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Vickie Moored can walk again. Her words don’t slur, her vision isn’t blurred, and she no longer collapses every day.

The 65-year-old from Elkton, Va., says she has her life back, thanks to the stunning efficacy of a cheap, but unapproved, chemical called 3,4-DAP. It’s a medication she would not have but for the largesse of a tiny, family-run drug maker that has been giving it out, for free, for decades.

But Moored, along with others who share her rare neuromuscular disorder, are concerned that their fortunes could change. The cost of the treatment will likely skyrocket — from next to nothing to potentially more than $100,000 without insurance — now that the Food and Drug Administration has approved a medicine to treat their disease, Lambert-Eaton Myasthenic Syndrome.


“There has been a long-standing need for a treatment for this rare disorder,” the FDA said in a statement this week announcing the approval.

The reality is that physicians have been prescribing 3,4-DAP, a nearly identical treatment, off-label, since the 1980s. It was never formally approved by the FDA; instead, LEMS patients pay about $300 to $500 a month to get the drug from a compounding pharmacy. That, or they’ve been supplied with the drug — for no charge — from the New Jersey-based Jacobus Pharmaceutical Co.


With an FDA approval in hand, however, Florida-based Catalyst Pharmaceuticals now has the ability to charge what could be hundreds of thousands of dollarsfor a medicine that costs almost nothing to make. What’s more: Catalyst could block older forms of the drugs from being given to patients, even though they didn’t invent the formulation in the first place.

Catalyst, which declined to respond to requests for comment for this article, will release specific pricing information in mid-December. It says that an approved drug will help more patients get access to therapy — and some members of the LEMS community agree. Others, however, are devastated.

“So many of us are feeling desperate and scared and outraged,” said Sharon Southern, a LEMS patient in Australia who has been getting her 3,4-DAP from Jacobus since 2004. “Catalyst is sweeping in to look like the heroes — when they’re actually the opposite.”

Drug pricing is an increasingly contentious issue in the U.S. Take the case of Turing Pharmaceuticals, which hiked the price of AIDS drug daraprim 5,000 percent when led by former CEO Martin Shkreli. Or Valeant Pharmaceuticals, which routinely increased the prices of all its drugs.

The Trump administration has launched a handful of measures in an effort to curb price hikes. Some of those efforts have been led by the FDA, but the FDA is a regulatory agency tasked with ensuring the safety and efficacy of drugs, and has not historically taken price into consideration when deciding whether to approve new treatments.

“This is a very political environment, and this sort of decision will raise a lot of interest — and a lot of criticism — in the FDA,” said Dr. Donald Sanders, a professor of neurology at Duke University who has treated LEMS patients for many years. He’s also worked as a consultant for Jacobus. “I’ve found it very difficult to believe anyone would risk creating a public firestorm by allowing a very expensive drug to replace another that’s already available — safely and for free.”

Dr. Ted Burns, a professor of neurology at the University of Virginia, says Catalyst is “profiteering” off of vulnerable patients, and calls its business model for Firdapse, the brand name of the drug, “exploitative.” In 2015, he penned an editorial — signed by more than 100 neurology experts — expressing grave concern that an FDA approval for Firdapse would spike treatment costs for LEMS patients. It’s unclear how much of the cost insurers will cover.

“This feels like a win for Wall Street and a loss for the rest of us,” Burns said. “It’s unfair. The patients pay. We all pay. Deductibles, premiums, and taxes will go up because of this. None of this makes sense.”

Patients say the unapproved version of the drug works perfectly well — and has been transformative.

“I wouldn’t be alive today if not for 3,4-DAP: It allows you to move and breathe,” said Southern, who recalled that before treatment she used to feel profound weakness and a slowing of her reflexes. “It’s absolutely life-changing. And lifesaving.”

