Celgene's Once-Nightmare Drug Turned Blockbuster Returns

Celgene's Once-Nightmare Drug Turned Blockbuster Returns August 22, 2016
By Mark Terry, BioSpace.com Breaking News Staff

For people who grew up in the 1960s and 1970s, the drug thalidomide has extremely negative associations. Which is why it’s startling that the drug, using the name Thalomid, is an extremely successful drug marketed by Summit, New Jersey’s Celgene Corporation . Recently unsealed documents from a whistleblower lawsuit show details about how the drug associated with a generation of birth defects, had come to be a highly touted cancer drug.

Thalidomide was first marketed as an over-the-counter sedative in West Germany in the 1950s under the trade name Contergan. A little later it was marketed as an anti-nausea medication and to alleviate morning sickness in pregnant women. In 1960, the U.S. Food and Drug Administration (FDA) rejected the drug for morning sickness over concerns about side effects.

It was also marketed in the UK, Australia and New Zealand as Distaval. Although never approved for marketing in the U.S., it was distributed for testing purposes.

Not long after the drug was approved in Germany, children began being born with phocomelia, a malformation of the arms and hands. Approximately 40 percent of those afflicted survived. Worldwide, about 10,000 cases were reported, with a global survival rate of 50 percent. The most common defects were “flipper-like hands” or even stumps. There were other effects as well, including eye and heart deformities.

After being pulled from the market, thalidomide was studied in a number of different diseases. Eventually it was used to treat leprosy in other parts of the world. During the 1990s, a study suggested it could be used to treat HIV-related weight loss. Because it was an immunomodulator, it was also considered as a possible treatment for blood cancers, such as multiple myeloma.

The FDA didn’t approve it for use against leprosy until 1998. And in 2005, Celgene got approval for its version of thalidomide, Revlimid, for mantle cell lymphoma, and a year later, for multiple myeloma.

“There aren’t a lot of lepers in the United States,” said Robert D’Amato, a surgery director at Boston Children’s Hospital told Bloomberg, “but there are a lot of people who wanted to use it for other uses.”

Thalomid, Revlimid and Pomalyst, all variations on thalidomide, brought in $6.97 billion for Celgene in 2015, more than 75 percent of the company’s revenue. It expects to double those sales by 2020 to more than $21 billion.

Analysts have indicated that Celgene is the fastest growing biotech stock.

A whistleblower lawsuit was filed in 2010 by Beverly Brown, arguing that Celgene “defrauded government insurance programs by marketing its drugs for off-label uses.” The U.S. Justice Department didn’t join the lawsuit, but it was cleared for trial by a California judge.

“These allegations, which date as far back as 15 years, are baseless,” Brian Gill, a Celgene spokesman, said in a statement. “Celgene is committed to patient safety, and its products have improved and extended the lives of many thousands of cancer patients.”

Brown’s lawsuit argues that Celgene hired 100 sales staff and trained them specifically to push Thalomid and its other thalidomide-based drugs to oncologists, even though it had not been approved for use in cancer. Bloomberg notes, “From the beginning, off-label prescriptions drove Thalomid’s sales. In the first 12 months after its U.S. debut for leprosy, about 70 percent of the prescriptions were for oncology patients, Celgene said in an annual filing. In 2000, six years before Thalomid was approved as a cancer treatment, oncological cases accounted for 92 percent of prescriptions, according to the filing.”

As some of the now unsealed paperwork suggest, there are indications that at least some of the company’s staffers were covering up evidence of possible blood clot risks in cancer patients. Brown brought up an October 2000 email from Todd Clark, who was involved in drug safety at Celgene, which seems to chastise another employee about reporting side effects in an email because it “is potentially a glaring red flag to the FDA.”

Clark apparently also was warned by Jerry Zeldis, Celgene’s chief medical officer, in a February 2001 email, noting that “this is a non-erasable message that can be audited by the FDA.”

At this time, Celgene is apparently arguing that Brown’s interpretation of the emails is incorrect, and that the email messages were a reminder to employees to keep the FDA informed of possible side effects. Clark, in a court document, also stated that the company had instructed sales staff to report any issues.

In addition, Celgene launched a speakers program to physicians to try and overcome the bad PR associated with thalidomide. In 2015, Celgene paid 159 doctors $2.1 million to speak about Revlimid, Thalomid and Pomalyst.

Brown’s lawsuits also accused the company of colluding with medical charities in order to increase sales of the drugs.

“Ms. Brown is wrong and her allegations are baseless,” a Celgene spokesperson told Fortune. “[The government] has issued guidance related to donations by medical innovators to charitable patient assistance programs. Celgene complies with that guidance with respect to its donations to patient assistance programs.”

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