June 2021 decisions expected from the FDA

Your snapshot of new drugs expecting FDA decisions in June 2021

May 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).

6/1/2021: relugolix, estradiol and norethindrone

The FDA is reviewing Myovant’s relugolix, estradiol and norethindrone for women with heavy menstrual bleeding associated with uterine fibroids. Relugolix is a gonadotropin-releasing hormone-receptor antagonist that can be used daily without hypoestrogenic effects. In two clinical trials, relugolix, estradiol and norethindrone significantly reduced menstrual bleeding in women with uterine fibroids when compared to placebo. A total of 73% (trial 1) and 71% (trial 2) of women in the relugolix combination therapy groups met the study primary endpoint of ?50% reduction in blood loss volume from baseline, compared to 19% (trial 1) and 15% (trial 2), respectively, in the placebo groups.1 In a continuation Phase 3 study, 78.4% of women who continued on relugolix combination therapy remained responders through Week 76 compared with 15.1% of women who discontinued treatment at Week 52. Additionally, 88.3% of women who discontinued treatment relapsed with heavy menstrual bleeding approximately 5.9 weeks after stopping the relugolix combination.2 The combination therapy also preserved bone mineral density in women and provided relief from other uterine fibroid symptoms. Relugolix was approved by the FDA in December 2020 for the treatment of advanced prostate cancer. Similar products include oral contraceptives which are first line treatment for uterine fibroid symptoms and Abbvie’s Oriahnn® (elagolix/ estradiol/ norethindrone acetate and elagolix).

6/1/2021: Lybalvi® (olanzapine and samidorphan)

Alkermes’ Lybalvi is being reviewed by the FDA as olanzapine, an antipsychotic, co-formulated with samidorphan, a novel opioid antagonist for treatment of schizophrenia, for treatment of manic or mixed episodes associated with bipolar I disorder as a monotherapy or as adjunct to lithium or valproate, and for maintenance treatment of bipolar I disorder. Samidorphan reduces the weight gain associated with olanzapine. The ENLIGHTEN-2 study compared Lybalvi-treated patients to patients taking olanzapine alone and found that olanzapine alone had a 57% higher weight gain from baseline compared to patients taking Lybalvi. Olanzapine had twice the risk of gaining 10% or more of their baseline body weight compared to patients on Lybalvi.3 Similar products include Lilly’s Zyprexa® (olanzapine) and its generics.

6/1/2021: infigratinib

The FDA has granted priority review of BridgeBio’s infigratinib for the treatment of cholangiocarcinoma, or cancer of the bile ducts. Infigratinib is an oral FGFR1-3 selective inhibitor. FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and there is only a 9% survival rate at five years. Infigratinib is seeking approval based on a clinical trial that demonstrated patients treated with infigratinib as first-line treatment had a 50% objective response rate (ORR) and a median overall survival of 22 months.4 Additionally, in a retrospective analysis, infigratinib demonstrated that third- and later-line infigratinib therapy led to a superior improvement in median progression free survival (PFS) compared with pre-infigratinib, second-line chemotherapy at 6.77 months versus 4.63 months.5 Similar products include generic gemcitabine/cisplatin.

6/1/2021: Brexafemme® (ibrexafungerp)

Scynexis’ Brexafemme is being reviewed by the FDA as a triterpenoid antifungal for treatment of vulvovaginal candidiasis (VVC). Brexafemme is a once-daily oral medication for drug-resistant infections. Brexafemme’s application for approval is supported by positive results from two Phase 3 trials, VANISH-303 and VANISH-306, in which Brexafemme demonstrated statistically superior efficacy compared to placebo in women with VVC. VANISH-306 demonstrated that 63.3% of treated patients achieved cure at the primary 10-day test-of-cure endpoint compared to placebo.6 If approved, Scynexis is seeking to launch in the second half of 2021. Similar products include fluconazole.

6/5/2021: Ryplazim® (plasminogen human plasma-derived purified)

The FDA is reviewing Prometic Life Sciences’ Ryplazim for treatment of congenital plasminogen deficiency. Patients with congenital plasminogen deficiency experience an accumulation of fibrin growths or lesions on mucosal surfaces throughout the body. Many cases are first diagnosed in the pediatric population, and if left untreated, disease manifestations may be organ-compromising. Ryplazim’s BLA resubmission was based on a Phase 2/3, open-label study evaluating the safety and efficacy of Ryplazim in patients, which demonstrated 100% of the patients enrolled met both primary efficacy endpoints of the clinical study.7 Primary endpoint was a surrogate target of increase in blood plasma concentration level of plasminogen. Additionally, all patients who had active visible lesions had complete healing of lesions, a 100% response rate, within one week. There are currently no approved therapies for the treatment of congenital plasminogen deficiency.

