Waukeshahealthinsurance.com-
–
A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, the manufacturer announced Wednesday.
Lenmeldy was approved by the US Food and Drug Administration on Monday as the first treatment for the rare and devastating disease, which commonly kills affected children before they are 7 years old. About 40 babies are born with MLD each year in the US.
The bulk cost is not usually borne by patients, but is assumed and covered by public and private health insurance plans, including state Medicaid plans, which cover 4 out of 10 children in the United States.
Manufacturers of gene therapies say the big prices reflect the big benefits — the potential for a cure from a crippling or even fatal disease — and should be able to recoup the high costs of developing, testing and manufacturing their products.
Health policy experts say that as the list of gene and cell therapies continues to skyrocket, states and other insurers may be unable to cover their costs and ultimately limit patient access. Cover.
Dr. Bobby Gaspard, founder and CEO of Orchard Therapeutics, the company that makes Lenmeldin, said the treatment is “paradigm-changing medicine and has the potential to stop or reduce this devastating childhood disease with a single treatment.” ”
“We are committed to enabling broad, beneficial and sustainable access to this important treatment for eligible patients with early-onset MLD in the US,” Gaspar said in a statement.
Lenmeldi took stem cells from a person with MLD and used a harmless virus to insert working copies of the faulty gene. The corrected cells are returned to the patient, and then the children with the disease begin to produce the missing enzyme. Some cells eventually migrate to the bone marrow, where they live and make new cells and enzymes, providing lasting benefits to patients.
The first patients treated with Lenmeldi have now been followed for more than 12 years, and researchers continue to find gene-modified cells that make the missing enzyme, Orchard Therapeutics' Gaspar said.
“At the moment we cannot say that this will last a lifetime, but what we are saying is that there is a long-term lasting effect,” said Gaspar.
MLD is an inherited disease, and children born with it lack an enzyme needed to break down fatty substances called sulfatides. The accumulation of these fatty substances eventually becomes toxic to the nerves, causing a gradual loss of movement and thinking.
Children with MLD grow normally for a while, but by age 2 they lose the ability to walk and talk. The disease progresses rapidly, causing children to deteriorate into a vegetative state.
“We're over the moon about what this means to other families,” said Kendra Riley, 41, of Phoenix, who has two children with MLD.
For his family, the FDA's action this week was bittersweet.
Riley's 5-year-old daughter, Olivia, remains in the hospital. After being diagnosed with MLD in infants.
Riley said the first hint that something was wrong came when Olivia was about 2 years old. She had trouble walking, and her head regularly tilted to the side while watching TV.
“We thought we just needed some physical therapy,” Riley said.
“Then her eyes began to twitch. That's when we knew there was something else.”
By the time doctors diagnosed Olivia with MLD, it was too late for the gene therapy to help. But knowing that the disease is hereditary, they were able to get their youngest daughter Keira tested and diagnosed.
Keira, now 4, was the 32nd child in the world to receive the therapy; This is most effective before children show symptoms.
The treatment was not yet available in the United States, so the family raised funds to temporarily relocate to Italy to receive the therapy in 2020. Although the company offered the gene therapy for free, it cost them about $500,000 to live abroad and pay for Keira's medical care.
“Having this FDA-approved treatment means that if a child is diagnosed before symptoms start, they have a chance at a normal life,” Riley said.
“She's doing amazing. Zero symptoms,” she said Kira's. “You never know.”
Boston's nonprofit Institute for Clinical and Economic Review, or ICER, which evaluates the cost-effectiveness of new drugs, published one.Final reportLenmeldy estimated that the cost of the treatment would be in line with the expected benefit to patients if it was sold last fall and between $2.3 million and $3.9 million.
Experts agree that even such a high price is worth the result. Untreated children with the disease usually die within five years; The oldest patient treated with the therapy has now been followed for more than 12 years and appears to be progressing normally.
“He's taking a child who has a short life and can give them a normal life. And that's worth a lot of money,” said Dr. David Rhind, ICER's Chief Medical Officer. “This is one of the most interesting drugs we've seen.”
Still, Lenmeldi's price was hundreds of thousands of dollars higher than even Rand had expected.
“I think when you get into a number that big, people don't necessarily pay attention when it's a little bit bigger, but I think that price is too high,” Rind said.
ICER gave a range of $2.29 million to $3.94 million — that doesn't mean the top of the range is the right price, Rind said, “even if manufacturers are inclined to think so.”
“Going $310,000 over the top of that range is really a lot of money,” he said.
The last product to top the charts on a single-treatment basis is another gene therapy, HemGenix, approved in 2022 to treat hemophilia B, a blood clotting disorder. The cost was $3.5 million for a one-time treatment.
Close behind is Elvevid, approved in 2023 for muscular dystrophy at $3.2 million, and Skysona, approved in 2022 for MLD-related adrenoleukodystrophy, with a one-time cost of $3 million.
Despite its high cost, MLD treatment won't be a blockbuster, says Rind, because the disease is so rare. Some gene therapies are approved for diseasesThey are lost Because the companies that ended up with them couldn't make enough money to stay in business. Orchard Therapeutics struggled to market. He previously developed gene therapy.Leaving patients hanging.
Edwin Park, a research professor at Georgetown University's McCourt School of Public Health, is concerned about the impact on states that share the cost of covering patients on Medicaid with the federal government.
Medicaid requires lower rebates from drug manufacturers, but without competition there probably won't be much bargaining over treatment prices, Park said.
After being surprised by the cost of antiviral drugs that treat hepatitis C, more and more states are closely monitoring drug pipelines, he said.
But even paying Lenmeldy a year for one or two kids can be too much of a price to swallow for some states.
“Unless states give it their due and look at the drug pipeline, they may not be ready for something with a higher price,” Park said.
This latest approval comes as the FDA works to clear a backlog of cell and gene therapies that have been vying for attention since the pandemic. The agency hired more staff to evaluate new treatments and established a new superoffice, the Office of Therapeutic Products.
LenmeldiFound in Europe From 2020But it took four more years to bring it to America.
Dr. Nicole Verdun, who was hired to lead the FDA's new initiative last year, said Lenmeldi's approval is a sign that things are moving in the right direction.
“MLD is a devastating disease that greatly affects the quality of life of patients and their families,” said Verdun.
“This approval represents an important advance in the development and availability of effective treatments for rare diseases, including gene therapy.
Ann and Robert H. of Chicago. Dr. Barbara Burton, MD, assistant professor of genetics, genomics and metabolism at Lurie Children's Hospital, said it was a watershed for doctors, too.
“For a long time, my colleagues and I have comforted families at their most vulnerable times – often following a grueling odyssey of diagnosis, coping with dire prognoses and being told there is no cure, then having to watch their little one slip away.
“With this approval, we are taking a critical step toward ensuring that future generations of children, families and healthcare professionals will no longer have to witness the devastating effects of this disease on untreated patients,” she said.
Riley says Olivia is stable now, but Keira lives in fear that she might bring germs home.
“The common cold could kill her,” Riley said. “So every time her sister comes home from school with a cold, that's probably what gets her out.”
Olivia can neither walk nor speak, and is fed through a tube. Riley said she needs round-the-clock care. Physical and occupational therapists come to the home every week to “get her mind moving.” She understands what's going on.”
She said that when they watched Olivia sleep, they realized that her life had a deeper purpose. But they say they hope Lenmeldi's approval means families with MLD won't have to lose a child trying to save another child.
“We always call Livvy Kiera's guardian angel on earth,” Riley said. “She's here for a reason.”