Azafaros B.V.
Azafaros B.V. works on 4 rare diseases tracked on Trial Friend, including Gaucher Disease, Niemann-Pick Disease, Niemann-Pick Disease Type C and 1 more, with 4 recruiting clinical trials.
Azafaros is a clinical-stage biotechnology company developing brain-penetrant, oral small-molecule therapies for rare lysosomal storage disorders. The company was spun out from research at Leiden University and Amsterdam UMC. Lead program nizubaglustat targets both Niemann-Pick disease type C and GM1/GM2 gangliosidoses, with positive Phase 2 topline data reported.
Focus areas at Azafaros B.V.
As a rare disease specialist, Azafaros B.V. has active clinical trial programs and drug development efforts across 4 rare diseases, including Gaucher Disease, Niemann-Pick Disease, Niemann-Pick Disease Type C, Tay-Sachs Disease. 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 Azafaros B.V., 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.
Azafaros B.V. is headquartered in Leiden, Netherlands, founded in 2018. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Azafaros B.V. Drug Pipeline
Azafaros B.V. has 4 active clinical trials across 2 development stages, with 4 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 Azafaros B.V.'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.
Azafaros B.V. Clinical Trials (4)
Active and recruiting clinical trials sponsored by Azafaros B.V., 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.
FDA-Approved Drugs
Azafaros B.V. Trial Locations
Azafaros B.V. clinical trials are running at 103 sites in 17 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 (4)
Diseases targeted by Azafaros B.V.'s clinical trial and drug development programs
Gaucher disease is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme glucocerebrosidase, resulting in accumulation of fatty substances in the spleen, liver, and bone marro...
Niemann-Pick disease is a rare lysosomal storage disorder where the body cannot properly break down cholesterol and other lipids within cells. These fatty substances accumulate in the liver, spleen, b...
Niemann-Pick Disease Type C (NPC) is a rare, progressive lysosomal lipid storage disorder caused by mutations in the NPC1 or NPC2 genes, which encode proteins critical for cholesterol and lipid traffi...
Tay-Sachs disease is a rare autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme hexosaminidase A, leading to accumulation of GM2 gangliosides in nerve cells. This progres...
Patient Resources
Organizations and resources related to Azafaros B.V.'s rare disease focus areas
Frequently Asked Questions About Azafaros B.V.
Common questions about Azafaros B.V.'s rare disease programs, clinical trials, and treatments.