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Whenever a multi-million-dollar new drug comes to market, its price is widely reported. And while eye-watering prices seem exorbitant on paper, the reality behind these figures is much more nuanced.
The most expensive drugs are gene therapy drugs, which are typically one-off treatments aimed at sparing patients from repeated lifelong medical interventions. Prices are set based on the overall savings over the patient’s entire treatment journey and are intended to reflect the high value of the drugs and the long journey they take to develop.
Although these treatments have the potential to be curative, assuming their effectiveness is sustained over the long term, patients are not always keen to undergo them.
Orchard Therapeutics $4.25 million Remmerdi It was approved in March as a treatment for metachromatic leukodystrophy, making it the latest gene therapy to earn the title of “the world’s most expensive drug.” Launch PlanThis includes working with payers to implement innovative reimbursement structures that “appropriately balance the needs of patients and families for adequate access,” although it is too early to know the extent to which they will be accepted in the market.
But other news about gene therapy suggests future adoption could be a challenge. Here’s a look at the sales performance of some of the most innovative and expensive therapies approved in recent years.
Kasgevi
List price: $2.2 million
In December, Vertex Pharmaceuticals and CRISPR Therapeutics won the first FDA approval for a CRISPR-based drug, initially to treat sickle cell disease, and a month later also to treat beta-thalassemia.
The breakthrough treatment has been on the market for several months, but drug companies are still counting cells, not dollars, to gauge its adoption.
As of mid-July, cells from 20 patients had been harvested for the treatment, CRISPR Therapeutics said. Reported In its second-quarter results, the company reported that more than 35 licensed treatment centers have been opened to expand access.
Neither company has released specific sales figures for Casgevy or Vertex, but Full-year sales forecast raised Sales rose by $100 million this month to nearly $11 billion. While the company’s cystic fibrosis portfolio drove the growth, Vertex also attributed the increase to expected increased demand for Casgevy, saying: Potential patients: 35,000 For treatment.
Analysts expect Casgevy’s sales to grow Recovery in the third quarter It could eventually reach about $81 million.
HemGenics
List price: $3.5 million
CSL Behring FDA approves HemGenics The first gene therapy of its kind for hemophilia B is expected to be available in 2022. But uptake appears to be slow, according to sales reports.
CSL expects revenue to grow from $1.19 billion in 2023 to $1.09 billion in 2024 across its broad hemophilia portfolio. $1.3 billion this yearHowever, the company has not yet disclosed specific sales figures for HemGenics in its financial presentation.
company Gaining regulatory coverage Earlier this year, Hemgenix began clinical trials in the UK and Denmark under a performance-based payment model, which could help accelerate distribution of the treatment outside the US.
Loctavian
List price: $2.9 million
Hemgenix is not the only company struggling to gain traction in the gene therapy field. Hemophilia Market.
Since then, FDA approved BioMarin Pharmaceuticals Inc.’s Roctabian, a treatment for severe hemophilia A, saw sluggish sales last year, forcing the company to reassess its commercialization strategy.
Only five patients received Loctavian in the second quarter (three in the United States and two in Italy). BioMarin reportedThe company said its ultimate goal is to reduce Roctavian-related costs by $60 million and make the treatment profitable by the end of 2025.
BioMarin CEO Alexander Hardy also said earlier this year that the treatment It may eventually be sold From the company’s portfolio.
Erebdis
List price: $3.2 million
Analysts previously viewed Sarepta Therapeutics’ Erevidys as a gene therapy drug that could surge against the trend of mass adoption. The pinnacle of blockbuster successBut so far, sales of drugs set to be approved in 2023 to treat Duchenne muscular dystrophy have fallen short of some analysts’ expectations.
Erebidis is about $121.7 million That was down from second-quarter revenue of about $133.9 million. First.
Sarepta CEO Doug Ingram said on the earnings call that the flat sales were due to a months-long approval process for the treatment, rescheduling of infusions and other delays to treatment.
Company executives are also on a positive note, pointing to several indicators that could boost sales of Erebidis, including the recent approval of an expanded indication, increased demand for antibody testing kits needed for the treatment, and “unprecedented demand” at treatment centers. Overall, company executives expect sales to increase by a third in the third quarter and revenue to further double in the final three months of the year.
“With the broader label granted in June this year, the opportunity to serve patients and thereby reward committed investors will be enormous,” Ingram said. Said during the call.
