5 FDA Decisions to Watch in Q4 2024

After a historic third quarter, which saw noteworthy US Food and Drug Administration (FDA) approvals for schizophrenia, COPD, IgA nephropathy, and more, some of the year’s most anticipated decisions take place during the final 3 months of 2024. With several high-impact agents and therapies under review, both patients and healthcare providers are poised to witness decisions that could shape care for decades.

To help our audience stay informed, we have curated a list of 5 highly anticipated approvals expected by year-end, along with related content from our past coverage as well as sister publications within the MJH Life Sciences family.

Acoramidis (BridgeBio Pharma)

PDUFA: November 29, 2024

Acoramidis, a next-generation, orally administered, highly potent, small molecule stabilizer of transthyretin, has the potential to become the second approved therapy for management of transthyretin amyloid cardiomyopathy (ATTR-CM). An underdiagnosed condition characterized by stiffening heart tissue caused by amyloid fibril deposits, ATTR-CM is estimated to impact more than 120,000 US adults have ATTR-CM, with 5000 to 7000 new cases diagnosed each year.

Billed as a more potent tetramer stabilizer than tafamidis, which is currently the only approved therapy for ATTR-CM, the application for acoramidis is based on data from the phase 3 ATTRibute-CM trial. According to a November 2023 release from BridgeBio Pharma, acoramidis is estimated to have intellectual property protection out to at least 2039.

Expert Perspective: Ahmad Masri, MD, MS: AHA 2023 Data Highlights Potential of Acoramidis

Olezarsen (Ionis Pharmaceuticals)

PDUFA: December 19, 2024

Olezarsen, an investigational RNA-targeted medicine, could usher in a new era of management as the first therapy to receive approval from the FDA for treatment of familial chylomicronemia syndrome (FCS) in adult patients. A rare genetic disease, FCS is associated with elevated triglyceride levels and episodes of pancreatitis.

The recipient of Fast Track, Orphan Drug, and Breakthrough Therapy designations from the FDA, the agent was accepted for Priority Review in June 2024. The application for olezarsen is based on data from the phase 3 BALANCE trial, which was presented at the American College of Cardiology 2024 Scientific Sessions and simultaneously published in the New England Journal of Medicine. In their release announcing acceptance for Priority Review, Ionis noted it was also evaluating olezarsen for the treatment of severe hypertriglyceridemia in 3 phase 3 clinical trials.

Expert Perspective: Christie Ballantyne, MD: 'Exciting' Time for FCS Pipeline

Sotagliflozin (Lexicon Pharmaceuticals)


PDUFA: December 20, 2024

Sotagliflozin, the first SGLT1/2 inhibitor to receive approval from the FDA, is seeking an indication for use an adjunct to insulin therapy for glycemic control intype 1 diabetes and chronic kidney disease. Already approved by the FDA for the treatment of worsening heart failure in May 2023, sotagliflozin’s regulatory journey for type 1 diabetes dates back to 2018 when Sanofi and Lexicon Pharmaceuticals applied for an indication in type 1 diabetes. The FDA issued a Complete Response Letter for this application in July 2019.

The agent received approval for this indication within the European Union in 2019, but was never brought to market and, in September 2019, Sanofi moved to terminate its partnership with Lexicon Pharmaceuticals. With this, Lexicon Pharmaceuticals regained global rights for the development and commercialization of Zynquista in both type 1 and type 2 diabetes. As part of their review of the current application, the FDA has scheduled an Endocrinologic and Metabolic Drugs Advisory Committee meeting on October 31, 2024 to discuss the application.

Related: Diabetes Dialogue: Sotagliflozin for T1D and Other ADCES 2024 Highlights

Oral sulopenem (Iterum Therapeutics plc)

PDUFA: October 25, 2024

Oral sulopenem is seeking approval for the treatment of uncomplicated urinary tract infections in adult women. The application is supported by the phase 3 REASSURE trial and, if approved, would become the first oral penem approved in the US, according toIterum Therapeutics.

The agent was the subject of an FDA Antimicrobial Drugs Advisory Committee on September 09, 2024. Although the meeting did not include a vote, the committee determined, if approved, oral sulopenem would provide substantial benefit to a subset of patients; there is a risk for off-label use as well as a risk to the community and individuals in the spread of antimicrobial resistance; a plan regarding post-marketing analysis is essential; and observation of changes to the rates of antimicrobial resistance may be necessary.

