FDA Panel Rejects Vion Leukemia Drug

NEW HAVEN, Conn. (TheStreet) -- The FDA's cancer drug advisory panel voted unanimously against approval of Vion's leukemia drug Onrigin.

By a 13-0 vote, the panel recommended that a new randomized controlled trial be conducted before the drug is approved. The same panel rejected Genzyme's leukemia drug during a morning session for similar reasons.

Vion shares closed Tuesday down 23% at $2.60.

Vion Makes Case for Leukemia Drug to FDA Panel

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NEW HAVEN, Conn. (TheStreet) --After dinging Genzyme's leukemia drug this morning, the FDA's panel of cancer experts is back this afternoon to review Vion Pharmaceutical's(VION.OB Quote) leukemia drug Onrigin.

Vion is seeking Onrigin's approval as a treatment for elderly, poor-risk patients with acute myeloid leukemia. This is the same cancer type discussed this morning on the Genzyme panel but the patients treated with Onrigin are much sicker.

But like this morning's session, Vion and the FDA are at odds over the adequacy of the single-arm trials designed to support Onrigin's approval. In addition, the FDA has raised serious safety concerns about Onrigin.

In its presentation to the advisory panel, Vion's representatives made the case that the elderly patients enrolled in its Onrigin studies had a number of risk factors making it difficult if not impossible to treat them with standard chemotherapy drugs.

Despite the poor prognosis for these patients, treatment with Onrigin led to a 32% remission rate. For those patients who responded to Onrigin treatment, the median overall survival was greater than one year.

Fourteen percent of patients treated with Onrigin died within a month of treatment, but Vion said this rate of mortality was not much different from that seen with other drugs used to treat these patients. Moreover, lung toxicity and higher infection rates were also not surprising given the poor health status of these patients as they entered the study.

The FDA's presentation was more negative. The agency's reviewer said that about one third of the patients who responded to Onrigin were likely healthy enough to be treated with standard chemotherapy. More than half of responding patients also received another chemotherapy drug in addition to Onrigin, which makes it difficult to separate out the contribution of Onrigin.

Moreover, the responses in Onrigin-treated patients were brief, with 38% lasting less than 90 days.

The FDA's review spent a lot of time discussing lung toxicity associated with Onrigin, which led to patient deaths and a large part of the serious adverse events reported in the study. Again, without a randomized controlled trial, FDA said it was difficult to ascertain whether the drug was to blame for the lung toxicity or whether it was due to the underlying disease of the patients. The FDA noted that other lung toxicity was also an reported adverse event in other Onrigin studies.

Like Genzyme, FDA recommended to Vion that it conduct a randomized controlled study in elderly, high-risk AML patients, but the company did not heed that advice.

With the FDA and Vion presentations completed, the advisory panel will spend the rest of the meeting asking questions and debating the Onrigin clinical data. Check back later for another update.

Vion shares were down 19% to $2.73 in recent trading.

FDA Panel Turns Down Genzyme Cancer Drug

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CAMBRIDGE, Mass. (TheStreet) -- The FDA advisory panel reviewing Genzyme's(GENZ Quote) cancer drug Clolar recommended that a new randomized controlled trial be conducted before the drug is approved.

The panelists, in a 9-3 vote, expressed their belief that Clolar is an active drug for elderly patients with acute myeloid leukemia (AML) but that the data submitted by Genzyme from a single-arm study wasn't sufficient for approval.

In this regard, the cancer experts on the panel concurred with FDA officials who criticized Genzyme for not conducting a randomized clinical trial that would have compared Clolar against some other treatment.

The negative vote by the panel was likely buffered by the fact that Clolar is already an approved drug for pediatric cancer patients, which means doctors can use the drug off-label to treat elderly AML patients today.

The panel also seemed concerned that Genzyme has yet to start a confirmatory study of Clolar in pediatric patients -- a commitment the company made to FDA when the drug was granted accelerated approval in 2004.

Genzyme officials said that confirmatory trial in children would start next year. The company also said it is already conducting randomized, controlled studies of Clolar in elderly AML patients.

Genzyme shares were down 84 cents to $54.87 in recent trading.

Sequenom Down Syndrome Test Rumor False, Report says

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SAN DIEGO, Calif. (TheStreet) -- The market rumor that pushed Sequenom(SQNM Quote) shares sharply higher Monday was false, according to newspaper report.