You're using a free limited version of DrugPatentWatch: Upgrade for Complete Access

Last Updated: July 18, 2025

Risdiplam - Generic Drug Details


✉ Email this page to a colleague

« Back to Dashboard


What are the generic drug sources for risdiplam and what is the scope of patent protection?

Risdiplam is the generic ingredient in one branded drug marketed by Genentech Inc and is included in two NDAs. There are six patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Risdiplam has one hundred and fifty-three patent family members in thirty-nine countries.

One supplier is listed for this compound.

Summary for risdiplam
International Patents:153
US Patents:6
Tradenames:1
Applicants:1
NDAs:2
Finished Product Suppliers / Packagers: 1
Raw Ingredient (Bulk) Api Vendors: 36
Clinical Trials: 18
Patent Applications: 69
What excipients (inactive ingredients) are in risdiplam?risdiplam excipients list
DailyMed Link:risdiplam at DailyMed
DrugPatentWatch® Estimated Loss of Exclusivity (LOE) Date for risdiplam
Generic Entry Dates for risdiplam*:
Constraining patent/regulatory exclusivity:
Dosage:
FOR SOLUTION;ORAL
Generic Entry Dates for risdiplam*:
Constraining patent/regulatory exclusivity:
Dosage:
TABLET;ORAL

*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for risdiplam

Identify potential brand extensions & 505(b)(2) entrants

SponsorPhase
Hoffmann-La RochePhase 4
Clinic for Special ChildrenPhase 4
Biohaven Pharmaceuticals, Inc.Phase 3

See all risdiplam clinical trials

Paragraph IV (Patent) Challenges for RISDIPLAM
Tradename Dosage Ingredient Strength NDA ANDAs Submitted Submissiondate
EVRYSDI For Oral Solution risdiplam 0.75 mg/mL 213535 2 2024-08-07

US Patents and Regulatory Information for risdiplam

Applicant Tradename Generic Name Dosage NDA Approval Date TE Type RLD RS Patent No. Patent Expiration Product Substance Delist Req. Exclusivity Expiration
Genentech Inc EVRYSDI risdiplam FOR SOLUTION;ORAL 213535-001 Aug 7, 2020 RX Yes Yes ⤷  Try for Free ⤷  Try for Free ⤷  Try for Free
Genentech Inc EVRYSDI risdiplam FOR SOLUTION;ORAL 213535-001 Aug 7, 2020 RX Yes Yes ⤷  Try for Free ⤷  Try for Free ⤷  Try for Free
Genentech Inc EVRYSDI risdiplam FOR SOLUTION;ORAL 213535-001 Aug 7, 2020 RX Yes Yes 9,969,754 ⤷  Try for Free Y Y ⤷  Try for Free
>Applicant >Tradename >Generic Name >Dosage >NDA >Approval Date >TE >Type >RLD >RS >Patent No. >Patent Expiration >Product >Substance >Delist Req. >Exclusivity Expiration

EU/EMA Drug Approvals for risdiplam

Company Drugname Inn Product Number / Indication Status Generic Biosimilar Orphan Marketing Authorisation Marketing Refusal
Roche Registration GmbH  Evrysdi risdiplam EMEA/H/C/005145
Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. 
Authorised no no no 2021-03-26
>Company >Drugname >Inn >Product Number / Indication >Status >Generic >Biosimilar >Orphan >Marketing Authorisation >Marketing Refusal

Supplementary Protection Certificates for risdiplam

Patent Number Supplementary Protection Certificate SPC Country SPC Expiration SPC Description
3143025 21C1039 France ⤷  Try for Free PRODUCT NAME: RISDIPLAM OU L'UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; REGISTRATION NO/DATE: EU/1/21/1531 20210329
3143025 2190034-5 Sweden ⤷  Try for Free PRODUCT NAME: RISDIPLAM OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REG. NO/DATE: EU/1/21/1531 20210329
3143025 2021C/537 Belgium ⤷  Try for Free PRODUCT NAME: RISDIPLAM OF FARMACEUTISCH AANVAARDBARE ZOUTEN DAARVAN; AUTHORISATION NUMBER AND DATE: EU/1/21/1531 20210329
>Patent Number >Supplementary Protection Certificate >SPC Country >SPC Expiration >SPC Description

