February 2022 decisions expected from the FDA

Your monthly synopsis of new drugs expecting FDA decisions in February 2022

January 13, 2022
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).

2/2022: GC5107 (immune globulin intravenous [human], 10% liquid)
The FDA is reviewing GC Pharma’s GC5107IV immune globulin (IVIG) for treatment of patients with primary humoral immunodeficiency to reduce serious bacterial  . GC5107 is seeking approval based on the Phase 3 clinical study assessing its efficacy and safety. GC5107 demonstrated an incidence of 0.02 acute serious bacterial infection (aSBI) events per patient year which met the FDA efficacy requirement of <1 aSBI per patient year.1 Similar products include Takeda’s Gammagard® (immune globulin infusion (human) 10%), Bio Products Laboratory’s Gammaplex® (immune globulin intravenous (human) 10%) and Grifols’ Gamunex-C® (immune globulin injection (human) 10%).

2/17/2022: mitapivat
Agios Pharmaceuticals’ mitapivat is being reviewed by the FDA for the treatment of adults with pyruvate kinase (PK) deficiency. Pyruvate kinase (PK) deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells. In the open-label ACTIVATE-T trial which included patients with PK deficiency who are dependent on transfusions, 10 of 27 pyruvate kinase deficiency patients receiving oral mitapivat met the primary endpoint, a 33% or better reduction in transfusion burden at 24 weeks of twice-daily treatment, compared with individual historical transfusion burden. Six of those responders were transfusion-free during that period.2 The clinical trial ACTIVATE which included patients with PK deficiency who are not dependent on blood transfusions found mitapivat met its primary endpoint by demonstrated 40% of patients in the group receiving mitapivat had a response of ≥1.5 g/dL increase in hemoglobin concentration at weeks 16, 20 and 24. Currently there are no other FDA-approved treatment.

2/23/2022: tebentafusp (IMCgp100)
The FDA is reviewing Immunocore’s tebentafusp (IMCgp100) for treatment of HLA-A*02:01-positive adults with metastatic uveal melanoma (mUM). Tebentafusp is seeking approval based on a clinical trial that tested tebentafusp compared to the control group of Keytruda®, Yervoy® or dacarbazine. Overall survival (OS) at one year was 73% in the tebentafusp group and 59% in the control group. Progression-free survival was also significantly higher in the tebentafusp group compared to the control group (31% vs. 19% at six months). At a median follow-up of 14.1 months, the median OS with tebentafusp was 21.7 months vs 16.0 months with the control group.3 Similar products include Merck’s Keytruda® (pembrolizumab), Bristol-Myers Squibb’s Yervoy® (ipilimumab) and generic dacarbazine.

2/25/2022: bardoxolone methyl
Reata Pharmaceuticals’ bardoxolone methyl is seeking approval from the FDA to slow the loss of progression of kidney disease caused by Alport syndrome in patients 12 and older. Bardoxolone methyl is seeking approval based on the clinical trial CARDINAL which demonstrated that patients treated with bardoxolone methyl experienced a statistically significant improvement in kidney function as measured by eGFR at Week 100 and Week 104, compared to patients treated with placebo.4 Reata Pharmaceuticals engaged in an advisory committee meeting with the FDA. The FDA stated that Reata Pharmaceuticals ignored the FDA’s trial design advice and found the Phase 3 study was not designed adequately to evaluate the potential of bardoxolone methyl to slow the progression of decline in kidney function. The U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 13-0 against FDA approval of Reata Pharmaceuticals’ bardoxolone methyl. If approved, FiercePharma estimates that bardoxolone methyl will quickly reach blockbuster status in 2024 with an estimated $1.12 billion in sales, further accelerating to $2.5 billion in 2026.5 Similar products include generic ACE inhibitors.

2/28/2022: lenacapavir
Gilead Science’s lenacapavir is applying for approval from the FDA for treatment of HIV-1 infection in heavily treatment-experienced (HTE) patients with multi-drug resistant (MDR) HIV-1 infection. Lenacapavir is a health care administered HIV-1 capsid inhibitor that is injected subcutaneously every six months along with other antiretrovirals. Lenacapavir is seeking approval based on clinical trial CAPELLA, a Phase 2/3, placebo-controlled study. The study achieved its primary endpoint by demonstrating that a significantly higher proportion of participants randomly allocated to receive lenacapavir in addition to other antiretrovirals achieved a clinically meaningful viral load reduction of at least 0.5 log10 copies/mL from baseline compared with those receiving placebo plus antiretrovirals during the 14-day functional monotherapy period (88% compared to 17%).6 Similar products include ViiV’s Cabenuva® (cabotegravir; rilpivirine).

