CLINICAL TRIALS PROFILE FOR RADICAVA

All Clinical Trials for RADICAVA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03272503 ↗	A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS)	Recruiting	ALS Canada	Phase 2	2017-10-27	This study will look at whether Pimozide may help to slow the progression of Amyotrophic Lateral Sclerosis. 100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo. Participants will be on study medication up to 22 weeks, and on study up to 26 weeks. There are 8 clinic visits and 1 phone visit over the course of the Treatment Phase of the study. The second phase which is Observational, is optional with follow-up for up to 5 years from the end of the Treatment Phase.
NCT03272503 ↗	A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS)	Recruiting	Brain Canada	Phase 2	2017-10-27	This study will look at whether Pimozide may help to slow the progression of Amyotrophic Lateral Sclerosis. 100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo. Participants will be on study medication up to 22 weeks, and on study up to 26 weeks. There are 8 clinic visits and 1 phone visit over the course of the Treatment Phase of the study. The second phase which is Observational, is optional with follow-up for up to 5 years from the end of the Treatment Phase.
NCT03272503 ↗	A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS)	Recruiting	University of Calgary	Phase 2	2017-10-27	This study will look at whether Pimozide may help to slow the progression of Amyotrophic Lateral Sclerosis. 100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo. Participants will be on study medication up to 22 weeks, and on study up to 26 weeks. There are 8 clinic visits and 1 phone visit over the course of the Treatment Phase of the study. The second phase which is Observational, is optional with follow-up for up to 5 years from the end of the Treatment Phase.
NCT03272802 ↗	Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS)	Unknown status	Isfahan University of Medical Sciences	Phase 2/Phase 3	2017-03-16	Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that causes the death of 30,000 affected individual every year. Complex nature and unknown pathogenesis of this disease are 2 major reasons for failure of therapeutic interventions. Edaravone is a free radical scavenger that slows down functional decline and prevents from disease progression in ALS patients. FDA newly approved this drug in these patients (2017/5/5). In this study, investigators aimed to assess the treatment effect of this newly approved drug in patients with ALS in a representative Iranian population.
NCT03664544 ↗	PK Study in Subjects With Severe Hepatic Impairment	Completed	Mitsubishi Tanabe Pharma Corporation	Phase 1	2018-11-06	This is an open-label, single-dose study in male and female subjects with severe hepatic impairment and in male and female subjects with normal hepatic function.
NCT06107205 ↗	Bioequivalence of TTYP01 Tablets in Healthy Adult Subjects	Completed	Auzone Biological Technology Pty Ltd	Phase 1	2023-11-07	This is a Phase 1, Randomized, Open-Label, Three-Treatment, Three-Period Crossover Study to Assess Bioequivalence and Safety of TTYP01 Tablets to Radicava® Injection, and Radicava ORS® in Healthy Adult Subjects Under Fasting Conditions.The objective is To characterize the bioequivalence、safety and tolerability of TTYP01 tablets and Radicava® injection or Radicava ORS®in healthy adult subjects under fasted conditions.In this study, 30 healthy adult subjects will receive TTYP01, or Radicava, orRadicava ORS in each period according to the randomization sequence.
NCT06249867 ↗	A Study to Assess the Safety, Tolerability, and Pharmacology of Darifenacin in Patients With ALS	RECRUITING	Université de Montréal	PHASE2	2024-11-08	Amyotrophic lateral sclerosis (ALS) is a progressive neurological disorder characterized by selective death of upper and lower motor neurons, which leads to severe disability and fatal outcomes. One of the major hallmarks of ALS is the denervation of neuromuscular junctions (NMJs), which is one of the earliest events seen in ALS patients and mouse models of ALS. Under healthy conditions, glial cells called Perisynaptic Schwann Cells (PSCs) have a key role in regulating the stability and maintenance of NMJs, but they only participate in NMJ repair once denervation occurs. Denervation and the subsequent decline in synaptic activity triggers a loss of muscarinic acetylcholine receptors (mAChRs) in the PSC, and the resulting decrease in mAChR-mediated gene expression drives the "repair mode" of the PSC. In assessing the NMJ under conditions of ALS, a scarcity of process extensions in PSCs was observed for months prior to disease onset in the superoxide dismutase 1 (SOD1) mouse model of ALS, indicating inadequate glial repair. Collectively, these preclinical findings support the hypothesis that dampening glial mAChRs will restore the anticipated "repair" response of PSCs in the NMJ. Hence, the use of a selective M3 muscarinic receptor antagonist, Darifenacin, as a disease-modifying therapeutic in familial and sporadic ALS could improve NMJ function, resulting in a beneficial impact on the autonomy and quality of life of ALS patients. The purpose of the current Phase 2 trial is therefore to test the safety, tolerability, and pharmacology of Darifenacin in patients with ALS. Specifically, 30 eligible subjects between 18 and 85 years of age will take 7.5 mg of darifenacin or placebo daily (by mouth) for two weeks followed by an increased dose of 15 mg for the next 22 weeks. The trial will evaluate the effects of this medication on several outcome measures including patient safety, physical and neurological function, muscle strength, depression levels, and NMJ innervation of patients with ALS. Detailed clinical assessments will be conducted at regular intervals throughout the study in order to achieve these objectives.

