VGL101 Granted Orphan Drug Designation

Exciting news for ALSP patients!

VGL101 received U.S. Food and Drug Administration (FDA) orphan drug designation: In July, the FDA granted orphan drug designation to VGL101 for the treatment of patients with ALSP. The FDA’s Orphan Drug Designation program provides orphan designation to drugs and biologics that are intended to treat, diagnose or prevent rare diseases affecting fewer than 200,000 people in the U.S. This designation provides the potential to obtain certain benefits, including financial incentives to support clinical development and seven years of marketing exclusivity upon FDA approval. Vigil Neuroscience Reports Second Quarter 2022 Financial Results and Provides Business Update (

What is an orphan disease? A disease affecting so few people that:

  • there is no reasonable expectation that the cost of developing and making available a drug for such disease or condition will be recovered from sales.


  • it is not cost effective to spend money on research
  • it has been virtually ignored with little medical attention or other support

What is an orphan drug? An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. 

What does it mean to be granted orphan status? The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. In the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel.

Additional information can be found at: Designating an Orphan Product: Drugs and Biological Products | FDA