 2/28/2022: ciltacabtagene autoleucel (cilta-cel)
The FDA is reviewing Janssen’s ciltacabtagene autoleucel (cilta-cel) for treatment of relapsed and refractory multiple myeloma. Ciltacabtagene autoleucel is a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T-cell (CAR-T) therapy. The Phase 1b/2 CARTITUDE-1 trial demonstrated ciltacabtagene autoleucel had an overall response rate (ORR) of 97.9% with an 80.4% rate of stringent complete response (sCR).7 At 18 months, 66% of patients were alive and progression free with an sCR of 75.9%. Similar products include Bristol Myers Squibb’s Abecma® (idecabtagene vicleucel).

2/28/2022: Enpaxiq® (pacritinib)
CTI BioPharma’s Enpaxiq is seeking priority review from the FDA for treatment for myelofibrosis patients with severe thrombocytopenia (platelet counts less than 50 x 109/L). Enpaxiq is an oral kinase inhibitor with specificity for JAK2, IRAK1, and CSF1R. The FDA decision will be made based on results from the Phase 3, PERSIST-2 and PERSIST-1 trials. PERSIST-2 demonstrated that Enpaxiq at 200mg twice daily was significantly more effective in treating patients with myelofibrosis and thrombocytopenia than the existing best available therapies. The results suggest that Enpaxiq may provide a treatment option for patients whose options are limited.8 Specifically, a reduction in spleen volume of at least 35% was observed in 29% of patients treated with Enpaxiq compared with only 3% of patients treated with the best available therapy. Similar products include Incyte’s Jakafi® (ruxolitinib).

2/28/2022: Filsuvez® (oleogel-S10 formerly AP101)
Amryt Pharma’s Filsuvez is seeking approval from the FDA for treatment of cutaneous manifestations of junctional and dystrophic epidermolysis bullosa (EB). Filsuvez is a topical gel that can be applied at home. Filsuvez’s application was based on the Phase 3, EASE trial that met its primary endpoint by demonstrating statistically significant increase in speed of wound healing in EB with a complete closure of the target wound within 45 days compared to placebo.9 Several secondary measures were missed including 90-day target wound closure, and the pain endpoint. Current treatments aim to help alleviate symptoms, including bandaging and bathing open wounds and trying to manage patients’ pain, but there are no FDA approved products for this indication.

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.businesswire.com/news/home/20210811005915/en/GC-Pharma-Announces-Publication-of-Results-From-Phase-III-Clinical-Trial-of-%E2%80%98GC5107%E2%80%99-Immune-Globulin-in-Frontiers-in-Immunology
  2. https://www.biospace.com/article/more-positive-phase-iii-data-sets-up-mitapivat-for-regulatory-filings-this-year/
  3. https://www.cancernetwork.com/view/tebentafusp-improves-os-in-frontline-setting-of-metastatic-uveal-melanoma
  4. https://www.reatapharma.com/investors/news/news-details/2021/Reata-Announces-FDA-Accepted-for-Filing-the-NDA-for-Bardoxolone-for-the-Treatment-of-Patients-With-Chronic-Kidney-Disease-Caused-by-Alport-Syndrome/default.aspx
  5. https://www.fiercepharma.com/special-report/bardoxolone-methyl-10-most-anticipated-drug-launches-2021
  6. https://www.gilead.com/news-and-press/press-room/press-releases/2021/6/gilead-submits-new-drug-application-to-us-food-and-drug-administration-for-lenacapavir-an-investigational-longacting-capsid-inhibitor-for-the-tre
  7. https://www.businesswire.com/news/home/20210201005446/en/Janssen-Announces-CAR-T-Therapy-Ciltacabtagene-Autoleucel-Cilta-cel-Accepted-for-Accelerated-Assessment-in-Europe-for-the-Treatment-of-Patients-with-Heavily-Pretreated-Multiple-Myeloma
  8. https://www.cancernetwork.com/view/fda-grants-priority-review-to-pacritinib-for-treating-myelofibrosis-with-severe-thrombocytopenia
  9. https://www.globenewswire.com/en/news-release/2020/10/29/2116547/0/en/AMRYT-ANNOUNCES-POSITIVE-RESULTS-FROM-PHASE-3-TRIAL-OF-FILSUVEZ-IN-EPIDERMOLYSIS-BULLOSA.html

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