Approved gene therapy Vyjuvek for DEB soon to be available in US
Krystal's new distribution network will allow treatment at home, clinic
The just-approved gene therapy Vyjuvek (beremagene geperpavec) soon will be available to people with dystrophic epidermolysis bullosa (DEB) in the U.S. at home and in the clinic.
Krystal Biotech, the treatment’s developer, has set up a limited distribution network in the country that includes Option Care Health and Orsini Specialty Pharmacy, two Illinois-based pharmacies.
“Orsini Specialty Pharmacy is deeply committed to helping DEB patients and their families gain access to this revolutionary treatment and provide ongoing support in managing their condition,” Brandon Tom, Orsini’s president and CEO, said in a company press release.
Vyjuvek is indicated to treat wounds in patients 6 months and older who have a genetic diagnosis of DEB. Its approval earlier this month made it the first and so far only medicine for this type of epidermolysis bullosa.
Applied onto the skin as a gel, its dose can be adjusted depending on the wound size. It comes as a biological suspension that must be mixed with a gel at a pharmacy before use. The application is then done by healthcare professionals in a clinic or even at home.
Nurses can apply Vyjuvek to skin at patients’ homes
Option Care Health, which boasts 95 specialty pharmacies across the U.S., will be the only pharmacy provider that can mix and distribute the ready-to-use gel to healthcare professionals. The company has teamed up with Amedisys, which will have nurses deliver and apply the medicine in the home setting.
“The ability to administer this non-invasive treatment in the comfort of one’s home will help ensure that this medicine gets to those who need it the most,” John C. Rademacher, Option Care Health’s president and CEO, said in another company press release.
“We are honored to partner with Amedisys on this important endeavor, building on a history of successful collaboration between our two companies that brings innovative solutions to provide high-quality care in the home,” Rademacher added.
The ability to administer this non-invasive treatment in the comfort of one’s home will help ensure that this medicine gets to those who need it the most.
Epidermolysis bullosa causes the skin to become very fragile and likely to blister and develop wounds in response to any trauma or friction. In DEB, this is caused by mutations in COL7A1, a gene coding part of a protein called type VII collagen. This protein is essential for holding the layers of skin onto each other.
Vyjuvek works by delivering healthy copies of the COL7A1 gene directly into cells of the skin. This provides the cells with the instructions they need to make working type VII collagen, which is expected to help heal the wounds in people with DEB.
The approval by the U.S. Food and Drug Administration (FDA) was based on the results of two clinical studies: the Phase 1/2 GEM-1 study (NCT03536143) and the Phase 3 GEM-3 study (NCT04491604). In both of these trials, Vyjuvek brought about complete wound healing after three and six months.
Berkshire Sterile Manufacturing produces a gel that it calls “a key component” of Vyjuvek.
“Being part of this FDA-approved project fills me with pride,” Lynne Anstett, a project manager at Berkshire, said in a separate company press release.
Now that Vyjuvek is approved, its developer has made an agreement to sell some of the company’s shares to investors, according to a Krystal press release. By selling these shares, Krystal expects to receive around $160 million in revenue.
“These additional funds, together with our existing cash, cash equivalents and investments, should allow us to fund the Vyjuvek launch, future operations, and the advancement of our growing pipeline through the end of 2026,” said Krish S Krishnan, Krystal’s chairman and CEO.
“It also allows us to retain the optionality of monetizing the Priority Review Voucher we received in connection with the FDA approval of Vyjuvek at a future date with favorable terms and without any dilution,” Krishnan added.
A priority review voucher is issued by the FDA upon approval of a medicine for a rare disease. The company can use it to guarantee expedited review of another medicine at some later date, or transfer it to another company at a profit.