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Vyjuvek – the First and Only FDA-Topical Gene Therapy

July 25, 2023 by Ernesto Munoz, PharmD

As advancements in technology are made, unique treatment options have emerged for diseases that were once believed to be unmanageable or untreatable. Innovative treatments like gene therapy can modify a patient’s genes with the intent to treat or cure a disease. One such drug, Vyjuvek, was approved on May 19, 2023.

Vyjuvek is the first and only FDA-approved topical gene therapy that can be re-dosed and is not a one-time treatment like the current gene therapies on the market.  It is indicated to treat wounds from dystrophic epidermolysis bullosa (DEB). DEB results from a mutation in the gene that affects the production of collagen. The lack of collagen leaves patients with exposed wounds that cannot heal properly. Severity differs between the different types of DEB, with recessive DEB resulting in the most extreme symptoms and decreased life expectancy for patients.

DEB is a condition that causes the skin to be fragile and easily blister with minor trauma or friction which leads to pain and infection. It is an extremely rare disease with a prevalence of about 3.26 per million live births or 0.00326 per thousand live births in the United States.  These numbers help showcase how rare DEB is and illustrate how unlikely it is that a plan sponsor will see this therapy hitting their plan. Most patients eligible for this therapy are under the age of 18 and about 40-50% are covered by Medicaid while 40-50% are covered by commercial insurance.

Currently, this therapy will be billed under the medical benefit only, as a health care professional must apply the gel to the open wounds and wrap the area with a hydrophobic dressing. Since the gel must be applied to open wounds, the surface area covering the wounds will vary from patient to patient, which also affects how many vials a patient may utilize each year. It is estimated that the average patient will utilize 26 vials per year. At a wholesale acquisition cost (WAC) of $24,250 per vial, the annual cost of treating one patient with Vyjuvek is projected to be about $630,500. It is possible for patients to utilize more vials than estimated, thus increasing the annual cost.

One of the main concerns around gene therapy is the durability of the therapy, where a patient could receive the expensive treatment but not see the intended therapeutic effects. This concern exists around all gene therapies, but specifically for those treatments that are a one-time infusion or injection. Since Vyjuvek is re-dosed, a slightly different payment model was utilized by the manufacturer, Krystal Biotech. It has stated that it is offering all commercial payers a price cap of $900,000 per patient per year to account for patients requiring large amounts of Vyjuvek. Some patients will utilize many vials per year, while others may utilize less than the predicted 26 per year, thus $900,000 appeared to be middle ground as a pricing cap.

It is expected that there could be as many as 60 approved gene therapies by 2030, with many more expected in the years to come. It is important now more than ever with the rising approvals, that plan sponsors review their stop-loss policy to ensure that there is protection in place if a costly therapy, like Vyjuvek, were to be utilized by a member of the plan.

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