Biogen
Biogen works on 31 rare diseases tracked on Trial Friend, including Alport Syndrome, Amyotrophic Lateral Sclerosis, Angelman Syndrome and 28 more, with 34 recruiting clinical trials and 9 FDA-approved rare disease drugs.
Biogen is a neurobiological company with strong focus on rare neurodegenerative and neurological diseases including spinal muscular atrophy, Friedreich's ataxia, and ALS. The company develops disease-modifying therapies for rare inherited neurological conditions.
Focus areas at Biogen
Within its broader pharmaceutical portfolio, Biogen has active clinical trial programs and drug development efforts across 31 rare diseases, including Alport Syndrome, Amyotrophic Lateral Sclerosis, Angelman Syndrome, Dermatomyositis, Eosinophilic Granulomatosis with Polyangiitis, and 26 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 Biogen, 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.
Biogen is headquartered in Cambridge, United States, founded in 1978, publicly traded under the ticker symbol BIIB. 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.
Biogen Drug Pipeline
Biogen has 34 active clinical trials across 5 development stages, with 34 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 Biogen'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.
Biogen Clinical Trials (34)
Active and recruiting clinical trials sponsored by Biogen, 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.
Biogen FDA-Approved Drugs (9)
Medications developed or marketed by Biogen 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 |
|---|---|---|---|
| INTERFERON BETA-1A Interferon beta [EPC] | Avonex intramuscular | May 17, 1996 | |
| PEGINTERFERON BETA-1A Interferon beta [EPC] | Plegridy Pen subcutaneous, intramuscular | Aug 15, 2014 | |
| TOFERSEN Antisense Oligonucleotide [EPC] | QALSODY intrathecal | Apr 25, 2023 | |
| OMAVELOXOLONE | SKYCLARYS oral | Feb 28, 2023 | |
| NUSINERSEN Survival Motor Neuron-2-directed RNA Interaction [EPC] | Spinraza intrathecal | Dec 23, 2016 |
Biogen Trial Locations
Biogen clinical trials are running at 723 sites in 43 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 (31)
Diseases targeted by Biogen's clinical trial and drug development programs
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...
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...
Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints. It involves inflammation of muscles and skin blood vessels. Abo...
Eosinophilic granulomatosis with polyangiitis (EGPA, formerly Churg-Strauss syndrome) is a systemic necrotizing vasculitis characterized by asthma, blood eosinophilia, and inflammation of small- and m...
Inherited erythromelalgia is a rare autosomal dominant pain disorder caused by gain-of-function mutations in the SCN9A gene, which encodes the Nav1.7 voltage-gated sodium channel. Patients experience ...
Patient Resources
Organizations and resources related to Biogen's rare disease focus areas
Frequently Asked Questions About Biogen
Common questions about Biogen's rare disease programs, clinical trials, and treatments.