FSGS can have many different causes, and can be classed as either:
- Primary FSGS: This type of FSGS means that the disease happened spontaneously without a known or obvious cause.
- Secondary FSGS: This type is caused by another disease or chronic drug use. Examples include: viruses such as HIV or drugs such as anabolic steroids that some people use to speed up their muscle growth
FSGS affects at least 210,000 people globally and is a disease for which there is a recognised medical need for a new or improved treatment.1 The disease progresses rapidly and results in kidney failure, requiring dialysis or kidney transplant. A kidney transplant costs in the region of $260,000 per patient, with ongoing and expensive anti-rejection drugs also costing thousands of dollars per year, and dialysis costs in the region of $100,000 per patient per year and requires regular visits, totaling over 12 hours per week to the medical facility. This is a huge burden on both the patient and the healthcare system.
DMX-200 for FSGS is currently in a Phase 2 clinical trial and has been granted Orphan Drug Designation by the FDA and EMA. Orphan Drug Designation is granted by the FDA and EMA in order to support the development of products for rare diseases. Orphan drug designation qualifies the sponsor of the drug for various development incentives by the FDA and EMA, including: seven years and ten years respectively of market exclusivity if regulatory approval is received, exemption from certain application fees, and an abbreviated regulatory pathway to approval.