Browsing Tag


HD Clinical Trials

The FDA Orphan Drug Act/Huntington’s Disease

In 1983, Orphan Drug Act (ODA) was set up to encourage increased development of drugs for rare diseases. It was amended in 1984 to define rare diseases as those that affect less than 200,000 people in the United States, but it also included drugs for diseases affecting more than 200,000 people as long as there was no commercial viability—that is, that the cost of development and making available in the United States a drug for the disease would exceed revenue from the US sales. Huntington’s disease fits that criteria.  Overview-of-huntingtons-disease/

Wave Life Sciences has seen the FDA grant it an orphan drug designation for its lead candidate WVE-120101.  The experimental drug, which targets rs362307–a single nucleotide polymorphism that is associated with the disease-causing mutation in the huntingtin gene.

The orphan status gives it 7 years of exclusivity in the U.S., and other boosts, including tax credits related to clinical trial expenses, an exemption from the FDA user fee, and FDA assistance in clinical trial design.

To read more about the Orphan Drug Act, visit:  https://www.ajmc-the-orphan-drug-act

We Can Never Lose HOPE………………

The author website for the nonfiction book I published is

Watching Their Dance on many book websites like Amazon