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news.The US Food and Drug Administration (FDA) has granted fast track designation for GC Biopharma and Novel Pharma's GC1130A, a biological drug for the treatment of MPSIIIA (Sanfilippo syndrome Type A).
Sanfilippo syndrome Type A is a rare genetic disorder that leads to central nervous system damage due to the accumulation of heparan sulfate. Credit: Pete Linforth from Pixabay.
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This development follows the clearance for an investigational new drug (IND) application by the FDA for the asset last month.
The fast track status is expected to expedite the development and review of GC1130A significantly.
This designation also provides benefits including more frequent meetings with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval.
GC1130A is developed using advanced high-concentration protein formulation technology. It is administered directly into the brain’s ventricles via intracerebroventricular (ICV) injection.
This delivery method was first globally implemented by GC Biopharma in its Hunter syndrome treatment, ‘Hunterase’, which has been approved in Japan.
In 2023, the FDA awarded both rare pediatric disease and orphan drug disease (odd) designations to GC1130A.
The European Medicines Agency (EMA) also granted ODD status to the drug earlier this year.
GC Biopharma and Novel Pharma are preparing to launch a multinational first-in-human clinical trial to assess GC1130A’s safety and tolerability.
The trial is set to take place in Korea, the US, and Japan.
Sanfilippo syndrome Type A is a rare genetic disorder that leads to central nervous system damage due to the accumulation of heparan sulfate, causing progressive neurological decline.
In a press statement, a Nobel Pharma official said: “Following the FDA’s designation as an orphan drug (ODD) and orphan drug for children (RPDD), we have also received fast-track support, allowing us to further accelerate the development of the world’s first MPS IIIA treatment.
“Starting with this, we are making every effort to prepare for clinical trials in multiple countries, including Korea and Japan.”
