Treatment of glioma The FDA grants pan-PI3K inhibitor paxalisib fast-track eligibility.

In addition, over the weekend, the FDA granted paxalisib the status of orphan drugs (ODDs), including DIPG, for the treatment of malignant gliomas.
month, the FDA also granted paxalisib the status of treating DIPG for rare pediatric diseases (RPDD).
DIPG is a rare and highly invasive malignant tumor in children, which lacks effective treatment and has a very high fatality rate.
currently, an Phase I study of paxalisib's treatment of DIPG is being conducted at St Jude Children's Research Hospital, with preliminary efficacy data expected in the second half of 2020.
Paxalisib is Kazia's lead drug, a small molecular inhibitor of the PI3K/AKT/mTOR path through the blood-brain barrier, which was approved from Roche's Genentek at the end of 2016 and entered Phase II clinical trials in 2018.
glioblastoma is the most common and invasive type of primary brain cancer.
the target patients of this FTD accurately reflect the patient population studied in paxalisib's ongoing Phase II clinical study, is the leading recommended group for the key GBM AGILE study, and is also the expected adaptive disorder at the time of commercial release.
the United States, FTD was established to accelerate drug development and rapid review of serious diseases to address critically unsolved clinical needs in key areas.
paxalisib's access to FTD means an opportunity to accelerate the review process in various forms, including more frequent meetings and communication with the FDA during the development phase, a rolling review mechanism that allows for the phased submission of new drug application (NDA) filings without having to wait for all the information to be completed, and the granting of FTDs is expected to further qualify for priority review and accelerated approval.
FDA granted paxalisib FTD, which means the agency has recognized the drug's potential to significantly improve prognosis in patients with glioblastoma, which is a very strong recognition.
Kazia has plans to start preparations for the new drug application (NDA) for paxalisib in fiscal 2021.
The opportunities created by FTD are of tremendous value and have the potential to significantly accelerate the commercialization of paxalisib as we move forward with our submission to the NDA," said Dr. James Garner, CEO of Kazia, in response to the FDA's award of paxalisib to treat glioblastoma.
, in particular, the "rolling review" process allows Kazia to complete and submit most of our NDA applications ahead of schedule, saving time and reducing product risk.
look forward to working closely with the FDA as paxalisib enters the final phase of research and development.
", in response to the FDA's award of PAxalisib for the treatment of DIPG, Dr James Garner said: "Combined, RPDD and ODD provide a powerful set of incentives, opportunities and protections for the development of paxalisib in DIPG.
looking forward to seeing preliminary data on the DIPG Phase I study conducted at St Jude Children's Research Hospital in the second half of this year.
, we are working closely with our partners, consultants and researchers to determine the best way to treat DIPG, a devastating disease.
ODD qualification is the result of a regulatory optimization program that Kazia has launched for paxalisib over the past six months.
, we are moving toward the goal of commercializing paxalisib, and these FDA special qualifications will enable us to move forward in the fastest and most effective way."
early April, Kazia released positive interim data from the ongoing evaluation of paxalisib's treatment of glioblastoma (GBM) Phase II study (NCT03522298).
The study, conducted in newly diagnosed GBM patients with non-methylated MGMT initiation status, is evaluating the safety, toerability, recommended stage II dose (RP2D), pharmacodynamics (PK) and clinical activity of paxalisib as an auxiliary therapeutic drug after receiving maximum surgical excision and combined simultaneous chemotherapy of temozolomide (TMZ).
TMZ is currently the standard treatment for GBM.
results showed that (1) the medium total survival (OS) of paxalisib-assisted therapy was 17.7 months, representing an extended clinically significant life compared to the 12.7 months associated with existing standard care TMZ.
(2) the medium non-progressive lifetime (PFS) of paxalisib-assisted therapy was 8.5 months, representing a favourable outcome compared to the 5.3 months associated with existing standard care TMZ.
(3) who received the longest treatment remained disease-free 19 months after diagnosis.
(4) of the patients in the group are still receiving paxalisib treatment, and OS and PFS data are likely to improve further as research continues.
data from the study are expected to be released in the second half of fiscal 2020 and final data are expected in the first half of fiscal 2021.
"gold standard" is the ability to prolong life for any new cancer drug - a particularly challenging goal in diseases such as glioblastoma (GBM).
new data provide the first clinical evidence that paxalisib has the potential to achieve this goal in a very challenging patient population.
more than two decades, newly diagnosed glioblastoma patients have not had any new medications.
paxalisib is fast becoming one of the most promising drug candidates in the global pipeline of this challenging disease.
original origin: US FDA Awards Fast Track Designation (FTD) to Paxalisib for Glioblastoma Original title: Glioma New Drug! FDA grants pan-PI3K inhibitor paxalisib fast-track qualification, first-line efficacy beyond tymoamine (TMZ)