The approvals follow a protracted period in which Genzyme suffered repeated manufacturing gaffes that led to a consent decree with the FDA, a $20 billion takeover by Sanofi and litigation by some Fabry patients over rationing that allegedly caused to at least three deaths and prompted a request to the National Institutes of Health for a license to develop an alternative source of production.
Now, though, Genzyme hopes to restore supplies over the next few months to patients in both the US and Europe. Following the EMA approval, the most severely affected patients in Europe are expected to receive full doses between now and the end of March. And beginning in March, all existing patients in the US will be returned to full dosing, according to the drugmaker (see the statement).
Wall Street cheered, given that production problems were hanging over Sanofi and prompted some to question whether the Genzyme acquisition would pay off. "This is a critically important step in the validation of the acquisition as Framingham was the key gating factor in a recovery toward historic peak sales of $5 billion," Leerink Swann analyst Seamus Fernandez writes in an investor note. Of course, this places pressure on Shire Pharmaceutical, which is seeking FDA fast-track approval for Replagal, a rival medication.
However, an attorney who represents some Fabry patients, who had filed a lawsuit against Genzyme over the shortages, had a mixed reaction to the plant approval. On one hand, Allen Black says his clients welcome the news that supplies should soon be restored to proper levels. On the other hand, he remains troubled that European patients will have access to Fabrazyme before US patients, given that Replagal is available in Europe.
"If you read the statement (from Genzyme) closely, it says that European patients are being triaged ahead of US patients. Why is that happening when (Europeans) have access to Replagal? It's the same issue we've been raising for some time and has been a fundamental problem," he says, noting that Replagal is sold in Europe by Shire Pharmaceuticals, which is seeking FDA approval. "Americans paid for the invention (of the drug), so why are they waiting in line behind Europeans?" His clients petitioned the NIH to override use patents held by Genzyme and have been seeking a so-called march-in petition. Their hope had been to secure rights that will attract a partner interested in producing a sufficient supply of the medication in light of ongoing shortages. They had also sued Mt. Sinai School of Medicine, which had been awarded an NIH grant and invented Fabrazyme (back story).
Despite the regulatory approvals, the lawsuit, Black adds, will proceed and his clients intend to seek damages from the drugmaker and the medical institution. "Hopefully, they can get patients back on supply before they die," he says. "The whole point of the lawsuit was to get recovery for injuries that Genzyme caused by not providing full doses."
One of his clients readily agrees. "With the capacity this facility will have, we'll hopefully only be talking about a difference of a few weeks" between restored supplies for US and European patients, says Michael Masula, a construction project manager in Pittsburgh whose supply of Fabrazyme has been rationed for nearly three years. "But there was negligence. We shouldn't have been in this situation. Nobody should be allowed to get away with what they've done."