Retrophin Will Acquire Worldwide Rights
As a result of the approval,
The
"With FDA approval, Cholbam will be available to patients suffering from several life-threatening diseases that until now, had no approved treatment. Cholbam complements Retrophin's existing bile acid therapy, Chenodal (chenodeoxycholic acid), and will position us as the leading provider of treatments for patients with these bile acid synthesis and peroxisomal disorders," said
The effectiveness of Cholbam has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. There are approximately 30 patients currently receiving Cholbam through an open label extension of these trials. The estimated incidence of bile acid synthesis disorders due to single enzyme defects is 1 to 9 per million live births. Peroxisomal disorders are believed to affect approximately 1 in 50,000 live births.
Cholbam will have seven years market exclusivity in
About
Forward-Looking Statements
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995, regarding the research, development and commercialization of pharmaceutical products. Without limiting the foregoing, these statements are often identified by the words "may", "might", "believes", "thinks", "anticipates", "plans", "expects", "intends" or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the Company's ability to consummate the acquisition of Cholbam or the Pediatric PRV, the effectiveness of Cholbam in treating bile acid synthesis disorders or peroxisomal disorders, the Company's ability to leverage Cholbam as a complement to the Company's existing bile acid therapy, the Company's ability to position itself as the leading provider of treatments for patients with bile acid synthesis and peroxisomal disorders, the incidence rate of bile acid synthesis disorders and peroxisomal disorders, Cholbam receiving an approval for a CTX indication, the Company's business and finances in general, as well as risks and uncertainties associated with the Company's sales and marketing strategies. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in the Company's filings with the
Manager, Investor Relations
IR@retrophin.com
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