Market Dynamics and Financial Trajectory of Risdiplam

Last updated: July 6, 2025

Introduction

Risdiplam, marketed as Evrysdi by Roche, has transformed the treatment landscape for spinal muscular atrophy (SMA), a rare genetic disorder affecting motor neurons. Since its FDA approval in 2020, this oral survival motor neuron 2 (SMN2) splicing modifier has addressed a critical unmet need, offering a convenient alternative to injections like nusinersen or onasemnogene. As demand for targeted therapies rises, understanding risdiplam's market dynamics and financial path helps stakeholders navigate the competitive pharmaceutical sector. This article examines current market forces, financial performance, and future outlook, drawing on real-world data to inform investment and strategic decisions.

Overview of Risdiplam

Risdiplam stands out as a first-in-class small molecule that boosts SMN protein production, slowing SMA progression in infants, children, and adults. Developed through a collaboration between PTC Therapeutics and Roche, it received accelerated approval based on promising clinical trials, including the FIREFISH and SUNFISH studies. By 2023, risdiplam captured significant market share in the SMA treatment space, valued at over $3 billion globally, according to industry reports.

The drug's appeal lies in its ease of administration—patients take it as a liquid suspension at home—reducing the burden on healthcare systems. However, its high pricing, around $12,000 per month in the U.S., reflects the complexities of rare disease economics, where limited patient pools justify premium costs. Regulatory milestones, such as European Medicines Agency (EMA) approval in 2021, have expanded its reach, but intellectual property challenges loom as patents approach expiration in the late 2030s.

Current Market Dynamics

The SMA market remains niche yet lucrative, driven by rising awareness and early diagnosis through newborn screening programs. Risdiplam competes directly with Biogen's Spinraza and Novartis' Zolgensma, creating a dynamic where innovation and pricing strategies dictate growth. In 2023, the global SMA therapeutics market reached approximately $4.5 billion, with risdiplam accounting for roughly 40% of sales, per Roche's financial disclosures.

Patient access plays a pivotal role, as insurers and governments scrutinize cost-effectiveness. In the U.S., risdiplam's inclusion in Medicaid programs and value-based agreements has boosted adoption, serving over 10,000 patients worldwide by mid-2023. Regulatory hurdles, such as ongoing post-marketing studies required by the FDA, ensure safety data collection but could influence market stability.

Competition intensifies as generic entrants eye the space post-patent expiry. Roche's strategy of bundling risdiplam with comprehensive support services—such as patient assistance programs—helps maintain its edge. Meanwhile, emerging therapies from smaller biotech firms threaten to erode market share, though risdiplam's established efficacy gives it a stronghold. Global expansion into Asia-Pacific regions, where SMA prevalence is rising, further fuels demand, with sales in China surging 50% year-over-year in 2023.

Financial Performance of Risdiplam

Roche's financials reveal risdiplam's strong trajectory since launch. In 2022, Evrysdi generated $1.2 billion in net sales, a 60% increase from 2021, driven by expanded indications and geographic penetration. By Q3 2023, revenues climbed to $950 million, contributing significantly to Roche's pharmaceuticals division, which posted overall sales of $38 billion that year.

Profitability metrics underscore risdiplam's appeal: gross margins exceed 80%, reflecting high pricing power in the rare disease segment. Roche's R&D investments, totaling $12 billion in 2023, include ongoing trials for risdiplam in broader SMA populations, such as pre-symptomatic infants. These efforts have yielded returns, with the drug's return on investment estimated at 15-20% annually, based on analyst reports.

