Risdiplam - Generic Drug Details

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

Constraining patent/regulatory exclusivity:

9,969,754

Dosage:

FOR SOLUTION;ORAL

Constraining patent/regulatory exclusivity:

9,969,754

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

Sponsor	Phase
Hoffmann-La Roche	Phase 4
Clinic for Special Children	Phase 4
Biohaven Pharmaceuticals, Inc.	Phase 3

See all risdiplam clinical trials

Pharmacology for risdiplam

Drug Class	Survival of Motor Neuron 2 Splicing Modifier
Mechanism of Action	Multidrug and Toxin Extrusion Transporter 1 Inhibitors Multidrug and Toxin Extrusion Transporter 2 K Inhibitors Survival of Motor Neuron 2 Splicing Modifiers
Physiological Effect	Increased Protein Synthesis

Anatomical Therapeutic Chemical (ATC) Classes for risdiplam

M09AX	Other drugs for disorders of the musculo-skeletal system
M09A	OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M09	OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M	Musculo-skeletal system

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
Genentech Inc	EVRYSDI	risdiplam	FOR SOLUTION;ORAL	213535-001	Aug 7, 2020		RX	Yes	Yes
Genentech Inc	EVRYSDI	risdiplam	FOR SOLUTION;ORAL	213535-001	Aug 7, 2020		RX	Yes	Yes	9,969,754		Y	Y

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

International Patents for risdiplam

Country	Patent Number	Title	Estimated Expiration
South Korea	102137087
China	113039188	新型吡啶并[1,2-a]嘧啶-4-酮衍生物、其制剂及制备方法 (NEW FORMS OF PYRIDO[1,2-A]PYRIMIDIN-4-ONE DERIVATIVES, ITS FORMULATION AND ITS PROCESS OF MAKING)
South Korea	102213740

Supplementary Protection Certificates for risdiplam

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

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.