Marathon Pharmaceuticals
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 | |
| Company type | Private |
|---|---|
| Industry | Pharmaceuticals |
| Founder | Robert Altman[1] |
| Headquarters | Northbrook, Illinois , USA |
Area served | Worldwide |
Key people | Robert Altman Jeffrey S. Aronin, former Chairman and CEO |
| Products | Developed 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]
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.