RDT Products
Rare Disease Therapeutics, Inc., (RDT) is currently developing Orphan products, acquiring Orphan products through strategic alliances, and distributing Orphan products throughout the Americas. RDT works closely with the FDA’s Office of Orphan Product Development, National Organization for Rare Disorders (NORD), the National Institutes of Health, large international pharmaceutical companies, and patient advocacy groups to identify the unmet needs of patients with rare diseases and potential products to meet these needs.
Orfadin® (www.Orfadin.com)
A classic example of the kind of product Rare Disease Therapeutics, Inc., pursues is Orfadin® (nitisinone), or 2-(2-nitro-4-trifluoromethylbenzoyl) cyclohexane-1,3-dione, for the treatment of Hereditary Tyrosinemia Type 1/HT-1, which is an extremely rare disorder afflicting less than 500 children worldwide with less than 100 in the United States. Present at birth, acute tyrosinemia manifests within weeks or months as the infant fails to thrive. Frequent symptoms include hepatomegaly, edema, ascites, melena, and hemorrhagic diathesis. If untreated, the disorder is fatal in the first year of life.
Orfadin® was launched by Rare Disease Therapeutics, Inc., in the United States for the treatment of Hereditary Tyrosinemia Type 1 as an adjunct to dietary restriction of tyrosine and phenylalanine on January 18, 2002.
* Please see full prescribing information for a complete discussion of indications and usage, contraindications, warnings, precautions, adverse reactions, and overdosage.
Cystadane® (www.Cystadane.com)
A prescription drug product, Cystadane® is the first agent for the treatment of homocystinuria, a rare genetic disorder and has been demonstrated to be safe and effective for the treatment of the three primary types of homocystinuria. Cystadane® has been licensed by Rare Disease Therapeutics, Inc., from Orphan Europe effective April 13, 2007.
Cystadane® is available in plastic bottles containing 180 grams of betaine anhydrous. Each bottle is equipped with a plastic child-resistant cap and is supplied with a polystyrene measuring scoop.
* Please see full prescribing information for a complete discussion of indications and usage, contraindications, warnings, precautions, adverse reactions, and overdosage.
Anascorp® (www.anascorp-us.com)
Anascorp® [Centruroides (Scorpion) Immune F(ab')2 (Equine) Injection], a Centruroides immune F(ab’)2 is the first of a series of antivenoms in the Rare Disease Therapeutics, Inc., pipeline. Anascorp®, an F(ab’)2 antibody, is FDA approved for the treatment of scorpion envenomation from the Centruroides scorpions. Centruroides sculpturatus is the only scorpion species with vertebrate neurotoxins whose natural range includes the United States.
It is found in southeastern California, Arizona, Nevada, southern Utah, and southwestern New Mexico. It is also found throughout the Baja California Peninsula and western Sonora, Ensenada, B.C in México. A typical “bark” or “crevice” scorpion, C. sculpturatus is commonly encountered under rocks, logs, the bark of trees, and other surface objects.
Centruroides scorpion envenomation produces a pattern of neurotoxicity with a spectrum of severity ranging from the trivial to life threatening. Severe envenomation, more common in small children, may involve neuromotor hyperactivity, pulmonary edema, and ventilatory compromise occasionally resulting in death.