Two top lawmakers are questioning a pharmaceutical company after it said it would charge $89,000 a year for a drug that treats a deadly form of childhood-onset muscular dystrophy.
Sen. Bernie SandersBernie SandersStudy: Test detects signs of dementia at least six months earlier than standard method The Hill's Morning Report - Presented by Uber - Omicron tests vaccines; Bob Dole dies at 98 Democrats see Christmas goal slipping away MORE (I-Vt.) and Rep. Elijah Cummings (D-Md.), two vocal critics of high drug prices, demanded answers from Marathon Pharmaceuticals Monday, calling the price of Deflazacort "unconscionable."
“Marathon’s apparent abuse of government-granted exclusivity periods and incentives to sell what should be a widely available drug for $89,000 a year is unconscionable,” Sanders and Cummings wrote in the letter to the CEO of Marathon.
“Exorbitantly pricing potentially life-saving medications that should be widely available for a fraction of the price hinders patient access and drives up costs for the entire health care sector.”
The Food and Drug Administration approved Deflazacort for use in the U.S. Thursday as a treatment for Duchenne muscular dystrophy, a rare early-onset genetic disorder that causes progressive muscle deterioration and weakness.
Marathon subsequently said it would charge $89,000 for the drug, although it is sold for $1,000 in other countries.
Sanders and Cummings noted that the drug has been imported by patients in the U.S. for "decades."
“Marathon did not develop Deflazacort,” Sanders and Cummings wrote.
“Rather, Marathon acquired the rights to historical clinical trial data from the 1990s and completed some additional analyses to gain approval from the Food and Drug Administration (FDA) to sell the drug in the U.S.”
Sanders and Cummings accused the company of taking advantage of the FDA's orphan drug program, which will give it seven years of exclusive sales rights on the treatment.
“We believe Marathon is abusing our nation’s ‘orphan drug’ program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases — not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades,” they wrote.
Babar Ghias, Marathon's chief financial officer, told the Chicago Tribune patients will pay far less than $89,000 for the medication because it will be covered by insurers.
Ghias also said patients without insurance can get the drug for free through a Marathon program.
Marathon announced Monday that it would halt its rollout of the drug amid backlash.
"Since last week’s approval, we have heard both support from the community and concerns about how the pricing and reimbursement details will affect individual patients and caregivers, such as how it affects coverage of other components of Duchenne treatment," Marathon Pharmaceutical's CEO Jeffrey Aronin said in a statement.
"We are pausing our commercialization efforts in order to meet with Duchenne community leaders and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization based on the resulting plan of action."