The shifting of gears is happening before our eyes. Already leaves are falling and the morning alarm goes off in the final moments of night time darkness rather than the first rays of morning sunshine. The trees are signalling that they are ready for their slumber. I have been too – away on a Equinox vacation. I’m back, so let’s see what happened not just over the past week, but two.
Or first, what didn’t happen. There were no new OPDP action letters posted these past few weeks, but we are now up to four for what is shaping up to be a new low in the number of enforcement letters posted by the office.
But a few things did happen and here are some that I wanted to highlight:
- FDA Approves First Drug to Treat Duchenne’s Muscular Dystrophy – It was perhaps one of the more anticipated decisions by FDA on a product this year. That was because it treats a rare, yet devastating condition in children where there were no options. And it was because the clinical trial was small. And because these and other factors would cause FDA to step outside of its usual approach in considering new approvals. Amid media reports of internal dissension within the agency, and after some delay, FDA ultimately announced approval of Exondys 51 for the treatment of a subset of people with Duchenne’s Muscular Dystrophy (DMD) who have a specific gene mutation (about 13 percent of the DMD population). The compound had fast track, orphan drug and priority review designation. The company stated in its release on the approval that a clinical benefit has not yet been established for the treatment and that continued approval may be contingent upon seeing that benefit in future confirmatory trials.
- Automated Insulin Delivery Device Approved – Hailed as the first “artificial pancreas”, FDA this week announced that it was giving the ok to the first device designed to do what the pancreas does – to deliver insulin to the body on a regular basis. The device, the MiniMed 670G hybrid closed loop system, is equipped with a sensor to monitor glucose levels and deliver insulin through an infusion patch connected to an insulin pump, worn externally. The device was studied in a trial consisting of 123 people. The indication is in patients 14 years and older with Type 1 diabetes and the agency will be looking for a post-market study to get a clearer picture on actual use in the real world. Details and photos on the use and range of the device can be found on the company site. The company says the new system will be ready for shipping in Spring, 2017.
- The “Close-Hold Embargo” and FDA – Anyone working in media is familiar with a news embargo. It allows an entity with news to share it with reporters prior to an official announcement so that stories can be ready for publication when the news actually happens. It is a handy tool for both sides. Add the words “close-hold” prior to the word embargo, however, and you have controversy. That is what happened with the publication of an article detailing a practice at FDA known as the “close-hold embargo” whereby the news was not only embargoed, but the ability for a reporter to talk to others to get input and point of view to help shape the story was also proscribed. The idea that a federal agency would tell reporters who to report the news – or to intervene in the reporting process brought to light by the article, generated a good deal of critical comment during the course of the week. Agree with it or not, it may not be perceived as bolstering the notion of transparency.
That’s it for me this week. Have a good weekend everyone. Happy Autumn!