LEMS is an autoimmune disease, in which the body’s own defenses turn against the region connecting a person’s nerves and muscles. This disturbs the nerves’ ability to send signals to muscle cells, leading to severe muscle weakness and pain, and difficulty with walking, breathing, and eyesight. About half the time, it’s linked to non-small cell lung cancer. There are an estimated 3,000 patients with LEMS in North America, and another 6,000 in the European Union.

In the 1970s, Scottish scientists discovered 3,4-DAP — also called amifampridine — and by the 1980s, Swedish scientists were demonstrating that it worked in LEMS patients. The Muscular Dystrophy Association later asked Jacobus to manufacture 3,4-DAP because doctors wanted to study its use in LEMS, but didn’t have a steady supply of the chemical.

Jacobus agreed, and ever since has provided the drug to more than 1,000 of patients with LEMS, asking them only to cover postage costs, said Laura Jacobus, who runs the company with her father, David.

In 2000, Parisian researchers developed a shelf-stable version of 3,4-DAP by adding a phosphate group onto the amifampridine compound. The older version required refrigeration, which could be inconvenient for patients.

Amifampridine phosphate was licensed to a biopharma company, and the drug switched hands from one to another — until it was acquired by Bay Area-based Biomarin in 2009. Amifampridine phosphate was approved by the European Medicines Agency that year, and was given a new name: Firdapse.

It was also given a new price tag: $60,000.

Although it’s rare for private pharma companies to display generosity like Jacobus has, “it’s dirt cheap to manufacture the drug,” Sanders said.

When he first started prescribing 3,4-DAP back in the 1990s, his team at Duke would buy it in bulk from a chemical company for about $100 per gram. The school’s pharmacy would put the drug into capsules, and then Sanders would distribute it to his patients with LEMS.

Moored is one of them: Every six months she drives four hours, from her home in Virginia to Duke University in North Carolina, to pick up a half year’s supply of her medication. She pays a small fee to cover shipping costs for the refrigerated drug. She takes four pills a day.

To get 3,4-DAP, patients like Moored must work with their doctor to appeal to the FDA’s compassionate use program, which allows access to unapproved therapies if no other options are available. It’s not easy, and patients and physicians have had to wade through lengths of red tape to get 3,4-DAP. Still, the net result is a free and effective drug to treat LEMS.

Other patients that don’t have ready access to the Jacobus product can get the drug from about a half-dozen compounding pharmacies around the country, Sanders said. They’ll charge about $300 to $500 per month.

Firdapse will be much more costly.

In a 2015 presentation to investors, Catalyst Pharma estimated that it could make $300 million to $900 million on Firdapse sales, suggesting a price tag of perhaps $100,000 per person based on the estimated patient population. Other analysts have estimated the cost could run up to $300,000 per year.

There’s a divide among some in the LEMS community. Some patients and their families say they are grateful for Firdapse — and believe the FDA approval will guarantee access for all patients.

Leigh Shell, whose 24-year-old daughter, Sarah, has a congenital form of LEMS, is thrilled.

Sarah began showing symptoms of LEMS when she was just 15 — losing weight, having difficulty swallowing, passing out frequently. By the time she was a freshman in college, Sarah had to use a wheelchair. Her family had taken her to specialists in Atlanta, New Orleans, and Birmingham, Ala., for a diagnosis, and she’d been told she could have anything from a brain tumor to a rare, fatal genetic disease. When a doctor finally came upon the correct diagnosis, he retired.

“We were left without a doctor who knew what LEMS was, and how to treat it,” Shell said. “So there was no medicine available for her.”

Shell managed to get the drug from a compounding pharmacy, but was never instructed that the drug required refrigeration. So it quickly denatured, and never worked for her. Shell also tried to get the drug from Jacobus, but came up short.

“Nobody responded to us, because we didn’t have a doctor that was connected with Jacobus to get it for free,” Shell said.

(Jacobus says it currently provides 3,4-DAP for about 350 LEMS patients.)

The family eventually enrolled in a clinical trial Catalyst was running.

