January 17, 2024

Mitsubishi Tanabe Pharma America, Inc. (MTPA) Statement on Ferrer’s Phase 3 ADORE Clinical Trial of FNP122 (FAB122)

JERSEY CITY, N.J. January 17, 2024 – Mitsubishi Tanabe Pharma America, Inc. (MTPA) is aware of the recent outcome of Grupo Ferrer Internacional (Ferrer’s) Phase 3 ADORE clinical trial of FNP122 (FAB122), an investigational oral formulation of edaravone. It is important to note that the formulation of oral edaravone used in this study is not RADICAVA ORS® (edaravone) or RADICAVA IV® (edaravone).1 In addition, the ADORE clinical trial, which is not an MTPA sponsored study, had many significant differences from the RADICAVA IV pivotal Phase 3 trial (MCI186-19), including study design, inclusion criteria and dosing.2

RADICAVA ORS is an oral suspension of 105 mg per 5 mL, which has been shown to be equivalent to RADICAVA IV in pharmacokinetic testing3,4, whereas FNP122 (FAB122) consists of granules packaged in sachets to be dissolved in water for a total of 100 mg per dose5; as such any data and/or conclusions relative to FNP122 (FAB122) cannot be directly compared to RADICAVA IV or RADICAVA ORS.

RADICAVA IV was shown in the pivotal Phase 3 trial (MCI186-19) to slow the loss of physical function by 33 percent in 68 patients vs. placebo (n=66 patients), measured over a 24-week time period by the ALS Functional Rating Scale-Revised (ALSFRS-R).3 On average, patients on RADICAVA IV lost 2.49 fewer points on the ALSFRS-R vs. those in the placebo arm.3 RADICAVA ORS offers the same efficacy as RADICAVA IV in an oral formulation. 3, 4 The safety profile of RADICAVA IV was demonstrated in pooled placebo-controlled trials in which 184 patients with ALS were administered RADICAVA IV (60 mg) in 24-week treatment cycles.3 The safety profile of RADICAVA ORS was demonstrated in a 6-month, Phase 3, open-label clinical trial in 185 patients.3 Please see Important Safety Information below and Full Prescribing Information here.

Additionally, a large, exploratory, retrospective, observational, real-world evidence (RWE) study was conducted to evaluate overall survival in patients with ALS receiving RADICAVA IV compared with patients with ALS not receiving RADICAVA IV and who were continuously enrolled for 12 months in an administrative claims database. The data demonstrated that initiation of RADICAVA IV resulted in a 6-month longer median survival compared with the non−RADICAVA IV-treated controls and that the risk for death was 27% lower among RADICAVA IV-treated cases compared with non−RADICAVA IV-treated controls (HR, 0.73; 95% CI, 0.59–0.91; P=0.005).6 This study was limited only to patients with ALS who had commercial health coverage. Real-world data analyses have inherent limitations and are not intended to replace prospective clinical trials.

RADICAVA ORS is the only U.S. Food and Drug Administration (FDA)-approved formulation of oral edaravone in the U.S. and the outcome of the FNP122 (FAB122) ADORE study does not impact the U.S. commercial availability of RADICAVA ORS. In addition, RADICAVA ORS is also the only Health Canada-approved formulation of oral edaravone in Canada and has been approved in Japan and Switzerland.

To date, RADICAVA ORS and RADICAVA IV have been studied in multiple clinical trials, post-hoc analysis and RWE studies evaluating the efficacy and safety of both the ORS (oral) and IV formulations and have been published in over 40 peer-reviewed articles.

IMPORTANT SAFETY INFORMATION

Hypersensitivity Reactions
RADICAVA (edaravone) and RADICAVA ORS (edaravone) are contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals, and erythema multiforme) and cases of anaphylaxis (urticaria, decreased blood pressure, and dyspnea) have occurred with RADICAVA.

Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA or RADICAVA ORS, treat per standard of care, and monitor until the condition resolves.

Sulfite Allergic Reactions
RADICAVA and RADICAVA ORS contain sodium bisulfite, a sulfite that may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible people. The overall prevalence of sulfite sensitivity in the general population is unknown but occurs more frequently in asthmatic people.

Adverse Reactions
The most common adverse reactions (≥10%) reported in RADICAVA-treated patients were contusion (15%), gait disturbance (13%), and headache (10%). In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS. 

Pregnancy
Based on animal data, RADICAVA and RADICAVA ORS may cause fetal harm.

To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You may also report suspected adverse reactions to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

INDICATION
RADICAVA and RADICAVA ORS are indicated for the treatment of amyotrophic lateral sclerosis (ALS).

For more information, including full Prescribing Information, please visit www.RADICAVA.com.

About Mitsubishi Tanabe Pharma America, Inc.
Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit www.mt-pharma-america.com or follow us on X (formerly Twitter), Facebook and LinkedIn.

About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), the pharma arm of Mitsubishi Chemical Group (MCG), is one of the oldest pharmaceutical companies in the world, founded in 1678. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan’s pharmaceutical industry. MCG has positioned health care as its strategic focus in its management policy, “Forging the future”. MTPC sets the MISSION of “Creating hope for all facing illness”. To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on “precision medicine” to provide drugs with high treatment satisfaction and additionally working to develop “around the pill solutions” to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to https://www.mt-pharma.co.jp/e/.

Media inquiries:
Media_MTPA@mt-pharma-us.com

1 Hulskotte EGJ, et al. Pharmacokinetics and safety of TW001, the oral formulation of edaravone. Poster presented at: 28th International Symposium on ALS/MND; December 8-10, 2017; Boston, MA.
2 Moolenaar SH, et al. Treatment comprising oral or gastric administration of edaravone. International patent WO 2018/134243 (A1). July 26, 2018.
3 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.
4 Shimizu H, et al. Bioequivalence study of oral suspension and intravenous formulation of edaravone in healthy adult subjects. Clin Pharmacol Drug Dev. 2021;10(10):1188-1197.
5 van den Berg L, et al. Design of an international, phase 3, randomized, placebo-controlled trial with daily oral edaravone (FNP122) in ALS: the ADORE study. Poster presented at: 2022 ENCALS meeting; June 1-3, 2022; Edinburgh, Scotland.
6 Brooks, B, et al. Intravenous edaravone treatment in ALS and survival: An exploratory, retrospective, administrative claims analysis. eClinicalMedicine. 2022; 52: 101590.