Curatis will apply for a Biologic License for C-PTBE-01 in the USA - this will significantly increase the duration of market protection in the USA in the event of approval
14.11.2024 07:00:00 CET | Business Wire | Press release
Curatis Holding (SIX:CURN) has explored in detail which regulatory pathway should be pursued in view of a possible approval of C-PBTE-01 in the USA. The detailed clarifications in collaboration with a renowned US law firm and their regulatory experts have shown that C-PTBE-01 meets the criteria for a Biologic License Application (BLA). If approved in the USA, this would result in a market protection of 12 years, significantly longer than the 7 years market protection under orphan drug status. This would extend the market protection for C-PTBE-01 in the USA by 70% or 5 years.
According to the US Public Health Service Act, the status of “biologic” is intended for peptides with a length of more than 40 amino acids. The molecule C-PTBE-01 has a length of 41 amino acids. The orphan drug designation for C-PTBE-01 will be pursued as planned.
About C-PTBE-01
Curatis is focusing its development activities for C-PTBE-01 on an very rare group of aggressive brain tumours (Diffuse Midline Glioma, DMG). These tumours mainly affect children, with most cases being diagnosed between the ages of 5 and 9. In the USA, around 800 patients are diagnosed with DMG every year; in Europe, the number is in the same order of magnitude, which is why the disease is considered a ‘rare disease’ for regulatory purposes.
In connection with DMG, indirect brain damage regularly occurs due to an accumulation of extracellular fluid in the vicinity of the tumour. This peritumoural brain edema (PTBE) can cause symptoms such as headaches, vomiting and neurological dysfunction such as paralysis, speech disorders, visual problems and altered mental status and can be life-threatening.
The current typical treatment method for PTBE is the use of corticosteroids. Corticosteroids often have serious side effects such as severe myopathies, muscle wasting, abnormal weight gain, osteoporosis, gastritis, gastrointestinal bleeding, hypertension and personality changes. The already serious side effects are exacerbated in children.
C-PTBE-01 has demonstrated a strong steroid-sparing effect in two clinical safety and efficacy studies, which may lead to a significant reduction or complete replacement of corticosteroid use and thus alleviation of the severe side effects associated with steroid use in children.
A detailed analysis of Curatis and its product candidates is available in a research report at https://ir.curatis.com/equity-research-reports.
About Curatis:
Curatis Holding AG is a publicly listed company (CURN.SW) specialising in the development and commercialisation of drugs for rare and very rare diseases. Curatis has a sales portfolio of more than 30 drugs and a pipeline of orphan drug products and speciality products that can make a significant contribution to cash flow from 2025 onwards. Further information can be found on the website www.curatis.com.
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