US FDA Grants Rare Pediatric Disease Designation (RPDD) to Paxalisib for DIPG
Key Points
With RPDD granted, Kazia may now be eligible to receive a 'rare pediatric disease priority review voucher' (PRV) if paxalisib is approved for DIPG
A PRV grants the holder an expedited six-month review of a new drug application by FDA.
PRVs can be sold to other companies and have historically commanded prices between US$68 million and US$350 million
RPDD has been awarded following positive emerging preclinical data in DIPG, and with initial clinical efficacy data expected in 2H CY2020; positive clinical data may substantially enhance likelihood of a potential future PRV
The FDA's RPDD program is intended to advance the development of drugs and biologics for certain serious and life-threatening rare pediatric diseases by providing incentives to industry. Most significant among these incentives is the potential access a priority review voucher at the time of a marketing authorization for the rare paediatric disease.
RPDD may be granted to drugs in development for diseases which primarily affect children (under the age of 18 years), have an incidence of less than 200,000 new cases per annum in the United States, and which are serious or life-threatening.
In effect, the PRV shortens the FDA review period for a future marketing application of any drug from 12 months to 6 months.
finance.yahoo.c...-143000744.html?guce_refer...
Key Points
With RPDD granted, Kazia may now be eligible to receive a 'rare pediatric disease priority review voucher' (PRV) if paxalisib is approved for DIPG
A PRV grants the holder an expedited six-month review of a new drug application by FDA.
PRVs can be sold to other companies and have historically commanded prices between US$68 million and US$350 million
RPDD has been awarded following positive emerging preclinical data in DIPG, and with initial clinical efficacy data expected in 2H CY2020; positive clinical data may substantially enhance likelihood of a potential future PRV
The FDA's RPDD program is intended to advance the development of drugs and biologics for certain serious and life-threatening rare pediatric diseases by providing incentives to industry. Most significant among these incentives is the potential access a priority review voucher at the time of a marketing authorization for the rare paediatric disease.
RPDD may be granted to drugs in development for diseases which primarily affect children (under the age of 18 years), have an incidence of less than 200,000 new cases per annum in the United States, and which are serious or life-threatening.
In effect, the PRV shortens the FDA review period for a future marketing application of any drug from 12 months to 6 months.
finance.yahoo.c...-143000744.html?guce_refer...