Sarah’s symptoms improved almost instantaneously after her first dose of Firdapse. After being in a wheelchair for a year, she was able to stand up and walk again. When Sarah was at her worst, she would rarely leave her home. Now, she’s 24, married, and works as a special needs tutor in Oceanside, Calif.

Along with other patients who have LEMS, the Shell family has received travel vouchers from Catalyst to attend conferences for patients with this rare disease. Sarah, and her mother, have also spoken at Catalyst, sharing their experiences as patient and caregiver.

They are grateful that Catalyst offered her the drug when they felt they had no options. Shell isn’t concerned about the cost: She called her insurer, Blue Cross Blue Shield, and they assured her they would pay for the drug.

“Our insurance company said they view this as a specialty drug — saying that because so few people need it, they’ll cover it,” Shell said. “They told us our out-of-pocket cost would only be $60 per month.”

Catalyst has said it plans to set up programs to help patients navigate insurance questions and apply for financial assistance once the drug is launched in the first quarter of 2019.

Burns, the University of Virginia neurologist, says Catalyst is taking advantage of a loophole in the Orphan Drug Act, which gives incentives to companies that develop drugs for rare diseases. Most of the time, biotechs use these provisions to invent new medicines — spending tens, even hundreds, of millions of dollars on research and development.

Catalyst did not invent Firdapse. But it did pay the necessary millions needed to test it in clinical trials, and apply for FDA approval.

“The big problem is, this is not what the Orphan Drug Act was designed to do,” Burns said. “It was designed to incentivize innovation. That’s not what’s happening here, because there’s no biopharmaceutical innovation.”

All the same, Catalyst could use its newly gained intellectual property to prevent patients from otherwise accessing 3,4-DAP, if it chose to do so. Compounding pharmacies can’t touch drugs that are protected under patent.

Catalyst disclosed in its 2017 annual report that it’s leaning heavily on the notion that insurers will pay for the drug: The company said that if payors don’t view Firdapse as cost-effective, and refuse to reimburse them for the drug, they may have to cost-cut — to the detriment of shareholders. And there’s no guarantee that insurance will pay for Firdapse.

There’s some precedent suggesting that could happen. In the U.K., the National Health Service will not reimburse for the Biomarin drug, leaving British patients to either pay for it out-of-pocket — or find an alternative source for 3,4 DAP. Citing this example, Moored is leery that Medicare would cover a costly drug like Firdapse.

“There is no reason in principle why the NHS should be required to prescribe a more expensive licensed drug when a pharmacologically identical drug is unlicensed for the treatment in question,” the NHS said back in 2010.

In other European countries, including Germany and Italy, regulatory bodies turned a blind eye to the LEMS situation, Sanders said — allowing patients to keep using whatever source for 3,4-DAP they’d been using all along. That was preferable to spending a large chunk of their national health budgets unnecessarily on this rare disease, Sanders said.

“Most European countries have figured out how to get it through some back door,” Sanders said.

In 2012, Biomarin licensed out Firdapse to Catalyst, investing $5 million in the Florida pharmaceutical company and handing over U.S. distribution rights. Catalyst plans to expand the drug’s use in other neuromuscular disorders, and is currently doing the clinical trial and regulatory legwork to get there.

In response to Catalyst’s plan to get Firdapse approved in the U.S., Jacobus decided to conduct a clinical trial of its own. Both companies published a spate of clinical data in recent years, and submitted applications for FDA approval.

Laura Jacobus said her family’s version of 3,4-DAP is still up for FDA review, but would not say when. But, in any case, Catalyst got its submission in first.

“It took too long for us to have enough of a war chest,” Jacobus said. The small company sells drugs to treat diseases like tuberculosis and leprosy, but lacks the financial backing — and the stockholders — to quickly raise money.

“From a commercial point of view, we didn’t do it right,” she added. “We thought we were doing the right thing by always making our drugs affordable —  making this drug available in a compassionate use basis — and we got stung.”

