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Monthly News Roundup - September 2019

Rybelsus Cleared as First Oral GLP-1 Agonist for Type 2 Diabetes

The US Food and Drug Administration (FDA) has approved Novo Nordisk’s once-daily Rybelsus (semaglutide) oral tablets used with diet and exercise to improve blood sugar control in adults with type 2 diabetes. Rybelsus, in 7 mg and 14 mg tablets, is the first oral glucagon-like peptide-1 (GLP-1) receptor agonist approved in the U.S. The injectable form of semaglutide is known as Ozempic.

  • In head-to-head studies, Rybelsus was compared with sitagliptin, empagliflozin and liraglutide in over 9,500 patients with type 2 diabetes. Rybelsus reduced A1C and showed reductions in body weight up to 4.4 kg (9.7 lb).
  • The safety is similar to the subcutaneous forms of GLP-1) receptor agonists. The most common side effects reported in at least 5% of patients are nausea, abdominal (stomach) pain, diarrhea, decreased appetite, vomiting and constipation.
  • Rybelsus is expected to be available in Q4 2019. 

Ibsrela Approved as a First-in-Class Agent for IBS with Constipation

Irritable bowel syndrome with constipation (IBS-C) leads to chronic stomach pain and constipation and affects roughly 11 million people in the U.S. This month the FDA approved Ibsrela (tenapanor) from Ardelyx, Inc., a first-in-class, sodium/hydrogen exchanger 3 (NHE3) inhibitor indicated for adults with irritable bowel syndrome with constipation (IBS-C). 

  • Ibsrela, dosed at 50 mg orally twice a day, acts locally in the gastrointestinal (GI) tract to lower sodium absorption and increase water secretion into the intestinal lumen, a unique mechanism.
  • In Phase 3, placebo-controlled studies, patients met the primary endpoints, defined as a 30% or greater reduction in the abdominal pain score and an increase of at least 1 complete spontaneous bowel movement for at least 6 of the first 12 treatment weeks.
  • Common side effects (≥2%) include diarrhea, stomach distension, flatulence (gas) and dizziness.
  • Ibsrela use is for adults; do not use in children under 6 years of age, and avoid in children 6 to less than 12 years of age due to the risk of severe diarrhea and dehydration.

Gvoke: First Ready-To-Use Liquid Glucagon for Severe Hypoglycemia

Severe hypoglycemia can result in heart disease, seizure, coma, and, if left untreated, death. In September, the FDA approved Gvoke (glucagon) injection from Xeris Pharmaceuticals, a ready-to-use, room-temperature stable liquid glucagon for the treatment of severe hypoglycemia in pediatric and adult patients with diabetes ages 2 years and above.

  • Gvoke is given via a prefilled syringe (Gvoke PFS) or auto-injector (Gvoke Hypopen) with fewer preparation and administration steps.
  • In Phase 3 studies that compared Gvoke to conventional glucagon emergency kits in children and adults with type 1 diabetes, treatment success was met in 100% of children and 99% of adults.
  • The most common side effects included nausea, vomiting, hypoglycemia, injection site reactions, headache, and stomach pain, among others.

FDA OKs Jynneos Vaccine for Smallpox, Monkeypox Prevention

In September the FDA approved Jynneos (Smallpox and Monkeypox Vaccine, Live, Non-replicating) for the prevention of smallpox and monkeypox disease in adults 18 years of age and older determined to be at high risk. Jynneos is given in two doses four weeks apart. This is the only FDA-approved vaccine for the prevention of rare monkeypox disease, which can be fatal in up to 10% of cases.

  • Routine smallpox vaccination of Americans was stopped in 1972 after the disease was eradicated in the U.S.; therefore, the intentional release of this highly contagious virus could have a devastating public health effect.
  • In a clinical study with 400 healthy adults who had never been vaccinated for smallpox, the Jynneos immune response was shown to be non-inferior to (not worse than) ACAM2000, another FDA-approved smallpox vaccine. However, because it is non-replicating, Jynneos can be used in people with weakened immune systems, those with eczema, and those with household members who have eczema.
  •  Common side effects included pain, redness, swelling, and itching, among others.
  • This vaccine is also part of the Strategic National Stockpile (SNS), the largest U.S. supply of potentially life-saving pharmaceuticals for a public health emergency.

Nucala Gains New Pediatric Use in Severe Eosinophilic Asthma

The FDA has cleared GSK's Nucala (mepolizumab), an interleukin-5 (IL-5) antagonist, to be used as add-on maintenance treatment in children aged six to 11 years with severe eosinophilic asthma. Nucala works by preventing IL-5 from binding to its receptor on the surface of eosinophils which may help to control inflammation.

  • The Nucala 40 mg dose, given as a subcutaneous injection once every 4 weeks, is now the only U.S. targeted biologic approved for this condition in children aged six to 11 years.
  • The approval is supported by an open-label study that investigated Nucala’s pharmacology and long-term safety as well as from previous adult and adolescent studies. The safety profile in younger children was similar to the known safety profile in older children.
  • Nucala is also approved as add-on maintenance treatment for severe eosinophilic asthma in patients aged 12 years and older and for eosinophilic granulomatosis with polyangiitis (EGPA) in adults.


Posted: September 2019