Emergency Drug Fills Vaccine Void

Believing that the possible benefit outweighs any risk, FDA is allowing BioCryst Pharmaceuticals’ unapproved drug peramivir to be used in treating hospitalized flu patients. The new antiviral agent has been made available to battle the novel H1N1 influenza virus at a time when vaccines are overdue.

Just about a week ago, FDA issued an emergency use authorization for peramivir, following a request from the Centers for Disease Control & Prevention (CDC). With no adequate approved alternative, FDA says, the drug is meant for patients who aren’t responding to or can’t take Roche’s oral drug Tamiflu or GlaxoSmithKline’s inhaled product Relenza.

Discovered by Birmingham, Ala.-based BioCryst, peramivir inhibits neuraminidase, an enzyme that is critical to the replication of the flu virus. In the U.S., the company has the drug in Phase III clinical trials, which are being supported by $180 million in Department of Health & Human Services (HHS) funding. BioCryst’s partner, Shionogi, has completed late-stage studies in Japan and is filing for approval there.

“BioCryst has worked with HHS to enable the government to rapidly deploy an initial supply of peramivir, and we are prepared to deliver more,” says the firm’s CEO, Jon P. Stonehouse. The company already has provided HHS with 1,200 courses of treatment, each consisting of 600 mg per day for five days.

In preparation for orders from the U.S. or other governments, BioCryst is completing production of about 130,000 more treatment courses. According to analysts at the investment firm Leerink Swann, this could translate into about $39 million in sales for the company. Under the emergency use authorization, BioCryst is not allowed to promote its drug. CDC will control how and where peramivir is distributed.

Meanwhile, producers of H1N1 vaccines continue to have difficulty supplying as many doses as they once promised. As of Oct. 28, about 23.2 million doses were available, HHS Secretary Kathleen Sebelius said in a press briefing. Although the number of doses grew by about 9 million in one week, it is much below the 45 million doses that were anticipated to be on hand by mid-October and the total of 250 million doses reserved under contracts.