Related Content: FDA Advisory Committee Meets to Discuss NDA of Oral Sulopenem for uUTI

DFD-29 (Journey Medical)

PDUFA: November 04, 2024

DFD-29 is seeking approval for the treatment of inflammatory lesions and erythema in adult patients with rosacea. A low-dose, modified-release capsule formulation of minocycline, the application for DFD-29 is based on data from the MVOR-1 and MVOR-2, which concluded use was associated statistical superiority over both the current standard-of-care treatment and placebo for Investigator’s Global Assessment treatment success as well as the reduction in the total inflammatory lesion count. If approved, potential to become the only oral, systemic therapy to address both inflammatory lesions and erythema from rosacea.

Related Content: FDA Accepts Journey Medical’s NDA for DFD-29 for Rosacea

References:

1. BridgeBio Pharma. BridgeBio shares recurrent event analysis of attribute-cm, demonstrating a 42% reduction by ACORAMIDIS on the composite endpoint of all-cause mortality and recurrent cardiovascular-related hospitalization events. BridgeBio. September 27, 2024. Accessed October 4, 2024. https://bridgebio.com/news/bridgebio-shares-recurrent-event-analysis-of-attribute-cm-demonstrating-a-42-reduction-by-acoramidis-on-the-composite-endpoint-of-all-cause-mortality-and-recurrent-cardiovascular-related-hospitalizat/.
2. ICER. Disease Modifying Therapies for the Treatment of Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM). Institute for Clinical and Economic Review. April 5, 2024. Accessed October 4, 2024. https://icer.org/wp-content/uploads/2024/04/ATTR-CM_Revised-Scope_For-Publication_04052024.pdf.
3. Bonaguro AM, Caballero VT, Narang N. Clarifying the natural history of transthyretin cardiac amyloidosis: Insights from the attribute-cm trial. American College of Cardiology. April 9, 2024. Accessed October 4, 2024. https://www.acc.org/Latest-in-Cardiology/Articles/2024/04/04/10/30/Clarifying-the-Natural-History-of-Transthyretin-Cardiac-Amyloidosis.
4. BridgeBio Pharma. Bridgebio Pharma presents additional clinical outcomes data from the phase 3 attribute-cm study of Acoramidis in patients with transthyretin amyloidosis cardiomyopathy (attr-cm). BridgeBio. November 12, 2023. Accessed October 4, 2024. https://bridgebio.com/news/bridgebio-attribute-cm-phase-3-data-aha.
5. Ionis Pharmaceuticals. Ionis announces Olezarsen FCS New Drug Application accepted for Priority Review and enrollment in phase 3 shtg program completed. PR Newswire: press release distribution, targeting, monitoring and marketing. June 25, 2024. Accessed October 4, 2024. https://www.prnewswire.com/news-releases/ionis-announces-olezarsen-fcs-new-drug-application-accepted-for-priority-review-and-enrollment-in-phase-3-shtg-program-completed-302181549.html.
6. Campbell P. FDA approves Sotagliflozin for treatment of heart failure. HCP Live. May 26, 2023. Accessed October 4, 2024. https://www.hcplive.com/view/fda-approves-sotagliflozin-for-treatment-of-heart-failure.
7. Lexicon Pharmaceuticals, Inc. Lexicon Pharmaceuticals announces FDA Advisory Committee meeting for zynquista. GlobeNewswire News Room. August 21, 2024. Accessed October 4, 2024. https://www.globenewswire.com/news-release/2024/08/21/2933458/0/en/Lexicon-Pharmaceuticals-Announces-FDA-Advisory-Committee-Meeting-for-Zynquista.html.
8. Iterum Therapeutics plc. Iterum Therapeutics reports second quarter 2024 financial results. Iterum Therapeutics plc. August 14, 2024. Accessed October 4, 2024. https://ir.iterumtx.com/press-releases/detail/131/iterum-therapeutics-reports-second-quarter-2024-financial.
9. Clarke H. FDA Advisory Committee meets to discuss NDA of Oral Sulopenem for uuti. Urology Times. 9AD. Accessed October 4, 2024. https://www.urologytimes.com/view/live-updates-from-fda-advisory-committee-meeting-on-oral-sulopenem-for-uuti.
10. Stewart J. DFD-29 (minocycline hydrochloride): What is it and is it FDA approved? Drugs.com. March 20, 2024. Accessed October 4, 2024. https://drugs.com/history/dfd-29.html.
11. Bader K. FDA accepts Journey Medical’s NDA for DFD-29 for rosacea. Dermatology Times. March 18, 2024. Accessed October 4, 2024. https://www.dermatologytimes.com/view/fda-accepts-journey-medical-s-nda-for-dfd-29-for-rosacea.