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Last Updated: June 13, 2025

CLINICAL TRIALS PROFILE FOR RISDIPLAM


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All Clinical Trials for Risdiplam

Trial ID Title Status Sponsor Phase Start Date Summary
NCT02633709 ↗ A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers Completed Hoffmann-La Roche Phase 1 2016-01-07 The objective of this study is to assess the safety and tolerability of Risdiplam (RO7034067) in healthy people. The study will assess what the body does to Risdiplam (RO7034067) and what Risdiplam (RO7034067) does to the body. Risdiplam (RO7034067) will be given by mouth in gradually increasing doses. The data from this study will help to define the dose to further explore Risdiplam (RO7034067) in patients with Spinal Muscular Atrophy.
NCT02908685 ↗ A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants Active, not recruiting Hoffmann-La Roche Phase 2/Phase 3 2016-10-20 Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part (Part 2) of Risdiplam for 24 months.
NCT02913482 ↗ Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy Active, not recruiting Hoffmann-La Roche Phase 2/Phase 3 2016-12-24 Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.
NCT03032172 ↗ A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy Active, not recruiting Hoffmann-La Roche Phase 2 2017-03-03 This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Risdiplam

Condition Name

Condition Name for Risdiplam
Intervention Trials
Muscular Atrophy, Spinal 9
Spinal Muscular Atrophy 8
Neuromuscular Diseases 2
SMA 2
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Condition MeSH

Condition MeSH for Risdiplam
Intervention Trials
Muscular Atrophy, Spinal 17
Muscular Atrophy 17
Atrophy 17
Neuromuscular Diseases 2
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Clinical Trial Locations for Risdiplam

Trials by Country

Trials by Country for Risdiplam
Location Trials
United States 23
Italy 16
Brazil 6
Belgium 5
Poland 5
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Trials by US State

Trials by US State for Risdiplam
Location Trials
New York 5
Florida 4
California 4
Texas 3
Massachusetts 3
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Clinical Trial Progress for Risdiplam

Clinical Trial Phase

Clinical Trial Phase for Risdiplam
Clinical Trial Phase Trials
Phase 4 5
Phase 3 3
Phase 2/Phase 3 3
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Clinical Trial Status

Clinical Trial Status for Risdiplam
Clinical Trial Phase Trials
Not yet recruiting 9
Completed 4
Active, not recruiting 3
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Clinical Trial Sponsors for Risdiplam

Sponsor Name

Sponsor Name for Risdiplam
Sponsor Trials
Hoffmann-La Roche 13
Genentech, Inc. 2
Biohaven Pharmaceuticals, Inc. 1
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Sponsor Type

Sponsor Type for Risdiplam
Sponsor Trials
Industry 18
Other 1
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Risdiplam: Clinical Trials, Market Analysis, and Projections

Introduction to Risdiplam

Risdiplam, marketed as Evrysdi, is a groundbreaking treatment for Spinal Muscular Atrophy (SMA), a genetic disorder that affects muscle strength and movement. Developed by Genentech, Inc., a member of the Roche Group, risdiplam has been making significant strides in clinical trials and the market.

Clinical Trials Overview

FIREFISH Trial

The FIREFISH trial, initiated in December 2016, focuses on infants with SMA Type 1. This trial has two parts: the first part enrolled 62 infants to explore an exploratory dose for 12 weeks, completed in November 2021. The second part, with 41 infants, further investigated the selected dose for 24 months, with five-year data announced in June 2024[1][4].

SUNFISH Trial

The SUNFISH trial, started in October 2016, involves children and adults with SMA Type 2 or Type 3. The trial has two parts: the first part enrolled 231 participants to investigate an exploratory dose for 12 weeks, completed in September 2019. The second part, with 180 participants, is ongoing to evaluate the selected dose for 24 months, with an estimated completion date in early September 2023[1][4].

JEWELFISH Trial

The JEWELFISH trial, which began in March 2017, includes participants with SMA Type 1, 2, or 3 who have previously received other SMA treatments like nusinersen or onasemnogene abeparvovec (Zolgensma). This trial has shown a sustained increase in SMN protein levels and has demonstrated a strong safety profile[3][4].

RAINBOWFISH Trial

The RAINBOWFISH trial, initiated in August 2019, is an open-label study focusing on pre-symptomatic infants with genetically diagnosed SMA. This trial evaluates the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in these infants[3][4].

