FDA Updates for the Week of Feb. 5, 2024: Review Dates and Advisory Committee Meetings Scheduled

FDA Assigns Action Date for Arexvy to Prevent RSV in 50 to 59 Population

The FDA has assigned priority review to GSK’s Arexvy and assigned a Prescription Drug User Fee Act date of June 7, 2024, to be prevent respiratory syncytial virus (RSV) in adults aged 50 to 59 who are at increased risk. Arexvy (respiratory syncytial virus vaccine, adjuvanted) is currently approved in the United States for adults aged 60 and over for the prevention of lower respiratory tract disease caused by RSV.

The application is supported by results from a phase 3 trial that assessed the immune response in 570 participants aged 50 to 59 with stable chronic diseases. Additionally, a group of 570 participants aged 50 to 59 without these chronic diseases were also evaluated compared with adults aged 60 and older. Data released in October 2023 showed Arexvy in 50-to-59-year-olds met the endpoint of being non-inferior to adults over the age of 60. GSK said in a press release that results from this trial will be presented at upcoming medical conferences and submitted for peer-reviewed publication.

GSK used a priority review voucher to reduce the FDA review period of this supplemental biologics license application (sBLA) by four months.

Arexvy was approved in May 2023 and was the first RSV vaccine approved. The CDC’s Advisory Committee on Immunization Practices (ACIP) has recommended the use of Arexvy in the over 60 population.

FDA Sets PDUFA Date for Acoramidis in Heart Failure Indication

The FDA has accepted BridgeBio’s new drug application (NDA) for acoramidis to treat patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a type of heart failure in older adults. ATTR-CM is a rare disease that leads to a build-up of the protein transthyretin in the left ventricle of the heart, leading to a thickening of the heart muscle.

The agency has set a Prescription Drug User Fee Act (PDUFA) action date of Nov. 29, 2024. Regulators are not currently planning to hold an advisory committee meeting.

Acoramidis is an oral small molecule stabilizer of transthyretin. It was designed to mimic the protective effects of T119M mutation, which has been associated with reduced risk of vascular events.

The application was based on positive results from ATTRibute-CM, the phase 3 study, in which 421 patients received acoramidis and 211 patients received placebo. The trial allowed for patients to take Vyndamax (tafamidis) after month 12. Vyndamax was approved by the FDA in 2019 to treat adults with transthyretin mediated amyloidosis.

FDA Grants Priority Review to Engineered Tissue for Vascular Replacement

The FDA has granted priority review to Humacyte’s biologics license application (BLA) for Human Acellular Vessel (HAV), which would be used to repair arteries in patients with extremity vascular trauma when synthetic graft is not indicated. The FDA has set a Prescription Drug User Fee Act (PDUFA) date of Aug. 10, 2024.

The HAV is a bioengineered tissue that is being researched as an implantable vascular replacement that does not require immune suppression. Designed to be ready for off-the-shelf use, the HAV can be produced at commercial scale in Humacyte’s existing manufacturing facilities.

The BLA, which was submitted in December 2023, is supported by positive results from the V005 phase 2/3 clinical trial, as well as real-world evidence from the treatment of wartime injuries in Ukraine under a Humanitarian Aid Program supported by the FDA.

FDA Sets Advisory Meeting for Abecma in Supplemental Multiple Myeloma Indication

The FDA will convene a meeting of the Oncologic Drugs Advisory Committee (ODAC) on March 15, 2024, to review data supporting Bristol Myers Squibb’s supplemental biologics license application (sBLA) for Abecma (idecabtagene vicleucel). BMS and partner 2seventy bio are seeking approval of Abecma to treat earlier lines of relapsed or refractory multiple myeloma (RRMM).

The advisory committee will review data related to the secondary endpoint of overall survival from the phase 3 KarMMa-3 study, which met its primary endpoint of improvement in progression-free survival (PFS) compared with standard regimens and reducing the risk of disease progression or death.

Abecma is currently approved to treat patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy. It is a B-cell maturation antigen (BCMA)-directed CAR-T cell immunotherapy. It binds to BCMA, a protein that is expressed on cancer cells in multiple myeloma.

The median overall survival was 41.4 months with Abecma and 37.9 months with standard regimens. The study allowed for cross over to Abecma treatment for patients originally assigned to standard regimens. BMS said that when adjusted for crossover, the data showed a median overall survival of 41.4 months for Abecma and 23.4 months for standard regimens.

Amneal Resubmits Application for Novel Parkinson’s Therapy

Amneal has resubmitted its application for an oral formulation of carbidopa/levodopa (CD/LD) extended-release capsules for the treatment of Parkinson’s disease. The FDA had issued a complete response letter (CRL) in July 2023 for IPX203. The FDA had asked for additional pharmacokinetic data of carbidopa, one of the drugs in IPX203. Pharmacokinetic data provides information about how the drug is adsorbed, distributed, metabolized and excreted from the body.

The FDA’s CRL for IPX203 indicated that although an adequate scientific bridge was established for the safety of one ingredient, levodopa, based on pharmacokinetic studies, it was not adequately established for carbidopa. The letter did not identify any issues with respect to the efficacy or manufacturing of IPX203.

IPX203 contains extended-release beads that consist of levodopa, coated with a sustained release polymer, to allow for slow release of the drug. Additionally, this formulation contains immediate-release granules that consist of carbidopa and levodopa, with a disintegrant polymer to allow for rapid dissolution. This formulation of IPX203 is different from Rytary extended-release capsules, Amneal’s extended-release carbidopa/levodopa treatment for Parkinson’s disease that the FDA approved in 2015.