IQ-AI Ltd (LSE:IQAI, OTCQB:IQAIF)'s subsidiary, Imaging Biometrics, has secured orphan drug designation from the US Food and Drug Administration (FDA) for gallium maltolate (GaM) for treating atypical teratoid rhabdoid tumours.
This marks the second time this year that GaM has received orphan designation from the FDA.
An atypical teratoid rhabdoid tumour (ATRT) is a rare and aggressive form of cancer that primarily affects the central nervous system, including the brain and spinal cord.
This type of tumour is most commonly diagnosed in young children, although it can occur at any age.
ATRT is characterised by its rapid growth and propensity to spread, making it particularly challenging to treat effectively.
Due to its aggressive nature and the current lack of highly effective treatments, the prognosis for patients with ATRT is generally poor.
As IQ-AI points out, currently, patients diagnosed with ATRT face a bleak prognosis.
So, the FDA's decision to grant ODD status to GaM signifies a potential breakthrough in addressing this unmet medical need.
"This additional orphan drug determination by the FDA is another regulatory milestone towards providing an effective, well-tolerated treatment alternative for patients with limited options, and we look forward to sharing additional information on our clinical data as the phase I nears completion," said Trevor Brown, CEO of IQ-AI.
Securing orphan drug designation from regulatory agencies such as the FDA offers a number of advantages to treatment developers and, ultimately, to patients:
- Financial Incentives: Companies receive various financial benefits, including tax credits for clinical research costs, grants for drug development, and potential fee waivers, making the development process more economically viable.
- Market Exclusivity: Upon approval, the drug enjoys a period of market exclusivity, typically seven years in the United States, during which competitors cannot market a generic version. This exclusivity can be a significant financial advantage.
- Regulatory Support: Companies often receive more extensive guidance and support from regulatory agencies during the drug development process, which can expedite the time to market.
- Lower Regulatory Hurdles: The criteria for clinical trial success may be more flexible for orphan drugs, given the rarity and severity of the conditions they treat. This can make it easier to demonstrate efficacy and safety.
- Access to Funding: ODD can make a drug candidate more attractive to investors, as the designation often implies a faster, less expensive, and less risky development pathway.