Bone drug based on rare disease could get FDA reprieve

It’s the disease that became a Big Pharma “eureka moment.”

Sclerosteosis only affects about 100 people around the world who can carry a defective gene that makes their bones keep growing to the point of life-threatening pressure on the brain.

When a biotech company in the UK started looking into sclerosteosis, however, they didn’t find a cure for this rare genetic condition… but rather a new drug for a much more common ailment — osteoporosis.

And while the FDA just put the brakes on this med due to the finding of some serious heart problems, it also left the door wide open for drugmaker Amgen to try another way to get it on the market.

Amgen must have thought romosozumab would be well on its way to FDA approval by now. The drugmaker probably even paid a small fortune to an ad agency to come up with a fancy name for it.

But while we don’t know what they’re going to end up calling it, here’s what I think it should be named — Rejecta. That would remind patients just how risky this new med must be for the FDA to say “No” — because you know how seldom that happens!

Rejected — but for how long?
Amgen got its inspiration for its med romosozumab from the extremely rare condition called sclerosteosis that I mentioned before.

Those with the condition — just a handful of people, mostly descendants of the Dutch settlers of South Africa (a.k.a. “Afrikaners”) — have inherited a gene that blocks an important protein that, when working properly, keeps our bones from growing out of control.

The “plan” was that, by inhibiting that same protein with this drug, women who take it will lose less bone and might actually form more new bone.

But, in the case of sclerosteosis, the problem is that the bone growth could occur all over your body — including your skull.

With that protein blocked, the abnormal bone growth eventually starts pinching on the nerves coming from the brain, causing a total loss of smell, facial paralysis, deafness, blindness, and death.

That’s certainly a great idea to model a drug after! What could possibly go wrong with that, right?

Yet Amgen has conducted a total of three trials on the drug — none of which tells a very encouraging story on its own.

One called FRAME showed that the med didn’t work too well, though no serious risks were found. The next, dubbed ARCH, showed its effectiveness to be comparable to the drug Fosamax — but, as trade pub FierceBiotech noted, that slightly more positive data was “tarnished” when some big heart risks came to light.

That’s why the FDA officially rejected it. (And thank goodness it did!)

The FDA has previously denied requests to “pool” data from multiple studies, but never say never when it comes to the agency. In the case of Amgen, it has agreed to allow the company to try and razzle-dazzle the feds into saying yes by combining those results with ones from a third trial (called BRIDGE), done on just a few hundred men.

And that may prove to be the ace up Amgen’s sleeve.

Despite the fact that this med will eventually be prescribed to hundreds of thousands of women, testing it on 245 men may be how Amgen researchers will finally find a way to spin that data so it looks amazing.

What all this means is that a risky med, discovered and developed based on a terrible disease, may soon hit your doctor’s office and be handed out to women worried about breaking a bone.

And, in the process, you won’t hear a word about its strange and troubled past.

All this is especially disturbing because there are some very effective ways to naturally keep your bones strong.

One is to harness the power of sunshine. Ten minutes or so outside on a sunny day will allow your body to make its own bone-protecting vitamin D.

You can also regularly take supplements of vitamin D, which works well on its own but will protect your bones even more if you take it with vitamin K.

Also, adding K (which you can get in the form of supplements made from natto or nattokinase) is especially important if you’re taking calcium — as it sends the mineral to your bones where it’s needed and not to your arteries, where it can cause trouble.

“Safety scare prompts FDA to reject Amgen’s romosozumab, Nick Paul Taylor,” July 17, 2017, FierceBiotech, fiercebiotech.com