Orphan Drugmaker|Wins Exclusivity

(CN) - The U.S. Food and Drug Administration must grant orphan-drug marketing exclusivity to the maker of a medicine that treats a rare complication of the shingles virus, a federal judge ruled.

Enacted in 1983, the Orphan Drug Act encourages pharmaceutical companies to develop medicines to treat diseases that affect fewer than 200,000 people in the United States, by offering financial incentives, such as seven-year exclusivity. How drugs qualify for this exclusivity is at the heart of the matter.

Newark, Calif.-based Depomed developed the drug Gralise to treat post-herpetic neuralgia (PHN), a rare complication of shingles that causes lasting nerve pain after other symptoms have cleared up.

Though the FDA designated Gralise as an orphan drug and approved it for public marketing, it refused to recognize Gralise's exclusivity period because it had previously given market approval to Pfizer's PHN drug, Neurontin.

Depomed sued the FDA and the U.S. Department of Health and Human Services in September 2012, claiming that their failure to grant Gralise market exclusivity when it met the two statutory requirements for qualification violated the Administrative Procedures Act.

The FDA moved to dismiss, claiming it does not have to grant Gralise orphan-drug exclusivity because Depomed cannot not prove that Gralise, whose active ingredient is gabapentin, is superior to Neurontin, which has the same active ingredient.

U.S. District Judge Ketanji Brown Jackson in Washington, D.C., granted Depomed summary judgment on Sept. 5, finding that "the plain language of the Orphan Drug Act requires the FDA to recognize exclusivity for Gralise."

Jackson rejected the FDA's argument that the Orphan Drug Act does not say whether the agency must automatically grant exclusivity to a designated orphan drug despite the existence of previously approved drugs, and that the court must therefore defer to its decision.

"There is nothing ambiguous about Congress's statutory statement that if the FDA designates a drug as an orphan drug and approves that drug for marketing, the agency cannot approve another application for the marketing of a new such drug for a period of seven years," the 33-page ruling states.

Though the FDA controls when marketing exclusivity begins because it controls which drugs it designates as orphan drugs and approves for public use, "the language of the act's exclusivity provision does not permit or invite any discretion on the part of the FDA regarding whether or not to continue authorizing new such drug marketing applications once an orphan drug has been so designated and approved," the ruling adds.

In fact, Congress specifically allowed for only two situations in which the FDA can ignore automatic marketing exclusivity: if a manufacturer cannot make enough of a drug to meet demand, or if the manufacturer gives the FDA written consent to allow another company to market the same drug, according to the ruling.

The FDA's claim that the statute contains a "gap" concerning whether a new drug with the same active ingredient can be given market exclusivity if another drug for the condition has previously been approved also failed.

"As the D.C. Circuit has noted repeatedly, the fact that Congress has 'not mentioned' a particular agency concern in a governing statute does not mean that 'Congress is 'silent or ambiguous' as to that issue," Jackson wrote.

Citing the ruling in Baker Norton Pharmacies v. FDA, the FDA argued that the word "drug" is ambiguous enough to demonstrate a statutory gap that justifies its use of discretionary authority.

Jackson shot down this line of reasoning by pointing out that the Baker Norton ruling revolved around how the FDA defines the term "such drug" to decide if it can approve the drug during a period of marketing exclusivity.

In this case, whether Gralise counts as a "such drug" is irrelevant because there are no other designated gabapentin drugs to treat PHN, so the FDA cannot use the "such drug" ambiguity to demand that Depomed demonstrate clinical superiority before obtaining exclusivity for Gralise, Jackson wrote.

"Try as they might, defendants cannot square their insistence that the FDA has the discretion to address this situation with the fact that, under the statute, Congress did not give the FDA any discretionary authority to grant or deny exclusivity at all - rather, as mentioned previously, Congress forbade the FDA from granting any further approvals when the statutory conditions were met."

The government also argued that granting exclusivity to Gralise would hinder rather than promote the development of new orphan drugs.

Though Jackson acknowledged that the FDA "struggle[d] valiantly" to make its case, its "policy argument misses the mark by a mile."

Granting a seven-year exclusivity period to Depomed would not be unfair to other manufacturers because they had the same chance to obtain orphan designation and public use approval for their drugs, but chose not to do so, the ruling states.

Moreover, Jackson wrote, the exclusivity statute is specifically designed to prevent competitors from developing and marketing new drugs during a company's exclusivity period, to encourage companies to develop orphan drugs in the first place. The FDA may think this structure is "absurd," but that is the structure that Congress intended, according to the ruling.

Jackson nixed the FDA's other policy argument: that drug manufacturers could use the exclusivity statute to maintain exclusivity indefinitely by modifying their formulas and resubmitting their applications.

The FDA can easily stop this from happening by passing regulations forbidding such abuse of process, or requiring manufacturers to demonstrate clinical superiority, the ruling states.

Jackson ordered the FDA to recognize Gralise's seven-year market exclusivity period without forcing Depomed to submit additional proof.

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