FDA Says Drug Doesn’t Work – Then Approves It Anyway!

Vyndaqel—a drug that has been shown not to limit a disease’s progression and has not been compared with other drugs that treat the same symptoms—was just approved by the FDA. I think that most readers can figure out why it happened.

by Heidi Stevenson

The FDA has replaced the two-faced god, Janus, in these modern times. In spite of finding that Pfizer had been unable to demonstrate that their new drug, tafamidis meglumine (Vyndaqel), works, they approved it anyway!

By a vote of 13 to 4, the FDA’s Peripheral and Central Nervous Systems Drug Advisory Committee, acknowledged that Vyndaqel doesn’t work. Then, they again voted, by the same margin of 13 to 4, to approve it.

Apparently, lack of another drug to treat a rare disorder justifies approving one that simply doesn’t work. By the FDA’s thinking,  filling a gap with empty space has make sense.

Bizarre and Twisted Justification

The disorder in question is terrible. Familial amyloid polyneuropathy (FAP) is inherited and results in progressive deterioration of nerve function resulting in a wide array of miserable symptoms, including numbness and pain, and loss of function of limbs and organs.

Inadequate Trial

The justification used for approving Vyndaqel is that, though it failed to slow FAP’s progression, it does ease some symptoms. However, there are significant reasons to doubt the validity of these results:

• Symptoms were not the primary endpoint of the study, but only secondary. The study, therefore, was not designed to provide accurate information about them.
• The study was of the intention-to-treat variety, which means that the results for people who do not continue the treatment for any reason, including dropping out of the study, are included as if they had, thus skewing the results.
• About 20% of the subjects dropped out of the study.

FDA Requirement to Compare with Other Drugs Not Met

Not only are the results questionable, but the testing process did not include a primary FDA requirement. Vyndaqel was not compared with any existing drugs to see if it provides better results. The FDA’s own rules require such a trial comparison before granting approval. In their industry guidance for the drug approval process, Section I(iii)(a) states:

There is no comparable or satisfactory alternative drug or other therapy available to treat that stage of the disease in the intended patient population.

No other drug was used for comparison, so a primary rule— written by the FDA itself—was broken in the rush to approve a drug that they admit wasn’t beneficial!

It makes no difference that a drug can ease a symptom unless it’s either the only drug that can do so or is compared with others to see if it does better. Without such a comparison, we have no way of knowing if the drug is worthwhile.

Why the Rush to Approval?

A factor that makes Vindaqel unique is that it’s classed as an orphan drug. Such drugs are expected to have limited use because there is limited application for them. FAP is a rare disorder, so Vindaqel was given a fast-track status under the Orphan Drug laws. Orphan Drugs are, therefore, given some leeway in the approval process. However, if there are other drugs that accomplish the same thing, then it still must be compared with existing drugs.

Apparently, Pfizer doesn’t expect Vindaqel to outperform any existing drugs for neurological symptoms, so they didn’t try to gain approval through that method. Instead, they asked for Orphan Drug status as a treatment for FAP. That gives them an easy ride to approval—and must have come as quite a surprise.

By the way, once a drug is on the market, it can be prescribed by doctors for anything they wish. Thus, you can be fairly sure that there will be some sort of pseudo science that gives the impression of Vyndaqel’s ability to benefit this disorder or that disorder. Once an Orphan Drug, there’s no law keeping it in that status.

How the sleight-of-hand that got approval in spite of not meeting the basic qualifications for approval would be an interesting tale—but without the view of a fly on the wall, it’s unlikely we’ll ever hear it.

The simple fact, though, is that Vyndaqel—a drug that has been shown not to limit the progression of FAP and has not been compared with other drugs that treat the same symptoms—was just approved by the FDA. I think that most readers can figure out why it happened.

Sources:

• FDA Panel Sends Mixed Message on Pfizer Drug
• Familial amyloid polyneuropathy
• Tafamidis meglumine Advisory Committee Briefing Materials
• Orphan Drug Act
• FDA’s Guidance for Industry

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