FDA Approval

New FDA approval: retacrit, to improve anemia of kidney disease

New FDA approval: retacrit, to improve anemia of kidney disease

Erythropoietin is a key hormone produced by the kidney to stimulate bone marrow production of red blood cells. FDA has now approved the second erythropoietin analog, retacrit, in addition to epogen. It shows similar efficacy and side effects to epogen. This approval allows both medications to be more accessible to patients, at a lower cost. It is indicated for treatment of anemia of chronic kidney disease or from use of chemotherapy and zidovudine. It could also be used preemptively prior to surgeries with expected major blood loss.

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New FDA approval: drug therapy for anaplastic thyroid cancer

New FDA approval: drug therapy for anaplastic thyroid cancer

Anaplastic thyroid cancer (ATC) is a rare and aggressive type of thyroid cancer. It accounts for about 1-2% of all thyroid cancers. NIH estimates 54,000 new cases of thyroid cancer and 2,000 deaths from the disease in the US in 2018. 

The tafinlar + mekinist combination is now approved for the treatment of BRAF V600E mutation-positive ATC that cannot be addressed surgically or has already spread to other organs. Tafinlar and Mekinist have also been approved for two other BRAF V600 mutation-positive metastatic malignancies; melanoma and non-small cell lung cancer.

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New FDA approval: a more convenient test for adult growth hormone deficiency

New FDA approval: a more convenient test for adult growth hormone deficiency

FDA has approved the oral agent macrilen for evaluation of growth hormone deficiency in adults. Macrilen is a ghrelin agonist which leads to the provocative release of growth hormone by the anterior pituitary gland. The diagnostic procedure is safe, efficient and comparable with the gold standard isulin tolerance test (ITT). This is a meaningful advancement in the field of endocrinology.

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New FDA approval: artificial intelligence for diabetic eye disease.

New FDA approval: artificial intelligence for diabetic eye disease.

Diabetic retinopathy occurs when high levels of blood sugar lead to damage in the blood vessels of the retina, the light-sensitive tissue in the back of the eye. Diabetic retinopathy is the most common cause of vision loss among the more than 30 million Americans living with diabetes and the leading cause of vision impairment and blindness among working-age adults.

FDA

New FDA approval: the first drug for x-linked hypophosphatemia

New FDA approval: the first drug for x-linked hypophosphatemia

Crysvita or burosumab is the first drug to be approved by FDA for treatment of x-linked hypophosphatemia in adults and children older than one year of age. It is inherited, rare and unresponsive to vitamin D supplementation. It is a form of ricket and osteomalacia leading to low blood phosphorus levels.  Clinical manifestations are disabled bone growth and development in children and impaired bone mineralization in adults.

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New FDA approval: Dexcom G6, an integrated continuous glucose monitor

New FDA approval: Dexcom G6, an integrated continuous glucose monitor

The Dexcom G6 is a patch device, about the size of a quarter, that is applied to the skin of the abdomen and contains a small sensor that continuously measures the amount of glucose in body fluid.

The device transmits real-time glucose readings every five minutes to a compatible display device such as a mobile medical app on a cell phone. The patch device should be replaced every 10 days.

The Dexcom G6 version is factory calibrated and does not require users to calibrate the sensor with fingerstick blood glucose measurements. In addition, it has an updated sensor probe that minimizes interference with the pain reliever acetaminophen.

FDA

New FDA approval: intrarosa helps with pain during sex

New FDA approval: intrarosa helps with pain during sex

DHEA is the main testosterone-like substance produced by the adrenal glands. Naturally it declines with age. Once a day vaginal insert of DHEA (intrarosa or prasterone) helps postmenopausal women experience less pain during intercourse. Side effects include abnormal pap smear and vaginal discharge. Currently there is no evidence that over-the-counter oral DHEA supplementation helps with such symptoms.

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New FDA approval: daily SGLT-2 inhibitor for type 2 diabetes

New FDA approval: daily SGLT-2 inhibitor for type 2 diabetes

Steglatro (ertugliflozin) is a sodium glucose co-transporter 2 inhibitor. SGLT-2 is the predominant transporter responsible for reabsorption of glucose from the glomerular filtrate back into the circulation. By inhibiting SGLT2, ertugliflozin reduces renal reabsorption of filtered glucose and lowers the renal threshold for glucose, and thereby increases urinary glucose excretion.

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New FDA approval: once a week GLP-1 agonist for type 2 diabetes

New FDA approval: once a week GLP-1 agonist for type 2 diabetes

The FDA approval of Ozempic was based on the results from a global Phase IIIa clinical trial comprised eight clinical trials involving more than 8,000 adults with type II diabetes, including a two-year cardiovascular outcomes trial that evaluated safety in adults with type II diabetes at high risk of cardiovascular events.

Ozempic showed clinically meaningful and statistically significant reductions in A1c compared with placebo, sitagliptin and exenatide extended-release. As a secondary endpoint in the trials, treatment with Ozempic resulted in reductions in body weight. 

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New FDA approval: no more fingersticks for adults with diabetes

New FDA approval: no more fingersticks for adults with diabetes

FDA approved FreeStyle Libre Flash Glucose Monitoring System in late September 2017. It allows patients to get instantaneous glucose measurements without finger-sticks. The sensor is small, placed subcutaneously and lasts up to 10 days. No more painful pricks, blood, lancets, strips, or alcohol swipes.

FreeStyle Libre Flash CGM has not been indicated for the pediatric population. It also does not have the capability of providing real-time alerts for severely low or high blood sugars.

Overall this is a great technological advancement for patients with diabetes. I anticipate widespread use of the device.

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New FDA approval: Tymlos (abaloparatide) for Osteoporosis

New FDA approval: Tymlos (abaloparatide) for Osteoporosis

In April 2017, FDA approved Tymlos/AbaloParatide for treatment of postmenopausal osteoporosis. Tymlos has potent bone anabolic properties (PTH1 receptors). It is administered once a day subcutaneously for up to 2 years. Side effect could be nausea and palpitations. The original study published in JAMA June 2016 suggests overall tymlos superiority over forteo in fracture reduction and bone density improvement during an 18 month observation.

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New FDA approval: parsabiv for hyperparathyroidism

New FDA approval: parsabiv for hyperparathyroidism

In February 2017, FDA approved Parsabiv intravenous infusion for treatment of hyperparathyroidism in patients undergoing hemodialysis.

End stage kidney disease can cause severe PTH rise, leading to worsening of hyperphosphatemia, higher bone turnover (ex, osteitis fibrosa cystica) and eventually renal osteodystrophy. Secondary kidney-related hyperparathyroidism can increase mortality and morbidity in affected patients.

Parsabiv is supposed to break the cycle of hyperphosphatemia by augmenting the action of calcium thus reducing secretion of PTH by parathyroid chief cells. Results are impressive.

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