While not the official one, the summer I knew as a kid – the one beginning after Memorial Day – has arrived. Officially I guess we have to wait a few more weeks. Once again a week without an intervening posting apart from the Weekly Roundup, but it has been a busy season and I do have a few in the works on deck. In the meantime, as the stragglers among us get busy planning summer getaways, here is a bit of what happened this week:

• The Eyes Have It – Designated as a Breakthrough Device, this week the agency announced approval in the U.S. of the first standalone prosthetic surgically implantable iris to be used in patients who have a damaged iris either as a congenital condition or as a result of injury. While the iris is the colored part of the eye, it also serves the function of controlling the amount of light entering the eye. A damaged iris is not only therapeutic, but is also a cause of light sensitivity. The device is made of a thin, flexible silicon and can be customized for the eye color and appearance of the patient. In reporting on the clinical trial, one heard something the agency does not often refer to in clinical outcomes which was reference to improved quality of life in patients. FDA said adverse events were low, but did note several conditions where use of the device would be contraindicated. The device went through the Pre-Market Application process – the most thorough regulatory pathway for devices. 

• Duchenne Muscular Dystrophy Treatment Gets Negative Opinion in Europe – In 2016 Sarepta was granted FDA approval for its treatment – eteplirsen – for Duchenne Muscular Dystrophy under the rare circumstance (about 10-11 percent of the time by my count) whereby the agency goes against the opinion of an advisory committee recommendation.  This week the company announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had issued a negative opinion on the treatment. The company had previously anticipated the outcome in an earnings call earlier this month and will request a re-examination of the opinion, a process that will take place by year-end. The U.S. decision has been described as controversial. 

• Gottlieb Statement off REMS and Generics – Commissioner Gottlieb continued his narrative related to the use of REMS by brand name drug makers as a means to block timely generic drug market entry this week by issuing a statement to echo prior statements on the subject and to further outline the agency’s approach and releasing new guidance documents to try to ease the situation. He stated that REMS programs are exploited in two ways to block generics by some players. He stated one occurs at the front-end when a generic manufacturer is seeking sufficient doses of a brand name drug to determine bio-equivalence and cannot get access and the other is at the backend of the process when a generic drugs seeks approval. He announced steps to address the latter. Current law requires that under certain circumstances a brand name and generic drug share a single REMS program and the process for doing so requires negotiation between the companies. He said the agency was issuing two new guidance documents to address potential delays – the first – Development of a Shared System REMS that describes principles and recommendations to improve clarity and efficiency for the development of a single REMS program between companies. The second – Waivers of the Single, Shared System REMS Requirement outlines when and how FDA will consider waiving the requirement. 

Things to Keep an Eye on This Week

• June 6 – House Energy and Commerce SubCommittee on Health – Legislation to Ra-authorize the Pandemic and All-Hazards Preparedness Act
• June 12 – Senate HELP Committee Hearing – The Cost of Prescription Drugs: Examining the President’s Blueprint ‘American Patients First’ to Lower Drug Prices 

Regulatory Developments in Drugs/Biotech and Devices

• FDA Puts Clinical Hold on CRISPR Trial
• FDA Opens Docket to Get Feedback on Digital Health Software
• Priority Review for Postpartum Depression NDA

Photo by Patrick Baum on Unsplash