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Who: Questcor (QCOR: $10.02) is a small pharmaceutical company seeking an expanded indication for its treatment Acthar. Acthar is currently mainly used in multiple sclerosis and off-label for infantile spasms. The company only has two main products: Acthar and anti-anxiety treatment Doral.
What: Acthar has been submitted already to the FDA and rejected. The FDA requested more data.
Consequences: As recently as December 10, 2009, the price of Questcor was $4.08 a share.
Analysis: Questcor’s application has significant issues that may affect approval.
Conclusion: There is at least a 25% chance that Acthar will not be approved.
The drug application for Acthar’sapproval contains multiple issues relating to the collection of data and various irregularities. The studies it uses to support its drug as safe and effective each contain significant issues.
We present at the end selected citations from the FDA review panel which highlight these issues.
The company Questcor has presented three studies to the FDA in seeking approval of its treatment. The FDA in the previous application had requested data from five studies but says that the company did due diligence in obtaining the data it did.
But officially, the FDA can approve an “orphan drug” on the basis of just one strong study but the interpretation of the clause that says so is not fully determined.
Study 01 is the pivotal study, studies 04 and 05 are supporting.
There are problems with each of the studies and any one of them would probably not be adequate for approval. They are academic studies and not the clinical research studies typically used in drug applications – this means that the data from them was not collected normally and is “likely to be incomplete.”
Study 01 is from 1996 and is of only 29 patients. It was not a proper research study so it did not have the standard statistical analysis. Although it is the pivotal study, the safety data from that study is not acceptable as it was not collected properly.
Study 05 showed no difference in efficacy between a high dose of Acthar and a low dose.
Study 04 showed no superiority of Acthar over existing treatment. It seems that the data recording was flawed and that safety information from this study is also not included.
Safety data is mostly from hospital charts.
The FDA panel voted 22 to 1 to approve Acthar and 20 1 that it was safe. That said, a subvote on evidence of manageability of side effects was 12 to 10 against.
The vote in favor of Acthar could possibly be largely based off the existing use of Acthar as a treatment for IS. Panelists may have felt it would be wrong to vote against a drug for a use they were using it, but such common use did not prevent the FDA from rejecting it previously.
Because of these various irregularities and issues, it is likely that there is at least a 25% chance that the FDA will not approve Acthar.
Arguments for approval
Acthar is already being used for infantile spasms. It has orphan drug designation, the standards for which are significantly easier than normal. The panel voted strongly for approval.