Kazia Therapeutics Limited [NASDAQ:KZIA] was one of the top performers on Friday, closing the day with gains of 49%. This followed the company’s announcement that the United States Food and Drug Administration (FDA) had given a Rare Pediatric Disease Designation for its treatment called Paxalisib, in the treatment of rare and aggressive childhood brain cancer. Some of the key highlights of that came with this announcement are as below:
First, the company announced that now that it has been granted the RPDD, it was in a position to get the rare pediatric disease review voucher. This will happen if its treatment (Paxalisib) is approved for treating DIPG. The company further announced that if it gets the PRV grants, it would be in a position to fast-track a new drug application from the Food and Drug Administration.
The company added that with such approval, it could make money by selling PRVs to other pharmaceutical corporations, as such sales go for $68 million to $350 million in the markets. The company also noted that RPDD had been given after positive pre-clinical data on DIPG and that initial clinical data on the treatment was expected within the second half of 2020. Positive data may help improve the chances of a PRV in the future.
Commenting on the development, CEO Dr. James Garner stated that glioblastoma remained the company’s top priority for Paxalisib. However, the company was also increasingly focused on developing it as a treatment for aggressive brain cancer in children. He added that there currently are no treatments approved by the FDA for the treatment of DIPD and that survival rate from the time of diagnosis was around 9.5 months. In essence, by being granted the RPDD, the FDA had recognized the company’s efforts, and this positioned it well to push further in the development of Paxalisib as a treatment for DIPG. He further stated that the company continues to work with its partners in a bid to better understand Paxalisib as a treatment option for people suffering from pediatric illness.
Going forward, a breakthrough in the treatment of childhood brain cancer could see the value of this stock rocket in the near-term. That’s because it would be a hugely positive addition to the company’s bottom line.
However, even as the company continues with technical developments, it stands to benefit immensely short-term from the announcement it made. That’s because the market could be headed for a new wave of gains after President signed an executive order on the economy. Stocks that already have bullish momentum building could record even higher gains.
About Kazia Therapeutics
Kazia Therapeutics is a biotech firm focused on the development of cancer treatments. It is based in Sidney, Australia.