- Prescient Therapeutics (PTX) receives orphan drug designation for its PTX-100 product for the treatment of peripheral T-cell lymphomas (PTCL)
- Orphan drug designation is given to drugs that are intended to treat rare diseases, and it gives PTX seven years of market exclusivity for PTX-100, among other benefits
- PTCL is a blood cancer that has high unmet clinical needs, with current treatments characterised by a high occurrence of serious toxicities and a response rate of less than 30 per cent
- Prescient says so far, PTX-100 is showing a favourable safety profile, with one patient exhibiting a durable response and still on the therapy after 28 months
- Prescient is up 18.75 per cent with shares trading at 19 cents at 11:47 am AEST
Prescient Therapeutics (PTX) has received orphan drug designation for its PTX-100 product in the treatment of peripheral T-cell lymphomas (PTCL).
Orphan drug designation is given to drugs that are intended for the safe and effective treatment, prevention or diagnosis of rare diseases that affect less than 200,000 people in the United States.
Further, it grants seven years of market exclusivity and waives the prescription drug user fee act fees for orphan drugs.
PTCL is a blood cancer that has high unmet clinical needs, and survival following relapse is poor.
Current treatments are characterised by a high occurrence of serious toxicities, less than a 30 per cent response rate and a short duration of response.
Prescient said so far, PTX-100 was showing a favourable safety profile, with one patient exhibiting a durable response and still on the therapy after 28 months.
CEO and Managing Director Steven Yatomi-Clarke said the granting of orphan drug designation by the FDA was “significant” for the company’s development of PTX-100.
“Orphan drugs often enjoy shorter and cheaper development pathways,” Mr Yatomi-Clarke said.
“Additionally, the company now has the certainty of seven years of market exclusivity in the event of regulatory approval of PTX-100 for PTCL.”
Prescient was up 18.75 per cent with shares trading at 19 cents at 11:47 am AEST.