Dive Brief:
- Israel-based Taro Pharmaceuticals will distribute its rare disease drug Keveyis free of charge, after negligible sales and difficulty identifying patients. Taro will continue to manufacture the drug, but all commercial sales and marketing will be halted.
- Approved by the U.S. Food and Drug Administration in August 2015, Keveyis treats a hereditary paralytic disorder known as primary periodic paralysis. An estimated 5,000 patients are affected in the U.S., but many go undiagnosed - creating an apparently unsurmountable hurdle for Taro's commercialization plans.
- The company had already provided the drug free of charge to some patients through a financial assistance program. But commercial sales totaled less than $1 million dollars since launch last year, which Taro says is inadequate to support marketing for the drug.
Dive Insight:
Despite investing "significantly" in Keveyis, Taro has had difficulty in finding patients to treat. Less than 1,500 people are estimated to be diagnosed with periodic paralysis, limiting an already tiny patient population. Taro had hoped to treat a few hundred people with Keveyis, but now says identifying and marketing to patients has proven too difficult.
"Given the high costs and resources required to identify and reach a limited number of viable patients, Taro realizes that it cannot sustain its current level of investment," the company explained. While Taro will still produce the drug, it will be made available at no cost to distributors.
But Keveyis also has a substantial risk profile, with burning sensations, difficulty thinking, changes in taste, and confusion listed as the most common side effects. The drug also can cause falls in elderly patients.
Although Keveyis is the first medicine approved to treat the condition, Taro's short-lived attempt to market the drug underscores the difficulties in reaching patients and raising awareness for ultra-rare diseases.
Even Martin Shkreli, the notorious former CEO of Turing Pharma and KaloBios, didn't know what to make of it.
$TARO makes a rare disease drug free. Not sure if that's good or bad. Very interesting read: https://t.co/SXvCYE2qml
— Martin Shkreli (@MartinShkreli) May 2, 2016
Taro itself is an interesting company. Based in Israel, it primarily sells to wholesalers in the U.S., with almost all of its sales coming from its dermatology products. The Indian drug giant Sun Pharma owns a nearly 80% stake in the voting shares of Taro.
In February 2013, Sun dropped an effort to buy out the remainder of Taro and take it private (Sun owned just over 60% of Taro then). Taro's board had agreed to the deal in August 2012 but Taro shareholders rejected the price Sun offered. A number of Sun executives now run Taro, including its CEO and Chairman of the Board.