From Yahoo Finance
Reata's (RETA) Kidney Candidate Gets Orphan Drug Status
Reata Pharmaceuticals, Inc. RETA announced that the FDA has granted an orphan drug designation to its pipeline candidate, bardoxolone methyl (bardoxolone), for the treatment of autosomal dominant polycystic kidney disease (ADPKD), an inherited form of kidney disease.
Notably, the orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. The designation also makes the company entitled to certain other benefits including tax credits related to clinical trial expenses and exemption from the FDA user fee. The status also makes bardoxolone eligible for seven years of marketing exclusivity in the United States.
Shares of Reata Pharmaceuticals have soared 56.6% so far this year, outperforming the industry’s increase of 1%.
Last year, the company announced encouraging data from the phase II PHOENIX study on bardoxolone in the ADPKD cohort. Notably, the phase II study is evaluating bardoxolone for four rare forms of chronic kidney disease (CKD). Patients treated with bardoxolone for 12 weeks, experienced a mean increase from baseline in estimated glomerular filtration rate (eGFR) of 9.3 mL/min/1.73 m2, a measure of the kidney function. Moreover, 96% of the subjects showed an improvement in eGFR after a 12-week regimen.
Following positive results from the PHOENIX program, Reata Pharmaceuticals started the phase III FALCON study for treating patients with ADPKD. Last week, the company enrolled the first patient for the evaluation. The primary efficacy endpoint of this study is to see the change from baseline in eGFR as compared to placebo after 48 weeks of treatment followed by a 4-week drug withdrawal period.
ADPKD is a common form of inherited CKD, caused by mutations in PKD1 and PKD2 genes. Despite the standard of care treatment, it often causes end-stage kidney disease and almost 50% patients require dialysis or kidney transplant by the age of 60. Notably, this is the third orphan drug designation for bardoxolone to address kidney ailment distinguished by mitochondrial dysfunction and inflammation and the second designation for the treatment of patients with rare forms of CKD.
On approval, bardoxolone may provide an important new treatment option for ADPKD patients.
Dialysis
From KPLR, Channel 11, St Louis, MO
SSM Health Medical Minute: Breakthrough procedure for patients with kidney disease
ST. LOUIS - More than 460,000 people in the U.S. are living with End-Stage Renal Disease, the most severe form of chronic kidney disease. A brand-new option is now available for patients suffering from kidney disease. It’s the first innovative breakthrough for patients who require dialysis treatment in 50 years.
SSM Health St. Mary’s Hospital is the first hospital in the Midwest to offer a new, minimally invasive procedure to establish vascular access for patients suffering from kidney disease who require dialysis treatment. The procedure, known as WavelinQ™ (Wave-link) is the first innovative breakthrough in creating arteriovenous fistula (AVF), rather than a surgical fistula.
WavelinQ™ has reduced recovery time in half, allowing patients to go home the same day and start dialysis treatment sooner. It has also reduced infection rates and lowered the risk for painful inflammation.
Dr. Alejandro Alvarez, who helped to pioneer this new procedure and offers one of two training sites in the country at SSM Health St. Mary’s Hospital. “It’s going to have a big impact on our patients by reducing the risk of catheters, infection and even future procedures, as well as increasing recovery time so they can begin dialysis treatment faster.”
The new, minimally invasive WavelinQ™ procedure offers the same benefits as surgery as well as lower infection rates, plus lower the cost of delivery, without the painful inflammation and long recovery. In most cases, patients are able to go home and return to normal activity the next day. Furthermore, the time from fistula creation to first use for dialysis treatment is usually six weeks or less.
Surgical fistula creation has been the preferred technique for these patients. However, the surgery causes painful swelling that can last for weeks. Additionally, it increases the chances of a hospital stay, and the national average from creation to first use is 12 weeks.
“Our patients go through so much – they are tied to a dialysis machine three days a week – they deserve an alternative, more advanced option,” says Dr. Alvarez, MD. “As physicians and leaders in the community, it is our job to try to bring access to these kinds of procedures to our patients and to continue learning and innovating. We are proud to be one of the first hospitals in the area to offer the WavelinQ™ procedure.”
For more information about WavelinQ, click here.
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