Jacobus accepts the FDA’s judgment on Firdapse, and believes that their data must have demonstrated that it was safe and effective. It’s still unclear whether Jacobus will keep producing 3,4-DAP — moving forward, it all depends on FDA regulations around compassionate use. If it’s just for a handful of patients who live abroad, the company will most likely not be able to keep producing the drug, she said.

Jacobus said she’s happy that she’s been able to build up relationships with LEMS patients over the past 25 years, and help them live better lives. She says she will continue to wish them all the best.

An earlier version of this story incorrectly stated the estimated cost of 3,4-DAP in the 1990s.

  • Based on a 2014 study conducted by researchers of Tuft’s Center for the Study of Drug Development, the average direct and indirect cost of launching a drug was $2.87 billion, including $312 million which was spent on post-approval research

  • This was a GREAT USEFUL AND TRUTHFUL article! Thank you for writing it! So thankful to Senator Sanders for taking on the unconscionable, predatory Catalyst Pharmaceutical! I’m a patient who has been on 3,4 DAP since 2004 with NO side effects, so everything Catalyst is saying about Jacobus is NOT true. They need to be commended for their compassionate free distribution of this med which is NOT hard to get as per Catalyst.
    This is SHAMEFUL

    • You do realize that the Jacobus drug has never been properly approved and tested for safety and dosage? Would you still feel the same if something had gone wrong? Would you complain that the FDA “should not have allowed this to happen” if something had gone wrong? You don’t support the idea that drugs be properly tested for safety, dosage, efficacy and approved manufacturing facilities? This is a good read:

      In the article above, Jacobus acknowledges that the should have got the drug approved but did not have enough money as they are not a listed company. Do you think that if they spent tens of millions on getting it approved that they would have then continued to provide it for free? They would have needed to charge the same as Catalyst to get a return on the investment and not go bankrupt.

      Do you know that Catalyst is working to improve Firdapse and working on other uses that it may have for rare diseases?

  • . Let’s dive through this poorly written article that omits many facts and contorts the situation while painting Jacobus as some victim. I would only agree that the pricing is very surprising with today’s current climate and Catalyst deserves the blame on that full stop. However, your article furthers just ridiculous falsehoods that continue to propagate, so much so I want to cancel my STAT subscription.

    1- Jacobus didnt take their role seriously and that’s what happened, full stop. They were profiting by selling thru EAPs/CUPs and not abiding by the responsibility associated with doing so, which is your intention should be to seek FDA approval. They were distributing drug thru EAPs/CUPs but and charging for the product (albeit small $). These programs are designed where the sponsor is seeking *approval* of the product. It can’t and shouldn’t be used as a run around for approval. They failed in doing so.

    If everything is so well characterized and efficacy is understood, why didn’t FDA bless Jacobus’ with approval at some earlier date????? They ran a Ph2/3 study and clearly met with FDA. They just try and wiggle out of doing the work necessary for formal approval but still want to sell the product.

    2- Don’t give me this mom & pop spiel either. Jacobus sold Dapsone for years (sales ~10M for many years) as the only version available. They had the cashflow to take it seriously and get it approved to help patients.

    3- “Burns, the University of Virginia neurologist, says Catalyst is taking advantage of a loophole in the Orphan Drug Act, which gives incentives to companies that develop drugs for rare diseases. Most of the time, biotechs use these provisions to invent new medicines — spending tens, even hundreds, of millions of dollars on research and development.

    Catalyst did not invent Firdapse. But it did pay the necessary millions needed to test it in clinical trials, and apply for FDA approval.

    “The big problem is, this is not what the Orphan Drug Act was designed to do,” Burns said. “It was designed to incentivize innovation. That’s not what’s happening here, because there’s no biopharmaceutical innovation.”

    As you should know, US and EU regulators need a consistent label(dosing/safety/efficacy) and product characterization (CMC/nonclincal). Sorry regulators dont trust academics and their claims of efficacy/safety. (Also, who demonstrated the nonclinical profile was safe??)

