Top 5 Rare Disease Stories

James Radke, PhD

It has been a busy week for the rare disease community. Below are the 5 topics that have our interest this week.

Expanded access forms now available

FDA issues guidance for clinicians about compassionate use of drugs in development.  In a new release, the FDA noted they have a new Patient Expanded Access Investigational New Drug Application (Form FDA 3926) that clinicians can use to request expanded access to investigational drugs for individual patients who suffer from serious or immediately life-threatening diseases and for whom no comparable or satisfactory alternative therapy is available.

For more information, click here.

Approvals, disapprovals, and discontinuations

Lots of regulatory news this week regarding the fate of several orphan drugs and rare disease populations. In addition to Dr Robert Califf’s notice that the FDA has new forms for expanded access, the FDA announced the approval of a radioactive probe to help locate rare neuroendocrine tumors. The FDA also approved Ocaliva to treat primary biliary cholangitis. However, it is not all good news. The FDA sent a Complete Response Letter to Teva Pharmaceuticals regarding their SD-809 to treat chorea associated with Huntington’s disease.
Also,  BioMarin announced they have given up trying to get their exon skipping drug, drisapersen, approved and have stopped all clinical trials for that drug as well as three other drugs in development to treat Duchenne muscular dystrophy.

The walking dead

A rare psychiatric condition is making its way around the web this week. Cotard’s syndrome is a rare mental disorder in which patients – usually clinically depressed patients – believe they are dead or do not exist.
To read the original story, click here.

 #CuresNow - the video

Our BFF Max has been very busy the past couple of years trying to get the OPEN ACT approved. The OPEN ACT (Orphan Product Extensions Now Accelerating Cures and Treatments) Act of 2015 was passed through congress and committee and now awaits the Senate. The bill amends the Federal Food, Drug, and Cosmetic Act to require the Department of Health and Human Services (HHS) to extend by 6 months the exclusivity period for a drug or biological product approved by the Food and Drug Administration (FDA) when the product is additionally approved to prevent, diagnose, or treat a new indication that is a rare disease or condition (also known as an “orphan disease”).
And after walking through the Senate halls earlier this year to raise awareness of the OPEN ACT, 8-year old Max has a video that he wants people to send to their Senator.

To view the video and share with your Senator, click here.

And finally, Zika
Zika continues to make the news. While Zika infections are not a rare occurrence, the risk of developing rare birth defects in pregnant women exposed to the virus are a grave concern. Also of concern is the possible link between Zika virus and other rare conditions is only beginning to be understood. With that in mind, the debate on whether it is a good idea to send athletes and spectators to the Olympics in Rio heated up this week with 4 leading scientists urging the World Health Organization to move or postpone the Olympics.
To learn more about the doctors concerns, click here.
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