Christine Blank

 the first oral blood thinner for children: Boehringer Ingelheim’s Pradaxa (dabigatran etexilate).

The longstanding medicine for adults is now approved to treat children three months to less than 12 years old with venous thromboembolism directly after they have been treated with a blood thinner given by injection for at least five days and to prevent recurrent clots among patients three months to less than 12 years old who completed treatment for their first venous thromboembolism.

In addition, Pradaxa was approved in capsule form to treat blood clots in patients eight years and older with venous thromboembolism directly after they have been treated with a blood thinner given by injection for at least five days, and to prevent recurrent clots in patients eight years and older who completed treatment for their first venous thromboembolism.

The only other approved blood thinning medication for children is given by injection, FDA said in a 

. Pradaxa was originally approved in 2010 to reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation.

"The FDA is committed to helping our youngest patients with serious medical conditions have treatments that are relatively easy to take,” said Ann Farrell, MD, director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research. “With today’s approval of Pradaxa, pediatric patients have another therapeutic option to treat and prevent potentially deadly blood clots.”

The safety and efficacy of Pradaxa for treating blood clots in patients younger than 18 was evaluated in one study of 267 pediatric patients. Nearly 46% of the 177 people taking Pradaxa met the composite endpoint — they had not died from a blood clot, their blood clots had completely resolved, and they had no additional blood clots — compared to 42.2% of the 90 patients who received standard of care.

Pradaxa has a boxed warning cautioning that early treatment discontinuation may increase the risk of blood clots and that blood accumulation within parts of the spinal cord (spinal or epidural hematomas) in patients undergoing spinal procedures may cause serious side effects.

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The longstanding medicine for adults is now approved to treat children 3 months to less than 12 years old with venous thromboembolism.

FDA Oks First Oral Blood Thinner for Children

FDA recently approved 2 novel cancer treatments, including the second “precision medicine” cancer therapy based on a common biomarker across genes.

Here are the top 2 novel cancer drugs cleared by the agency:

1. Larotrectinib (Vitrakvi, Loxo Oncology) treats adult and pediatric patients whose cancers have with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity and have no satisfactory alternative treatments or that have progressed following treatment.

This is the second time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated. “The approval marks a new paradigm in the development of cancer drugs that are ‘tissue agnostic,’” FDA said in a 

FDA clears new type of leukemia treatment

"This new site-agnostic oncology therapy isn’t specific to a cancer arising in a particular body organ, such as breast or colon cancer. Its approval reflects advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine,” said FDA Commissioner Scott Gottlieb, MD, in the statement. “We now have the ability to make sure that the right patients get the right treatment at the right time.”

Vitrakvi has shown some benefit with a multitude of solid tumors, with the specific NTRK gene type, Mark Ginestro, principal at KPMG Strategy, told 

“This essentially has shown a greater degree of sophistication of connecting gene markers with specific medications. For more than a decade, a lot of drugs have been paired with diagnostics or have a biomarker to check to make sure the drug is appropriate. I wouldn’t call this a culmination of a trend, but progress on a path toward personalized medicine,” Ginestro added.

2.  Gilteritinib (Xospata, Astellas Pharma) tablets treats adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

FDA also approved an expanded indication for a companion diagnostic, LeukoStrat CDx FLT3 Mutation Assay (Invivoscribe Technologies) to detect the FLT3 mutation.

“Approximately 25% to 30% of patients with AML have a mutation in the FLT3 gene. These mutations are associated with a particularly aggressive form of the disease and a higher risk of relapse,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Xospata targets this gene and is the first drug to be approved that can be used alone in treating patients with AML having a FLT3 mutation who have relapsed or who don’t respond to initial treatment.”

The efficiency of Xospata was studied in a clinical trial of 138 patients with relapsed or refractory AML having a confirmed FLT3 mutation. Twenty-one percent of patients achieved complete remission or complete remission with partial hematologic recovery with treatment.

Of the 106 patients who required red blood cell or platelet transfusions at the start of treatment with Xospata, 31% became transfusion-free for at least 56 days.

