October 2021 decisions expected from the FDA

Your snapshot of new drugs expecting FDA decisions in October 2021

September 12, 2021

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).

10/1/2021: somatrogon

The FDA is reviewing Pfizer and Opko Pharmaceuticals’ somatrogon as a once-weekly treatment for pediatric patients with growth hormone deficiency (GHD). Somatrogon’s application is based on a Phase 3 trial evaluating the safety and efficacy of somatrogon administered once weekly which met its primary endpoint of non-inferiority compared to once daily Genotropin® (somatropin) for injection. The primary endpoint of the trial was height velocity at 12 months. Somatrogon demonstrated the least square mean was higher in the somatrogon group (10.12 cm/year) than in the Genotropin group (9.78 cm/year); the treatment difference between somatrogon and Genotropin groups in height velocity (cm/year) was 0.33.1 Similar products include daily Novo Nordisk’s Norditropin® (somatropin) and once daily Pfizer’s Genotropin® (somatropin).

10/7/2021: VynpentaTM (avacopan)

The FDA is reviewing Chemocentryx’s Vynpenta for treatment of patients with ANCA-associated vasculitis. Vynpenta is an orally administered selective complement 5a receptor inhibitor. Vynpenta is seeking approval based on the Phase 3 ADVOCATE trial that compared Vynpenta to generic prednisone. The primary endpoint demonstrated non-inferiority at week 26; 72% of patients receiving Vynpenta were in remission, compared to 70% of prednisone patients. At week 52, 66% of Vynpenta patients were in remission compared to 55% of prednisone patients which was superior.2 In May, the FDA Advisory Committee evaluated avacopan and voted a 9–9 split on whether the efficacy data supported approval of Vynpenta and 10–8 that the therapy’s safety profile adequately supported approval. In a third question, the committee voted 10–8 in favor of the benefit-risk profile supporting the approval at the proposed dose of 30mg twice daily. According to a briefing document released by the agency, “complexities of the study design… raise questions about the interpretability of the data to define a clinically meaningful benefit of Vynpenta and its role in the management of autoantibody-associated vasculitis.”2

10/8/2021: RVT-802 (allogeneic processed thymus tissue-agdc)

The FDA is evaluating Enzyvant’s RVT-802 (allogeneic processed thymus tissue-agdc) to treat pediatric patients with congenital athymia, which is associated with complete DiGeorge anomaly (cDGA), CHARGE syndrome and FOXN1 deficiency. This is an ultra-rare condition in which children are born without a thymus. Congenital athymia can lead to profound immunodeficiency, immune dysregulation, and high susceptibility to infections. RVT-802 is a one-time regenerative tissue-based therapy derived from cultured human thymus tissue, obtained during cardiac surgery, implanted in a single surgery for immune reconstitution.3 Similar products include bone marrow transplant or thymus tissue transplant.

10/10/2021: tisotumab vedotin

The FDA is reviewing Seagen’s tisotumab vedotin for treatment of recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. Tisotumab vedotin is antibody-drug conjugate composed of Genmab’s tissue factor (TF)-directed monoclonal antibody linked to the microtubule disrupting drug monomethyl auristatin E (MMAE). The application is based on data from the Phase 2 innovaTV 204 trial, which demonstrated patients treated with tisotumab vedotin achieved an objective response rate (ORR) of 24% in patients with recurrent or metastatic cervical cancer who had previously received double chemotherapy and bevacizumab (Avastin). Tisotumab vedotin had a complete response in 7% and a median duration response of 8.3 months with 62% of responders having an ongoing response at greater than six months. Median time to response was 1.4 months.4 Similar products include Merck’s Keytruda® (pembrolizumab).

10/15/2021: FT218 (sodium oxybate, controlled-release)

The FDA is reviewing Avadel Pharmaceuticals’ FT218 (sodium oxybate, controlled-release) as a once-nightly formulation of the CNS depressant using Avadel’s Micropump microparticulate technology for treatment of excessive daytime sleepiness and cataplexy in adults with narcolepsy. FT218 is applying for approval via the 505(b)2 pathway using Jazz Pharmaceuticals’ Xyrem® as its reference product.5 Jazz Pharmaceuticals is seeking a preliminary injunction to block the launch of FT218. Similar products include Jazz Pharmaceuticals’ Xyrem® (sodium oxybate) and Jazz Pharmaceuticals’ Xywav® (calcium, magnesium, potassium, and sodium oxybates).

