Marathon Pharmaceuticals

Last updated
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

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.[ citation needed ] 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]

Contents

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 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]

Related Research Articles

<span class="mw-page-title-main">Duchenne muscular dystrophy</span> Type of muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, and worsens quickly. Muscle loss typically occurs first in the thighs and pelvis followed by the arms. This can result in trouble standing up. Most are unable to walk by the age of 12. Affected muscles may look larger due to increased fat content. Scoliosis is also common. Some may have intellectual disability. Females with a single copy of the defective gene may show mild symptoms.

<span class="mw-page-title-main">Becker muscular dystrophy</span> Genetic muscle disorder

Becker muscular dystrophy is an X-linked recessive inherited disorder characterized by slowly progressing muscle weakness of the legs and pelvis. It is a type of dystrophinopathy. This is caused by mutations in the dystrophin gene, which encodes the protein dystrophin. Becker muscular dystrophy is related to Duchenne muscular dystrophy in that both result from a mutation in the dystrophin gene, but has a milder course.

Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. Several ASOs have been approved in the United States, the European Union, and elsewhere.

<span class="mw-page-title-main">Vertex Pharmaceuticals</span> American pharmaceutical company

Vertex Pharmaceuticals is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headquarters in South Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, Oxfordshire, England.

Sarepta Therapeutics, Inc. is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States. Incorporated in 1980 as AntiVirals, shortly before going public the company changed its name from AntiVirals to AVI BioPharma soon with stock symbol AVII and in July 2012 changed name from AVI BioPharma to Sarepta Therapeutics and SRPT respectively. As of the end of 2019, the company has two approved drugs.

<span class="mw-page-title-main">Deflazacort</span> Pharmaceutical drug

Deflazacort is a glucocorticoid used as an anti-inflammatory and immunomodulatory agent. It was patented in 1965 and approved for medical use in 1985. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication for Duchenne Muscular Dystrophy.

<span class="mw-page-title-main">Ataluren</span> Chemical compound

Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics.

<span class="mw-page-title-main">PTC Therapeutics</span> Pharmaceutical company

PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.

In molecular biology, exon skipping is a form of RNA splicing used to cause cells to “skip” over faulty or misaligned sections (exons) of genetic code, leading to a truncated but still functional protein despite the genetic mutation.

Prosensa was a biotechnology company engaged in the discovery, development and commercialization of RNA-modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as Duchenne muscular dystrophy (DMD), myotonic dystrophy, and Huntington's disease. Prosensa was acquired by BioMarin

Drisapersen is an experimental drug that was under development by BioMarin, after acquisition of Prosensa, for the treatment of Duchenne muscular dystrophy. The drug is a 2'-O-methyl phosphorothioate oligonucleotide that alters the splicing of the dystrophin RNA transcript, eliminating exon 51 from the mature dystrophin mRNA.

<span class="mw-page-title-main">Eteplirsen</span> Medication

Eteplirsen is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation. Eteplirsen only targets specific mutations and can be used to treat about 14% of DMD cases. Eteplirsen is a form of antisense therapy.

<span class="mw-page-title-main">Ezutromid</span> Chemical compound

Ezutromid is an orally administered small molecule utrophin modulator involved in a Phase 2 clinical trial produced by Summit Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). DMD is a fatal x-linked recessive disease affecting approximately 1 in 5000 males and is a designated orphan disease by the FDA and European Medicines Agency. Approximately 1/3 of the children obtain DMD as a result of spontaneous mutation in the dystrophin gene and have no family history of the disease. Dystrophin is a vital component of mature muscle function, and therefore DMD patients have multifarious forms of defunct or deficient dystrophin proteins that all manifest symptomatically as muscle necrosis and eventually organ failure. Ezutromid is theorized to maintain utrophin, a protein functionally and structurally similar to dystrophin that precedes and is replaced by dystrophin during development. Utrophin and dystrophin are reciprocally expressed, and are found in different locations in a mature muscle cell. However, in dystrophin-deficient patients, utrophin was found to be upregulated and is theorized to replace dystrophin in order to maintain muscle fibers. Ezutromid is projected to have the potential to treat all patients suffering with DMD as it maintains the production of utrophin to counteract the lack of dystrophin to retard muscle degeneration. Both the FDA and European Medicines Agency has given ezutromid an orphan drug designation. The FDA Office of Orphan Products and Development offers an Orphan Drug Designation program (ODD) that allows drugs aimed to treat diseases that affect less than 200,000 people in the U.S. monetary incentives such as a period of market exclusivity, tax incentives, and expedited approval processes.

Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis.

Golodirsen, sold under the brand name Vyondys 53, is a medication used for the treatment of Duchenne muscular dystrophy (DMD). It is an antisense oligonucleotide drug of phosphorodiamidate morpholino oligomer (PMO) chemistry.

Viltolarsen, sold under the brand name Viltepso, is a medication used for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen is a Morpholino antisense oligonucleotide.

<span class="mw-page-title-main">Cure Rare Disease</span>

Cure Rare Disease is a non-profit biotechnology company based in Boston, Massachusetts that is working to create novel therapeutics using gene therapy, gene editing and antisense oligonucleotides to treat people impacted by rare and ultra-rare genetic neuromuscular conditions.

Toshifumi (Toshi) Yokota is a medical scientist and professor of medical genetics at the University of Alberta, where he also holds the titles of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair and the Henri M. Toupin Chair in Neurological Science. He is best known for his studies of antisense oligonucleotide-based therapeutics for muscular dystrophy that led to the development of an FDA-approved drug viltolarsen. His research interests include precision medicine for muscular dystrophy and genetic diseases. He has co-edited two books both published in the Methods in Molecular Biology series from Humana Press, Springer-Nature, and has published more than 100 refereed papers and patents. He is a member of the editorial boards for the International Journal of Molecular Sciences, Genes, Frontiers in Genome Editing, Frontiers in Physiology, and Nucleic Acid Therapeutics, a member of the Medical and Scientific Advisory Committee of Muscular Dystrophy Canada, and a co-founder of the Canadian Neuromuscular Network (CAN-NMD).

<span class="mw-page-title-main">Ultragenyx</span>

Ultragenyx is an American biopharmaceutical company involved in the Research and Development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases.

Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO). Duchenne muscular dystrophy is a rare disease that primarily affects boys. It is caused by low levels of a muscle protein called dystrophin. The lack of dystrophin causes progressive muscle weakness and premature death.

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. 1 2 Joseph Walker; 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, 2017
  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; 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 boys
  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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