Rare Disease Therapeutics Product Portfolio
Approved treatments and pipeline compoundsRare Disease Therapeutics Product Portfolio
Approved treatments and pipeline compounds
We work closely with the Office of Orphan Product Development at the FDA, National Organization for Rare Disorders (NORD), the National Institutes of Health, international pharmaceutical manufacturing companies, and patient advocacy groups to identify unmet needs in rare disease. RDT helps bring orphan drugs to market by partnering with manufacturing companies to meet the needs of patients.
North American Pit Viper Envenomation
Status:
Available
Scorpion Envenomation
Status:
Available
Links:
Prescribing Information
Website
Black Widow Envenomation
Status:
Not Approved – Phase 3 complete
Links:
ClinicalTrials.gov
Acute
Lymphoblastic
Leukemia
Status:
Available
Sickle Cell Disease
Status:
Not approved — Phase 3 complete
Links:
Website
Product History
Products previously sold by RDT
Product History
Products previously sold by RDTORFADIN® (nitisinone) Capsules
- Orphan drug approved for the treatment of hereditary tyrosinemia type 1 (HT-1)
- US distribution rights acquired by RDT in 1989
- Pivotal clinical trials executed globally by RDT
- Approved by the FDA in 1991
- Transformed treatment of this rare disease
- Replaced liver transplantation as the first-line treatment1
- Liver failure is controlled in 90% of patients1
CYSTADANE® (betaine anhydrous for oral solution)
- Specialty treatment approved for the treatment of homocystinuria in 1996
- Previously available in small doses in health food stores, but RDT helped create a ‘medical-
grade’ dosage - Consulted and executed clinical trials for approval
- Very small, but very at-risk patient population (~400 patients)
Reference:
1. McKiernan PJ. Nitisinone in the treatment of hereditary tyrosinaemia type 1. Drugs. 2006;66(6):743-750.