6/7/2021: aducanumab

Biogen Pharmaceuticals’ aducanumab is being reviewed by the FDA to reduce the clinical decline of Alzheimer’s disease (AD). Aducanumab is seeking to become the first treatment to change the course of AD, rather than just treating the symptoms; however clinical trials have not demonstrated this. In November 2020, The Peripheral and Central Nervous System Drugs Advisory Committee overwhelmingly voted no on several counts regarding the efficacy and evidence supporting the use of aducanumab in AD. Based on clinical trials, EMERGE and ENGAGE, eight experts voted no; aducanumab did not provide “strong evidence” in treating patients with Alzheimer’s, one expert voted yes, and two voted uncertain.8

The Institute for Clinical and Economic Review (ICER) assessed the comparative clinical effectiveness and value of aducanumab for AD. In the midpoint assessment of evidence on aducanumab, ICER found that there is insufficient evidence to determine whether aducanumab will provide health benefits to people with AD. ICER estimates that based on the findings, aducanumab should cost as low as $2,500 for a year of treatment to be considered cost-effective. Even at the high end of the cost-effective threshold, ICER’s designated price of $8,300 would still come way below what a typical antibody drug would cost on the U.S. market. It’s far less than the $50,000 market industry watchers have estimated aducanumab could cost.9 There are currently no disease-modifying drugs available for this indication.

6/17/2021: Miplyffa® (arimoclomol)

Orphazyme’s Miplyffa is being reviewed by the FDA for patients with Niemann-Pick type C patients (NPC). NPC is a rare, genetic disease that is progressively debilitating and often fatal. It is part of a family of lysosomal storage diseases caused by a defective NPC protein which means lipids are not able to be cleared out by lysosomes and therefore accumulate in tissues and organs, including the brain. NPC is found in approximately one in 100,000 live births, affecting about 1,800 people in the United States and Europe.10 In a Phase 2/3 clinical trial, Miplyffa failed to hit both its primary and secondary endpoints for the treatment of inclusion body myositis (IBM). Orphazyme expects data from a Phase 3 trial of Miplyffa in Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease, this spring. There are currently no FDA-approved medications for this indication, although Zavesca® (miglustat) is used off label.

6/2021: ruxolitinib, topical

The FDA is reviewing Incyte Corporation’s ruxolitinib topical for treatment of mild-to-moderate atopic dermatitis (AD). Ruxolitinib topical is a twice-daily cream formulation of the selective JAK1/JAK2 inhibitor. Ruxolitinib is approved by the FDA as oral Jakafi® (ruxolitinib) for other indications. Ruxolitinib topical met its primary endpoint in two clinical studies, TRuE-AD1 and TRuE-AD2, which demonstrated a statistically significant proportion of patients achieved Investigator’s Global Assessment Treatment Success (IGA-TS), which is defined as a score of 0 (clear) or 1 (almost clear), as well as demonstrating at least a 2-point improvement from baseline at Week 8.11 The first trial, TRuE-AD1, showed 50% of the ruxolitinib cream 0.75% group and 53.8% of the ruxolitinib cream 1.5% group achieved the primary endpoint compared to 15.1% of patients treated with vehicle. The second trial, TRuE-AD2, showed 39% of patients treated with ruxolitinib cream 0.75% and 51.3% treated with 1.5% met the primary endpoint compared to 7.6% of the vehicle group. Similar products include Jakafi® (ruxolitinib) and Pfizer’s Eurisa® (crisaborole).

6/23/2021: Ycanth® (cantharidin 0.7% topical solution)

Verrica Pharmaceuticals’ Ycanth is being reviewed by the FDA as a drug-device combination for topical treatment of molluscum contagiosum. Molluscum contagiosum is a highly contagious viral skin infection that affects close to 6 million people in the United States each year. Cantharidin is currently available as a compounded agent. Verrica Pharmaceuticals is seeking FDA approval based on the Phase 3 CAMP trial that demonstrated Ycanth to be statistically significant when compared to placebo. Efficacy was measured by the percentage of subjects with complete clearance of lesions in each location (head, chest, back, groin, upper and lower extremities) by day 84 compared to vehicle.12 When used on patient’s head/neck, 81.8% of Ycanth patients experienced complete clearance compared to 39.6% with vehicle. Overall, 50% of patients using Ycanth experienced complete clearance compared with 15.6% response with use of the vehicle. Verrica Pharmaceuticals intends to conduct additional clinical trials later this year to evaluate Ycanth for treatment of common warts and external genital warts. Similar products include cryotherapy, cantharidin compounded solution, and Allergan Pharmaceuticals’ Condylox® (podofilox).

6/25/2021: TransCon hGH® (lonapegsomatropin)

The FDA is reviewing Ascendis Pharma’s TransCon hGH for treatment of pediatric growth hormone deficiency (GHD). TransCon hGH is a long-acting once weekly injection prodrug of somatropin. TransCon hGH’s application was based on a Phase 3 trial in treatment-naïve children with GHD which demonstrated once-weekly treatment with TransCon hGH was superior to Pfizer’s once-daily Genotropin® (hGH) in annualized height velocity. TransCon hGH had a height growth of 11.2 cm/year compared with 10.3 cm/year with daily hGH, treatment difference: 0.86 cm/year.13 The FDA is not planning on holding an advisory committee meeting. Ascendis Pharma is also pursuing an autoinjector for TransCon hGH which would deliver a single, low-volume injection. Similar products include other daily subcutaneous injectable growth hormones.

