MAESTRO: First Treatment for NASH

Rezdiffra (Resmetirom), an oral medication targeting the liver and selective for thyroid hormone receptor β, demonstrated promising results. At doses of 80mg and 100mg, it achieved NASH resolution in 25% and 30% of participants, respectively, compared to just 10% in the placebo group.

Leqvio – Revolutionary Cholesterol Medication

Atherosclerotic cardiovascular disease (ASCVD) is the leading cause of death in the United States and worldwide. In the US alone, a person dies from cardiovascular-related causes every 33 seconds. Therefore, it is crucial to develop new therapies that aim to reduce the risk of cardiovascular disease.

Novel Drug for Hot Flashes

Menopause is a natural change that occurs in a woman’s life when her ovaries stop producing estrogen and progesterone. Typically, this happens between the ages of 45 and 55. Menopause is generally defined as not having a period for 12 consecutive months. Hot flashes, which affect 80% of women, are one of the most common symptoms of menopause and can significantly disrupt a woman’s life.

FIDELIO: Type 2 Diabetes & CKD

Kerendia (Finerenone) is an oral non-steroidal mineralocorticoid receptor (MR) antagonist. Excessive activation of mineralocorticoid receptors can result in hypertension, fluid retention, and cardiovascular harm, all of which have been directly linked to the deterioration of kidney function.

ORION: Gene Silencing, PCSK9 & LDL Cholesterol

Leqvio (Inclisiran) has received FDA approval as an additional therapy to diet and maximally tolerated statin medications for adults diagnosed with either heterozygous familial hypercholesterolemia (HeFH) or severe ASCVD who require further reduction of LDL cholesterol.

Possible Treatments for Covid-19

The FDA has been working closely with other government agencies and academic centers that are investigating the use of the drug CHLOROQUINE, which is already approved for treating malaria, lupus and rheumatoid arthritis, to determine whether it can be used to treat patients with mild-to-moderate COVID-19 to potentially reduce the duration of symptoms, as well as viral shedding, which can help prevent the spread of disease. Studies are underway to determine the efficacy of using chloroquine to treat COVID-19.

FDA

Point of care testing for Covid-19

The test we’re authorizing today will be able to provide Americans with results within hours, rather than days like the existing tests, and the company plans to roll it out by March 30, which is an incredibly rapid timeline for such an effort. With new tools like point-of-care diagnostics, we are moving into a new phase of testing, where tests will be much more easily accessible to Americans who need them.

With the development of point of care diagnostics, Americans who need tests will be able to get results faster than ever before. More and more options for reliable, convenient testing are becoming available at an incredibly rapid pace, thanks to the hard work of our FDA team and the ingenuity of the American industry.

Today marks an important step in expanding the availability of testing and, importantly, rapid results. Point-of-care testing means that results are delivered to patients in the patient care settings, like hospitals, urgent care centers, and emergency rooms, instead of samples being sent to a laboratory. With today’s authorization, there is now an option for testing at the point of care, which enables patient access to more immediate results.

FDA

Clinical Trials on Diabetes Safety

In 2008, the Food and Drug Administration mandated the pharmaceutical industry to prove cardiovascular safety for all new anti-diabetes medications. As a consequence, we presently have critically useful safety data from clinical trials like LEADER, SUSTAIN, CANVAS, PIONEER 6, DECLARE-TIMI 58, and REWIND.

New FDA approval: oral medication for Cushing’s disease

Cushing’s syndrome (CS) is a rare condition. It’s health consequences can be severe, leading to visceral adiposity, muscle weakness, depression, insulin resistance, diabetes, blood clots, reduced immune system, and cardiovascular disease.

A portion of patients diagnosed with CS has Cushing’s disease (CD). In Cushing’s disease, the root cause of hypercortisolism is a pituitary tumor producing excessive ACTH hormone. The preferred treatment is pituitary surgery. Unfortunately, in a small subset of patients, the operation is not successful or indicated.

To address the health concerns for individuals who are not responsive or candidates for pituitary tumor resection, the FDA has now approved a backup treatment via the oral drug Isturisa.

Isturisa, taken twice daily, is a potent inhibitor of 11-β-hydroxylase, a key enzyme in cortisol production. Side effects can include relative adrenal insufficiency, swelling, nausea, vomiting, and headaches. Clinical studies have shown that about 50% of patients respond to therapy.

Isturisa is an excellent addition to the arsenal of combating refractory Cushing’s disease.

Dr. Tashko

New FDA approval: fish oil (vascepa) for cardiovascular risk reduction

Vascepa is a form of omega-3 fish oil named icosapent ethyl. It was first approved by the FDA in 2012 for the treatment of severe hypertriglyceridemia, as defined by blood triglyceride levels >500 mg/dL.

The FDA has now added a second indication for Vascepa. It can be utilized in patients with established cardiovascular disease or in those who have diabetes plus two or more other risk factors (e.g., hypertension, hypercholesterolemia, smoking, kidney dysfunction) for cardiovascular disorder. Before treatment, patients must also have baseline triglyceride measures >150 mg/dL.

This significant approval comes after reviewing the results of REDUCE-IT, a landmark randomized clinical trial published in NEJM, January 2019. REDUCE-IT confirmed significant cardiovascular outcome reduction of 25% in patients receiving icosapent ethyl when added to statin therapy.

I anticipate widespread use of the fish oil, icosapent ethyl, especially in patients with diabetes, who, by its nature of insulin resistance, frequently have both the increased risk of ASCVD and high triglycerides. Clinicians should be aware of possible induction or worsening of atrial fibrillation, atrial flutter, and bleeding with Vascepa, particularly in the predisposed individuals.

GT

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.

GT