Here are the top 2 novel cancer drugs cleared by the agency.

FDA clears novel cancer treatments

	FDA is permitting marketing of the first medical device that uses artificial intelligence to detect greater than a mild level of the eye disease diabetic retinopathy.

	IDx-DR (IDx LLC), a software program that uses an artificial intelligence AI) algorithm to analyze images of the eye taken with a retinal camera, is the first device authorized for marketing that provides a screening decision without the need for a clinician to also interpret the image or results, FDA said in a statement. That capability makes it usable by healthcare providers who may not normally be involved in eye care.

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	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.

	“Early detection of retinopathy is an important part of managing care for the millions of people with diabetes, yet many patients with diabetes are not adequately screened for diabetic retinopathy since about 50% of them do not see their eye doctor on a yearly basis,” said Malvina Eydelman, MD, director of the Division of Ophthalmic, and Ear, Nose and Throat Devices at the FDA's Center for Devices and Radiological Health. “Today’s decision permits the marketing of a novel artificial intelligence technology that can be used in a primary care doctor’s office.”

	Here’s how the AI device works: a doctor uploads the digital images of the patient’s retinas to a cloud server on which IDx-DR software is installed. If the images are of sufficient quality, the software provides the doctor with one of two results: (1) “more than mild diabetic retinopathy detected: refer to an eye care professional” or (2) “negative for more than mild diabetic retinopathy; rescreen in 12 months.”

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	If a positive result is detected, patients should see an eye care provider for further diagnostic evaluation and possible treatment as soon as possible.

	FDA evaluated data from a clinical study of retinal images obtained from 900 patients with diabetes at 10 primary care sites. In the study, IDx-DR was able to correctly identify the presence of more than mild diabetic retinopathy 87.4% of the time and was able to correctly identify those patients who did not have more than mild diabetic retinopathy 89.5% of the time.

	IDx-DR was granted Breakthrough Device designation, meaning the FDA provided intensive interaction and guidance to the company on efficient device development, to expedite evidence generation and the agency’s review of the device.

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FDA is permitting marketing of the first medical device that uses artificial intelligence to detect greater than a mild level of the eye disease diabetic retinopathy.

FDA approves landmark AI device to detect diabetic eye disease

	FDA approved a new indication for Bupivacaine Liposome injectable suspension (Exparel, Pacira) for use as a nerve block to produce post-surgical regional analgesia following shoulder surgery in adults.

	Exparel is intended for use as a nerve block to relieve pain associated with shoulder surgery for a period of 48 to 72 hours following administration. The drug was approved in 2011 for local administration to provide post-surgical analgesia.

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	“Today’s action helps to fulfill a need for additional nonaddictive pain management tools by providing a new option for certain patients that can last up to 72 hours following surgery,” said Douglas Throckmorton, MD, deputy center director for regulatory programs in FDA’s Center for Drug Evaluation and Research, in a statement from FDA.

	However, Exparel’s new use is limited to individuals who will undergo shoulder surgeries. “We are taking careful steps to make sure that healthcare professionals treating pain are armed with this important information about the product’s approved uses as well as its limitations in order to make the best pain management decisions for a patient’s unique needs,” Thockmorton said.

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	The Interscalene brachial plexus nerve block works by anesthetizing the body’s nerves nearest the shoulder in order to help curb pain.

	Exparel’s new indication is being approved based on the results of one multicenter clinical study, which demonstrated that the product is safe and effective for use as an interscalene brachial plexus nerve block to provide post-surgical regional analgesia for shoulder surgeries, such as total shoulder arthroplasty and rotator cuff repair.

	FDA determined that clinical trial data is not sufficient to support the general use of Exparel for regional nerve blocks for post-surgical analgesia other than shoulder surgery, according to FDA. “As such, the product’s updated labeling approved today clearly articulates both Exparel’s limitations of use as well as the most up-to-date safety and efficacy data associated with its new interscalene brachial plexus nerve block for post-surgical analgesia indication,” FDA said.

