FDA Greenlights New Diabetes Device; and Treatments for Hypoparathyroidism, Thyroid Cancer

Next generation tubeless Omnipod DASH Insulin Management System coming to market, a novel nasal spray product for diabetes gastoparesis is under review., and orphan drug status granted for treatment of hypoparathyroidism and anaplastic thyroid cancer..

Editor’s note: Don’t miss out on a great AACE CME webinar (see information at bottom of this article).

Omnipod DASH Insulin Managment System Ready for Patient Use

The Omnipod DASH Insulin Management System for diabetes management was cleared by the Food and Drug Administration (FDA),1 enabling the Insulet Corporation to bring the product to market.

"Our mission is to reduce the burden of diabetes for both our users and the clinicians who care for them,” Shacey Petrovic, Insulet's President and Chief Operating Officer told EndocrineWeb, “Our new Omnipod DASH Insulin Management System embodies what those on multiple daily injections and their healthcare providers have been seeking in diabetes management. The system is designed to be discreet and intuitive with a sleek, touch-screen Personal Diabetes Manager, a tubeless waterproof Pod, and a suite of mobile apps allowing for seamless communication of patient data. DASH gives both users and clinicians confidence in quality insulin management, allowing them freedom and flexibility in their everyday lives.”

This next generation Omnipod DASH system represents a major upgrade for the first tubing-free, automatic insulin delivery device with an enhanced Personal Diabetes Manager (PMD) that enhanced features including a color touch-screen display and Bluetooth wireless technology.

Insulin delivery is controlled wirelessly with the PMD handheld device that will host the timing of bolus and basal insulin settings as well as insulin-on-board calculations.

With the DASH PDM, users and their caregivers will have immediate and easy access to insulin data and blood glucose readings through the Omnipod display and view apps, which will have the latest technology to integrate with current and future diabetes devices (eg, meters, continuous glucose monitors [CGMs], and apps).1

The smartphone “display” app will allow users to view both their meter and continuous glucose data and the “view" app will permit remote users to access information in similar fashion to that of the Dexcom CGMs.

Another feature included in the DASH system is the iOS Today View widget, which will permit access to insulin and glucose data on a patient’s (and caretakers’) smartphone.1   And the Contour Next One blood glucose meter (Ascencia) replaces the original meter to facilitate transmission of blood glucose readings to the PDM’s bolus calculator.

Insulet plans a limited market launch with the DASH PDM provided free to current users when new Pods are purchased. A full rollout is anticipated in early 2019 in the United States.

Orphan drug status granted to treat hypoparathyroidism; and FDA diabetes tools approved.

Nasal Spray Application to Manage Symptoms of Diabetic Gastroparesis

Evoke Pharma submitted a new drug application (NDA) to the FDA for Gimoti (metoclopramide) nasal spray candidate for the relief of symptoms arising from acute and recurrent diabetic gastroparesis in women.3 If approved, Gimoti will be the first intranasal formulation of metoclopramide developed to provide promobility and anti-emetic effects by systemic administration to manage gastroparesis in individuals with diabetes.

Currently, metoclopramide is administered orally (Reglan tablets) or intravenously.4 Unlike the current tablet, an intranasal application would bypass both the stomach and liver, allowing for much better absorption. As well, an intranasal route would be much more practical and less invasive and cumbersome as compared to the IV. 

Many of the women—who represent 80% of all patients with the disease—cannot use oral therapy, which is often ineffective during acute symptom flares, including episodes of nausea and vomiting, which will make a non-oral delivery method extremely well received and beneficial.

According to the company,4 a pharmacokinetics comparative exposure study is underway in healthy subjects to demonstrate exposure of the intranasal preparation against the oral tablet. Its worth noting that in a phase 3 study conducted in 2016, the primary endpoint was not attained but “Gimoti still demonstrated clinically meaningful benefits in patients with moderate to severe symptoms at baseline,”among 51% of trial participants (n=201). 5

 

“Gimoti is the first outpatient non-oral product candidate for the treatment of symptoms associated with diabetic gastroparesis in adult women. This nasal formulation of the only approved medication, metoclopramide, allows patients to receive predictable systemic delivery, regardless of stomach delays or vomiting, which are both common issues experienced by these patients," Matt D’Onofrio, Evoke Pharma’s Co-Founder and Chief Business Officer, told EndocrineWeb, "Evoke believes that Gimoti will make a significant difference in the lives of these patients and we look forward to the FDA review.”