10/15/2021: bimekizumab

UCB Pharma’s bimekizumab is being reviewed by the FDA for the treatment of psoriasis. Bimekizumab is a humanized monoclonal immunoglobulin (Ig)G1 antibody that selectively inhibits both interleukin (IL)-17A and IL-17F, both of which are considered to be key cytokines driving inflammatory processes. Bimekizumab is supported by data from three Phase 3 studies, BE READY, BE SURE, BE VIVID. Bimekizumab demonstrated positive responses by meeting all primary endpoints across the three trials. Bimekizumab-treated patients demonstrated superior skin clearance at week 16 when compared to patients treated with AbbVie’s Humira® (adalimumab) as well as placebo, as measured by the Psoriasis Severity Index (PASI 90) and Investigator Global Assessment (IGA) response of clear (0) or almost clear (1). Eighty-five percent of bimekizumab patients had a 90% or greater reduction in the area and severity of their psoriasis symptoms at 16 weeks. Complete skin clearance, indicated by a score of PASI 100, happened in 59% of patients. Similar products include Novartis’ Cosentyx® (secukinumab) and Lilly’s Taltz® (ixekizumab).

10/17/2021: narsoplimab

Omeros’ narsoplimab has been granted priority review by the FDA for treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). If untreated TMA has a mortality rate of up to 90%. Narsoplimab is an IgG-4 monoclonal antibody that inhibits the effector enzyme MASP-2 of the lectin pathway. Narsoplimab’s application is based on a three-stage, uncontrolled, multiple dose-escalation Phase 2 study, in which it demonstrated an improvement in overall survival (OS) as compared with historical controls. Patients treated with narsoplimab achieved a complete response rate (CRR) of 61%, compared to the 15% efficacy threshold agreed with FDA. The 100-day survival was 68% in the full analysis set of patients treated with narsoplimab and the median overall survival was 274 days.7 There are currently no FDA-approved medications for this indication.

10/17/2021: OC-01 (varenicline nasal spray)

Oyster Point Pharma’s OC-01 (varenicline nasal spray) as a preservative-free aqueous nasal spray formulation of the highly selective cholinergic agonist is for treatment of signs and symptoms of dry eye disease. OC-01 is applying for approval via the 505(b)2 pathway using Pfizer’s Chantix® (varenicline) as its reference product.8 Similar products include Allergan’s Restasis® (cyclosporine).

10/23/2021: ranibizumab Port Delivery System (PDS)

The FDA is reviewing Genentech’s ranibizumab Port Delivery System (PDS) for treatment of wet, or neovascular, age-related macular degeneration (nAMD). Ranibizumab port delivery systems would allow six months between treatments. The BLA submission is based on positive results from the Phase 3 ARCHWAY study primary analysis, which demonstrated that of those nAMD patients being treated with PDS, more than 98% were able to go six months without needing additional treatment prior to the refill-exchange. In addition, these patients achieved vision outcomes equivalent to patients receiving monthly ranibizumab eye injections. In the study, PDS was generally well-tolerated, with a favorable benefit-risk profile.9 Similar products include monthly administered Genentech’s Lucentis® (ranibizumab).

10/26/2021: mobocertinib

Takeda Pharmaceuticals’ mobocertinib is being reviewed by the FDA for the treatment of adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC), as detected by an FDA-approved test, who have received prior platinum-based chemotherapy. Mobocertinib is a small-molecule tyrosine kinase inhibitor designed to selectively target EGFR and human EGFR 2 (HER2) exon 20 insertion mutations. Results from the ongoing Phase 1/2 trial of mobocertinib in previously treated patients showed mobocertinib produced an ORR of 43% in patients with locally advanced or metastatic EGFR exon 20 insertion-mutant NSCLC.10 Overall, 35% of individuals responded to treatment with an average duration of 17.5 months. There are currently no similar FDA-approved products.

