Friday, Mar 6, 1987

Genentech Files Lawsuit Against FDA for Legalization of Orphan Drug Standards for Human Growth Hormone

South San Francisco, Calif. -- March 6, 1987 --

Genentech, Inc., announced today that it has filed suit against the Food & Drug Administration in the U.S. District Court for the District of Columbia seeking a clarification of what constitutes a drug under the standards of the Orphan Drug Act.

"We are taking the unusual action of filing a lawsuit for clarification because the Orphan Drug Act does not adequately define what constitutes a drug," said Robert A. Swanson, chief executive officer of Genentech. "The new protein pharmaceuticals developed using recombinant DNA technology are different than conventional pharmaceutical, and we want the FDA to consider their special characteristics in regulating them.

"The Orphan Drug Act was intended to reward pioneering drug development efforts for rare diseases with a period of marketing exclusivity, during which the pioneer could recover development costs. Without the clarification we are suggesting, this may not happen."

That exclusivity means that no other company's version of the same drug can be approved for seven years for the same indication after the approval of the orphan drug status, which includes the right to seven years of exclusive marketing. Protropin is a recombinant DNA product with a 192 amino acid sequence.

Genentech has since developed a recombinant human growth hormone with a 191 amino acid sequence and applied for approval to the FDA. A second manufacturer, Eli Lilly & Company, has also applied for approval for a recombinant human growth hormone with the same 191 amino acid sequence. However, the two drugs are manufactured by different processes. The FDA is considering both drugs for approval.

"Since we began developing human growth hormone in 1978, Genentech has maintained its commitment to providing the best possible product for the most possible children," said Swanson. "Taking this action reflects our continuing commitment to this goal."

In November 1986, Genentech filed a Citizens Petition with the FDA requesting a clarification of the standards for determining orphan drug status for human growth hormone and for the right to notice of proposed FDA actions and an opportunity to respond in advance to those actions. Genentech met with officials of the FDA twice in recent days to discuss the situation and gain full consideration of its position.

"We have exhausted every other option available to us, leaving us no alternative but to force the issue with a lawsuit," said Swanson. "It is our hope that this matter will be resolved promptly and in the interest of having the best possible human growth hormone products available."

Genentech, Inc. is a leading biotechnology company, focusing on the development, manufacture and marketing of pharmaceuticals produced by recombinant DNA technology.

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