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FDA Grants Orphan Drug Status to Novartis for Anaplastic Thyroid Cancer

The Food and Drug Administration granted both a priority review and a breakthrough orphan drug designation to Novartis Pharmaceuticals for treatment of anaplastic thyroid cancer (ATC) that has an abnormal gene, BRAF V600E (BRAF V600E mutation-positive), conferring rare disease status.6

Approved was granted for co-administration of Tafinlar (dabrafenib) and Mekinist (trametinib) for the treatment of ATC, when surgery is not an option or the cancer has spread (metastatic).6

“This is the first FDA-approved treatment for patients with this aggressive form of thyroid cancer, and the third cancer with this specific gene mutation that this drug combination has been approved to treat,” said Richard Pazdur, MD, director of the Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research at the FDA,6 in a prepared statement. “This approval demonstrates that targeting the same molecular pathway in diverse diseases is an effective way to expedite the development of treatments that may help more patients.”

The efficacy of using Tafinlar and Mekinist in combination to treat ATC was confirmed in an open-label trial of patients who had rare cancers with the BRAF V600E mutation.7 Results of the trials of BRAF V600E mutation-positive, metastatic melanoma and lung cancer as well as other BRAF V600E mutation-positive rare cancers supported the data for patients with ATC.

The ATC study evaluated the proportion (overall response rate) of patients demonstrating a complete or partial reduction in tumor size.8 Of the 23 patients assessed, more than half showed a partial response and 4 percent experienced a complete response; in nine of the 14 patients with responses, there were no significant tumor growths for six months or longer.8

The side effects of combination Tafinlar and Mekinist among patients with ATC were consistent with those experienced when treated for other cancers. Reports of typical side effects include: fever (pyrexia), rash, chills, headache, joint pain (arthralgia), cough, fatigue, nausea, vomiting, diarrhea, myalgia (muscle pain), dry skin, decreased appetite, edema, hemorrhage, high blood pressure (hypertension) and difficulty breathing (dyspnea). 

Both Tafinlar and Mekinist are already approved for use both individually or in combination to treat BRAF V600 mutation-positive metastatic melanoma and BRAF V600E mutation-positive, metastatic non-small cell lung cancer.

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Orphan Drug Designation Granted for TransCon PTH to Treat Hypoparathyroidism

The Food and Drug Administration (FDA) granted orphan drug status to Ascendis Pharma for TransCon PTH, a long-acting product of parathyroid hormone (PTH), for improved management of hypoparathyroidism.9 Hypoparathyroidism, a rare endocrine disorder that affects an estimated 80,000 individuals in the US, presents as low serum calcium and elevated serum phosphate levels, placing patients at 4-fold higher risk of developing kidney disease.

Until recently, hypoparathyroid disease remained one of the few hormone deficient states not treatable with hormone replacement. TransCon PTH is designed to restore PTH to physiologic levels, normalizing blood and urinary calcium levels, serum phosphate levels, and lessening bone turnover.

"We are pleased about the [orphan drug designation] for TransCon PTH, which reflects the need for a true PTH replacement therapy that more fully addresses all aspects of hypoparathyroidism," said Jonathan Leff, MD, chief medical officer at Ascendis Pharma, in a corporate statement. "We have recently completed a phase 1 trial in healthy adults, which reinforced the potential of TransCon PTH to restore PTH to physiological levels for 24 hours per day, and look forward to advancing TransCon PTH."10

The FDA grants orphan drug designation (ODD) to treatments developed for the safe and effective management, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The granting of an orphan drug designation does not alter the FDA's regulatory requirements to establish safety and effectiveness of a drug through adequate and well-controlled studies to support approval and commercialization.

Ascendis Pharma plans to initiate a phase 3 program for TransCon PTH in the first quarter of 2019,11 following discussions with the FDA and other regulatory agencies.

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AACE Webinar on Coding Strategies for Endocrinologists

  • Tuesday, June 26, 2018
  • 1:00 pm – 2:30 pm (EST)

Evaluation and Management Coding Strategies for Endocrinology” designed for endocrinology practices and focused on the specific rules relevant to E/M coding. Learn how to determine the appropriate E/M code level for the service provided and the criteria used in making that determination. Produced by Practice Management Institute. CEUs: 1.5

Presented by: Audrey Coaxum, CHI, CMC, CMIS, CMOM, CMCO, CMCA-E/M, CPC. For more information or to register:  AACE Webinar.

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Managing MEN2—Multidisciplinary Approach to Hypoparathyroid Diseases
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