Rare Disease
Therapeutics Product
Portfolio
Approved treatments and
pipeline compounds
Rare Disease Therapeutics Product Portfolio
Approved treatments and pipeline compounds
RDT helps bring orphan drugs to market by partnering with companies to provide life-saving therapies for patients with rare diseases.
With over 30 years of experience in orphan drug commercialization, including experience in antivenoms, oncology, hematology, metabolic diseases, and genetic disorders, RDT maintains a foundational presence in rare disease outreach.
ANTIVENOM—FDA-APPROVED PRODUCTS
Name
Status
Links
![ANAVIP® [crotalidae immune F(ab′)₂ (equine)]](https://www.raretx.com/wp-content/uploads/2022/06/logo_anavip_R001_no-icon-e1655406781557.png){kind=icon}
![ANASCORP [centruroides (scorpion) immune F(ab’)₂ (equine)] injection](https://www.raretx.com/wp-content/uploads/2022/06/logo_no-icon.png){kind=icon}
ANTIVENOM—FDA-APPROVED PRODUCTS
Name
Status
Available
Links
Name
![ANASCORP [centruroides (scorpion) immune F(ab’)₂ (equine)] injection](https://www.raretx.com/wp-content/uploads/2020/11/tagline_w-injection369x55-pms143n.png)
Status
Available
Links
HEMATOLOGY/ONCOLOGY—FDA-APPROVED PRODUCTS
Name
Status
Links
HEMATOLOGY/ONCOLOGY—FDA-APPROVED PRODUCTS
Name
Status
AVAILABLE
Links
HEMATOLOGY/ONCOLOGY—RESEARCH PRODUCTS
Name
Status
Links
To report a suspected adverse reaction or to order any of our approved products, please call 1-844-472-7389.
If you prefer to report adverse reactions to the FDA, either visit www.FDA.gov/medwatch or call 1-800-FDA-1088.
HEMATOLOGY/ONCOLOGY—Research products
Name
Status
NDA Pending
Links
To report a suspected adverse reaction or to order any of our approved products, please call 1-844-472-7389.
If you prefer to report adverse reactions to the FDA, either visit www.FDA.gov/medwatch or call 1-800-FDA-1088.
Product History
Products previously sold by RDT
ORFADIN® (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 (2006). “Nitisinone in the treatment of hereditary tyrosinaemia type 1”. Drugs. 66 (6): 743–750.