Newest Proposed Treatment for Duchenne Muscular Dystrophy Abruptly Changes Hands.

Less than one month after our exploration of the recent colossal price markup of a simple drug used by a vulnerable and desperate group of children and their families, a dramatic turn of events occurred which may make things better or worse. In February, the pharmaceutical company Marathon announced its marketing plans for Emflaza, its brand name for deflazacort, a simple corticosteroid widely available in other countries but not in the United States. Emflaza had recently been approved by the FDA for the treatment of symptoms of Duchenne muscular dystrophy (Duchenne MD) in the United States.   Duchenne MD is a usually fatal genetic muscular disorder of young boys for which no curative therapy is currently available, but for which corticosteroids such as deflazacort and prednisone slow the progression of weakness.  Deflazacort– the most commonly prescribed all-purpose steroid in some countries– has been used in Canada and elsewhere for the treatment of Duchenne MD for some time.  The wrinkle in Marathon’s release was that it declared an annual charge of $89,000 per child for a drug sold in many other countries for a price a measured in pennies instead of dollars.

I have already expressed my puzzlement that the FDA gave its approval to Marathon based on clinical data collected many years old by another drug company that for undisclosed reasons walked away from FDA approval. I am still looking for reliable studies that confirm that deflazacort is superior to, or safer than prednisone to treat Duchenne MD. [Prednisone is the most prescribed corticosteroid in the United States. To say that it is inexpensive would be an exaggeration!]  Perhaps a clinician without ties to the pharmaceutical industry will provide us with evidence-based data that Emflaza is an essential or even a better drug for Duchenne MD, or worth the monumental cost for any incremental benefit.

The free market at work. Did it?
At a time when the public is becoming more aware of the industry strategy of cornering the market for existing drug and charging exorbitant prices, Marathon’s plans lit a firestorm of outrage and promises of congressional hearings.  Even the threat of such a reaction seemed to have caused Marathon to delay its product launch.  According to a report last week in the Wall Street Journal, and perhaps in part to jump out of the frying pan of bad public relations, Marathon sold the rights to market Emflaza in the United States to PTC Therapeutics, Inc. for $140 million in cash and stock up-front, a 20% cut of sales after 2018, and for other considerations.  Marathon thus gets to keep part of the financial action which is estimated to reach $630 million in total sales, but passes the risk on to PTC which has reasons of its own to take control of Emflaza.  Marathon still has a “priority review voucher” it gained as part of its Emflaza approval that it can sell to another company for prices estimated at greater than $100 million.  It will be of interest to see how this voucher gets used.  A reasonable person might speculate that this major transaction had been in the works earlier.  I genuinely admire the nimbleness of a company that could turn around such a major deal in so short a time!

Searching for drugs that are truly disease-modifying.
I have not previously heard of PTC Therapeutics.  Although it is apparently based in the United States, it markets no drug here.  As far as I can determine, the company sells only a single product in Europe– Translarna (ataluren)– which is used to treat some forms of Duchenne MD, and cystic fibrosis– another serious genetic disease. PTC had further hopes that their drug would prove important in the treatment of all forms of Duchenne MD in the United States and has been seeking FDA approval for this purpose.  Unfortunately, the company revealed earlier this month that a major late-phase controlled study of Translarna in Duchenne MD failed both of its critical major endpoints.  This had to have been a major disappointment for the company and raises the question of how its use had been justified to treat Duchenne MD in Europe.  I do not know if PTC plans to press its application before a new FDA which now has leadership at both the Cabinet and Agency level that is much more business friendly than before– too friendly in my opinion.  Letting the pharmaceutical industry have its way with the rest of us is not a good idea!  Approval of drugs whose effectiveness has not been proven definitively would be a step backwards to the dark-ages of potions and poultices.

It is reported that following this failed late-stage clinical study that the market value of PTC fell from $2.6 billion to $373 million.  I anticipate that PTC may want to keep a developmental foothold in the Duchenne MD market. I wish it luck in its search for a more definitive treatment. The company has not yet announced its pricing plans for Emflaza. Controlling two (?both) of the only drugs approved specifically for treatment of Duchenne MD could be a good business strategy, but not necessarily one conducive to pricing restraint. I remain to be pleasantly surprised by a new price that is at least comparable to the price charged in Canada where many Duchenne MD families now go to obtain the drug. There are, however, development and business costs to recoup and investors to satisfy.

Children with Duchenne MD and their families deserve effective treatments that do not bankrupt them. Translarna has unfortunately not to this point been shown to satisfy either criteria for common forms of Duchenne MD.  Emflaza may at least to a limited degree satisfy the former, but not the latter criteria.  It is my opinion that expensive drugs of little or no incremental value are not what children with Duchenne MD need.  Such monies are better spent plowed back into basic and clinical research related to the disease itself.  How to finance such efforts for Duchenne MD and other diseases is a problem which we as a society have not yet solved– indeed can hardly begin to talk about.

If I have made an error of fact, medical or financial, please allow me to correct it. My experience with the use of steroids in muscle disease was with autoimmune polymyositis where the adverse effects of the treatment were profound. I would appreciate hearing from actual clinical experts in inherited muscular dystrophy.  I am not one of those.

Peter Hasselbacher, MD
President, KHPI
Emeritus Professor of Medicine, UofL
20 March 2017

2 thoughts on “Newest Proposed Treatment for Duchenne Muscular Dystrophy Abruptly Changes Hands.”

  1. PTC Drops the price of Emflaza to only $35,000!!
    PTC Therapeutics finally set a price for its newly acquired version of a corticosteroid, Emflaza. PTC purchased the drug from Marathon Pharmaceuticals after Marathon pulled down a firestorm of bad publicity and legislative attention by setting a yearly price-per-patient of $89,000. For this drug that is relatively inexpensive elsewhere in the world, PTC will now change only a minimum of $35,000 per child or adult patient! Since the dose of the drug is based on a patients weight, heavier patients may have to pay upwards of $100,000. Since one of the side effects of this steroid is weight-gain, PTC gets a fortuitous bonus!

    In my opinion, as with many cancer and other drugs, the pharmaceutical industry takes advantage of the desperation of patients and their families. I am still waiting for someone to show me the evidence that that Emflaza is clinically better than prednisone, a drug that is given out like it is candy in the US and costs pennies.

    I will write soon about another pharmaceutical company with another drug that in my opinion has inappropriately taken advantage of the situation of individuals with muscular dystrophy.

  2. Since I prepared this article, updates have been published elsewhere that give more facts. I will add links and summaries at a later time. For now, I refer my readers to articles by Sarah Jane Tribble and Sydney Lupkin in Kaiser Health News, and Joseph Walker in the Wall Street Journal (subscription.)

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