Curatis on track: submission of orphan drug designation application in the US for lead product candidate
LIESTAL, Switzerland, August 27, 2024--(BUSINESS WIRE)--Curatis Holding (SIX:CURN) has submitted an orphan drug designation application to the US Food and Drug Administration (FDA) for its lead product candidate C-PBTE-01. The product is intended to alleviate the great suffering of children with side effects from a specific brain tumour.
Curatis is focusing its development activities for C-PTBE-01 on an extremely 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.
The FDA grants Orphan Drug Designation (ODD) to products that treat rare diseases, incentivising companies to develop drugs or biologics. The FDA defines rare diseases as those that affect fewer than 200,000 people in the United States at any given time. Through orphan drug designation, C-PBTE-01 would be eligible for certain benefits and incentives, including seven years of market exclusivity if regulatory approval is ultimately granted for the designated indication, potential tax credits for certain activities, eligibility for orphan drug grants and waiver of certain administrative fees. The FDA's decision regarding ODD of C-PBTE-01 is expected within 3 months.
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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