February 2021 decisions expected from the FDA

Your snapshot of new drugs expecting FDA decisions in February 2021

January 15, 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).

2/2/2021: StrataGraft®

The FDA is reviewing Mallinckrodt’s StrataGraft for the treatment of adult patients with deep partial-thickness thermal burns. Approximately 40,000 patients in US require hospitalization for the treatment of severe burns. The StrataGraft skin tissue biologics license application (BLA) is based on data from the pivotal Phase 3, STRATA2016, clinical trial which met both primary endpoints including autograft sparing and durable wound closure. The trial demonstrated that treatment with StrataGraft required a smaller amount of burn wounds to be treated with autografting after three months, compared to burn wounds treated exclusively with autograft.1 Autograft, the current standard of care, involves the surgical harvesting of healthy skin from an uninjured site on the patient, and transplanting the skin graft to the injury. This leaves patients with more wounded areas requiring care.

2/11/2021: evinacumab

The FDA is reviewing Regeneron’s evinacumab as an adjunct to other lipid-lowering therapies for treatment of homozygous familial hypercholesterolemia (HoFH). HoFH is an ultra-rare inherited disease that affects approximately 1,300 patients in the United States. If approved, evinacumab will be the first medication to show efficacy in patients with HoFH, including patients with little to no low-density lipoprotein (LDL) receptor function, by binding to and blocking the function of angiopoietin-like 3 (ANGPTL3). A Phase 3 trial demonstrated that evinacumab had a 49% reduction in LDL cholesterol from baseline, when compared to placebo (47% reduction for evinacumab compared to a 2% increase for placebo).2 Evinacumab 15mg/kg is administered intravenously every four weeks by a health care professional. Similar products include Aegerion’s Juxtapid® (lomitapide) capsules, lipoprotein apheresis, or a liver transplant.

2/15/2021: trilaciclib

Trilaciclib is being reviewed by the FDA as a CDK4/6 cell cycle inhibitor to be administered prior to chemotherapy for myelopreservation in patients with small cell lung cancer (SCLC). Myelopreservation refers to preserving bone marrow function, making chemotherapy safer and more tolerable for patients. Trilaciclib is being reviewed, based on three placebo-controlled clinical trials where trilaciclib was administered prior to chemotherapy treatment in patients with SCLC. Clinical trials demonstrated trilaciclib significantly reduced chemotherapy-induced myelosuppression. Additionally, patients receiving trilaciclib experienced fewer dose delays/reductions, infections, hospitalizations, and need for rescue therapies, compared to patients receiving chemotherapy alone.3 The FDA is not planning on holding an advisory committee meeting to discuss the application. Currently there are no FDA approved medical therapies to protect patients from chemotherapy-induced toxicities before they occur.

2/15/2021: umbralisib

Umbralisib is being reviewed by the FDA for patients with previously treated marginal zone lymphoma (MZL) and follicular lymphoma (FL), both indolent forms of non-Hodgkin lymphoma. Umbralisib is a once daily, oral, dual inhibitor of PI3K-delta and CK1-epsilon. The FL indication has been accepted for standard review with a Prescription Drug User Fee Act goal date of June 15, 2021. Umbralisib is seeking approval based on Phase 2b clinical trial, UNITY-NHL, that demonstrated umbralisib met its primary endpoint of overall response rate (ORR) of 40-50%.4 Similar products include Genentech’s Rituxan® (rituximab), Genentech’s Gazyva® (obinutuzumab), and Bristol Myers Squibb’s Revlimid® (lenalidomide).

2/25/2021: Amondys 45® (casimersen)

Sarepta Therapeutics’ Amondys 45 is being reviewed by the FDA for treatment of patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene. Amondys 45 is a phosphorodiamidate morpholino oligomer (PMO) that binds to exon 45 of dystrophin pre-mRNA. Amondys 45 is intended to skip over exon 45 of the DMD gene, the largest known human gene with 79 exons in total. Amondys 45 is being evaluated based on a Phase 3 clinical trial, ESSENCE, that demonstrated boys given once-weekly intravenous infusions of Amondys 45 at a dose of 30mg/kg experienced a significant increase in dystrophin levels in muscle biopsies, compared both with measures taken at the study’s start and the placebo group.5 The Institute for Clinical and Economic Review (ICER) states that, “The clinical efficacy of exon-skipping therapies (including Amondys 45) is still unclear. There is limited or no evidence demonstrating improvements in functions, as comparison with historical controls with conditions such as DMD.”6 Similar products include Sarepta Therapeutics’ Vyondys 53® (golodirsen), Sarepta Therapeutics’ Exondys 51® (eteplirsen) and Nippon Shinyaku’s Viltepso® (viltolarsen).