    Also, is Burns an expert on the Orphan Drug Act???? Why should we take his view seriously? This is what ODA was designed for, to spur companies like Catalyst

    The FDA made Catalyst run not only 1 Phase 3 study, but an *additional* Phase 3 study! This is on top of the all the work that Biomarin had done previously. If it were so obvious, FDA would have approved based just on the work that Biomarin had done and spared patients/doctors the time of the Phase 3’s.

    4- They are actually running studies to broaden approval to other disorders (CMS as an example) that Jacobus was just trying to sell product thru (again abusing EAPs/CUPs) and not further the science to help out patients.

    5- FDA does deserve some blame, but maybe goes back to Congress. If as you say, this was so easy and we could rely on 3rd party reports of its wonderful efficacy, maybe we should re-write the laws to make it easier for approval……

    • Your comments ring hollow.

      There is absolutely no justification for firdapse’s $375,000 annual price tag.

      Biomarin gave up their dreams of playing the system in Europe because there was so much blowback on price. Catalyst is attempting to take advantage our opaque, “charge what the market can bear” pricing system. This has got nothing to do with patient care. This has everything to do with increased earnings and profitability. (Catalyst estimates it will rake in $325,000,000 PER YEAR on this drug by 2025.)

      How can Catalyst claim to be “patient focused” when it’s arbitrary pricing of an orphan drug will bankrupt the very patients it’s trying to “save”? Perhaps the executives at Catalyst should re-examine what happened at Turning Pharmaceuticals when Skhreli raise the price of daraprim 5000%. Patients and doctors were livid. Public blowback immediate. Notably, both Skhreli and his company went down in flames.

  • Is the Shell family paid for their fond words? I’m sorry but it sounds like one bad refrigeration experience led them to giving up on the drug and then praising the 60,000 version. SMH. Microinfluencers?

  • You either support the clinical testing and approval process or you don’t. You should. Also with the comments about the orphan drugs Act. Even if they didn’t get that designation they would have got it approved slower via the regular process. So the lack of innovation is not a problem or real issue either.

  • There are drugs that many small biotech and big pharma’s take big risks to develop from scratch, trial for many many years in the various phases, and spend sometimes billions to get it from concept to approval at the risk of investors over 6-10 years.

    This drug is NOT one of those. It is a compound that was developed by universities and distributed in-kind by folks who cared to the few suffering from the disease. It was a simple drug with no side effects and had no huge risks or investments.

    Biomarin and now Catalyst, are simply taking advantage of this compound and the FDA is not considering the history as they believe they are doing the right thing by certifying it.

    This will come full circle and this article will be one of many and Catalyst will be made an example of a company that took advantage. There management is a bunch of dimwits who cannot manage the most simple business tasks and are puppets of Biomarin and the CEO keeps buying cheap shares to what he thinks is an easy cash-in.

    Scam of a company and it will get exposed.

  • The “Business” model of healthcare is anything but health”care”. It’s an absolute horror show where profit is more important than people. Shame on all of you who participate in these crimes against humanity!

  • In the name of safety US pharma industry in conjunction with our lawmakers and FDA are basically raping the patients in broad day light.

    What if Metformin which is diabetes drug and is being tested for breast cancer has some positive results would Metformin be not sold or sold at higher than 3 cents per pill. Arn’t we being stupid or naive?

    • I agree with Girish Malhotra. Kerry King is way off base with her comment.

      This drug has been around since the 1980s. It’s not an orphan drug and this drug should Not be patented.
      When will the FDA start working for patients/taxpayers who are paying their salaries? Or are they paid more by the pharmaceutical companies?

