Azafaros B.V.
Rare disease drugmaker · based in Leiden, Netherlands · founded 2018 · azafaros.com
Azafaros B.V. works on 4 rare diseases we track, including Gaucher Disease, Niemann-Pick Disease and 2 more. It has 4 active clinical trials, and 2 are looking for volunteers now.
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.
Azafaros B.V. pipeline
Each row lists the diseases Azafaros B.V. is studying at that stage, from the ones closest to approval down to the earliest work.
Niemann-Pick Disease 2 trials recruiting · Gangliosidoses, GM2
Azafaros B.V. clinical trials
These are Azafaros B.V. trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials looking for volunteers now
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
Tests Nizubaglustat, Placebo
For Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
Tests AZ-3102, Placebo
For Niemann-Pick Type C Disease, GM1 Gangliosidosis and GM2 Gangliosidosis
2 trials underway, not taking new volunteers
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Tests AZ-3102
For GM2 Gangliosidosis and Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
Tests AZ-3102, Placebo
For Gangliosidoses, GM2 and Gangliosidosis, GM1
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More about 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 active and recruiting rare disease 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.
Where Azafaros B.V. trials take place
The trials above run at 103 study sites in 17 countries. Tap a country to see its states, cities and hospitals.
Rare diseases Azafaros B.V. works on
Each disease page lists every open trial, not only Azafaros B.V.'s, plus approved treatments.
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...
How common: 1 in 40,000 to 60,000 in general population; 1 in 850 among Ashkenazi Jewish population
Niemann-Pick disease is a rare lysosomal storage disorder where the body cannot properly break down cholesterol and other lipids within cells.
How common: Type A: 1 in 250,000; Type B: 1 in 100,000 to 150,000; Type C: 1 in 100,000 to 150,000
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...
How common: Approximately 1 per 100,000 to 1 per 150,000 live births; higher in certain populations
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.
How common: 1 in 30,000 births; 1 in 3,500 in Ashkenazi Jewish population
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Azafaros B.V.'s own site is azafaros.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- NIH GARD - Gaucher DiseaseGaucher Disease and Niemann-Pick Disease and 1 more
- Gaucher Disease Support GroupGaucher Disease
- National Niemann-Pick Disease FoundationNiemann-Pick Disease
- National Niemann-Pick Disease FoundationNiemann-Pick Disease Type C
- National Center for Advancing Translational Sciences (NCATS)Niemann-Pick Disease Type C
- National Tay-Sachs and Allied Diseases AssociationTay-Sachs Disease