A Rare Partner in Rare Diseases

A Rare
Partner in
Rare Diseases

Pioneers in orphan drug commercialization

Pioneers in orphan
drug commercialization

A Partner to Patients

A Partner to Patients

Helping people inspires us. Patients who have rare diseases often have few or no options to turn to for hope. Knowing that we may be able to extend, or even save lives, while also bringing a sense of optimism where there wasn’t one before, reminds us why we work with rare disease treatments every day.

A Partner to Manufacturers

A Partner to Manufacturers

Our mission is to bring treatments for rare diseases to market and to make sure the people who need those treatments can get them.

A Partner for Progress

Everything we do moves the rare disease treatment space forward. We have helped pioneer legislation in favor of rare disease treatments for 30 years.

As America’s first exclusively orphan drug company, RDT has been a trailblazer in many ways.

  • Helped one of the first orphan drugs achieve approval under the Orphan Drug Act of 1983
  • Successfully lobbied to reduce annual FDA fees for orphan drugs that served unmet medical needs
  • Worked with international manufacturers to bring rare disease treatments to market in the U.S. by navigating FDA regulatory requirements
  • Two-time award winner from NORD for achievements in humanitarianism and progress

RDT: An Award-winning Orphan Drug Company

Two-time NORD award winner

RDT: An Award-winning Orphan Drug Company

Two-time NORD award winner

2012 Partner In Progress Award

This amazing story puts into context how impactful ANASCORP® [centruroides (scorpion) immune F(ab’)2 (equine)] injection has been, leading to recognition from NORD. Wayne and Dawn Bray live in Arizona, where the majority of 20,000 scorpion stings in the U.S. occur each year. In 2002, a scorpion stung their two-year-old son Dally, leading to complications that ultimately took his life. This devastating event left the Brays with painful memories of desperately seeking help for sudden and frightening symptoms.

A few years later, their son Morgan also suffered a scorpion sting. The family rushed him to the nearest rural hospital and, from there, he was taken by helicopter to a Tucson hospital where he was given an experimental antivenom that saved his life.

That product was ANASCORP. Morgan was included in the clinical trial of ANASCORP that helped bring it to market.

After learning of the FDA approval, Dawn Bray said she felt “joy and elation” that this treatment would now be available to other families going through what her family experienced with Dally. It leaves her with a feeling that her son “didn’t pass away in vain,” she says.

NORD recognized RDT for their ability to bring new rare treatments to market that can change families’ lives.

For more info about ANASCORP,
including safety information, visit www.anascorp-us.com

Watch this video from the award event:

Play Video
The Bray family.

2000 Humanitarian Award

In June 2000, NORD recognized the President of RDT, Milton Ellis, for the development of ORFADIN®(nitisinone), a treatment for children with hereditary tyrosinemia type 1. This condition is a life-threatening genetic metabolic disease that affects infants and children.

Abbey Meyers, then President of NORD, said, “It’s an honor for me to present Mr. Ellis with this Humanitarian Award. His efforts to help find relief for those suffering from a variety of rare disorders goes way beyond the norm. Mr. Ellis and his company, Orphan Pharmaceuticals USA, Inc. (now named Rare Disease Therapeutics, Inc.) are continually searching for ways to assist patients and their families.”

Milton Ellis accepting NORD Humanitarian Award

Pictured from right to left: Abbey Meyers, NORD President Emeritus; Milton Ellis, Rare Disease Therapeutics; Danielle Barckett; Pam Barckett.