HINALEA Trials

Two new open-label trials, HINALEA 1 and HINALEA 2, were initiated in mid-2023 to assess the safety and efficacy of risdiplam in infants and children with SMA who have previously received Zolgensma. These trials will enroll approximately 28 participants each and are expected to complete by mid-2028[1].

Key Findings from Clinical Trials

  • SMN Protein Levels: Risdiplam treatment has led to a median two-fold increase in blood SMN protein levels, sustained for at least 12 months in several trials[3][4].
  • Motor Milestones: Infants with SMA Type 1 treated with risdiplam have achieved major motor milestones, and patients with Type 2 and 3 SMA have shown clinically meaningful increases in muscle function[3][4].
  • Safety Profile: All clinical trials have demonstrated a strong safety profile for risdiplam, with no drug-related safety findings leading to withdrawal from any study[3][4].

Market Analysis

Market Size and Growth

The global spinal muscular atrophy treatment market is expected to reach USD 13.09 billion by 2030, growing at a CAGR of 18.0% from 2025 to 2030. This growth is driven by increasing product approvals and launches, including the approval of risdiplam (Evrysdi) by regulatory bodies in various regions[2].

Regional Market

The Asia Pacific region is anticipated to be the fastest-growing market during the forecast period, driven by the entry of new products such as risdiplam into the region. For instance, risdiplam was launched in India in July 2021[2].

Competitive Landscape

Roche, with its diversified neurology portfolio, is expected to expand its foothold in the neurology market, with risdiplam being one of its key compounds. However, Roche's reliance on its blockbusters, particularly Ocrevus, whose patents are set to expire, may pose a challenge[5].

Market Projections

Approval and Licensing

Risdiplam has received significant regulatory approvals, including FDA approval in August 2020 for pediatric patients aged two months and older with SMA, and EMA approval in March 2021 for patients with Type 1, 2, and 3 SMA[2][3].

New Formulations

A Phase 1 study completed in 2023 demonstrated the efficacy and safety of a tablet formulation of risdiplam, which showed similar absorption profiles whether taken whole or dispersed in water. Roche plans to apply for licensing of this tablet formulation to the EMA and FDA in the first half of 2024[1].

Long-Term Effectiveness

The ACTIVENESS study, a multi-country, non-interventional, prospective, longitudinal study, aims to assess the long-term effectiveness of risdiplam in SMA patients. This study will include approximately 600 patients and will provide valuable insights into the long-term benefits of risdiplam[1].

Key Takeaways

  • Clinical Trials Success: Risdiplam has shown significant efficacy and safety in various clinical trials, including FIREFISH, SUNFISH, JEWELFISH, and RAINBOWFISH.
  • Market Growth: The SMA treatment market is expected to grow substantially, driven by approvals and launches of new treatments like risdiplam.
  • Regulatory Approvals: Risdiplam has received key regulatory approvals and is expected to expand its market presence with new formulations.
  • Long-Term Studies: Ongoing and planned studies like ACTIVENESS will provide crucial data on the long-term effectiveness of risdiplam.

FAQs

What is risdiplam used for?

Risdiplam, marketed as Evrysdi, is used for the treatment of spinal muscular atrophy (SMA) in patients aged two months and older.

What are the key clinical trials for risdiplam?

The key clinical trials include FIREFISH, SUNFISH, JEWELFISH, RAINBOWFISH, and the newly initiated HINALEA trials.

How has risdiplam performed in clinical trials?

Risdiplam has shown a significant increase in SMN protein levels, improvement in motor milestones, and a strong safety profile across various trials.

What is the projected market size for SMA treatments by 2030?

The global SMA treatment market is expected to reach USD 13.09 billion by 2030, growing at a CAGR of 18.0% from 2025 to 2030.

Is risdiplam available in tablet form?

Yes, a Phase 1 study has demonstrated the efficacy and safety of a tablet formulation of risdiplam, and Roche plans to apply for licensing in the first half of 2024.

Sources

  1. SMA UK: Results and Updates for Each Risdiplam Trial.
  2. Grand View Research: Global Spinal Muscular Atrophy Treatment Market.
  3. PTC Therapeutics: Risdiplam Spinal Muscular Atrophy Data Demonstrating Continued Benefit.
  4. SMA UK: Risdiplam Clinical Trials and Their Results.
  5. Pharmaceutical Technology: Roche and Biogen to go head-to-head in neurology market in 2025.
Last updated: 2024-12-31

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