FDA Advisory Committees - FDA Center for Drug Evaluation and Research (CDER) - Peripheral and Central Nervous System Drugs Advisory Committee (PCNS)
Upcoming Meetings
Past Meetings
On Friday, June 9th, 2023, the Peripheral and Central Nervous System Drugs Advisory Committee ([PCNS]) will discuss supplemental biologics license application (sBLA-761269/s-001), for LEQEMBI (lecanemab) solution for intravenous infusion, submitted by Eisai, Inc., for the treatment of Alzheimer's disease, initiated in patients with mild cognitive impairment or mild dementia stage of disease. The committee will also discuss the confirmatory study, BAN2401-G000-301, conducted to fulfill post-marketing requirement.
The Psychopharmacologic Drugs Advisory Committee (PDAC) and the Peripheral and Central Nervous System Drugs Advisory Committee (PDAC) of the USFDA has scheduled a meeting on Friday, April 14th, 2023. During the meeting, the committee will discuss supplemental new drug application (sNDA 205422 s009), efficacy supplement for REXULTI (Brexpiprazole) tablets, submitted by Otsuka Pharmaceutical Company, Ltd., and Lundbeck, Inc., for the proposed treatment of agitation associated with Alzheimer’s dementia.
On Wednesday, March 22, 2023, the PCNS advisory committee will discuss an NDA -215887, for tofersen (BIIB067) intrathecal injection, submitted by Biogen for the treatment of ALS associated with a mutation in the superoxide dismutase 1 gene.
The Peripheral and Central Nervous System Drugs Advisory Committee ([PCNS]) on 7th September 2022, Wednesday, will reconvene to review a new drug application (NDA) 216660, for oral formulation (suspension powder) of sodium phenylbutyrate/taurursodiol (AMX0035) for the treatment of Amyotrophic lateral sclerosis (ALS). The advisory committee is reconvening to debate over additional analyses of data from Amylyx’s clinical studies that were determined by the FDA to constitute a major amendment to the NDA.
The Committee voted unanimously, by a vote of 13-Yes to 0-No, with no abstentions that the benefit-risk profile of Epidiolex (cannabidiol oral solution), by GW Pharmaceuticals (GW), is favorable for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome in patients 2 years of age and older.
The Committee met to discuss the proposed use of Translarna (ataluren) by PTC Therapeutics (PTC) for the treatment of dystrophinopathies resulting from nonsense mutations in the dystrophin gene, including nonsense mutation Duchenne Muscular Dystrophy (nmDMD). A majority of the Committee, 10 of 11 members, voted that “although it is possible that ataluren may be effective, the data are inconclusive, and more work would be needed to establish whether ataluren is effective.”
The Committee voted that Sarepta Therapeutics, Inc. has not provided substantial evidence from adequate and well-controlled studies that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit, by a vote of 3-Yes to 7-No, with 3 abstentions.
The Committee also narrowly voted that the clinical results of the single historically-controlled study (Study 201/202) do not provide substantial evidence that eteplirsen is effective for the treatment of Duchenne muscular dystrophy (DMD), by a vote of 6-Yes to 7-No, with no abstentions.
Sarepta had submitted an application for eteplirsen for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping, under new drug application (NDA) 206488.
The PDUFA goal date for the FDA decide on whether to approve the application is May 26, 2016.
The Committee was scheduled to discuss a new drug application for eteplirsen injection for intravenous infusion, submitted by Sarepta Therapeutics, Inc., for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Tarius will monitor the Federal Register for FDA's announcement of a new date for the meeting. See the FDA Announcement
At this meeting, the FDA did not ask the Committee to vote on whether to recommend approval of drisapersen but to vote on whether key aspects of the data strengthen, weaken, or have no effect on the interpretation of the clinical results. The Agency also asked the Committee to discuss the overall strengths and weaknesses of the data for the proposed indication, in a ninth question that was added on the day of the meeting.
Peripheral and Central Nervous System Drugs Advisory Committee (PCNS)
PCNS reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of neurologic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs
he committee will discuss biologics license application (BLA) 761178, for aducanumab solution for intravenous infusion, submitted by Biogen Inc., for the treatment of Alzheimer’s disease.