Washington Editor

The FDA has approved Genzyme Corp.'s cultured epidermal autograft product Epicel to treat deep-dermal or full-thickness burns covering 30 percent or more of a patient's body.

People who are burned severely over a large area of the body often die within days after sustaining the injury.

About 20,000 people are hospitalized in the U.S. each year for major burns involving at least a quarter of their body surface, according to the Centers for Disease Control and Prevention. About 4,500 of those people die.

A severe burn can destroy the top two layers of skin, leaving the skin unable to regenerate on its own to cover the wound and protect the underlying muscle and organs.

The Epicel graft can replace the epidermal destroyed by a severe burn injury and can close the wound.

Rajiv Sood, medical director of the Richard M. Fairbanks Burn Center at Indiana University School of Medicine in Indianapolis, called Epicel "the most important advance in burn care for the coverage of large total body surface area burn wounds in this decade."

The product is made from sheets of autologous keratinocytes, or a patient's skin cells, and then grown on a layer of irradiated mouse cells.

Epicel is classified as a xenotransplantation product and is the first product in that class to be approved in the U.S., according to Genzyme.

The product, which originally was made by BioSurface Technologies before the firm was acquired by Cambridge, Mass.-based Genzyme in 1994, has been available to patients since the late 1980s. However, the FDA in 1996 determined that manipulated autologous cell-based products used for structural repair or reconstruction required regulatory approval.

Genzyme submitted an approval application for Epicel in 1999. The product was approved under the FDA's Humanitarian Device Exemption (HDE), a program for devices that treat fewer than 4,000 people in the U.S. annually.

The FDA established the HDE program in 1996 to provide an incentive for manufacturers whose research and development costs could exceed their market returns for diseases or conditions affecting small patient populations.

An HDE application is similar in form and content to a premarket approval application (PMA), but is exempt from the effectiveness requirements of a PMA.

HDE applications are not required to contain the results of scientifically valid clinical investigations demonstrating that a product is effective for its intended purpose. However, the application must contain sufficient information for the FDA to determine that the product does not pose an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use, taking into account the probable risks and benefits of currently available devices or alternative forms of treatment.

Because Epicel is a "humanitarian product, Genzyme has never profited" from its sales, spokeswoman Maria Cantor told BioWorld Today. The company, she added, has "no expectations" for any future profits from the product, but is committed to ensuring it remains available to burn victims.

"We are very proud of Epicel," Cantor declared, noting that the graft has been used in more than 1,000 patients.

She said that Epicel's FDA-approved labeling contains data collected about patients from 1989 to 1996, which was used as the basis for the HDE approval.

According to the FDA's Oct. 25 approval letter, Genzyme must construct and maintain a database to collect Epicel patient information, which will be provided to the National Xenotransplantation Database.

The firm must track Epicel, the FDA stated, because it is a "life-sustaining or life-supporting" product likely to be used outside of "device user facilities" and the failure of the product "would be reasonably likely to have serious adverse health consequences."

Shares of Genzyme (NASDAQ:GENZ) fell $1.51 Monday to close at $73.50.