Skip to content
Search AI Powered

Latest Stories

Lenmeldy, a lifesaving gene therapy, now the world's most expensive drug

Striking about 1 in 100,000 newborns, MLD is a rare, rapidly worsening, and fatal nervous system disorder.

Lenmeldy, a lifesaving gene therapy, now the world's most expensive drug

Lenmeldy, a new gene therapy for the rare and fatal childhood disease metachromatic leukodystrophy (MLD), has become the world's most expensive drug, at a whopping $4.25 million (£3.350 million).

MLD is a brutal illness that strikes toddlers and affects their ability to walk and talk. It's a heartbreaking reality for families, with roughly half of the afflicted children succumbing to the disease, while the others live on in a vegetative state.


However, MLD is also incredibly rare, affecting only around 40 children annually in the United States. This very rarity lies at the heart of the soaring price tags associated with new gene therapies like Lenmeldy. Consider the economics: Orchard employs a staff of 160, a stark contrast to the limited number of children they can treat over several years.

“My heart wants to talk about what an effect this therapy has had on these children. Without it, they will die very young or live for many years in a vegetative state. But kids who get the gene therapy, mostly end up being able to walk and do well cognitively. The ones we treat are going to school, they’re playing sports, and they are able to tell their stories,” says Orchard’s chief medical officer, Leslie Meltzer.

While the manufacturer, Orchard Therapeutics, justifies this astronomical cost by highlighting the treatment's potential to cure a deadly disease, affordability and accessibility remain major concerns.

This raises questions about the commercial viability of Lenmeldy, even at its current price. Experts predict a challenging journey, citing the past struggles of gene therapies on the market. The European experience with Lenmeldy, approved there three years ago at a slightly lower price, reflects this. Orchard generated a mere $12.7 million (£10.04 million) from sales for most of last year, a clear indication of the limited number of children treated.

Despite the financial concerns, Lenmeldy offers a glimmer of hope. The treatment works by adding a missing gene to a child's bone marrow cells, effectively reversing the root cause of the disease in the brain. Children who participated in trials that began in 2010 are now living normal lives, a testament to the therapy's effectiveness.

Independent groups also support the value of Lenmeldy. The Institute for Clinical and Economic Review, which assesses drug value, estimates the treatment to be cost-effective within a range of £1.8 million to £3.08 million. However, super-high prices can signal economic unsustainability. A previous record holder, the gene therapy Glybera for a different condition, was purchased only once before being pulled from the market due to its $1 million price tag, which couldn't be justified by its limited effectiveness.

gene therapy Five specialised treatment centres being qualified across the US to minimise travel burden on eligible patients and their families (Representative image: iStock)

Hemgenix, a £2.7 million gene therapy for haemophilia, held the title of the most expensive drug until Lenmeldy's arrival. Although these therapies were envisioned to generate billions in sales, news reports suggest a lower-than-expected uptake.

Orchard itself discontinued another gene therapy, Strimvelis, which offered a cure for a specific immune deficiency. The limited patient pool and the existence of an alternative treatment proved to be insurmountable hurdles, even with a money-back guarantee. Orchard was subsequently acquired by the Japanese drug company Kyowa Kirin.

The success of Lenmeldy hinges on early testing. Once symptoms develop, it might be too late. Currently, many cases are identified only because an older sibling succumbed to the inherited condition.

Early detection through newborn screening programs could be a game-changer, allowing for timely intervention and potentially saving countless lives. A decision on newborn screening for MLD is expected in May 2024.

Amy Price, a parent who lost one child to MLD but saved two others with Lenmeldy, exemplifies the human impact of this treatment. Her two treated children are now thriving teenagers.

While acknowledging the high cost, this treatment emphasises the long-term benefits compared to the financial burden of caring for an untreated child.

Lenmeldy represents a significant breakthrough in gene therapy. However, its high cost and the rarity of the disease it treats create significant challenges. Increased access through early testing and insurance coverage will be crucial for maximising the life-saving potential of this groundbreaking treatment.

Teddi Shaw, a toddler with a rare genetic disease, became the first child in the UK in 2023 to receive life-saving gene therapy on the NHS. last year, in February. The treatment, Libmeldy, is very expensive but could not help Teddi's older sister Nala, who was diagnosed too late.

Both girls have MLD, a fatal condition that attacks the brain and nervous system. Nala started showing symptoms first, like uncoordinated walking and tremors. The doctors initially dismissed the parents' concerns but later confirmed it was MLD.

Sadly, by the time Nala was diagnosed, the new gene therapy was no longer an option for her. Now terminally ill, Nala can only receive supportive care.

More For You


Coffee, a widely consumed beverage, has been associated with reduced risks of several conditions, including heart disease, cognitive decline, and chronic illnesses. (Photo: iStock)
Coffee, a widely consumed beverage, has been associated with reduced risks of several conditions, including heart disease, cognitive decline, and chronic illnesses. (Photo: iStock)

Coffee could extend healthy lifespan by nearly two years, study finds

DRINKING coffee may extend a person’s healthy lifespan by almost two years, according to a study published in the journal Ageing Research Reviews. The research, which reviewed previously published studies, concluded that moderate coffee consumption could correspond to an average increase in healthspan of 1.8 years.

Researchers from Portugal highlighted coffee’s potential in promoting a healthy lifestyle, especially as the global population continues to age. "We know that the world's population is ageing faster than ever, which is why it's increasingly important to explore dietary interventions which may allow people to not only live longer but also healthier lives," said Rodrigo Cunha from the University of Coimbra, who led the study.

Keep ReadingShow less
Focus on ultimate goal
Sadhguru

Focus on ultimate goal

A FUNDAMENTAL requirement for anybody who wishes to grow on the spiritual path is to create an undeviated flow of energy towards the one and only goal in life – to attain to the highest and not settle for anything less than that.

If there is a person who is capable of channelling himself without any break, he is a blessed one.

Keep ReadingShow less
Helpful healing wisdom
Learning life lessons from one another

Helpful healing wisdom

LAUNCHED in January 2024, the Healing Place podcast has been a journey of discovery, connection, and inspiration.

Over the past year, I have had the privilege of hosting 29 episodes featuring 30 incredible guests, each bringing unique perspectives and insights. The conversations and stories shared have been enriching, not only for listeners but also for me as a host.

Keep ReadingShow less
Surviving a credit crunch Christmas
This Christmas, find joy in simple and meaningful celebrations.

Surviving a credit crunch Christmas

THE ongoing cost-of-living crisis means many families will have to scale back their Christmas celebrations this year.

However, having less money to spend does not mean losing the joy of the festive season. In fact, it can be a perfect opportunity to rediscover the true meaning of Christmas.

Keep ReadingShow less
Muslim leaders urge communities to prioritise winter vaccines

Muslim leaders urge communities to prioritise winter vaccines

THE UK Health Security Agency (UKHSA) and the British Islamic Medical Association (BIMA) have urged members of the Muslim community to prioritise their flu and Covid vaccinations to strengthen their immunity against winter viruses.

People eligible for Covid vaccinations are encouraged to book their jabs before the NHS online booking system closes on 20 December.

Keep ReadingShow less