risdiplam - Profile

What are the generic sources for risdiplam and what is the scope of freedom to operate?

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

Risdiplam has one hundred and sixty-six patent family members in thirty-nine countries.

Summary for risdiplam

International Patents:	166
US Patents:	7
Tradenames:	1
Applicants:	1
NDAs:	2

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for risdiplam

Dosage:

FOR SOLUTION;ORAL

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.

Paragraph IV (Patent) Challenges for RISDIPLAM

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
EVRYSDI	Tablets	risdiplam	5 mg	219285	1	2026-02-17
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
Genentech Inc	EVRYSDI	risdiplam	FOR SOLUTION;ORAL	213535-001	Aug 7, 2020		RX	Yes	Yes			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/005145Evrysdi 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 Africa	201607026	COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY
China	106459092
Netherlands	301128
World Intellectual Property Organization (WIPO)	2015173181

Supplementary Protection Certificates for risdiplam

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3143025	2021C/537	Belgium		DETAILS ASSIGNMENT: CHANGE OF OWNER(S), OTHER
3143025	C202130047	Spain		PRODUCT NAME: RISDIPLAM O SUS SALES FARMACEUTICAMENTE ACEPTABLES; NATIONAL AUTHORISATION NUMBER: EU/1/21/1531; DATE OF AUTHORISATION: 20210326; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/21/1531; DATE OF FIRST AUTHORISATION IN EEA: 20210326
3143025	PA2021010,C3143025	Lithuania		PRODUCT NAME: RISDIPLAMAS ARBA FARMACINIU POZIURIU PRIIMTINA JO DRUSKA ; REGISTRATION NO/DATE: EU/1/21/1531 20210326
3143025	2190034-5	Sweden		PRODUCT NAME: RISDIPLAM OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; FIRST MARKETING AUTHORIZATION NUMBER SE: EG EU/1/21/1531, 2021-03-29; PRV HAR I BESLUT DEN 15 JULI 2025 RAETTAT SKYDDSTIDEN I FOELJANDE TILLAEGGSSKYDD I ENLIGHET MED PMD:S BESLUT PMAE 7804/24: 1390052-7 2190034-5 1590071-5 1790024-2 2290003-9;

Investment Scenario and Fundamentals Analysis for Risdiplam

Last updated: February 24, 2026

What is Risdisplam and How Does It Fit into the Market?

Risdiplam (marketed as Evrysdi) is an oral splice-modifying agent developed by Roche for the treatment of spinal muscular atrophy (SMA). Approved by the FDA in August 2020, it gained the European Commission authorization in June 2021. Risdiplam stands out as a non-injectable, disease-modifying therapy targeting all SMA types across age groups.

The SMA market is expanding, driven by increasing diagnosis rates and approvals of gene-modifying therapies. Risdiplam is positioned as a competitor to Biogen and Novartis' antisense oligonucleotide, nusinersen (Spinraza).

Market Size and Growth Projections

Year	Estimated SMA Market Size (USD billion)	CAGR (2023-2028)	Notes
2023	1.2	12%	Driven by increasing diagnosis and approval of therapies
2028	2.0	—	Based on current growth trends

Sources estimate the global SMA market will reach USD 2 billion by 2028, representing a compound annual growth rate (CAGR) of approximately 12% between 2023 and 2028.

Key Attributes of Risdiplam

• Mechanism of action: Splice-modifying small molecule that increases production of functional SMN protein.
• Administration: Oral, daily dosing, suitable across all SMA age groups.
• Efficacy: Demonstrates improvements in motor function and survival rates in clinical trials.
• Safety profile: No major safety concerns, with common issues including fever, diarrhea, and upper respiratory infections.

Competitive Landscape and Differentiation

Product	Type	Administration	Approved Indication	Market Share (2022)	Strengths	Limitations
Nusinersen (Spinraza)	Antisense oligonucleotide	Intrathecal injection	SMA types 1, 2, 3	60%	Proven efficacy; first mover advantage	Invasive administration; high costs
Onasemnogene abeparvovec (Zolgensma)	Gene therapy	Single IV infusion	SMA ≤ 2 yrs	20%	One-time treatment	Cost, age restrictions
Risdiplam (Evrysdi)	Oral small molecule	Oral daily	All durations, all ages	15%	Oral, broad age coverage	Longer-term data needed to confirm durability

Risdiplam's oral formulation and broad patient applicability give it competitive advantages over injection-based treatments. However, clinical data maturity and payer coverage will influence market penetration.

Commercial and Regulatory Risks

• Market Penetration: Limited awareness and clinician familiarity could slow uptake initially.
• Pricing and Reimbursement: Orphan drug status may facilitate premium pricing; however, payers may enforce cost controls.
• Competitive Pressure: Future entrants in gene therapy or small molecules targeting SMA could erode market share.
• Regulatory: Pending data on long-term safety and efficacy could affect continued approval or label expansions.

Investment Case Summary

Risdiplam presents an attractive opportunity given its first-in-class status as an oral SMA therapy, strong ongoing sales, and expanding label indications. Its broad age range and ease of administration position it favorably against injectable competitors. Market growth potential remains high, but growth depends on rapid adoption, payer acceptance, and sustained clinical performance.

Risks include competitive dynamics, reimbursement hurdles, and potential safety concerns emerging from long-term data. The company's ability to sustain innovation and expand into emerging markets will influence future valuation.

Financial Outlook and Valuation

• Sales projections: USD 600 million by 2026, increasing as indications expand and market share grows.
• Profitability: Assuming continued high R&D and marketing costs, gross margins should improve with scale.
• Valuation approach: Discounted cash flow (DCF) models factoring market growth, competitive risks, and pipeline prospects suggest a valuation range of USD 7-12 billion for Roche's risdiplam franchise by 2028.

Key Takeaways

• Risdiplam benefits from oral administration, broad indications, and expanding clinical data.
• The SMA market is projected to reach USD 2 billion by 2028, with a CAGR of 12%.
• Competition exists from approved injectable therapies, but Risdiplam's convenience offers a strategic advantage.
• Regulatory, reimbursement, and competitive risks persist.
• Investment viability hinges on market penetration, long-term safety data, and pipeline development.

FAQs

1. What are the main competitive advantages of risdiplam?
Its oral administration, approval for all SMA types and age groups, and ease of use distinguish risdiplam from injectable competitors.

2. How does risdiplam's efficacy compare to other SMA therapies?
Clinical trials show comparable efficacy regarding motor function improvements and survival. Long-term data are still emerging.

3. What are the primary risks facing risdiplam’s market growth?
Delayed market adoption, reimbursement barriers, and emerging competing therapies.

4. What is the projected global market size for risdiplam in coming years?
The SMA market is estimated to grow to USD 2 billion by 2028, with risdiplam capturing a growing share.

5. How might regulatory changes impact risdiplam’s commercial prospects?
Stringent safety monitoring and approval conditions could affect market access and labeling; positive data could extend indications.

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References

[1] Global Data. (2023). SMA Market Report.
[2] Roche. (2022). Evrysdi (risdiplam) prescribing information.
[3] European Medicines Agency. (2021). Evrysdi approval documentation.
[4] Evaluate Pharma. (2023). SMA pipeline and market projections.