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FDA approved a new indication for Exparel Bupivacaine Liposome injectable suspension (Exparel, Pacira) for use as a nerve block to produce post-surgical regional analgesia following shoulder surgery in adults.

FDA approves non-opioid drug for shoulder surgery

	FDA okayed the marketing of a new continuous glucose monitoring system for diabetics-the first to be used as part of an integrated system with other compatible medical devices and electronic interfaces.

	The G6 integrated continuous glucose monitoring (iCGM) system (Dexcom), which determines blood glucose levels in children aged two and older and adults with diabetes, can be used with automated insulin dosing systems, insulin pumps, blood glucose meters or other electronic devices used for diabetes management.

	FDA also classified this new type of device in class 2, which subjects it to certain criteria called “special controls”, FDA said in a statement. “This enables developers of future iCGM systems to bring their products to market in the least burdensome manner possible,” FDA said.

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	“The ability of this device to work with different types of compatible devices gives patients the flexibility to tailor their diabetes management tools to best meet personal preferences,” said Donald St. Pierre, acting director of the Office of In Vitro Diagnostics and Radiological Health in FDA’s Center for Devices and Radiological Health, in the statement. “In addition, the FDA has taken steps to expedite the review process for similar, integrated CGMs and make these types of systems available to patients as quickly as possible while also helping to ensure their safety and reliability.”

	In early 2017, FDA also expanded approval of Dexcom’s G5 Mobile Continuous Glucose Monitoring System to allow for replacement of fingerstick blood glucose (sugar) testing for diabetics.

	“This is the first FDA-approved continuous glucose monitoring system that can be used to make diabetes treatment decisions without confirmation with a traditional fingerstick test,” FDA said in a statement at the time. The system was previously approved to complement, not replace, fingerstick testing for diabetes treatment decisions.

	Meanwhile, the Dexcom G6 is a patch device, about the size of a quarter, which 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 and will trigger an alarm when a patient’s blood sugar enters a danger zone soaring too high or dropping too low. If it is integrated with an automated insulin dosing system, a rise in blood sugar would trigger the release of insulin from the pump. The patch device should be replaced every 10 days.

	FDA evaluated data from 2 clinical studies of the Dexcom G6, which included 324 adults and children aged 2 years and older with diabetes. Both studies included multiple clinical visits within a 10-day period where system readings were compared to a laboratory test method that measures blood glucose values. No serious adverse events were reported during the studies.

New device could be breakthrough for diabetics

FDA okayed the marketing of a new continuous glucose monitoring system for diabetics-the first to be used as part of an integrated system with other compatible medical devices and electronic interfaces.

FDA approves first-of-its kind diabetes management device

	As FDA considers new guidelines on pharmaceutical marketing, a recent study found that pharma makers may not be following current FDA ad regulations.

	None of the television drug ads included in the 

, published in the February 26 issue of the 

, described the drugs’ risks quantitatively. In addition, 13% of the ads-all for diabetes medications-suggested off-label uses for weight loss and blood pressure reductions, according to the authors.

	“Few broadcast DTC [direct-to-consumer] ads were fully compliant with FDA guidelines,” the authors wrote. “The overall quality of information provided in ads was low, and suggestions of off-label promotion were common for diabetes medications.”

	Meanwhile, FDA prioritized a “robust social and behavioral science research program to help us understand how people make decisions about the products we regulate,” said FDA Commissioner Scott Gottlieb, MD, in a statement.

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 on how drug risks are presented in TV ads suggested that a more-targeted presentation of risks may lead to better retention of the potential side effects that consumers should weigh as they consider their treatment decisions,” Gottlieb said. “As a result, we’ve been exploring new guidelines that would recommend more focused disclosure of risk information in TV ads, to better emphasize potential side effects that are severe, serious and actionable.”

	Several research projects from FDA will help the agency understand the effectiveness of disclosures, “communications aimed at health care providers and consumer understanding of the accelerated drug approval process,” Gottlieb said.

As FDA considers new guidelines on pharmaceutical product marketing, a new study found that pharma makers may not be following current FDA ad regulations.