Clinical Trial Conditions for RADICAVA

Condition Name

Condition Name for RADICAVA
Intervention	Trials
Amyotrophic Lateral Sclerosis	2
ALS	1
Healthy	1
Healthy Adult Subjects	1

Condition MeSH

Condition MeSH for RADICAVA
Intervention	Trials
Amyotrophic Lateral Sclerosis	3
Sclerosis	2
Motor Neuron Disease	2
Liver Diseases	1

Clinical Trial Locations for RADICAVA

Trials by Country

Trials by Country for RADICAVA
Location	Trials
Canada	6
Hungary	1
Czechia	1
United States	1
Iran, Islamic Republic of	1

Trials by US State

Trials by US State for RADICAVA
Location	Trials
Texas	1

Clinical Trial Progress for RADICAVA

Clinical Trial Phase

Clinical Trial Phase for RADICAVA
Clinical Trial Phase	Trials
PHASE2	1
Phase 2/Phase 3	1
Phase 2	1

Clinical Trial Status

Clinical Trial Status for RADICAVA
Clinical Trial Phase	Trials
Completed	2
RECRUITING	2
Unknown status	1

Clinical Trial Sponsors for RADICAVA

Sponsor Name

Sponsor Name for RADICAVA
Sponsor	Trials
Brain Canada	1
University of Calgary	1
Isfahan University of Medical Sciences	1

Sponsor Type

Sponsor Type for RADICAVA
Sponsor	Trials
Other	6
Industry	2

Last updated: May 7, 2026

RADICAVA (edaravone): Clinical-trials update, market analysis, and forward projection

What is RADICAVA and what is its current regulatory footprint?

RADICAVA is edaravone, a free-radical scavenger approved for amyotrophic lateral sclerosis (ALS). The key branded products in commercial use are:

• RADICAVA (edaravone) IV: historically approved for ALS in the US (under earlier eligibility rules that limited use to a specific patient subgroup).
• RADICAVA ORS (edaravone oral suspension): is the newer US formulation designed to expand administration practicality versus IV infusions.
• RADICAVA (edaravone) subcutaneous and/or other regional formulations: exist in some markets, with uptake driven by local reimbursement rules and prescribing patterns (not all formulations are uniformly approved in every geography).

Regulatory label evolution (US) that matters commercially

• The US approval history for edaravone included restrictive inclusion criteria early on.
• Later label expansion removed or relaxed parts of the eligibility constraints, improving addressable patient volume (and lowering the friction for prescribers).

Which clinical trials currently matter for RADICAVA, and what is the update status?

A full “current active trials” inventory requires a live registry pull. This response cannot provide a complete and accurate trials update without that registry-level dataset.

What can be stated with confidence from the durable, widely documented program outline

• Edaravone’s clinical development has centered on ALS efficacy and functional outcomes using endpoints such as ALSFRS-R trajectories and survival-related measures.
• Trials for edaravone have historically compared against placebo in ALS populations with specific baseline characteristics.
• The later-generation product (oral suspension) primarily addresses route of administration, bioavailability, and tolerability, rather than changing the core evidence base for efficacy.

Where does RADICAVA generate revenue, and what drives demand?

Demand is driven by four practical variables:

1. Eligibility and label constraints

   • Earlier restrictiveness limited eligible patients and capped conversion.
   • Relaxations expanded the treatable population.

2. Administration burden

   • IV requires clinic or infusion infrastructure.
   • Oral suspension reduces operational friction and improves adherence feasibility.

3. Reimbursement and prior authorization

   • In the US, access is mediated by insurer policy and step-therapy/prior authorization pathways.
   • Patient assistance programs and contracting terms influence net paid price.

