Mitsubishi Tanabe Pharma America Receives U.S. Food and Drug Administration Orphan Drug Exclusivity for RADICAVA ORS® (edaravone)

Rhea-AI Summary

Mitsubishi Tanabe Pharma America, Inc. receives Orphan Drug Exclusivity for RADICAVA ORS, an oral form of edaravone, for ALS treatment. The FDA acknowledges its significant contribution to patient care. RADICAVA ORS offers a self-administered, oral suspension route of administration, providing a clinically superior option to IV administration. The approval was based on a pivotal Phase 3 study showing a 33% slowdown in physical function loss compared to placebo.

Positive

• FDA grants Orphan Drug Exclusivity for RADICAVA ORS for ALS treatment
• RADICAVA ORS offers a self-administered, oral suspension route of administration
• Approval based on pivotal Phase 3 study showing 33% slowdown in physical function loss
• Over 14,600 ALS patients in the U.S. treated with RADICAVA and RADICAVA ORS
• Supported by robust data from multiple clinical trials and real-world evidence studies

Negative

• None.

Medical Research Analyst

The FDA's granting of Orphan Drug Exclusivity (ODE) to RADICAVA ORS (edaravone) is a significant milestone for Mitsubishi Tanabe Pharma America. This decision by the FDA is based on the drug's novel oral suspension route of administration, which offers a less burdensome alternative to intravenous therapy for ALS patients. The ODE status provides market exclusivity for seven years, which can be a strategic advantage for MTPA in the competitive pharmaceutical landscape.

From a medical research perspective, the importance of this development lies in the potential improvement in the quality of life for ALS patients. The ease of administration and the elimination of the need for refrigeration or reconstitution of the drug are practical benefits that could lead to better adherence to treatment regimens. This, coupled with the drug's demonstrated efficacy in slowing the loss of physical function, as evidenced by the pivotal Phase 3 clinical trial, suggests that RADICAVA ORS could see increased utilization.

For stakeholders, the exclusivity period could result in sustained revenue growth for MTPA. However, dependency on a single drug for revenue generation can be risky and it's essential to consider the broader implications of market exclusivity, including the potential for generic competition post-exclusivity and the need for continued innovation.

Healthcare Policy Expert

The FDA's recognition of RADICAVA ORS for ODE underlines a supportive regulatory environment for treatments targeting rare diseases. The Orphan Drug program incentivizes pharmaceutical companies to invest in drug development for conditions like ALS, which affects a small patient population. This policy can lead to advancements in treatment options where they may otherwise be unprofitable due to the limited market size.

For the healthcare system, the availability of an oral formulation can potentially reduce the overall costs associated with ALS treatment. Intravenous administration often requires medical personnel and clinic resources, whereas an oral drug can be self-administered. Over time, this could lead to a reduction in healthcare spending on ALS patient care, though initial drug costs may be higher due to exclusivity.

Furthermore, the impact on healthcare providers will be twofold: there is a learning curve associated with educating patients on the new oral administration, but it may also alleviate some of the time and resource burdens associated with IV treatments. The long-term benefits for the ALS community and healthcare providers will hinge on the accessibility and affordability of RADICAVA ORS.

Pharmaceutical Market Analyst

The announcement of ODE for RADICAVA ORS represents a strategic win for Mitsubishi Tanabe Pharma America in the niche market of ALS treatments. The exclusivity period can lead to a temporary monopoly, allowing MTPA to set prices without immediate generic competition. The oral formulation could potentially capture a significant share of the market, given its convenience over IV administration.

Analyzing the business implications, the exclusivity could lead to increased stock market interest in MTPA, as investors often react positively to such regulatory milestones. It's important to note that while the exclusivity is beneficial, the company's valuation will likely reflect the anticipated revenue from RADICAVA ORS sales, adjusted for the size of the treatable population and market penetration rates.

On the downside, once the exclusivity period ends, the company may face generic competition, which could dramatically reduce the drug's price and market share. To mitigate this, MTPA may need to focus on continuous innovation and pipeline development. Additionally, the reliance on RWE studies and post-hoc analyses to support the drug's efficacy and safety highlights the importance of ongoing research and data collection in maintaining the drug's market position.

Orphan Drug Exclusivity recognized for RADICAVA ORS based on major contribution to patient care 

JERSEY CITY, N.J., April 8, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced that the U.S. Food and Drug Administration (FDA) has recognized seven years of Orphan Drug Exclusivity (ODE) for RADICAVA ORS^® (edaravone) based on their assessment that the oral form of edaravone constitutes a major contribution to patient care for people living with amyotrophic lateral sclerosis (ALS). The FDA's Orphan Drug program is designed to support the development of drugs that treat rare diseases which affect less than 200,000 people in the U.S. Previously, the FDA granted RADICAVA ORS Priority Review and Fast Track designations.

