FDA Approval

New FDA approval: glucagon nasal powder for severe hypoglycemia

New FDA approval: glucagon nasal powder for severe hypoglycemia

Although uncommon, severe hypoglycemia can be devastating. It can occur in patients with type 1 diabetes or those with type 2 diabetes receiving insulin or sulfonylurea. If a patient experiences loss of consciousness or seizure from profound low sugars, glucagon needs to be administered immediately by a friend, family member, or caregiver.

The FDA has now approved a glucagon nasal powder as the second form of glucagon delivery for patients with severe hypoglycemia. In clinical trials, glucagon nasal spray has demonstrated similar efficacy as the injectable counterpart in increasing blood glucose concentrations.

The glucagon nasal powder is an excellent additional tool to combat severely low sugars in individuals with type 1 or type 2 diabetes. It has been approved for patients age four or older.

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New FDA approval: semaglutide, the first oral GLP-1 agonist for type 2 diabetes

New FDA approval: semaglutide, the first oral GLP-1 agonist for type 2 diabetes

The FDA has now approved the first oral semaglutide, Rybelsus, for the treatment of type 2 diabetes. This approval marks a breakthrough advancement in the field of clinical diabetology. Rybelsus is the first "protein" based molecule to be administered orally and not subcutaneously via an injection.

Oral administration of semaglutide is made possible through the use of SNAC compound. SNAC helps escort and transport the semaglutide intact across the gastrointestinal epithelial cells. It assists in bypassing the harsh acidic environment of the stomach.

Various clinical trials, under the name PIONEER, have consistently shown A1c improvements and weight loss benefits with Rybelsus – thus leading to this landmark FDA acceptance.

Rybelsus comes at three doses; 3, 7, and 14 milligrams. Patients should start at 3 mg per day for one month before advancing to the 7 mg, and if needed, to the 14 mg daily dosage. Rybelsus should be taken in an empty stomach, with no more than 4 ounces of plain water, and at least 30 minutes before breakfast.

Similar to other GLP-1 agonists, oral semaglutide can cause gastrointestinal side effects like nausea and diarrhea. Providers should be cautious when prescribing Rybelsus in those with a predisposed risk for pancreatitis, diabetic retinopathy, or kidney injury. It should not be prescribed in people with a personal or family history of medullary thyroid carcinoma.

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Vyleesi for hypoactive sexual desire disorder

Vyleesi for hypoactive sexual desire disorder

Vyleesi has been approved for generalized hypoactive sexual desire disorder (HSDD) in women younger than age 50. It is self-injected subcutaneously 45 minutes prior to the anticipated sexual intercourse. It should not be used more than 8 times per month and more than once daily. In a short-term randomized clinical trial, Vyleesi performed better than placebo. Although Vyleesi activates melanocortin receptors, its precise mechanism of action in HSDD is unknown.

Side effects include increased blood pressure, nausea, vomiting, headache, and darkening of the gums. It should be avoided in patients with uncontrolled hypertension or cardiovascular disease. This approval occurs in the context of FDA’s 2012 ruling of considering female sexual dysfunction as one of the top 20 high priority conditions. Its cost and health insurance coverage will determine its accessibility and usage.

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New FDA Approval: hydrogel capsule for weight loss

New FDA Approval: hydrogel capsule for weight loss

About 450 overweight or obese adults with BMI 27-40 kg/m2 were randomized to receive Gelesis100 or placebo. Subjects were followed for 6 months. At the end of the trial, patients receiving Gelesis100 lost a significant amount of weight compared to placebo group: about 60% and 25% of the adults lost ≥5% (≥10 lbs) and ≥10% (≥20 lbs) body weight.

Gelesis100 comes in a capsule form. It is taken with plenty of water twice daily before meals. The capsule contains particles, that in the presence of water, have the capability of expanding massively in the stomach, thus triggering a sense of fullness and decreased appetite.

The particles are not absorbed in the bloodstream. No serious adverse events were seen. Gastrointestinal upset was the most common side effect.

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New FDA Approval: romosozumab for osteoporosis

New FDA Approval: romosozumab for osteoporosis

Romosozumab , brand name Evenity, has now been approved by the FDA as a viable option for patients with advanced osteoporosis.

Romosozumab is a monoclonal antibody that specifically inhibits sclerostin. The approval comes with a major black box warning as the sclerostin inhibitor elevates the risk of heart attack, stroke and cardiovascular deaths.

Prior to Evenity initiation, patients need to be screened carefully for background cardiovascular disease. As the prevalence of both CVD and osteoporosis increases with age, the most likely candidates for such a therapy would be those at the highest risk for fragility or compression fractures.

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New FDA approval: oral testosterone

New FDA approval: oral testosterone

In March 2019 FDA approved the first oral testosterone supplementation for male patients with documented central (primary) or peripheral (secondary) hypogonadism. The medication is given twice a day orally. The recommended starting dose is about 250 mg every 12 hours. The minimum dose is about 150 mg BID with a maximum dose of 400 mg BID.

Follow up blood work needs to be done at least 7 days after initiation and 6 hours after the morning dose. Side effects may include elevated blood pressure, increased red blood cell mass, prostate enlargement, and GI upset. The approval comes with a black box warning for increased risk of hypertension and cardiovascular events.

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New FDA Approval: Subcutaneous Testosterone Pen

New FDA Approval: Subcutaneous Testosterone Pen

FDA has now approved the first subcutaneous testosterone auto-injector pen (Xyosted) for symptomatic patients with low testosterone levels. It is self-administered once weekly. It comes in three doses, 50 mg, 75, and 100 mg. Recommended starting dose is 75 mg per week. It can increase blood pressure, thus caution is advised in patients predisposed to hypertension and those at increased risk for cardiovascular events. Patients need to be monitor carefully after its initiation. Xyosted is not approved for women or males younger than age 18.

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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.

FDA

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 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. 

FDA

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.

GT

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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