June 2024 decisions expected from the FDA

Your monthly synopsis of new drugs expected to hit the market

May 15, 2024

Your monthly synopsis of new drugs expected to hit the market

FDA DECISIONS EXPECTED

At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs likely to be approved by the United States (U.S.) Food and Drug Administration (FDA).

Drug pipeline for June 2024

June 2024: sofpironium

Botanix Pharmaceuticals (Botanix) is seeking FDA approval of sofpironium 15% gel for the treatment of primary axillary hyperhidrosis. Botanix resubmitted their application for the once-daily, topical anticholinergic agent after receiving a Complete Response Letter (CRL) from the FDA in September 2023. The CRL was related to patient instructions; no clinical concerns were identified in the CRL. Sofpironium was evaluated in the double-blinded, vehicle-controlled CARDIGAN I and CARDIGAN II studies, which showed statistically significant improvements in both co-primary endpoints.¹ In the first endpoint, more patients treated with sofpironium compared those who received vehicle achieved ≥ 2-point improvement in Hyperhidrosis Disease Severity Measure–Axillary (HDSM-Ax-7) from baseline to end of treatment (six weeks) (CARDIGAN I, 49.3% versus 29.4%, respectively [p<0.001]; CARDIGAN II, 63.9% versus 47%, respectively [p=0.003]). In the second endpoint, sofpironium demonstrated statistically significant responses compared to vehicle for change in gravimetric sweat production (GSP) (CARDIGAN I, -129.5 mg versus -99.3 mg, respectively [p=0.002]; CARDIGAN II, -145.9 mg versus -131.7 mg, respectively [p=0.03]).

6/10/2024: elafibranor

Elafibranor, a dual peroxisome proliferator-activated receptor alpha and delta (PPAR-α/δ) agonist by Genfit and Ipsen, was submitted to the FDA for the treatment of primary biliary cholangitis (PBC). The once-daily, oral agent was evaluated in the double-blind, phase 3 ELATIVE trial, which demonstrated 51% of patients who received elafibranor compared to 4% who received placebo achieved the primary endpoint of biochemical response (defined as an alkaline phosphatase [ALP] level of < 1.67 × upper limit of normal (ULN), with a reduction of ≥ 15% from baseline, and normal total bilirubin levels) at week 52.² Elafibranor was granted Breakthrough Therapy and Orphan Drug designations as well as a Priority Review by the FDA. If approved, elafibranor will be the first peroxisome proliferator-activated receptor (PPAR) agonist available to treat PBC in patients with an inadequate response to ursodeoxycholic acid and will be an additional therapeutic option to obeticholic acid (Ocaliva®).

For more information, read the elafibranor Deep Dive in our April 2024 Quarterly Pipeline.

6/12/2024: tarlatamab

Amgen submitted their first-in-class delta-like ligand 3 (DLL3)-directed bispecific T-cell engager (BiTE), tarlatamab, for the treatment of advanced small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy. In the open-label, phase 2 DeLLphi-301 trial, after a median follow-up of just over 10 months, the overall response rate (ORR) with tarlatamab 10 mg and 100 mg doses were, 40% (primary endpoint) and 32%, respectively.³ The progression-free survival (PFS) was 4.9 months and 3.9 months with each dose, respectively. In the clinical trial, tarlatamab was administered intravenously (IV) every two weeks. If approved, it will provide a new approach to combat SCLC, a disease with a large unmet need. The FDA granted tarlatamab Breakthrough Therapy and Orphan Drug designations as well as a Priority Review and Real-Time Oncology Review (RTOR).

For more information, read the tarlatamab Deep Dive in our April 2024 Quarterly Pipeline.

6/14/2024: imetelstat

Geron is awaiting FDA approval of imetelstat for the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndromes (MDS) who have failed to respond to, have lost response to, or are ineligible for erythropoiesis-stimulating agents (ESAs). The double-blind, phase 3 IMerge trial demonstrated that significantly more patients treated with imetelstat achieved the primary endpoint of red blood cell (RBC) transfusion independence of ≥ 8 weeks compared to those who were given placebo (40% versus 15%, respectively; p=0.0008).4 In the study, imetelstat was administered IV every four weeks. Imetelstat is a first-in-class telomerase inhibitor. If approved, it would provide a new mechanism of action for treating anemia due to MDS after failure of ESA therapy. The agent received Fast Track and Orphan Drug designations by the FDA and, on March 14, 2024, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted in favor (12:2) of imetelstat for the treatment of anemia for low-risk MDS patients who are refractory or intolerant to ESAs.

For more information, read the imetelstat Deep Dive in our April 2024 Quarterly Pipeline.

