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Regulatory Focus™ > News Articles > FDA Changes Policy Hours Before Approvals, Giving Companies Gift Potentially Worth Billions

FDA Changes Policy Hours Before Approvals, Giving Companies Gift Potentially Worth Billions

Posted 13 October 2014 | By Alexander Gaffney, RAC

FDA Changes Policy Hours Before Approvals, Giving Companies Gift Potentially Worth Billions

On Friday, two companies were the lucky recipients of gifts never before given by the US Food and Drug Administration (FDA)—gifts potentially worth millions, if not billions of dollars in value.


In the US, pharmaceutical manufacturers who obtain approval to market a "new" drug from FDA are eligible for varying degrees of market- (rather than patent-) based exclusivity.

For New Chemical Entities (NCEs), that means five years of protection during which time FDA will not approve any generic equivalents. For "new" drugs that represent a new use for an old drug, or a new dose of an old drug, FDA is able to grant just three years of patent protection.

Historically, that meant that a fixed-dose combination (FDC) product consisting of at least one already-approved entity was ineligible for five years of exclusivity—just three.

New Guidance

But on the morning of 10 October 2014, FDA released a new, final policy which markedly changed its interpretation of the drug marketing exclusivity provisions of the Federal Food, Drug and Cosmetic Act (FD&C Act) in response to multiple petitions from the pharmaceutical industry.

"Accordingly, a 5-year NCE exclusivity determination will be made for each drug substance in a drug product, not for the drug product as a whole," FDA wrote in the guidance, New Chemical Entity Exclusivity Determinations for Certain Fixed-Dose Combination Drug Products.

"As a result, an application for a fixed-combination submitted under section 505(b) of the FD&C Act will be eligible for 5-year NCE exclusivity if it contains a drug substance, no active moiety of which has been approved in any other application under section 505(b).30," FDA explained. "For example, a fixed-combination drug product that contains a drug substance with a single, new active moiety would be eligible for 5-year NCE exclusivity, even if the fixed-combination also contains a drug substance with a previously approved active moiety."

However, the policy came with a significant setback for industry: The new policy would only be effective as of 10 October 2014, and not retroactively.

New Approvals

But while that policy was a potential setback for the sponsors of 19 already-approved FDCs (some of which might not be eligible for the protection), it's now a boon for two FDCs approved on 10 October 2014.

Just hours after FDA released its final FDC exclusivity policy, it announced the approval of Gilead's Harvoni (ledipasvir and sofosbuvir), a FDC used to treat chronic hepatitis C virus genotype 1 infections, and Eisai's Akynzeo (netupitant and palonosetron), an FDC used to treat nausea and vomiting in patients undergoing cancer chemotherapy.

Had the drugs been approved just one day earlier, neither would have been eligible for NCE exclusivity. Gilead's Harvoni includes sofosbuvir, a drug approved in 2014 and marketed as Sovaldi. Eisai's Akynzeo includes oral palonosetron, approved in 2008 under the brand name Aloxi.

But in addition to the existing drugs, both products contain new chemical entities that boost the effectiveness of the products. Harvoni's ledipasvir, for example, allows the drug to be used without interferon or ribavirin. Akynzeo's netupitant apparently boosts the effectiveness of the drug, protecting a patient from nausea and vomiting "during both the acute phase and delayed phase after the start of chemotherapy."

For both companies, the additional two years of market exclusivity could potentially be valuable. Harvoni is expected to become a blockbuster, meaning Gilead will have at least two extra years to reap billions in revenue before it has to begin defending its patents on the drug in earnest. Gilead said it plans to sell the product for $94,500 for a 12-week course of treatment.

And while projected sale data for Akynzeo was not available, it's hard to envision a scenario in which an extra two years of exclusivity could possibly hurt a company's bottom line. Sales of the drug's predecessor, Alixo, reached $537 million in 2011 according to data from Evaluate.

Both Harvoni's and Akynzeo's market exclusivity periods end on 10 October 2019,


FDA Approval Notices: (Harvoni) (Akynzeo)


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