Retrophin, based in San Diego, announced that the U.S. Food and Drug Administration (FDA) had given its 100 mg and 300 mg tablets of Thiola EC (tiopronin) the thumbs-up to treat cystinuria. Cystinuria is a rare inherited disease that results in a buildup of cystine levels in the urine, causing the development of recurring cystine kidney stones.
Retrophin focuses on developing drugs for rare diseases. In addition to Thiola, it has Cholbam (cholic acid) for bile acid synthesis disorders caused by single enzyme defects and as adjunctive treatment of peroxisomal disorders such as Zellweger spectrum disorders, and Chenodal (chenodiol) for cholesterol gallstones.
“This approval of Thiola EC marks another step in our continued commitment to helping patients with cystinuria manage the threat of recurring cystine stones,” stated Eric Dube, chief executive officer of Retrophin. “This new formulation provides patients with the freedom to administer Thiola EC with or without food, an advancement over the original formulation which has limiting food restrictions, and also provides the potential to reduce the number of tablets necessary to manage cystinuria. We look forward to working with the cystinuria community as we make the new formulation available next month.”
And by next month, the company refers to July 2019.
Today’s approval is for another formulation of the drug.
“Thiola’s utility as the treatment of choice for cystinuria is well established,” stated David S. Goldfarb, clinical chief, Division of Nephrology at NYU Langone Health. “However, for certain patients, the challenges of administration one hour before or two hours after meals three times a day, coupled with a high pill burden, have been challenging. Having a new treatment option with the flexibility of dosing with or without food, as well as one that provides an opportunity for patients to take fewer pills, should meaningfully improve convenience and compliance.”
Two weeks ago, the company settled with the so-called “pharma bro,” Martin Shkreli. He founded Retrophin in 2011 but was fired by the company three years later. Shkreli is currently serving a seven-year prison sentence for fraud. In 2017, Shkreli was found guilty on two charges of securities fraud and one charge of conspiracy to commit conspiracy fraud.
Shkreli became publicly famous for increasing the price of a drug acquired by another pharma company he founded after leaving Retrophin. He increased the price of an AIDS drug by 5,000% and his smirking countenance in the media made him the poster child for greedy pharmaceutical companies.
The company indicated that the deal with Shkreli covered his $30 million lawsuit accusing three former colleagues of fraudulently collaborating to fire him and profit from his work.
The settlement, according to Reuters, “also covers claims Retrophin first raised in August 2015, in a $65 million lawsuit accusing Shkreli of repeatedly breaching his duty of loyalty to the San Diego-based company. That case moved to arbitration last year.”
Shkreli voluntarily dismissed the case on June 18. It was apparently “voluntarily dismissed, with prejudice,” according to CNBC, meaning that Shkreli can’t file a similar lawsuit in the future against the defendants.
The former colleagues included Stephen Aselage, who replaced Shkreli as chief executive officer, Gary Lyons, Retrophin’s chairman, and Margaret Valeur-Jensen, who took on the role of general counsel after Shkreli left the company.