It’s impossible to put a price on a child’s life. But if your child suffers from a rare blood disorder called atypical hemolytic-uremic syndrome, you can expect to shell out more than $400,000 per year because that’s the price of the antibody drug that just received regulatory approval in the US to treat the clotting disease.

Atypical hemolytic-uremic syndrome (aHUS) is a life-threatening genetic disease affecting fewer than 1,000 Americans in which red blood cells break apart as they squeeze through small blood vessels leading to anemia, abnormal bleeding and kidney failure. Plasma exchange has become the standard of care for more than 30 years, but many people with aHUS do not tolerate or respond to the therapy. And even when it works, the procedure is time-consuming and not totally risk-free. So doctors have long sought treatments that actually target the root of the disease — the excessive activation of the complement system used by the immune system to clear pathogens from the body.

In 2009, two single-patient case reports came out showing that Alexion’s Soliris (eculizumab) helped an 18-month-old boy and a 30-year-old woman stabilize their disease. (The monoclonal antibody, which is directed against the complement protein C5, had been on the market since 2007 for the treatment of another rare blood disorder called paroxysmal nocturnal hemoglobinuria.) Alexion then launched two prospective open-label trials and a third retrospective study in a total of 56 people with aHUS. Reporting this summer at the European Hematology Association annual meeting in London, the company showed that the vast majority of those study subjects had no blood clots, improved kidney function and a better quality of life.

Last week, off the back of those results, the US Food and Drug Administration approved Soliris for all age groups with aHUS. Alexion also announced that the European Medicine Agency’s Committee for Medicinal Products for Human Use had given the product a favorable review. A final decision from European regulators is expected within two months.
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Posted on behalf of Madhumita Venkataramanan

The stock prices of companies that report positive results for cancer drug trials tended to increase before the first public announcement of the findings, according to a study published today in the Journal of the National Cancer Institute. But experts are split as to whether the trend points to illegal ‘insider trading’ by people who know the results of these trials in advance — including physicians and scientists — or simply reflect a bias in the analysis.

In the study, a team led by pharmacoeconomist Allan Detsky of Mount Sinai Hospital in Toronto identified 59 cancer drugs in US phase 3 trials and those awaiting FDA approval from January 2000 to January 2009. Of these, 23 trials were positive and 36 were negative. The scientists retrospectively examined the stock prices of the pharmaceutical companies for 120 days before and after the first public announcement of the drug trial outcome.

The results of the study showed a surprising trend. “Companies that made a positive announcement showed a trending up in [stock] prices 60 days prior that was different from companies with a negative announcement,” says Detsky. “And the difference is statistically significant.” The stock price for companies that were due to report positive trials showed an increase of 9.4% while prices for those anticipating negative results showed a decrease of 4.5%.

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Hormones aren’t the only excuse for teenage moodiness anymore: psychiatrists are increasingly diagnosing adolescents with bipolar disorder, with 65% more prescriptions and 35% more patients between 2002 and 2009, according to the US Food and Drug Administration (FDA). Many people are wringing their hands over whether children are being misdiagnosed and, perhaps, unnecessarily medicated. But there’s another problem with the diagnosis spike: potential side effects from the antipsychotics doctors prescribe to treat the disorder.

Yesterday, the FDA’s Pediatric Advisory Committee reported new data about the occurrence of obesity and diabetes in children taking antipsychotics. The report, presented by Tobias Gerhard, an epidemiologist at Rutgers University in New Brunswick, New Jersey, compared the development of obesity and diabetes in children and adolescents taking the five antipsychotics currently approved for pediatric use by the FDA. Although they found no difference in side effect occurrence among the various drugs, the committee voted to continue to study the issue, and to update the warning label on only one of the five antipsychotics approved for pediatric use, Bristol-Myers Squibb’s Abilify (aripiprazole), despite the finding of clinical equivalence.

Yet even though the panel’s report revealed no difference in side effect severity between the medicines, other studies have come to different conclusions. For example, a meta-analysis published just last month found that some drugs had a greater effect than others, compared to placebo. For example, Eli Lilly’s Zyprexa (olanzapine) was associated with greater weight gain than Abilify, AstraZeneca’s Seroquel (quetiapine), or Janssen’s Risperdal (risperidone). And children taking Zyprexa had the highest cholesterol of all studied antipsychotics, the study authors found.

These conflicting results and the vague conclusion by the committee — to simply continue studying the side effects, as they similarly decided back in 2009 — are a bit befuddling, as the question of negative antipsychotic side effects should warrant more significant action. And the single dissenter in the 16-1 FDA committee vote, Jeffrey Wagener, a pediatric pulmonologist at the University of Colorado School of Medicine in Aurora, agrees. He voted against continued study because he doesn’t think it’s doing enough to confront the issue of metabolic side effects from antipsychotics in children and wants more action.

“I don’t see how the FDA is responding to the … request by this committee in a thorough fashion,” he told Reuters. “It’s taken them two years to not respond to [the fact] that we need to be more than in the observational role.”