10/29/2021: MydCombiTM (phenylephrine and tropicamide) Ophthalmic Solution

The FDA is reviewing Eyenovia’s MydCombi (phenylephrine and tropicamide) ophthalmic solution as a fixed combination micro-dose formulation for pharmacologic mydriasis administered in the eye care practitioner’s office. MydCombi’s application is supported by data from two Phase 3, MIST-1 and MIST-2 studies that evaluated the efficacy and safety of MydCombi. Findings from both studies demonstrated that approximately 94% of treated eyes achieved 6mm or greater dilation at 35 minutes post-instillation.11 Similar products include generic tropicamide.

10/30/2021: XipereTM (triamcinolone acetonide)

Clearside Biomedical and Bausch + Lomb’s Xipere (triamcinolone acetonide) is seeking approval from the FDA for treatment of macular edema associated with uveitis. Xipere is administered by a health care provider into the back of the eye by suprachoroidal injection. Xipere is applying for approval via the 505(b)2 pathway using triamcinolone as its reference product.12 Similar products include Alcon Pharmaceuticals’ Triesence® (triamcinolone acetonide) and triamcinolone generics.

10/31/2021: Kyzatrex™ (testosterone undecanoate)

Marius Pharmaceuticals’ Kyzatrex (testosterone undecanoate) is applying for approval from the FDA for treatment of primary and secondary hypogonadism in adult men. Kyzatrex is an oral, soft gelatin capsule formulation of a testosterone replacement therapy. Kyzatrex is applying for approval via the 505(b)2 pathway using Endo Pharms’ Aveed® (testosterone undecanoate) as its reference product. Kyzatrex’s application is being evaluated based on the multicenter Phase 3 ReTUne study, which assessed the efficacy and safety of Kyzatrex for primary and secondary hypogonadism in men patients with total testosterone less than or equal to 281ng/dL. Results showed that greater than 96% of patients who completed 90 days of treatment achieved average testosterone levels in the normal range.13 Similar products include Clarus Therapeutics’ Jatenzo® (testosterone undecanoate) and other generic testosterone products.

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.


1. https://www.pfizer.com/news/press-release/press-release-detail/ema-accepts-marketing-application-somatrogon-treat

2. https://ancavasculitisnews.com/2021/05/10/fda-advisory-committee-narrowly-supports-approval-of-avacopan/

3. https://www.biospace.com/article/releases/enzyvant-resubmits-biologics-licensing-application-bla-to-fda-for-rvt-802-for-pediatric-congenital-athymia/

4. https://ascopost.com/news/april-2021/tisotumab-vedotin-in-recurrent-or-metastatic-cervical-cancer/

5. https://www.globenewswire.com/news-release/2021/03/01/2184208/0/en/Avadel-Pharmaceuticals-Announces-FDA-Acceptance-of-New-Drug-Application-for-FT218-in-Adults-with-Narcolepsy-for-the-Treatment-of-Excessive-Daytime-Sleepiness-and-Cataplexy.html

6. https://www.prnewswire.com/news-releases/ucb-to-present-12-abstracts-on-bimekizumab-at-aad-vmx-2021-301275987.html

7. https://www.businesswire.com/news/home/20210520005392/en/Omeros-Announces-Extension-of-FDA-Review-Period-for-Narsoplimab-in-HSCT-TMA

8. https://www.ophthalmologytimes.com/view/oyster-point-pharma-receives-fda-filing-acceptance-of-nda-for-oc-01-nasal-spray-to-treat-ded

9. https://www.roche.com/media/releases/med-cor-2021-06-24.htm

10. https://www.takeda.com/newsroom/newsreleases/2021/takeda-announces-u.s.-fda-grants-priority-review-for-new-drug-application-for-mobocertinib-tak-788-as-a-treatment-for-egfr-exon20-insertion-metastatic-non-small-cell-lung-cancer/

11. https://www.empr.com/home/news/drugs-in-the-pipeline/mydcombi-phenylephrine-tropicamide-eyenovia-mydriasis/

12. https://www.marketwatch.com/press-release/bausch-health-and-clearside-biomedical-announce-us-fda-filing-acceptance-for-xiperetm-triamcinolone-acetonide-suprachoroidal-injectable-suspension-2021-06-02?siteid=bigcharts&dist=bigcharts&tesla=y

13. https://www.empr.com/home/news/drugs-in-the-pipeline/kyzatrex-testosterone-undecanoate-primary-secondary-hypogonadism-adult-males/

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