World Orphan Drug Congress LIVE: Development of Orphan Drugs

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live, Regulation & Government by Freya Smale

Day two of World Orphan Drug Congress begins with FDA’s Richard Moscicki presenting on building the cures of the future. Richard discussed FDA’s programs to boost orphan drug development

  • The FDA’s tools to expand rare disease treatments, from vouchers to incentives
  • Interface with pharmaceutical companies and expanding means of communication
  • Upcoming initiatives, such as precision medicine, and what it means for the FDA orphan guidelines

He begins his presentation stating that this past year was a banner year. 41 new drugs have been approved, of which 41% were first in class. Since its inception in 2012, CDER an dCBER have designated 86 new therapies as breakthrough therapies from almost 30 requests.

Richard follows with breakthrough therapies and its’ two-year assessment. Some important points about breakthrough therapies he brings up include:

  • “Bar” for approval remains unclear for applicants/public
  • The pace of requests for BT designation have remained steady
  • Clinical development is often not the rate-limiting step
  • Program commitments are a resource intensive for FDA
  • Common reasons for denial of BT requests

Targeted medicines are increasing and are common among therapies for Orphan disease with both advantages and challenges80% of breakthrough and about 60% of orphan drugs in recent years have used targeting therapies.

Patient centered drug development is important in orphan disease. People with chronic diseases are “experts” in that disease, as far as the symptoms and the impact on QOL, and what might be acceptable tradeoffs.

The FDA is continuing to develop B/R assessment framework that incorporates burden of diseases. There are many patient groups and non-profits getting involved in evaluating these issues.

An additional incentive the FDA is putting out is the rare pediatric disease voucher program. The FDA will award priority review voucher to sponsors of rare pediatric disease product application that meet certain criteria. The program sunsets 1 year after the third voucher is awarded, and the third voucher was awarded March 2015, so there is one last year this program will run.  Furthermore, there is an increased level of global collaboration on rare diseases and this is a fact to be noted.


Following Richard was Daniel Anderson Scientific Founder of CRISPR Therapeutics discussing RNA as an orphan drug. During the session, Daniel goes into technical detailed description about turning genes on or off, and correcting them to cure genetic disorders. A couple of points touched up by Daniel include:

  • How CRISPR’s inheritable gene editing technology can reverse a rare condition after a one-time treatment in mice
  • What RNAi delivery methods need to be explored to extend these approaches to humans
  • Long term, can CRISPR and RNAi help eradicate rare genetic diseases from existence?

Amongst the discussion, it is noted that intracellular delivery of biomolecule has enormous therapeutic potential.

Daniel mentions how turning nucleic acids into a drug is not easy. It involves a couple of key barriers to deliver for instance:

–       Sequence selection

–       Chemical modification

–       Encapsulation

An important question Daniel addresses is how mRNA can make commercial drugs?

Lastly, Daniel touches up on inn vivo CRISPR delivery with high pressure injection, however, high pressure injection causes liver damage. Can a clinically suitable formulation be found?