Stay up to date with the latest news and standards knowledge.

Blog > The Monday Morning Briefing: Approvals galore - a busy week for the FDA

The Monday Morning Briefing: Approvals galore - a busy week for the FDA

7 September 2015

Good morning! We trust you had a happy and safe Labor Day weekend. Sadly, it’s time for the labor to get underway once more, so ease yourself into the week by getting up-to-date with the week’s pharma headlines.

The FDA approved an application from Eko Devices to market its Eko Core, a digital device that brings the stethoscope into the smartphone era, the New York Times reported

Eko Core is a digital device that attaches to a conventional stethoscope and allows it to record, amplify and wirelessly send audio and sound wave images to an iPhone application. The University of California, San Francisco, medical school has begun enrolling patients for a clinical trial to test the diagnostic reliability of the Eko algorithm. In the trial, the predictions of the Eko algorithm will be compared with echocardiograms for the same patients. Dr. Chorba said the clinical trial would most likely take about a year. The decision support software will undergo a separate review by the FDA.

The Administration also handed a breakthrough drug designation to Roche for its hemophilia A treatment ACE910, FierceBiotech reports

In a Phase I study, ACE910 showed promising results as a prophylactic treatment administered as a weekly subcutaneous injection in people with severe hemophilia A with and without inhibitors to factor VIII Breakthrough therapy designation is designed to accelerate the development and review of medicines that demonstrate early clinical evidence of a substantial improvement over current treatment options for serious diseases. People with severe hemophilia A can be susceptible to uncontrolled or difficult to control bleeding including internal bleeding, especially into the joints, which can lead to the need for joint replacements.

And the Administration had good news for AstraZeneca too, as it approved its potential blockbuster blood-thinning drug Brilinta, said the Guardian

The FDA approved the treatment for longer-term use in patients with a history of heart attacks. The new 60mg dose can be taken twice a day along with aspirin beyond a year after a heart attack. Until now, the blood thinner could only be prescribed for one year after a heart attack, as a 90mg dose. The FDA decision will boost sales of the heart drug, which is used to prevent blood clots that can cause heart attacks, strokes and deaths. AstraZeneca has estimated that annual sales could reach $3.5bn (£2.29bn) by 2023 and a spokeswoman said the expanded label for Brilinta reinforces the company’s confidence in that forecast. Brilinta sales rose 23% to $144m in the second quarter, and totalled $476m last year.

However, Alexion wasn’t so lucky - the FDA has delayed its decision on approving its lysosomal acid lipase deficiency drug by three months, Reuters reported

The FDA, which has granted the drug "breakthrough therapy" status for LAL deficiency in infants, was expected to make its decision by Sept. 8. Alexion said on Friday it had submitted some additional "chemistry, manufacturing and controls" information that the FDA asked for, and that the timing of the submission had caused the delay. The regulator has not asked for any addition clinical data on the enzyme-replacement therapy for the ultra-rare, life threatening disease, the company said. European health regulators on Tuesday approved the drug, Kanuma, to treat lysosomal acid lipase deficiency (LAL-D), a progressive metabolic disease that often leads to liver failure, multi-organ damage and premature death.

Finally, PharmaTimes tells us that the agency gave the green light to Wellstat’s Xuriden, the first treatment for patients with a super-rare metabolic disorder affecting only 20 people in the world

The disease, hereditary orotic aciduria, affects children and occurs because of a defective or deficient enzyme which results in the body being unable to normally synthesise uridine. Signs and symptoms of the condition include blood abnormalities (anaemia, decreased white blood cell and neutrophil counts), urinary tract obstruction, failure to thrive, and developmental delays. Wellstat’s Xuriden (uridine triacetate) is an orally administered product intended to replace uridine, which, in clinical trials, was shown to stabilise blood-based markers in all four clinical trial participants, and caused no side-effects.

Topics: Biotech, News, Pharma