- Australian oncology-focused biotech company Kazia Therapeutics (KZA) has been awarded rare paediatric disease designation (RPDD) for paxalisib
- The designation comes from the U.S. Food and Drug Administration and follows positive preclinical data in the treatment of diffuse intrinsic pontine glioma (DIPG), a rare and highly-aggressive childhood brain cancer
- It allows Kazia to apply for a priority review voucher from the FDA which would shorten the review period for a new drug from twelve to six months
- Clinical efficacy data from the phase I clinical trial being conducted at St Jude Children’s Research Hospital in Memphis is expected later this year
- Following the news, Kazia Therapeutics shares jumped 57.1 per cent to close at 88 cents
Kazia Therapeutics (KZA) has obtained rare paediatric disease designation (RPDD) for paxalisib in treating a form of childhood brain cancer.
The RPDD comes from the U.S. Food and Drug Administration (FDA) and follows positive preclinical data in the treatment of an aggressive type of brain cancer in children, known as diffuse intrinsic pontine glioma (DIPG).
The Australian oncology-focused biotech company has been developing paxilisib, formally GDC-0084, for the treatment of glioblastoma, an aggressive form of primary brain cancer in adults.
Kazia Therapeutics' CEO Dr James Garner said the company has also increasingly focused on developing paxalisib to fill the gap in treating childhood brain cancer.
"For patients diagnosed with DIPG, there are currently no FDA-approved drug treatments, and the average survival from diagnosis is around 9.5 months. The granting of RPDD by the FDA recognises our efforts and achievements so far and leaves us well placed to move paxalisib forward as a potential therapy for DIPG," James said.
"We continue to be inspired by the dedication of our collaborators in this field and are committed to understanding whether paxalisib may be able to help in this enormously challenging paediatric disease," he added.
The RPDD initiative was established by the FDA to support the development of drugs for serious or life-threatening diseases affecting children, with less than 200,000 new cases recorded per year.
The designation means that Kazia can request a rare paediatric disease priority review voucher (PRV) during the marketing application for paxalisib, which would shorten the review period from 12 to six months.
An expedited review can offer significant economic value to a company. Alternatively, a PRV can be sold to another company and, in the past, these vouchers have fetched hundreds of million of dollars.
The phase I trial of paxalisib in DIPG at St Jude Children’s Research Hospital in Memphis has already reported top-line safety data and established the tolerated dose in children.
Initial efficacy data is also expected by the end of 2020 which will guide further development of the drug.
Following the news, Kazia Therapeutics shares jumped 57.1 per cent to close at 88 cents.