The FDA recognized ODE for RADICAVA ORS because it provides a clinically superior option for patients due to its oral suspension route of administration that can help reduce the burden patients face with intravenous (IV) administration of previously approved RADICAVA^® (edaravone). RADICAVA ORS is self-administered, can be taken orally or via feeding tube, comes in a portable bottle with an oral dosing syringe and no need for patients to refrigerate or reconstitute before taking. With appropriate instruction from a healthcare provider (HCP), RADICAVA ORS may take only a few minutes to administer on treatment days and should be taken in the morning after overnight fasting.¹

"I'm thrilled at the FDA's recognition of ODE to RADICAVA ORS," said Stanley H. Appel, MD, Houston Methodist Stanley H. Appel Department of Neurology. "This comes at a critical time in the ALS treatment landscape and helps ensure that our patients and HCPs have a treatment option validated for several years, including by the pivotal Phase 3 clinical trial."

The U.S. approval of RADICAVA^® (edaravone) was based on the pivotal Phase 3 study (Study 19 or MCI186-19), in which RADICAVA was shown 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).¹ On average, patients on RADICAVA lost 2.49 fewer points on the ALSFRS-R vs. those in the placebo arm (p=0.0013).^1,2 RADICAVA ORS offers the same efficacy as RADICAVA in a different formulation.^1,2 The safety profile of RADICAVA was demonstrated in pooled placebo-controlled trials in which 184 patients with ALS were administered RADICAVA (60 mg) in 24-week treatment cycles.¹ The safety profile of RADICAVA ORS was demonstrated in a 6-month, Phase 3, open-label clinical trial in 185 patients. Please see Important Safety Information below and Full Prescribing Information here.

"We recognize the profound impact that an oral formulation has on the ALS community, in part by offering a less burdensome option for people living with ALS," said Calaneet Balas, President & CEO, ALS Association. "We thank Mitsubishi Tanabe Pharma America for continuing their efforts to help provide treatments to people living with ALS."

To date, in the U.S., over 14,600 people living with ALS have been treated with RADICAVA and RADICAVA ORS and has been prescribed by over 2,300 HCPs.^3,4 Both formulations are supported by a robust set of data from multiple clinical trials, post-hoc analysis and real-world evidence (RWE) studies evaluating the efficacy and safety. These results have been published in over 40 peer-reviewed articles.

About RADICAVA^® (edaravone) and RADICAVA ORS^® (edaravone)
The U.S. Food and Drug Administration (FDA) approved RADICAVA^® (edaravone) on May 5, 2017, and the oral formulation RADICAVA ORS^® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA recognized RADICAVA ORS with Orphan Drug Exclusivity based on the major contribution to patient care of the innovative oral formulation. RADICAVA is administered in 28-day cycles by intravenous (IV) infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily for 10 days within a 14-day period, followed by a two-week drug-free period. RADICAVA ORS is taken daily for 14 consecutive days followed by a 14-day drug-free period for the initial treatment cycle. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.¹ 

Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as RADICUT^® for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), Malaysia (December 2021) and Brazil (February 2024). Marketing authorization for RADICAVA^® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT^® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022. To date, in the U.S., RADICAVA and RADICAVA ORS have been used to treat over 14,600 people with ALS, with over 1.8-million days of therapy, and have been prescribed by over 2,300 HCPs.^3-5

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 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.
² 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.
³ Data on file. Mitsubishi Tanabe Pharma America, Inc.
⁴ Data on file. Mitsubishi Tanabe Pharma America, Inc.
⁵ Data on file. Mitsubishi Tanabe Pharma America, Inc.

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SOURCE Mitsubishi Tanabe Pharma America

What is the significance of the FDA granting Orphan Drug Exclusivity for RADICAVA ORS (edaravone)?

The FDA acknowledged the major contribution of RADICAVA ORS to patient care for ALS, granting it seven years of Orphan Drug Exclusivity.

How does RADICAVA ORS differ from previously approved RADICAVA (edaravone)?

RADICAVA ORS offers a self-administered, oral suspension route of administration, providing a clinically superior option to IV administration.

What was the basis for the U.S. approval of RADICAVA (edaravone)?

The U.S. approval was based on a pivotal Phase 3 study showing a 33% slowdown in physical function loss compared to placebo.

How many ALS patients in the U.S. have been treated with RADICAVA and RADICAVA ORS?

Over 14,600 people living with ALS in the U.S. have been treated with RADICAVA and RADICAVA ORS.

What kind of data supports the efficacy and safety of RADICAVA and RADICAVA ORS?

Both formulations are supported by robust data from multiple clinical trials, post-hoc analysis, and real-world evidence studies, with results published in over 40 peer-reviewed articles.