4. Competitive pressure in ALS

   • RADICAVA competes in a crowded ALS landscape that includes agents affecting disease biology or supportive endpoints.
   • Competitive substitution risk is highest where payers prefer lower-cost alternatives or where new evidence favors competing mechanisms.

What is the market size and growth outlook for RADICAVA?

A quantitative forecast (revenue, patients, and share) requires current market tracking data (pricing, treatment rates, uptake of ORS vs IV, and geography-level reimbursement trends). This response cannot provide a complete and accurate market-sizing model without those inputs.

Structure of a credible RADICAVA projection model (what determines the curve)

• Addressable ALS population (US + major EU/JP geographies if modeling internationally)
• Treatable fraction under label criteria
• Penetration rate (share of eligible patients receiving edaravone)
• Formulation mix (IV vs ORS)
• Persistence (time-on-treatment)
• Net price (WAC less discounts, rebates, and payer contracts)

Without live pricing and payer-contract data, any numeric projection would not meet an accuracy standard required for investment or R&D planning.

How does ORS change the commercial trajectory vs IV?

Even without live uptake numbers, the directionality is clear: oral administration typically increases:

• Converting eligible patients who are otherwise deterred by infusion scheduling
• Persistence by reducing missed infusions or clinic dependency
• Payer willingness when operational burden is lower and adherence risk is reduced

Net impact depends on:

• ORS reimbursement parity vs IV
• prior authorization criteria stability
• patient co-pay support
• prescriber adoption and formulary placement

What are the key risks to RADICAVA revenue (business drivers)?

1. Payer tightening

   • Prior authorization and coverage limitations can reintroduce effective eligibility constraints even after label expansion.

2. Formulary substitution

   • Competitors can displace edaravone if payer contracts or clinical guidelines shift.

3. Pipeline or lifecycle saturation

   • If the oral switch satisfies most patient needs, growth becomes penetration-led rather than expansion-led.

4. Safety and tolerability monitoring

   • Ongoing pharmacovigilance and real-world tolerability can change prescribing behavior.

What is the key R&D and IP posture to track for RADICAVA?

For investment decisions, the critical IP question is when core composition, formulation, and method-of-use protections expire by geography, including:

• Composition of matter (ediravone itself is not new, so the practical IP likely concentrates on formulation and specific indications/uses)
• Formulation IP (oral suspension and dosing regimen protections)
• Process patents (manufacturing and stability)
• Orphan and regulatory exclusivity (where applicable, depending on jurisdiction rules)

A proper freedom-to-operate and lifecycle analysis requires a jurisdiction-by-jurisdiction patent estate pull, which is not included in the information provided in this conversation. This response therefore cannot produce a complete, accurate IP timeline.

---

Key Takeaways

• RADICAVA is edaravone for ALS; commercial demand is primarily shaped by label eligibility, administration burden, and reimbursement.
• RADICAVA ORS reduces operational friction versus IV, which typically supports higher penetration and persistence where reimbursement is stable.
• A complete clinical-trials update and market/revenue forecast requires a current, registry and payer-pricing dataset; without it, this response does not present numeric trial counts, enrollment changes, or financial projections.
• For forward planning, the decision-critical elements are treatable population growth, payer access policy, formulation mix, and the patent estate by geography.

---

FAQs

1) Is RADICAVA still prescribed in its IV form?
Yes. IV use persists where formularies and payer policies favor it or where ORS adoption is incomplete.

2) What clinical endpoints define RADICAVA’s evidence base in ALS?
Trials have focused on ALS functional decline, commonly using ALSFRS-R-related outcomes and time-to-event style endpoints in secondary analyses.

3) Does RADICAVA ORS expand eligibility or only convenience?
Primarily, ORS improves administration practicality; label eligibility is determined by the approved indication and coverage criteria, not by route alone.

4) What are the biggest commercial risks for edaravone in ALS?
Reimbursement tightening and formulary substitution are the highest-leverage risks to maintain share.

5) What should investors track next for RADICAVA?
Active-cycle evidence in ALS, ORS uptake vs IV, payer policy changes, and the patent expiration calendar across major markets.

---

References (APA)

[1] U.S. Food and Drug Administration. (n.d.). Drug approvals and safety information for RADICAVA (edaravone). FDA. https://www.fda.gov/
[2] European Medicines Agency. (n.d.). EudraGMDP and product information for edaravone (RADICAVA). EMA. https://www.ema.europa.eu/