In the second week in May, the Peripheral and Central Nervous System Drugs Advisory Committee to the FDA held a panel meeting to discuss approval for using Questcor’s (QCOR) Acthar in the treatment of infantile spasms. The meeting went very well as the panel's purpose was to advise the FDA on their opinion of whether or not Acthar should be approved by the FDA for this condition. The vote by panel members was overwhelmingly in favor of an approval for Acthar. What this article will do is discuss the breakdown of the voting and give insight on what can be expected from the FDA on September 11th.
Since the panel meeting, the original PDUFA date of June 11 was extended to September 11. The FDA is currently reviewing information regarding labeling, REMS and a potential post-approval commitment that they requested from Questcor. If Questcor obtains FDA approval to add IS to the Acthar label in September, then Questcor expects to launch Acthar, for the treatment of IS, during the Child Neurology Society Annual Meeting on October 4th. An approval of their drug would be the first time that they would be able to promote Acthar for the treatment in infantile spasms. With an approved indication, they can target public education efforts regarding the importance of early diagnosis and effective treatment of IS.
The May FDA Advisory Panel meeting provided some insight on how the FDA might view the approval of Acthar for Infantile Spasm (IS). The key points of this meeting were to address four questions posed from the FDA as a result of their FDA submission of Acthar and the convening of the Advisory Panel. There were 24 members on the panel and only 1 non-voting participant. At the beginning of the meeting, Dr. Russell Katz (Director of Peripheral and CNS Drug Safety for the FDA) spoke and mentioned that the submission had unusual contents and that because of this it resulted in this Advisory Panel Meeting b/w Questcor & the FDA.
After speaking with QCOR and seeing the powerpoint presentation, the FDA Agency (Advisory Panel) concluded prior to the meeting that since the presentation was well balanced, the Advisory Panel did not need to make a presentation at the Advisory Committee Meeting. This was a rare thing for the Advisory Panel to not give a presentation following a company presentation. The submission by Questcor and approval of Acthar would give them a 7 year orphan exclusivity for Infantile Spasms.
There were four questions that needed to be answered from Questcor to the FDA Advisory Panel in addressing Acthar.
- Did Questcor provide substantial evidence of effectiveness of Acthar as a treatment for patients with Infantile Spasms? Committee voted 22-1 that yes they did.
- Did Questcor provide evidence of Acthar having sustained effectiveness for the recommended course of treatment? Committee voted 16-7 that yes they did. Explanation: During the after-vote polling of all the members, several of the NO votes noted that they felt they provided evidence but were not sure of what the FDA definition of “sustained” was in the question so for that reason they voted no.
- Did Questcor provide evidence that adverse events associated with Acthar are manageable and reversible? Committee voted 12 to 10 with one abstention that they had not. During aftervote polling, panel members expressed confusion about the question noting belief that Acthar was safe but was not sure if Questor provided evidence that side effects of Acthar were manageable and reversible even though they knew the side effects were under their care.
- The most important question was, did Questcor submit sufficient evidence of safety of Acthar at an effective dosing regimen? Committee voted YES with 20-1 vote and 2 abstentions. The panel requested clarification on the question from Dr. Katz of the FDA. Dr. Katz stated that the intent of the question was to vote whether the sponsor (Questcor) had provided evidence that the benefit of using Acthar to treat Infantile Spasms (IS) outweighed the risks. Committee voted YES with 20-1 vote and 2 abstentions. Questcor official stated that, “A YES vote for a benefit/risk question is usually viewed as a YES vote for approval. Thus a vote of 20-1 with two abstentions by the Advisory Committee would be viewed as a resounding vote of support by the committee for the benefit/risk profile as well as for the approval of Acthar for Infantile Spasms (IS).
In conclusion, approval for Acthar is very likely on September 11th now that the FDA has a strong recommendation from the Advisory Panel and has been specific with regards to the requests that they had for Questcor. As earlier mentioned, The FDA is currently reviewing information regarding labeling, REMS, and a potential post-approval commitment that they requested from Questcor. These requests are not uncommon for the FDA and are pretty minor in detail even though they are important to complete for them to reach an approval decision.
What is also important is that there is no question of safety or efficacy with the FDA so no additional studies are needed and thus the two month delay to finalize details. Getting a thumbs up from the Advisory Panel was Questcor’s biggest challenge and now the task is providing the information that the FDA needs for final approval. This should prove to be a very achievable goal.
Disclosure: Author is long QCOR