2/25/2021: tepotinib

EMD Serono’s tepotinib is being reviewed for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition exon 14 (METex14) skipping, as detected by an FDA-approved test. Tepotinib is a once-daily, orally dosed MET inhibitor. Alterations of the MET signaling pathway are found in approximately 3 to 5% of NSCLC cases. Patient population is generally characterized as elderly with a poor clinical prognosis. Tepotinib is seeking approval based on ongoing Phase 2 clinical trial, VISION, which measured objective response as its primary outcome. Results demonstrate consistent response rate and durable anti-tumor activity across lines of treatment including in patients with brain metastases and in patients assessed by both liquid biopsy (LBx) and tissue biopsy (TBx).7 Novartis’ Tabrecta® (capmatinib) is a similar product.

2/28/2021: paclitaxel and encequidar

Athenex and Hanmi’s paclitaxel is being reviewed by the FDA as an oral treatment for metastatic breast cancer. It is a combination product of paclitaxel, a taxane chemotherapy, and encequidar, a novel highly selective P-glycoprotein pump inhibitor. Encequidar is a potent minimally absorbed p-glycoprotein inhibitor that a patient takes first in order to take oral paclitaxel, which is known to have poor oral bioavailability. Phase 3 clinical trial, KX-ORAX-001, demonstrated that the combination of oral paclitaxel and encequidar led to a 26.5% reduction in the risk of death compared with intravenous (IV) paclitaxel in patients with metastatic breast cancer. The combination had a 35.8% overall response rate (ORR) compared to a 23.4% ORR in IV paclitaxel patients. Additionally, the median overall survival with the combination was 23.3 months compared to 16.3 months with IV paclitaxel.8

2/28/2021: melflufen

Oncopeptides’ melflufen is seeking approval from the FDA in combination with dexamethasone for treatment of adults with relapsed refractory multiple myeloma (RRMM) whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one anti-CD38 monoclonal antibody. Melflufen is seeking approval based on the Phase 2, HORIZON trial, which demonstrated intravenous (IV) melflufen in combination with dexamethasone had an ORR of 29%. Melflufen is currently enrolling patients in a Phase 3 clinical trial that is evaluating the efficacy of melflufen plus dexamethasone in comparison to pomalidomide and dexamethasone. Similar products include Takeda’s Velcade® (bortezomib) and GlaxoSmithKline’s Alkeran® (melphalan).9

2/28/2021: Defencath® (taurolidine, heparin and citrate)

The FDA is reviewing CorMedix Inc.’s Defencath as prevention of catheter-related bloodstream infections (CRBSIs) in patients with end-stage renal disease who are receiving hemodialysis via a central venous catheter. Taurolidine is a non-antibiotic agent with broad-spectrum antimicrobial activity, which has been used as a lock solution to prevent catheter-related bloodstream infections in some settings. The primary objective of the Phase 3 trial, LOCK-IT-100, was to demonstrate the efficacy and safety of Defencath as a catheter lock solution (CLS) for prevention of catheter-related bloodstream infection (CRBSI) in subjects receiving hemodialysis (HD) for the treatment of end stage renal disease (ESRD) when compared with heparin. Defencath demonstrated a 71% reduction in catheter-related bloodstream infections relative to the heparin control arm.10 Currently, there are no FDA-approved pharmacological agents for prevention of CRBSIs, however heparin sodium is used off-label.

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.prnewswire.com/news-releases/mallinckrodt-announces-us-fda-filing-acceptance-of-biologics-license-application-for-stratagraft-regenerative-skin-tissue-for-treatment-of-adults-with-deep-partial-thickness-thermal-burns-301108836.html

2. https://investor.regeneron.com/news-releases/news-release-details/regeneron-announces-positive-topline-results-phase-3-trial

3. https://www.cancernetwork.com/view/fda-grants-priority-review-to-trilaciclib-to-treat-patients-with-sclc

4. https://www.biospace.com/article/releases/tg-therapeutics-announces-fda-acceptance-of-new-drug-application-for-umbralisib-as-a-treatment-for-patients-with-previously-treated-marginal-zone-lymphoma-and-follicular-lymphoma/

5. https://musculardystrophynews.com/2020/08/27/casimersen-exon-45-skipping-duchenne-therapy-under-fda-priority-review/

6. https://icer-review.org/assessment/duchenne-muscular-dystrophy-2019/

7. https://www.prnewswire.com/news-releases/fda-accepts-filing-of-new-drug-application-for-tepotinib-for-the-treatment-of-patients-with-metastatic-nsclc-with-metex14-skipping-alterations-301117906.html

8. https://www.ajmc.com/view/oral-paclitaxel-and-encequidar-vs-iv-paclitaxel-in-metastatic-breast-cancer

9. https://www.ashclinicalnews.org/news/latest-and-greatest/fda-grants-priority-review-melflufen-myeloma/


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