uncontrolled activation of the complement system, a part of the immune system that normally helps fight infection. This act...
Autoimmune Hepatitis (AIH) is a chronic liver disease characterized by persistent inflammation and progressive fibrosis of the liver due to loss of immune tolerance to hepatocyte antigens. The immune ...
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 Roche Holding AG / Genentech's rare disease focus areas
Frequently Asked Questions About Roche Holding AG / Genentech
Common questions about Roche Holding AG / Genentech's rare disease programs, clinical trials, and treatments.