A Lupus Drug and FDA Approval

Abstract

We are concerned about misinformation in the News Focus article “Lupus drug company asks FDA for second chance” (J. Couzin, 11 Feb., p. [835][1]). It is not true that the U.S. Food and Drug Administration (FDA) has rejected the medication LJP 394, which is under active development by La Jolla Pharmaceutical Company. LJP 394 is currently undergoing testing in an ongoing clinical benefit trial for lupus nephritis that could not occur without the approval and guidance of the FDA. In fact, we understand that this trial is being conducted under a Special Protocol Assessment with the FDA. It is also not true that the company has requested a “second chance” for the drug. The company has requested consideration of a special development pathway known as accelerated approval. Under this pathway, drugs might be marketed while further development studies are under way. Such an approach has been used numerous times by the FDA for diseases such as cancer and HIV. # Response {#article-title-2} As I reported, La Jolla Pharmaceutical Company submitted an application to the FDA detailing over a dozen clinical trials of its lupus drug, LJP 394. Last October, the FDA notified the company that it was declining to approve the drug on the basis of existing data and that an additional, large trial was needed before the drug could be approved. The FDA agreed that a multiyear trial begun the previous summer would suffice. (That trial was originally conceived by the company as a postmarketing trial that would continue while the drug was sold.) The FDA's decision not to approve LJP 394 without further data amounted to a rejection of the company's new drug application. After the FDA declined to approve LJP 394 without the additional study, La Jolla Pharmaceutical Company asked the FDA for “accelerated approval,” which would allow it to market LJP 394 while the trial was taking place. This request, initiated because the company was struggling to pay for costly clinical trials, formed the basis of the article. I believe that my article fairly characterized this appeal as requesting a second chance. [1]: /lookup/doi/10.1126/science.307.5711.835