    • Girish is exactly right- this drug existed for decades before them, and they are opportunistic and taking advantage of the system- Catalyst is about to get away with ripping everyone off- this drug already exists and costs next to nothing, but whatever generates the biggest cash cow is all these companies care about- and he who has the most money gets what he wants- i have a relative taking the jacobus version right now and he wouldn’t be able to function without it. Now this other company has stolen the drug, spent a bunch of money on tests, and the FDA is giving them them right to monopolize it, charging whatever they like. The FDA has also been compromised by large, greedy corps – see the story about Essure- there are many like it- someone is making a ton of money to help them screw people. And since there are so few people with this condition, it will hardly see the light of day- few people will even know that this happened.

      As far as the company making it available for more people? Well yeah, but only as long as they get paid a ton of money for something that already costs next to nothing. What are people who can’t afford it supposed to do now? Well, they create no cash flow, so i guess nobody cares about them.

  • Matthew Herder said it well: “At bottom, the ODA was intended to redistribute resources to medical needs that would otherwise be marginalized by market forces.” Resources to develop a drug for LEMS with proven efficacy were for decades marginalized by market forces. The ODA redistributed resources to allow a for-profit developer/manufacturer to meet this unmet need. The argument that an inexpensive drug existed for LEMS prior to this ODA-protected drug (Firdapse) is specious, ignoring not only the many benefits afforded patients from FDA scrutiny of Firdapse pre- and post-approval but also the collateral benefits (patient services, co-pay limits, investments in research,patient and physician education, etc.) to be reaped by the LEMS “ecosystem” now rendered non-orphan by the ODA. If Catalyst behaves ethically, as the public scrutiny demands it will, Firdapse is an ODA success story not exploitation of an ODA loophole. It’s egregious that some doctors will lie to their LEMS patients about the dangers of profit-mongering drug developers for their own selfish purposes. They should be thanking Catalyst for providing a reliable source of high-quality medication to their patients in need.

    • I don’t understand this, but willing to try-

      You have this drug that costs next to nothing to mfg, that many people can’t function without. It’s being provided at little or no cost to people, and most of those people get to live better or even nearly normal lives.

      Then you have this Catalyst company that spent a ton of money on a bunch of tests for the drug, and because of that, the FDA is stealing the right to distribute the drug from Jacobus and giving Catalyst monopoly rights to it. Catalyst has already said how much money they plan on making with the drug- that will make it impossible to get for a lot of people, and lots of other people will be financially f*’d. These companies generally will look at target demographics to figure what their baseline income is like, and use that to figure out how much money they think they can get from them- it’s got nothing to do with recovering the costs they’ve put into it and mfg costs- they want to maximize their profit, that’s what companies do. But the FDA is supposed to be keeping a safe/level playing field, not giving a company the right to steal a different company’s drug, put it through a bunch of tests and then monopolize it right from under the first company. This is legalized scamming.

      What should we be thanking catalyst for? Maybe i have the facts wrong..

    • …and hydroxyprogesterone (Makena) and deflazacort (Emflaza). The companies behind those did not have a fun time when they placed a really high price on those when they got approved. Hopefully someone at Catalyst is intelligent enough to pay attention to these case studies, otherwise they better get hip to Twitter.

    • This also happened with sapropterin for atypical phenylketonuria. It had been available for decades at a reasonable price from Schircks Laboratories, and then Biomarin got ahold of it and forced Schircks to stop selling the pharmaceutical dosage form and massively jacked up the price. Congress needs to do pass a law to prevent serial gougers like Biomarin, Turing, and Valeant from doing this.

    • There’s fear mongering, and then there is informing people about what will likely happen based on what’s happened in the past. This is what these companies do- anyone who takes the time can find that out, plus it’s common sense- a company just spent god knows how many millions to get FDA approval- did they do it to help people out? They’ve already stated how much money they plan on making off people and their insurance companies. few bother educating themselves about problems, and that’s a big reason why they get away with it.

      Kerry- how is this fear mongering? Everything stated in this article is verifiable fact. Though it’s easy for a company who’s invested tens of millions on their drug to post a message that suggests it’s all fear mongering- it would be foolish for the company not to- it’s easy, it’s cheap, and it might make a few people think Catalyst are the good guys.

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