Thermosome secures FDA Orphan Drug Designation for soft tissue sarcoma therapy
Ella Day | May 12, 2025 | News story | Research and Development | European Medicines Agency (EMA), FDA, Oncology, Thermosome
Thermosome has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its lead candidate, THE001, a treatment targeting soft tissue sarcomas. The designation, which complements a previous ODD from the European Medicines Agency, affirms the therapy’s clinical promise and strengthens Thermosome’s position in the US pharma market.
THE001 is a thermosensitive liposomal formulation of the chemotherapy drug doxorubicin, designed to release high concentrations of the drug at tumour sites when activated by mild heat. This targeted delivery aims to improve tumour penetration, overcome drug resistance and onset an additional immune response through regional hyperthermia. This presents potential advantages over conventional doxorubicin therapy.
A phase 1 clinical trial is currently underway at two German sites to assess the safety, tolerability and anti-tumour activity of THE001. Early results have been encouraging, with initial dose levels declared safe by an independent data safety board last year.
The FDA’s decision also enhances Thermosome’s commercial prospects by offering regulatory incentives such as tax credits and seven years of market exclusivity after approval. At the same time, the company has expanded its intellectual property portfolio with four new international patent applications, bringing its total to seven patent families.
“This recognition marks an important milestone and is a further step into the US market,” said Pascal Schweizer, CEO of Thermosome.
Ella Day
12/5/25
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