Rare Disease Therapeutics (RDT) specializes in the commercialization of orphan drugs, leveraging over 30 years of experience to bring life-saving treatments to patients with rare diseases. The company has developed a robust expertise in navigating the complex regulatory landscape associated with orphan drugs, including Investigational New Drug Applications (INDs), Biologics License Applications (BLAs), and New Drug Applications (NDAs). RDT employs targeted sales and marketing strategies tailored specifically for rare disease treatments, ensuring that these therapies reach the patients who need them most. Their extensive medical knowledge in clinical research and development further enhances their ability to support the successful launch and distribution of these specialized drugs.

RDT's product portfolio includes a range of FDA-approved treatments across various therapeutic areas, such as antivenoms, oncology, hematology, metabolic diseases, and genetic disorders. The company has played a pivotal role in transforming the treatment landscape for rare diseases, exemplified by their work with ORFADIN® (nitisinone) for hereditary tyrosinemia type 1, which has significantly improved patient outcomes since its approval in 1991. Additionally, RDT has been instrumental in developing medical-grade formulations, such as CYSTADANE® (betaine anhydrous), for conditions like homocystinuria, demonstrating their commitment to addressing the unique needs of small, at-risk patient populations.

United States

Rare Disease Therapeutics is a pharmaceutical company that brings orphan drugs to the US market for people with rare diseases and unmet medical needs.

Rare Disease Therapeutics

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