The share price of American biotech company BioMarin Pharmaceuticals dipped Wednesday after the U.S. Food and Drug Administration rejected its new gene therapy. The agency wants longer-term data before it can approve the treatment for distribution and use.
The company said Wednesday its new gene therapy treatment for bleeding disorder hemophilia A was rejected by the FDA. The rejection is expected to push the launch of the new treatment to sometime in 2022. Following the announcement, BioMarin's shares fell by as much as 31 percent.
From a close of $118.54 per share Tuesday the stock price fell to an opening of $85.75 per share Wednesday. BioMarin's shares eventually closed lower at $76.72 per share.
Investors, doctors, and the company had expected the new treatment to be approved this month. Initial clinical trials had mostly shown the treatment was effective in reducing the bleeding rate of patients suffering from hemophilia A. The treatment would have decreased or eliminated the need for further treatment for about 16,000 hemophilia A sufferers in the U.S.
The current treatment for the disorder involves the use of factor proteins - which have to be injected several times a week. The lifetime treatment costs patients hundreds of thousands of dollars a year. BioMarin's gene therapy treatment would have eliminated that - savings patients a lot of money.
The company's proposed gene treatment won't be cheap, however. BioMarin has said the new treatment could cost between $1 million to $3 million - making it one of the most expensive one-time treatments.
The FDA has demanded the company submit more data, BioMarin said. This includes two years' data for its continuing late-stage study. The company's chief executive officer Jean-Jacques Bienaime said he was very disappointed at the rejection and that the treatment was being subjected to the agency's sudden change in requirements.
The data the company originally submitted to the FDA included a three-year phase 1/2 data and a phase 3 interim analysis. The FDA claimed the data limited its ability to properly examine the long-term efficacy of the treatment.
