Marathon Pharmaceuticals

Marathon Pharmaceuticals LLC was a privately held biopharmaceuticals company focused on drugs for people with rare diseases.[2] The Illinois-based company[3] developed and manufactured therapeutics and brought them to market. It employed 100 people in four global locations. In 2017, PTC Therapeutics acquired rights to Marathon Pharmaceuticals' drug Emflaza (deflazacort) for $140 million after criticism about their plan to sell the drug at a list price of $89,000 per year to sufferers despite the fact that the same drug was available in Canada and the UK for around $1,000 per year.[4][5]

Marathon Pharmaceuticals
TypePrivate
IndustryPharmaceuticals
FounderRobert Altman[1]
Headquarters
Northbrook, Illinois
,
USA
Area served
Worldwide
Key people
Robert Altman Jeffrey S. Aronin, former Chairman and CEO
ProductsDeveloped drugs for high-need, small patient populations, and late stage

Business model

Marathon produced medicines for high-need, small patient populations, including patients with rare diseases as outlined by the U.S. Food and Drug Administration’s (FDA) Orphan Drug Act. The Act defines rare diseases as impacting fewer than 200,000 patients in the U.S.[6]

Marathon developed late-stage drugs, earned regulatory approvals, and then manufactured and commercialized medicines with input from patient advocacy groups. Marathon’s regulatory efforts centered on gaining FDA approval of New Drug Applications (NDA) or Biologic License Applications (BLA).[7]

The company provided assistance for eligible patients with financial hardship[8] and helped patients secure other assistance through the National Organization of Rare Disorders (NORD) and similar patient support groups.[9]

The company was criticized for their business model which uses regulatory loopholes and FDA incentives to cheaply acquire drugs to treat rare diseases and multiplying the list price to make a profit.[10][11]

Marathon distributed its products in North America.

Treatment pipeline

Marathon developed medications[12] that targeted neurological, muscular, gastrointestinal and blood disorders, including deflazacort, a new medication to slow the progression of Duchenne muscular dystrophy (DMD) in patients.[13] In January 2015, Marathon was granted fast track status from FDA for deflazacort as a potential treatment for DMD.[14] Exondys 51 and deflazacort are, as of 2/12/2017, the only FDA-approved drugs that treat DMD. Most patients usually die by age 25,[15] although recently some people have lived into their 30s and 40s[16] with assistive devices and support.

Deflazacort

On February 13, 2017, the CEO of Marathon, Jeff Aronin, announced that after four years of development and over 17 trials, the company was granted FDA approval for deflazacort (Emflaza)[17] to treat Duchenne muscular dystrophy, a rare disease that affects approximately 15,000 young boys in the U.S. Prior to the FDA approval, less than 10% of Duchenne patients in the U.S. had access to Emflaza. Post FDA approval, all Duchenne patients have access to an FDA approved and U.S. made drug that is being covered by over 170 payers and over 45 state Medicaid programs.[18]

Amid controversy over the proposed $89,000 wholesale price to payers, Marathon sold Emflaza and related assets to PTC Therapeutics, a firm with a 20-year history in Duchenne drug development. In Canada, the same drug, although never approved for treating Duchenne, could be purchased for around $1 per tablet and used off-label to treat the disease.[19] According to The Wall Street Journal, the price Marathon was proposing was "roughly 70 times" more than deflazacort would cost overseas, such as in Europe where it was approved to treat 23 indications for a patient base of 83 million.[20] In a 2015 Chicago Tribune article, parents had expressed concerns that if deflazacort were approved in the United States, FDA guidelines seemed to suggest that parents would not "be able to order it from another country." They feared a "U.S. drug could be vastly more expensive than current international rates, leaving a question about insurance coverage."[21] Senator Bernie Sanders and Rep. Elijah Cummings are investigating the price of the drug as part of a larger investigation about the cost of pharmaceuticals.[17]

References
  1. Walker, Joseph (2 May 2017). "Revolt Against Sky-High Drug Prices Prompts a Pioneer to Cash Out". WSJ.
  2. "NORD". National Organization for Rare Disorders. Retrieved 17 June 2014.
  3. "About Marathon". Marathon Pharmaceuticals. Retrieved 17 June 2014.
  4. "PTC Acquires DMD Med Emflaza From Marathon Pharma for up to $190 million". Muscular Dystrophy News.
  5. Mangan, Meg Tirrell, Dan (2017-02-13). "Marathon 'pauses' launch of $89,000 muscular dystrophy drug in United States amid pricing outrage". CNBC. Retrieved 2017-11-22.
  6. "Orphan Drug Act - Relevant Excerpts". U.S. Food and Drug Administration. Retrieved 17 June 2014.
  7. "Biologics License Applications (BLA) Process (CBER)". U.S. Food and Drug Administration. Retrieved 17 June 2014.
  8. "Patient Assistance Program". RxAssist. Retrieved 21 July 2014.
  9. "Patient Support Program". Marathon Pharmaceuticals. Retrieved 17 June 2014.
  10. "Marathon Pharmaceuticals to charge $89,000 for muscular dystrophy drug after 70-fold increase". Fox News. 2017-02-11. Retrieved 2017-11-22.
  11. Lowe, Derek (2017-04-26). "Marathon Pharmaceuticals Cashes Out". Seeking Alpha. Retrieved 2017-11-22.
  12. "Pipeline". Marathon Pharmaceuticals. Retrieved 17 June 2014.
  13. "Sarepta (SRPT) Lower as Marathon Pharma Receives Orphan Drug Status for Deflazacort". StreetInsider.com. Retrieved 21 July 2014.
  14. Hirst, Ellen Jean. "Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016". chicagotribune.com.
  15. "Duchenne muscular dystrophy". Medline Plus. U.S. National Library of Medicine and National Institutes of Health. Retrieved 17 June 2014.
  16. "Overview". Muscular Dystrophy Association. Retrieved 17 June 2014.
  17. Joseph Walker and Susan Pulliam (February 13, 2017), Firm Delays Muscular Dystrophy Drug U.S. Launch Amid Criticism of $89,000 Price: Sanders, Cummings investigating how Marathon Pharmaceuticals set hefty price tag for an old drug, The Wall Street Journal, retrieved February 13, 2017CS1 maint: uses authors parameter (link)
  18. Lopes, Jose Marques; PhD (24 August 2017). "PTC Therapeutics Highlights Duchenne Therapies Translarna and Emflaza in Corporate Report".
  19. Clifton Sy Mukherjee (February 10, 2017). "Brainstorm Health Daily". Retrieved February 13, 2017.
  20. Joseph Walker and Susan Pulliam (February 13, 2017), Marathon Pharmaceuticals to Charge $89,000 for Muscular Dystrophy Drug After 70-Fold Increase, The Wall Street Journal, retrieved February 13, 2017, FDA-approved deflazacort treats rare type of disease affecting boysCS1 maint: uses authors parameter (link)
  21. Ellen Jean Hirst (January 19, 2015), Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016, The Chicago Tribune, retrieved February 13, 2017, has been shown to prolong lives ... a progressive and fatal disease that has no drug treatment available in the US
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