Mitsubishi Tanabe Pharma America Receives U.S. Food and Drug Administration Orphan Drug Exclusivity for RADICAVA ORS® (edaravone)
- 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
- None.
Insights
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.
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.
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
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
"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
About RADICAVA® (edaravone) and RADICAVA ORS® (edaravone)
The
Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the
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 (≥
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
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,
Media inquiries:
Media_MTPA@mt-pharma-us.com
1 RADICAVA and RADICAVA ORS Prescribing Information.
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
FAQ
What is the significance of the FDA granting Orphan Drug Exclusivity for RADICAVA ORS (edaravone)?
How does RADICAVA ORS differ from previously approved RADICAVA (edaravone)?
What was the basis for the U.S. approval of RADICAVA (edaravone)?
How many ALS patients in the U.S. have been treated with RADICAVA and RADICAVA ORS?