6/26/2021: cyclosporine ophthalmic emulsion

Santen’s cyclosporine ophthalmic emulsion is being reviewed by the FDA as a topical ophthalmic emulsion 0.1% for treatment of severe vernal keratoconjunctivitis (VKC) in pediatric patients ages 4 to 18 years old. VKC is a rare and recurrent allergic eye condition, most common in children and adolescents, that if left untreated can cause severe inflammation of the surface of the eye which may result in corneal ulcers or potential vision loss. In the clinical trial, VEKTIS, cyclosporine ophthalmic emulsion was administered four times a day (high dose group) or two times a day (low dose group) compared to vehicle group for one month. Next, the patients in vehicle group were switched to either the high- or low-dose group from month 4 to month 12 and then was evaluated. Cyclosporine ophthalmic emulsion met its primary endpoint by demonstrating improvements in corneal fluorescein staining score, rescue medication use, key VKC symptoms and improvement in quality of life assessed by questionnaire.14 Similar products include Allergan’s Restasis® (cyclosporine ophthalmic emulsion 0.05%) and Novartis’ Xiidra® (lifitegrast ophthalmic solution 5%).

6/29/2021: NexoBrid®

The FDA is reviewing MediWound’s NexoBrid to remove nonviable burn tissue (eschar) within four hours of application without harming viable tissue. NexoBrid is a topically administered biological product that enzymatically removes nonviable burn tissue in patients with deep partial and full-thickness thermal burns. DETECT, a Phase 3 clinical trial, demonstrated that NexoBrid in adult patients with thermal burns up to 30% of total body surface area successfully met its primary endpoint of complete eschar removal compared to gel vehicle.15 Additionally, NexoBrid met all secondary endpoints compared to standard of care (SOC), including shorter time to eschar removal, a lower incidence of surgical eschar removal and lower blood loss during eschar removal. Similar products include escharotomy.

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.physiciansweekly.com/oral-gnrh-antagonist-relugolix-reduces-heavy-menstrual-bleeding/

2. https://www.globenewswire.com/news-release/2021/03/24/2198315/0/en/Myovant-Sciences-and-Pfizer-Announce-Positive-Data-from-Phase-3-LIBERTY-Randomized-Withdrawal-Study-of-Once-Daily-Relugolix-Combination-Therapy-in-Women-with-Uterine-Fibroids.html

3. https://www.fiercebiotech.com/biotech/virtual-fda-adcomm-for-alkermes-alks-3831-adds-new-focus-drug-drug-interactions

4. https://bridgebio.com/news/bridgebio-pharma-and-affiliate-qed-therapeutics-announce-fda-acceptance-of-new-drug-application-for-infigratinib-for-the-treatment-of-cholangiocarcinoma

5. https://www.onclive.com/view/later-line-infigratinib-outperforms-standard-chemotherapy-in-cholangiocarcinoma

6. https://www.biospace.com/article/releases/scynexis-presents-data-at-2021-acog-demonstrating-the-positive-outcome-and-sustained-response-of-investigational-oral-ibrexafungerp-brexafemme-in-difficult-to-treat-patients-with-vaginal-yeast-infections/

7. https://www.biospace.com/article/releases/liminal-biosciences-provides-update-on-progress-on-bla-for-ryplazim-and-174-plasminogen-/

8. http://www.pmlive.com/pharma_news/icer_extends_evaluation_of_biogens_aducanumab_after_fda_delay_1363093

9. https://alzheimersnewstoday.com/2021/05/10/insufficient-evidence-aducanumab-efficacy-alzheimers-disease-icer-draft-report-finds/

10. https://www.biospace.com/article/orphazyme-s-arimoclomol-flunks-inclusion-body-myositis-trial/

11. https://www.dermatologytimes.com/view/ruxolitinib-cream-nda-accepted-for-priority-review-by-fda

12. https://www.globenewswire.com/news-release/2020/01/18/1972223/0/en/Verrica-Pharmaceuticals-Announces-Presentation-of-Positive-Data-from-New-Pooled-Analysis-of-Phase-3-CAMP-Trials-of-VP-102.html

13. https://www.biospace.com/article/releases/ascendis-pharma-a-s-announces-u-s-food-and-drug-administration-fda-accepts-biologics-license-application-bla-for-transcon-hgh-for-pediatric-growth-hormone-deficiency-ghd-/

14. https://www.prnewswire.com/news-releases/santen-announces-us-fda-filing-acceptance-of-new-drug-application-nda-for-cyclosporine-topical-ophthalmic-emulsion-0-1-for-the-treatment-of-severe-vernal-keratoconjunctivitis-in-patients-ages-4-18–301159476.html

15. https://www.biospace.com/article/releases/vericel-announces-fda-acceptance-of-biologics-license-application-for-nexobrid-for-the-treatment-of-severe-thermal-burns/

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