Each time a brand new multimillion-dollar drug is launched to the market, headlines trumpet the associated fee. And whereas eye-popping value tags look exorbitant on paper, the truth behind these figures is rather more nuanced.
The most costly medicine are gene therapies, usually one-and-done remedies aimed toward rescuing sufferers from a lifetime of repeated medical interventions. Costs are set primarily based on the general financial savings all through a affected person’s journey and meant to symbolize the excessive worth of the medicine and their lengthy highway to improvement.
Despite the fact that these remedies could possibly be healing — assuming the therapeutic impact holds up over time — sufferers haven’t all the time clamored to take them.
Orchard Therapeutics’ $4.25 million Lenmeldy grew to become the latest gene remedy to take the “world’s priciest drug” mantle when it was accepted in March for metachromatic leukodystrophy. The corporate has outlined its launch plans, together with work with payers to implement progressive reimbursement buildings that “appropriately steadiness the wants of sufferers and households for sufficient entry,” but it surely’s too quickly to understand how properly it is going to be obtained within the market.
However different gene remedy tales trace on the potential uptake challenges forward. Right here’s a have a look at the gross sales efficiency for a few of the most progressive and costly therapies accepted lately.
Casgevy
Listing value: $2.2 million
Vertex Prescribed drugs and CRISPR Therapeutics in December scored the primary FDA approval for a CRISPR-based drug. Initially indicated for sickle cell illness, the businesses received an extra nod for beta thalassemia a month later.
The groundbreaking remedy has been available on the market for a number of months, however the firms are nonetheless counting cells — not {dollars} — on the subject of measuring uptake.
As of mid-July, 20 sufferers have had their cells collected for remedy, CRISPR Therapeutics reported in its second quarter earnings. And greater than 35 approved remedy facilities have come on-line to increase entry.
Though neither firm has reported particular gross sales numbers for Casgevy, Vertex raised its full-year gross sales forecast this month by $100 million to nearly $11 billion. Whereas development is being pushed by the corporate’s cystic fibrosis portfolio, Vertex additionally pointed to an anticipated uptick in Casgevy demand as a purpose for the increase, and mentioned it has recognized about 35,000 potential sufferers for the remedy.
Analysts additionally predict Casgevy gross sales will choose up within the third quarter and will finally hit about $81 million.
Hemgenix
Listing value: $3.5 million
CSL Behring raised its profile when it received an FDA OK for Hemgenix in 2022, the primary gene remedy of its form for hemophilia B. Nonetheless, gross sales experiences counsel uptake has been gradual.
Though CSL is forecasting income development throughout its intensive hemophilia portfolio from $1.19 billion in 2023 to $1.3 billion this 12 months, the corporate has but to record particular gross sales outcomes for Hemgenix in any of its monetary displays.
The corporate received regulatory protection in England and Denmark for Hemgenix beneath an outcomes-based fee mannequin earlier this 12 months, which may assist the remedy achieve traction exterior the U.S.
Roctavian
Listing value: $2.9 million
Hemgenix isn’t the one gene remedy struggling to construct momentum within the hemophilia market.
For the reason that FDA accepted BioMarin Prescribed drugs’ Roctavian for extreme hemophilia A final 12 months, gross sales have been so sluggish the corporate has needed to rework its commercialization technique.
Simply 5 sufferers — three within the U.S. and two in Italy — took Roctavian within the second quarter, BioMarin reported, producing $7 million in income. Given the excessive working bills related to producing the remedy, the corporate is now limiting entry to 3 international locations — the U.S., Germany and Italy. In the end, the aim is to slash Roctavian-related prices by $60 million and make the remedy worthwhile by the tip of 2025, the corporate mentioned.
BioMarin CEO Alexander Hardy additionally indicated earlier this 12 months that the remedy may finally be divested from the corporate’s portfolio.
Elevidys
Listing value: $3.2 million
Analysts as soon as pegged Sarepta Therapeutics’ Elevidys as a gene remedy that might defy bigger uptake developments and soar to blockbuster heights. However to date, gross sales for the Duchenne muscular dystrophy remedy accepted in 2023 have landed beneath some analysts’ expectations.
Elevidys pulled in about $121.7 million in income within the second quarter — down from about $133.9 million within the first.
On an earnings name, Sarepta CEO Doug Ingram blamed flat gross sales on the months-long approval course of for the remedy, rescheduled infusions and different remedy delays.
Firm executives additionally put a constructive spin on the scenario by pointing to a number of indicators Elevidys gross sales may ramp up, together with a latest expanded label approval, a rise in demand for the antibody testing kits wanted for the remedy, together with “unprecedented demand” at remedy facilities. Total, firm leaders predicted a one-third gross sales leap within the third quarter, and one other doubling of income within the remaining three months of the 12 months
“With the broader label granted in June of this 12 months … the chance to serve sufferers and in so doing reward dedicated traders shall be monumental,” Ingram mentioned through the name.
