FDA approved a lifesaving therapy for children with a rare genetic disease. It might become the world’s most expensive drug

FDA approved a lifesaving therapy for children with a rare genetic disease. It might become the world’s most expensive drug - Health - News

A New Lease of Life: FDA Approves Expensive Gene Therapy for Rare and Devastating Condition, Metachromatic Leukodystrophy

The United States Food and Drug Administration (FDA) has made a groundbreaking decision by approving the first-ever therapy for Metachromatic Leukodystrophy (MLD), a rare and devastating condition that typically claims the lives of affected children before they reach their seventh birthday. This one-time treatment, named Lenmeldy, utilizes a patient’s stem cells and a harmless virus to insert functioning copies of the faulty gene. The repaired cells are then infused back into the patient, enabling them to produce an enzyme that is severely lacking in children with MLD.

Metachromatic Leukodystrophy is a genetic disorder, where the affected children lack an essential enzyme needed to break down fatty substances called sulfatides. The accumulation of these fatty materials eventually becomes toxic to nerves, leading to the progressive loss of movement and cognitive abilities. Children diagnosed with MLD typically begin to lose their ability to walk and talk around age two, with the disease advancing rapidly, leaving them in a vegetative state.

Kendra Riley, a mother of two from Phoenix, expressed her elation regarding this FDA approval, stating, “We are over the moon about what this means for other families.” Her 5-year-old daughter, Olivia, was diagnosed with MLD as a toddler and is currently receiving hospice care. Although the therapy came too late for Olivia, it offers hope for their younger daughter, Keira, who was diagnosed at the age of 2 and received the treatment when she was 4. Lenmeldy is most effective when administered before children exhibit symptoms, ensuring a chance at a normal life for those diagnosed early.

The high cost of the personalized treatment is expected to be substantial, with analysts estimating that it could list for as much as $3.9 million. This would make Lenmeldy the world’s most expensive drug, surpassing Hemgenix, a one-time treatment for hemophilia B, priced at $3.5 million per dose. Lenmeldy is already approved in Europe, where it goes by the name Libmeldy and has a list price of 2.8 million pounds or approximately $3.5 million US dollars.

The Boston-based nonprofit Institute for Clinical and Economic Review (ICER) evaluated Lenmeldy last fall and estimated that the cost of the therapy would match its expected benefits if it was priced between $2.3 million and $3.9 million. Experts agree that this substantial price tag is worth the potential long-term benefits for MLD patients, as those who do not receive the treatment typically die within five years of diagnosis.

MLD is an inherited disease, and families like Riley’s have had to go to great lengths to access life-saving treatments. The family relocated to Italy in 2020, where the therapy was available, spending about $500,000 for their temporary stay and medical care. Although the company provided the gene therapy for free, the financial burden of living abroad took a heavy toll on them.

Dr. David Rind, ICER’s chief medical officer, explained, “It’s taking a child who would have had a miserable short life and likely giving them a normal life. And that’s worth a lot of money.” He further added, “This is one of the more exciting drugs that we’ve looked at.”

Despite the high cost, Orchard Therapeutics, the company that manufactures Lenmeldy, will announce its treatment’s price in the coming days. The exorbitant cost of Lenmeldy and other rare disease treatments has raised concerns about affordability and accessibility for patients. Some gene therapies developed for rare diseases have already disappeared, leaving patients without effective treatment options due to financial struggles on the part of the companies producing these treatments.

As the FDA continues its efforts to clear a backlog of cell and gene therapies, it has hired more staff to review new treatments and established a new superoffice, the Office of Therapeutic Products, to oversee their regulation. Lenmeldy has been available in Europe since 2020 but took four additional years to gain FDA approval. Dr. Nicole Verdun, who heads the new FDA initiative, believes this approval is a step in the right direction for the advancement and availability of effective treatments for rare diseases.

The development of Lenmeldy represents decades of research and progress in gene therapy, providing hope for families affected by this devastating condition. For Kendra Riley and her family, the approval of Lenmeldy signifies a significant step forward in ensuring future generations of children, families, and healthcare professionals do not have to endure the heartache and desperation that comes with an MLD diagnosis.