UPDATE 2-US FDA panel backs Questcor's Acthar

Thu May 6, 2010 5:28pm EDT

* Questcor seeking FDA approval of gel for infant use

* Shares halted pending advisory panel meeting

* FDA decision due by June 11 (Adds panel and company comments, details on drug)

ADELPHI, Md., May 6 (Reuters) - Questcor Pharmaceuticals Inc's QCOR.O Acthar gel is safe and effective for treating certain seizures in infants, a panel of medical experts told the U.S. Food and Drug Administration on Thursday.

Acthar is already widely used by doctors to treat infantile spasms, a form of epilepsy, even without FDA consent. Questor has been seeking the agency's approval for years, saying it will help standardize care and offer specific dose guidelines.

First approved in 1952, the injectable drug is already cleared for dozens of uses such as multiple sclerosis.

"It's better than just about anything out there, and it's been around for about 50 years," said panelist Eric Felner, an Emory University pediatrics professor in Atlanta.

The panel voted 22-1 in favor of the drug's efficacy in treating spasms in infants. It voted 20-1, with two members abstaining, that there was enough evidence backing the drug's safety, although panelists were mixed over how manageable and reversible the complications were.

Both the FDA and Questcor agreed that Acthar has already become the standard treatment for infant spasms by doctors, who can legally use it "off label." Questcor, which acquired Acthar from then-Aventis Pharmaceuticals in 2001, sought FDA approval for the infant spasm use in 2006 but was denied.

Winning FDA's backing is key so that doctors and parents have consistent and accurate dosing information as well as other information on side effects, Questcor told panelists.

It also allows the company to legally market the new use.

At the meeting, FDA's panel of outside advisors -- many of whom said they already use Acthar for their infant patients -- said the data was compelling but more follow-up safety information was needed.

Some FDA staff scientists had earlier expressed concern in interpreting Questcor's data, saying much of it was collected from previously published studies.

Questcor officials pointed to decades of successful Acthar use in infants with such seizures. More than 30,000 babies have received the drug for spasms since the drug's approval, it said, and about 40 percent of all infants with such epilepsy were given Acthar in the last two years.

The only other FDA-approved treatment for infantile spasms is vigabatrin, sold under the brand name Sabril by Danish drugmaker H Lundbeck A/S (LUN.CO). But Questcor said that rival product has a low success rate and that patients need other options. Another drug, prednisone, is also given "off-label."

More than 20 parents and physicians asked FDA's advisers to back the drug, many calling it life-saving therapy as they saw their children's or patients' seizures stop after just days. Questor paid for most of them to travel to the meeting.

Side effects are serious and can include infection, convulsions and hypertension. But many parents said it was worth the risk to head off possible brain damage and developmental delays from the seizures themselves.

"Even though the data isn't perfect, there was nothing that was unexpected in terms of of the side effects," said panelist Samuel Frank, a neurology professor at Boston University and the panel's acting consumer representative.

Although the FDA and its panelists are not supposed to consider pricing, Acthar's cost was an issue for some panelists and public speakers. While some health insurers cover the infant use based on doctors' recommendations, others have cited its unapproved status to reject reimbursement.

The drug's list price is $23,000 per vial, and treatment typically requires about four vials, the company said.

Questcor's Chief Science Officer David Young said the high price is necessary to maintain the complicated manufacturing process, but that the drugmaker works with various groups to ensure patients can obtain the drug, many at no or low cost.

The FDA is expected to make the final decision by June 11. The FDA usually, but not always, follows its panel's advice.

Shares of the drugmaker were halted on Thursday pending the outcome of the meeting. (Reporting by Susan Heavey. Editing by Robert MacMillan and Richard Chang)