How a risky med meant for a few got an unconditional FDA OK

It may go down as the fastest (and shiftiest) way ever to get a drug approved.

Vanda Pharmaceuticals managed to get its new drug, called Hetlioz, in and out the FDA’s well-oiled door in just eight months.

On top of that, the drug was only tested for a mere six months — in only 52 people!

And here’s the danger: This new med, rushed through the FDA in record time, was only meant to treat a very small and specific group: people who are totally blind and suffer with a single kind of sleep disorder.

But something happened right as the drug was being OK’d by the FDA.

Something that will put this risky med into millions of medicine cabinets

Hiding in plain sight

It’s called “orphan drug” status. It’s reserved for meds designed to treat rare diseases, ones that affect less than 200,000 people in the U.S. Orphan drugs never make blockbuster status or bring in the billions.

Drug companies that develop these limited-use meds get lots of benefits. Things like tax incentives, longer patent protection and even money to do the clinical trials.

It’s considered a humanitarian effort, one that deserves all those extra perks.

And that orphan drug status is what Vanda put down on its new drug application for Hetlioz, a med to treat a condition called “non-24 sleep wake disorder” in those who are blind.

Non-24 happens in the blind who get no light “cues” as to when it’s night or day. Their circadian rhythms – the body’s internal “clock” — go seriously off schedule.

So Vanda got lots of help from the FDA, including that fast-track approval I mentioned. To develop a drug for something that only affects around 50,000 to 70,000 people will get you red-carpet treatment.

But somehow, right as the drug was being approved, the word “blind” went missing from the label. It wasn’t a typo, either.

In a letter from the FDA to Vanda, letting it know the drug was approved, the agency says that it made an error on the drug’s indications section. That “error” is redacted so I can’t read it, but miraculously in the corrected statement that all-important word “blind” is gone.

So it looks like the FDA threw the drug maker a really big bone, because without that word, Hetlioz can be prescribed to just about anyone who is having trouble sleeping.

And Vanda is taking full advantage of that, too. On the Hetlioz web site, it never bothers to mention that the drug was intended for only patients who are blind.

All of which puts anyone with trouble sleeping at risk for the drug’s common side effects of nightmares, UTIs, upper respiratory tract infections and liver damage.

Of course, since Hetlioz was rushed through the FDA, and only studied for a very short time in as few people as possible, we have no idea what other side effects will show up when it starts being prescribed to millions.

And we probably will never learn the real reason why this “orphan” drug — one that really isn’t — got such special treatment by the FDA.

But something tells me this might not be the first time we’re seeing this new trick. And that other drug companies may follow suit and use it as a way to get risky drugs on the market as fast as possible.

And with even less scrutiny than ever before.

Source:

“Sleep disorder treatment for blind persons may be marketed more widely” Worst Pills Best Pills Newsletter, January 2015, worstpills.org