Move over Tolvaptan/Jynarque, there may be a new sheriff in town.
On 05 June 2019 Texas-based Reata Pharmaceutical received orphan drug status from the FDA for Bardoxolone Methyl (aka Bardoxolone).
So what does it mean if a drug is granted orphan status? It means that even though there are fewer than 200,000 people in the United States with the disease at any given time, the federal government has acknowledged the need for medical innovation to address the condition. Companies/drugs given this status receive certain development incentives such as tax credits for clinical testing, exemption from prescription drug user fees (used to fund new drug approval processes) and the ability to market the drug exclusively for seven years.
I guess that takes the sting out of having a limited market. It may also explain why drugs such as Tolvaptan (also with orphan drug status) are so flippin' expensive! It's hard to recoup an investment when your upside is limited.
So what's the difference between Tolvaptan and Bardoxolone?
From what I can tell, Tolvaptan slows the growth of kidney cysts while Bardoxolone reduces inflammation and increases the function of whatever kidney tissue remains after being displaced by cyst growth.
It seems like the two drugs might be complementary if drug interactions can be kept to a minimum (that's for the pharmacologists to determine).
The official statement from Reata is that "Bardoxolone is an experimental, oral, once-daily activator of Nrf2, a transcriptor that induces molecular pathways that promote restoration of mitochondrial function, reduction of oxidative stress, and inhibition of pro-inflammatory signaling."
Last year, Reata's FALCON Phase 2 study observed a mean eGFR INCREASE of 9.3 mL/min/1.73m2 (this represents a reversal of an average two year function decline). Additionally, 96% of the patients who took the drug for 12 weeks showed improvement. While promising, this trial was small - only 31 patients.
Based on the favorable results, Reata launched a much larger Phase 3 trial (300 ADPKD patients) called FALCON. This study also increases the length of treatment from 12 to 48 weeks.
Side effects appear to be limited to muscle cramps (think over-exercising) during initial weeks which decrease once patients reach their maintenance dose.
Hmmm... something to think about...
On 05 June 2019 Texas-based Reata Pharmaceutical received orphan drug status from the FDA for Bardoxolone Methyl (aka Bardoxolone).
So what does it mean if a drug is granted orphan status? It means that even though there are fewer than 200,000 people in the United States with the disease at any given time, the federal government has acknowledged the need for medical innovation to address the condition. Companies/drugs given this status receive certain development incentives such as tax credits for clinical testing, exemption from prescription drug user fees (used to fund new drug approval processes) and the ability to market the drug exclusively for seven years.
I guess that takes the sting out of having a limited market. It may also explain why drugs such as Tolvaptan (also with orphan drug status) are so flippin' expensive! It's hard to recoup an investment when your upside is limited.
So what's the difference between Tolvaptan and Bardoxolone?
From what I can tell, Tolvaptan slows the growth of kidney cysts while Bardoxolone reduces inflammation and increases the function of whatever kidney tissue remains after being displaced by cyst growth.
It seems like the two drugs might be complementary if drug interactions can be kept to a minimum (that's for the pharmacologists to determine).
The official statement from Reata is that "Bardoxolone is an experimental, oral, once-daily activator of Nrf2, a transcriptor that induces molecular pathways that promote restoration of mitochondrial function, reduction of oxidative stress, and inhibition of pro-inflammatory signaling."
Last year, Reata's FALCON Phase 2 study observed a mean eGFR INCREASE of 9.3 mL/min/1.73m2 (this represents a reversal of an average two year function decline). Additionally, 96% of the patients who took the drug for 12 weeks showed improvement. While promising, this trial was small - only 31 patients.
Based on the favorable results, Reata launched a much larger Phase 3 trial (300 ADPKD patients) called FALCON. This study also increases the length of treatment from 12 to 48 weeks.
Side effects appear to be limited to muscle cramps (think over-exercising) during initial weeks which decrease once patients reach their maintenance dose.
Hmmm... something to think about...
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