6/17/2024: 21-valent pneumococcal conjugate vaccine (V116)

V116, by Merck, was submitted to the FDA for immunization to prevent invasive pneumococcal disease and pneumococcal pneumonia in adults. The FDA granted it a Priority Review. V116 is being evaluated across the ongoing, phase 3 STRIDE trial program, which includes patients who have and have not received prior pneumococcal vaccination.5 Among the studies, V116 elicited comparable immune responses for the serotypes shared with pneumococcal 15-valent conjugate vaccine (PCV15) and polyvalent 23-valent (PPSV23) and elicited even higher immune responses for the serotypes covered by V116 only. These responses occurred irrespective of the previous pneumococcal vaccine received or time since prior pneumococcal vaccination in patients 50 years and older. Comparable immune responses were also observed with V116 in adults 18 years and older with human immunodeficiency virus (HIV) infection. The safety profile of V116 was also similar to the safety profiles of the comparator vaccines.

6/26/2024: ensifentrine

Verona Pharma submitted ensifentrine to the FDA for the treatment of chronic obstructive pulmonary disease (COPD). Ensifentrine is an inhaled selective, dual inhibitor of phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4) that has a combined effect on airway inflammation, bronchodilation, and ciliary beat frequency in bronchial epithelial cells. The agent was administered via a nebulizer in the ENHANCE-1 and ENHANCE-2 trials in patients with moderate to severe symptomatic COPD. In both trials, ensifentrine met the primary endpoint, demonstrating significant improvement from baseline to week 12 in average 12-hour forced expiratory volume in one second (FEV1) area-under-the-curve (AUC0–12 h) compared to placebo (difference from placebo: ENHANCE-1, 87 mL and ENHANCE-2, 94 mL; p< 0.001 for both).6 If approved, ensifentrine would be the first dual PDE3/PDE4 inhibitor indicated for the treatment of COPD.

For more information, read the ensifentrine Deep Dive in our January 2024 Quarterly Pipeline.

6/26/2024: patritumab deruxtecan

Daiichi-Sankyo and Merck are seeking approval of patritumab deruxtecan for the treatment of adults with locally advanced or metastatic epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC) previously treated with two or more systemic therapies. Patritumab deruxtecan was granted a Breakthrough Therapy designation and is undergoing a Priority Review and RTOR by the FDA. It is a first-in-class human epidermal growth factor receptor 3 (HER3)-directed antibody drug conjugate (ADC). The FDA submission was supported by the open-label, phase 2 HERTHENA-Lung01 trial that demonstrated patritumab deruxtecan resulted in an ORR of 29.8% (primary endpoint), including one complete response and 66 partial responses.7 The median duration of response was 6.4 months. If approved, patritumab deruxtecan could become the first HER3-directed agent available in the U.S. and the second deruxtecan ADC.

6/28/2024: CT-P42 and FYB203 (aflibercept biosimilars to Regeneron’s Eylea®)

Both Celltrion and Formycon are seeking FDA approval for their aflibercept biosimilar candidates to Regeneron’s Eylea, a vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of patients with neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy (DR) and retinopathy of prematurity (ROP). If approved, CT-P42 and FYB203 would be the first biosimilars approved in the U.S. for Eylea and may be able to launch in 2024.

6/29/2024: marnetegragene autotemcel (Kresladi™)

Marnetegragene autotemcel, by Rocket Pharmaceuticals, is a gene therapy submitted to the FDA for the treatment of severe leukocyte adhesion deficiency type 1 (LAD-1). Marnetegragene autotemcel is a one-time treatment administered via IV infusion that uses a lentiviral vector (LVV) to encode a functional copy of the integrin subunit beta 2 (ITGB2) gene into a patient’s own hematopoietic stem cells. In a global phase 1/2 study, marnetegragene autotemcel demonstrated a 100% survival rate at 12 months after infusion for all patients with 12 to 24 months of available follow-up. A significant reduction in all-cause hospitalizations and severe infections was also demonstrated. Marnetegragene autotemcel was given Fast Track, Orphan Drug, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations. Its Priority Review was extended by the FDA for three months to allow time to review the Chemistry, Manufacturing, and Controls (CMC) information. If approved, marnetegragene autotemcel will be the first agent indicated for patients with LAD-1. It has the potentially to be a curative option, particularly for those without a human leukocyte antigen (HLA)-matched sibling donor. Notably, Stelara® (ustekinumab) is also being evaluated for LAD-1 in a phase 1/2 trial.

For more information, read the marnetegragene autotemcel Deep Dive in our January 2024 Quarterly Pipeline.

6/29/2024: polyspecific immunoglobulin preparation (Yimmugo®; BT959)

Biotest is seeking FDA approval of Yimmugo, a ready-to-use polyvalent immunoglobulin G preparation from human blood plasma for IV administration (IVIG), for the treatment of primary immunodeficiencies (PID). In an open-label, phase 3 trial, the rate of acute serious bacterial infections (SBIs) (primary endpoint) was 0.015 per patient-year among 67 patients treated with Yimmugo every 3 or 4 weeks.8 If approved, Yimmugo will be one of several IVIG products available in the U.S. to treat PID.

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