We deal with the challenges, so you can focus on the opportunities
Not only due to a limited number of patients and qualified study sites, orphan disorders require unique approaches in study conduct. Knowing the exceptional challenges, we handle these projects with profound understanding of Orphan Drug Development.
Our core competencies:
- Thorough protocol design taking into consideration a potential lack of literature
- Searching and choosing motivated and experienced sites
- Efficient patient recruitment
- Acting as sponsor for EU Orphan Drug Designations
Therapeutic approaches include:
- Cystic fibrosis
- Systemic lupus erythematosus (SLE)
- Limbal stem cell deficiency (LSCD)
- Glioblastoma
- Neuroblastoma
- Hepatocellular carcinoma
- Fabry disease
- Primary progressive aphasia
- Haemophilia B
- Head and neck carcinoma
- Huntington’s disease
- Advanced renal cell carcinoma
- Malignant ascites
- Primary sclerosing cholangitis
- Acute-on-chronic liver failure (ACLF)
- Multiple myeloma
- Eosinophilic esophagitis
- Ehlers-Danlos syndrome
- Indolent systemic mastocytosis
- Granulomatosis with polyangiitis (Wegener`s)
- Acute myeloid leukemia
- Friedreich’s ataxia
- Schnitzler’s syndrome
- Epidermolysis bullosa (EB)
Pediatric Studies in Orphan Diseases
- Neuroblastoma
- Limbal stem cell deficiency (LSCD)
- Systemic lupus erythematosus (SLE)
- Childhood cerebral adrenoleukodystrophy (CCALD)
- Arginase 1 Deficiency
Prof. Michael Hudecek, University Hospital Wuerzburg
Chair of Cellular Immunotherapy
Since 2018, FGK has been an invaluable partner in our clinical early-phase CAR-T cell projects. Their extensive experience in advanced new therapies, like ATMP and cell therapy projects, has provided us with the expertise and support needed to navigate this complex field. Their commitment to high-quality research and their deep understanding of innovative therapies make them a trusted and essential partner in our development efforts. We look forward to continuing this successful collaboration.
Kristin orr, Q32 Bio
Senior Director Clinical Operations
I worked with the FGK team on Phase I study which went exceptionally well. Excellent communication at all levels and the collaboration between all parties was highly effective. FGK played a key role in the successful execution of the trial through their reliability, professionalism, and strong teamwork.
If you’re interested in our broad experience in overcoming the hurdles of rare disease trials, get in touch with us.
