Washington Editor
The FDA issued a not-approvable letter to IDM Pharma Inc. for its osteosarcoma treatment Junovan (mifamurtide) and told the company it needs to submit more clinical trial data.
Regulators followed the recommendation of an FDA advisory panel that voted 12-2 in May that the results of a single Phase III clinical trial, known as INT 0133, did not provide substantial evidence of the effectiveness of Junovan in the treatment of patients with nonmetastatic, resectable osteosarcoma receiving combination chemotherapy. (See BioWorld Today, May 10, 2007).
Osteosarcoma is an uncommon tumor of childhood but is the most common tumor of bone, according to the FDA. It is the fifth most common cancer of adolescents. About 400 new cases of the disease are reported annually in the U.S.
The FDA generally requires two clinical studies demonstrating efficacy to support a new drug application. However, regulators will accept data from a single trial if the study provides evidence of an important clinical benefit that is so highly reliable and statistically strong that confirmation in a second trial would be ethically impossible, the agency stated in documents.
But the results of IDM Pharma's single study were not strong enough to win approval of the drug. Wall Street took notice, with shares of IDM (NASDAQ:IDMI) falling 20 percent, or 41 cents, to close at $1.61.
Ciba-Geigy, which merged with Sandoz in 1996 to form Novartis, held the original investigational new drug application for Junovan from 1988 to 1996. In 1996, the agent was acquired by Jenner Technologies, now Jenner Biotherapies. IDM Pharma acquired it 2003.
The Irvine, Calif.-based firm submitted study results from INT 0133, an open-label, prospective multicenter, randomized study conducted by two pediatric cooperative groups, to the FDA in October 2006. Nearly 800 patients were enrolled in the study between 1993 and 1997.
The FDA told IDM Pharma that the agency wants data from additional clinical trials to demonstrate the benefit of Junovan, which received orphan drug status in 2001. Regulators also requested "information or clarification with respect to other sections of the NDA," the firm stated.
In July, the company announced its intent to amend its new drug application for Junovan with additional survival data. The firm said it had anticipated then that the FDA would issue a not approvable letter.
Although regulators have asked for more clinical trials, IDM Pharma said it believes that "capturing supplemental data will overcome the need for additional trials and further confirm the overall survival benefit" of Junovan in patients with osteosarcoma and will "provide evidence for approvability."
The firm said that is plans to continue working with the two pediatric cooperative groups and investigative sites involved in INT 0133 to collect survival information on study participants, which was not available at the time the NDA was originally submitted.
When that data have been collected, IDM Pharma said, it will analyze it and submit an amendment to its NDA by the first quarter of 2008.
The firm said it also will address other issues raised by the FDA in its not approvable letter.
IDM Pharma is also seeking marketing approval from the European Medicines Agency for Junovan.
Before IDM Pharma went public with the FDA's decision, the cancer patient advocacy group A Right to Live last week said that it plans to hold a rally at the agency's Rockville, Md., office on Sept. 18 to support the approval of Junovan and other cancer products, such as Dendreon's prostate cancer vaccine Provenge (sipuleucel T).
An FDA advisory panel in March recommended approval of Provenge. But in an unusual decision announced in May, the FDA ignored that advice and told Seattle-based Dendreon that more supporting efficacy data was needed. (See BioWorld Today, May 10, 2007).