In January 2010, the US Federal Drug Administration (FDA) approved AMPRYA (dalfampridine) extended release tablets to improve walking capabilities for patients with multiple schlerosis (MS). In clinical trials, patients who took AMPYRA had faster walking speeds than those who received a placebo. AMPYRA is the first drug that the FDA has approved for this purpose. This is exciting and promising news for people with MS, whose mobility is often very restricted by the effects of the disease.
AMPYRA is a product of Acorda Therapeutics, a biotechnology company based in Hawthorne, NY. Acorda’s stated mission is “to develop and market therapies to restore neurological function in people with spinal cord injury, MS and related conditions of the nervous system.” Recently, ABILITY Magazine’s editor-in-chief, Chet Cooper, talked science and corporate compassion with Tierney Saccavino, senior vice president of corporate communications for Acorda Therapeutics.
Chet Cooper: Tell me about how this drug works.
Tierney Saccavino: My boss describes AMPYRA as a “liquid bandage.” People with MS are disabled primarily due to demyelination, which is the stripping of the insulation around the nerves in the brain and spinal cord. The best analogy is that of an electrical wire that’s intact, but on which the insulation is stripped. When that happens, you get a short circuit. Even though the wire is there, the electrical impulse can’t be conducted without insulation.
You have bundles of nerves in the brain and spinal cord, and they are surrounded by packets of myelin, a sort of white, fatty insulation. In MS, the immune system causes the myelin to become damaged and worn away, and that causes a short circuit, because the insulation’s gone. The electrical impulse can’t be conducted any more.
Cooper: Are you saying that the myelin can actually regrow and that there would be no scarring?
Saccavino: No. When we say our drug is more like a liquid bandage, we mean that, while it’s active in your system, it temporarily covers the areas that have been destroyed. It eventually wears away, which is why you have to take a pill every 12 hours. But while it’s active in your system, it does cover over those exposed areas and allows the electrical impulses to continue on.
It doesn’t work for everybody. We haven’t been able to predict who will see a good result and who won’t, but we believe that has to do with the fact that some people may have too many areas of demyelination, or the little demyelination tracks may be too long, so, even with the drug, the electrical impulses can’t continue on their paths.
Cooper: The typical MS drugs— the ABCs (Avonex, Betaseron/Betaferon, Copaxone) or CRAB (the previous plus Rebif)—they don’t have this capability?
Saccavino: No. They do something very different. The currently approved immunomodulator drugs slow the progress of the disease. They stop the immune system from attacking itself.
Cooper: What I’m hearing, then, is that people would stay on their regular regime, because they want the immune system not to attack, and this would be an added value to mitigate the symptoms that might have occurred already from the reduction of myelin?
Saccavino: That’s exactly right. In our clinical trials, we found that people who were taking the immunomodulators had the same chances of responding well as those who didn’t. This was true no matter what kind of MS you had and no matter how long since you’d been diagnosed. So, it seems as though pretty much everybody has an equal chance at having a good response. A patient with MS can certainly continue to take it along with existing immunomodulators.
Chet Cooper: AMPYRA. Does that mean anything?
Saccavino: Not that I know. I think it is derived somewhat from the “amp” in Fampridine.
Cooper: It was previously called—?
Saccavino: It was originally called Fampridine, which is the former USAN, or US Adopted Name. The chemical name is 4-aminopyridine, and the FDA asked us to change it as we were approaching approval, because they thought that there might be some confusion between Fampridine and the USAN name of another product. So we agreed to change it to Dalfampridine.
Cooper: Had Fampridine been on the market before? Saccavino: It had not. It has certainly not been approved by regulatory authorities in the US before, and to my knowledge, not anywhere in the world. Compounding pharmacies have made it with raw ingredients for some time, but those formulations are not at all like ours.
Cooper: Are you saying that this drug has not been used by people with MS before now?
Saccavino: In clinical trials it has. And 4-aminopyridine, the chemical compound, has been used by prescription through the compounding pharmacies I mentioned. Scientists and physicians have been aware that this chemical compensates for demyelination for a couple of decades.
Cooper: And that compounding formula would not have the same results as AMPYRA?
Saccavino: In terms of results, I don’t know. Compounding is an unregulated industry, so there are no reports on results. But we can tell you that the FDAapproved formulation of AMPYRA is a very highquality, stable, extended release.
Cooper: I interviewed the actor, David Lander (“Squiggy” on Laverne and Shirley) for the last issue of the magazine. He has MS and was having some real struggles getting through the day without a walker or wheelchair. It would be cool if this thing worked for him.
Saccavino: It’s really such an awful disease, and you hear regularly from people that the effect it has on walking is probably the worst part. It really affects every area of their lives. So I hope that he does have a chance to take it. I hope his physician will be familiar with it and will think that it might be appropriate for him. I certainly hope he has a good result. You can tell pretty quickly.
Cooper: When you talk about his physician knowing about the drug, how does that work? Who are your target markets and how do you get the message about the drug to those markets?
Saccavino: We’re targeting neurologists who treat multiple sclerosis. For the launch, we doubled the sales force we had in place, for a total of 100 people in the field. We believe that number of sales people will be able to reach the vast majority of those physicians.
Cooper: And you’re talking to the MS societies?
Cooper: And of course you’re going to have to go global on this. Do you make this, or do you license it?
Saccavino: We licensed the formulation from Elan Pharmaceuticals, which continues to manufacture it for us. We held the worldwide rights to develop this product. We then licensed the rights to develop it outside the US to Biogen Idec. So, Acorda maintains the rights to develop and market it in the US, and Biogen Idec will develop and market it in all non-US markets.
Cooper: When does it actually launch? When you say “launch,” that means the physician can actually prescribe it?
Saccavino: That’s exactly right. We expect that to be in March. [AMPYRA was released on March 1, 2010, as expected. This interview was conducted shortly before that date.-Ed.] This product will be distributed through specialty pharmacies, which should be a substantial benefit to the patient, because that’s how most of their current MS drugs are delivered to them. The physician writes a prescription and submits it directly to the specialty pharmacy. The specialty pharmacy will have a desk with customer service folks who can work directly with patients’ insurance companies to make sure they get coverage. We will also have co-pay mitigation, so that if a patient has a very high co-pay, we can manage that for them on the front end so they won’t have to deal with it. Then we’ll send their prescription to them. We’ll also have a patient assistance program for people who are not insured to make sure that they have access to the drug.
Cooper: Do you work with a trade association for help with administering your patient assistance program?
Saccavino: We’re a pretty small company right now, with just over 200 people, so we are working with an outside expert for our patient assistance program. We are collaborating with them quite closely because we are very committed to ensuring broad access to the treatment.