September 2023 decisions expected from the FDA

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 U.S. Food and Drug Administration (FDA).

Drug pipeline for September 2023

9/2023: lebrikizumab
The FDA is reviewing Lilly’s lebrikizumab, a monoclonal antibody that binds interleukin-13 (IL-13), for treatment of atopic dermatitis. Lebrikizumab is seeking approval based on two Phase 3 trials, ADvocate 1 and 2, which met the co-primary endpoints of achieving an Investigator Global Assessment (IGA) score of clear (0) or almost clear (1) skin with a reduction of a least 2 points from baseline and at least 75% change in baseline in the Eczema Area and Severity Index (EASI-75) score at Week 16. Forty-three percent and 33% of patients receiving lebrikizumab achieved IGA 0 or 1 at 16 weeks compared to 13% and 11% of patients taking placebo in ADvocate 1 and ADvocate 2, respectively. Fifty-nine percent and 51% of patients receiving lebrikizumab achieved an EASI-75 response at 16 weeks compared to 16% and 18% of patients taking placebo in ADvocate 1 and ADvocate 2, respectively.¹ Similar products include Regeneron’s Dupixent^® (dupilumab).

9/9/2023: Aphexda^® (motixafortide) + G-CSF
The FDA is reviewing BioLineRx’s Aphexda (motixafortide) in combination with a granulocyte-colony stimulating factor (G-CSF) for stem cell mobilization for autologous bone marrow transplantation (ASCT) for multiple myeloma patients. Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for multiple myeloma. In the U.S., nearly 15,000 ASCTs are performed each year with the majority in patients with multiple myeloma. Aphexda (motixafortide) + G-CSF is seeking approval based on the GENESIS Phase 3 trial which evaluated motixafortide in combination with of G-CSF verses G-CSF alone in stem cell mobilization for autologous transplantation in multiple myeloma patients and demonstrated that more than 90% of patients collected an optimal number of cells for transplantation following a single administration of Aphexda compared to about 26% with G-CSF alone.² Similar products include G-CSF alone.

9/14/2023: zilucoplan
UCB’s zilucoplan is seeking approval from the FDA as a once daily self-administered subcutaneous injection for treatment of generalized myasthenia gravis (gMG) in adults who are acetylcholine receptor antibody positive (AChR+). The primary endpoint was met at Week 12 which demonstrated a placebo-corrected mean improvement of 2.12 points in the Myasthenia Gravis Activities of Daily Living (MG-ADL).³ Similar products include Argenx’s Vyvgart^® (efgartigimod alfa-fcab), Alexion’s Ultomiris^® (ravulizumab-cwvz) and Alexion’s Soliris^® (eculizumab).

9/16/2023: momelotinib
Sierra Oncology’s momelotinib is seeking approval from the FDA for the treatment of myelofibrosis. Momelotinib is a small-molecule inhibitor of the Janus kinases 1 and 2 (JAK1/2) and activin A receptor type-I (ACVR1). The Phase 3 MOMENTUM trial evaluated momelotinib compared with danazol in patients with symptomatic and anemic myelofibrosis who were previously treated with a JAK inhibitor (JAKi). The primary endpoint of Total Symptom Score (TSS) of >50% was higher in the momelotinib group (25%) versus the danazol group (9%). The SIMPLIFY-1 trial found momelotinib non-inferior in reduction in spleen volume at week 24 by at least 35% compared to ruxolitinib in JAKi-naïve patients with myelofibrosis. However, in a Phase 3 open-label trial in patients with myelofibrosis who had suboptimal responses or hematological toxic effects with ruxolitinib, momelotinib was not superior to best available therapy (89% ruxolitinib) for the reduction of spleen size by at least 35% compared with baseline.^4,5 Similar products include danazol, Incyte’s Jakafi^® (ruxolitinib), Bristol-Myers Squibb’s Inrebic^® (fedratinib), and CTI BioPharma Corp’s Vonjo^® (pacritinib).

9/23/2023: ATI-1501 (metronidazole oral suspension)
Saptalis Pharmaceuticals’ ATI-1501 (metronidazole oral suspension) taste-masked liquid oral suspension formulation for the treatment of anaerobic bacterial and parasitic infections.⁶ ATI-1501 is seeking approval via the 505(b)(2) pathway using Pfizer’s Flagyl^® (metronidazole) as its reference product.

9/26/2023: sofpironium bromide
Botanix Pharmaceuticals/Brickell Biotech’s sofpironium bromide is seeking approval from the FDA for treatment of severe primary axillary hyperhidrosis. Sofpironium bromide is seeking approval based on the Phase 3 Cardigan I and Cardigan II clinical trials evaluated sofpironium bromide applied topically to the underarms once daily at bedtime for six weeks compared to placebo. More patients treated with sofpironium bromide achieved at least a 2-point improvement in the Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) score and had a greater change in the measured weight of gravimetric sweat production from baseline to end of treatment compared to placebo.⁷ Similar products include Seaford Pharmaceuticals’ Drysol^® (aluminum chloride hexahydrate) and Journey Medical Corporation’s Qbrexza^® (glycopyrronium).

9/28/2023: Nyxol^® (phentolamine ophthalmic solution 0.75%)
The FDA is reviewing Ocuphire/Viatris’ Nyxol for reversal of pharmacologically-induced mydriasis (RM) produced by adrenergic agonist or parasympatholytic agents, or a combination thereof.⁸ Nyxol is seeking approval via the 505(b)(2) pathway using Novartis’s discontinued Regitine^® (phentolamine) as its reference product.

Contact your Prime representative for more information or with any questions you have about drugs in the pipeline.

While the information in this newsletter is from sources we believe to be reliable, we do not warrant that the information in this document is free from error. Use it only as a guide. Statements regarding drugs or manufacturers are not intended as promotion; those statements should not be used to make assumptions about formulary status. Each trademarked drug name is the property of its respective owner.

References

1. https://pubmed.ncbi.nlm.nih.gov/36920778/
2. https://www.prnewswire.com/news-releases/biolinerx-announces-us-fda-acceptance-of-new-drug-application-for-aphexda-motixafortide-in-stem-cell-mobilization-301674304.html
3. https://www.neurologylive.com/view/zilucoplan-demonstrates-consistent-efficacy-phase-3-raise-study-generalized-myasthenia-gravis

6. https://www.mdanderson.org/newsroom/targeted-therapy-momelotinib-provides-significant-symptom-and-anemia.h00-159615489.html
7. https://pubmed.ncbi.nlm.nih.gov/29275119/
8. https://appilitherapeutics.com/ati-1501/
9. https://botanixpharma.com/sofpironium-bromide/
10. https://www.ocuphire.com/product-pipeline/nyxol#:~:text=Nyxol%C2%AE%20is%20a%20preservative,smooth%20muscle%20of%20the%20iris