This is certainly big news. If my experience is any indicator, this will only serve to be a benefit to patients who are suffering from Bile Acid disorders. URL and text from press release follow.
NEW YORK & BALTIMORE–(BUSINESS WIRE)– Retrophin, Inc. (NASDAQ:RTRX) and Asklepion Pharmaceuticals, LLC, a privately held rare disease pharmaceutical development company, today announced the signing of a definitive agreement under which Retrophin has acquired the exclusive right to purchase from Asklepion, all worldwide rights, titles, and ownership of cholic acid for the treatment of bile acid synthesis defects, if approved by the U.S. Food and Drug Administration(FDA). Under the terms of the agreement, Retrophin will pay Asklepion an upfront payment of $5 million and up to $73 million in milestones based on approval and net product sales, plus tiered royalties on future net sales of cholic acid. Retrophinhas secured a line of credit from current lenders to cover necessary payments.
“We are very pleased with this agreement, which has the potential to significantly accelerate Retrophin’s growth in 2015 and beyond,” said Stephen Aselage, Chief Executive Officer of Retrophin. “Cholic acid is a natural complement to our current commercial portfolio and upon its approval, Retrophin would be positioned to become the leading provider of bile acid treatments for a number of rare diseases.”
Mr. Aselage continued, “The effort by Asklepion and its investigators is a testament to their dedication and hard work in bringing an important medication closer to the marketplace. The disorders treated in their clinical trial are devastating and we are grateful to have an opportunity to address those patients’ needs.”
Cholic acid is being considered for approval in a late-stage review by the FDA and is currently approved in Europe for the treatment of inborn errors in primary bile acid synthesis. It restores endogenous bile acid pool levels while inhibiting abnormal bile acid synthesis. The clinical use of cholic acid has been documented in medical literature for several single enzyme bile acid synthesis defects since the mid-1990s.
“This agreement could represent a significant milestone for both our company and patients suffering from bile acid disorders,” said Gary Pasternack, Chief Executive Officer of Asklepion. “If cholic acid is approved, we are confident that Retrophin’s expertise in this area would allow for the greatest number of patients to benefit from this treatment.