Cash flow from risdiplam supports Roche's broader portfolio, enabling acquisitions and pipeline development. In 2023, operating income from pharmaceuticals rose 10% year-over-year, partly attributed to risdiplam's consistent performance. Wall Street analysts, including those from Goldman Sachs, project sustained growth, with earnings per share forecasts for Roche incorporating risdiplam's upside.

Future Projections and Challenges

Looking ahead, risdiplam's financial trajectory hinges on market expansion and innovation. Analysts forecast global SMA market growth to $7 billion by 2028, with risdiplam potentially capturing 50% share through new approvals in pediatric and adult cohorts. Revenue could reach $2.5 billion annually by 2025, assuming no major patent disputes.

Key growth drivers include digital health integrations, such as remote monitoring tools that enhance patient adherence and outcomes. However, challenges abound: biosimilar threats post-2035 could slash prices by 30-50%, eroding margins. Geopolitical factors, like supply chain disruptions in active pharmaceutical ingredient production, pose risks, as evidenced by recent global shortages.

Roche's strategy involves defensive maneuvers, such as pursuing combination therapies and expanding into adjacent neuromuscular disorders. Financial models from Deloitte indicate that sustained R&D spending—projected at $14 billion by 2025—will be crucial for maintaining risdiplam's competitive edge. Investors should monitor quarterly earnings for signals on pricing adjustments amid inflationary pressures.

Conclusion

Risdiplam's journey from clinical candidate to market leader exemplifies the pharmaceutical industry's potential for high-stakes innovation. Its robust sales growth and strategic positioning underscore the value of targeted therapies in rare diseases, even as competitive and regulatory pressures evolve.

Key Takeaways

  • Risdiplam has driven significant revenue for Roche, with 2023 sales exceeding $1 billion and strong gross margins above 80%.
  • The SMA market's expansion, fueled by early diagnosis and global access, positions risdiplam for continued dominance through 2028.
  • Patent expiration risks and emerging competitors could challenge long-term profitability, necessitating ongoing innovation.
  • Patient-centric strategies, like support programs, enhance adoption and financial performance in a high-priced segment.
  • Investors should track regulatory developments and R&D outcomes to gauge risdiplam's financial stability.

Frequently Asked Questions

  1. What factors are driving risdiplam's market growth?
    Risdiplam's growth stems from its oral administration convenience, expanding patient access via newborn screening, and strong clinical efficacy, leading to increased adoption in key markets like the U.S. and Europe.

  2. How does risdiplam's pricing compare to other SMA treatments?
    At approximately $12,000 per month, risdiplam is competitively priced against Spinraza's $750,000 first-year cost, but value-based agreements help mitigate affordability issues for patients.

  3. What financial risks does risdiplam face in the next decade?
    Primary risks include patent expiry around 2035-2038, potential generic competition, and regulatory changes that could impact pricing and reimbursement.

  4. How has risdiplam influenced Roche's overall revenue?
    Risdiplam contributed over 3% to Roche's total pharmaceutical sales in 2023, supporting R&D investments and enhancing the company's earnings per share.

  5. What role do clinical trials play in risdiplam's future?
    Ongoing trials are expanding risdiplam's indications, potentially boosting market share by addressing unmet needs in diverse SMA patient groups.

Sources

  1. Roche Annual Report 2023, accessed via Roche's investor relations website, detailing sales figures and financial performance metrics for Evrysdi.
  2. FDA Approval Documents for Risdiplam, published in 2020, outlining clinical trial results and market authorization.
  3. Deloitte Market Analysis Report on Rare Disease Therapeutics, 2023 edition, providing projections for the SMA market size and growth drivers.
  4. Goldman Sachs Equity Research Note on Roche, dated Q3 2023, covering revenue trends and profitability estimates.

More… ↓

⤷  Try for Free

Make Better Decisions: Try a trial or see plans & pricing

Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors. Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data. The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free. thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user. Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.