EVRYSDI Drug Patent Profile

When do Evrysdi patents expire, and what generic alternatives are available?

Evrysdi is a drug marketed by Genentech Inc and is included in one NDA. There are six patents protecting this drug and one Paragraph IV challenge.

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

The generic ingredient in EVRYSDI is risdiplam. One supplier is listed for this compound. Additional details are available on the risdiplam profile page.

DrugPatentWatch^® Generic Entry Outlook for Evrysdi

Evrysdi was eligible for patent challenges on August 7, 2024.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be May 11, 2035. This may change due to patent challenges or generic licensing.

There is one Paragraph IV patent challenge for this drug. This may lead to patent invalidation or a license for generic production.

Indicators of Generic Entry

Summary for EVRYSDI

International Patents:	153
US Patents:	6
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	36
Clinical Trials:	3
Patent Applications:	41
Drug Prices:	Drug price information for EVRYSDI
What excipients (inactive ingredients) are in EVRYSDI?	EVRYSDI excipients list
DailyMed Link:	EVRYSDI at DailyMed

See drug prices for EVRYSDI

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

NDA:

213535

Dosage:

FOR SOLUTION;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 EVRYSDI

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

Sponsor	Phase
Hoffmann-La Roche	Phase 4
Genentech, Inc.	Phase 4
Hoffmann-La Roche	Phase 1

See all EVRYSDI clinical trials

Pharmacology for EVRYSDI

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

Paragraph IV (Patent) Challenges for EVRYSDI

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 EVRYSDI

EVRYSDI is protected by six US patents and five FDA Regulatory Exclusivities.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

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			Y	Y

EU/EMA Drug Approvals for EVRYSDI

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 EVRYSDI

When does loss-of-exclusivity occur for EVRYSDI?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Argentina

Australia

Brazil

Canada

Chile

China

Costa Rica

Croatia

Denmark

Eurasian Patent Organization

European Patent Office

France

Hungary

Israel

Japan

Lithuania

Malaysia

Mexico

Morocco

Netherlands

New Zealand

Norway

Peru

Philippines

Poland

Portugal

Serbia

Singapore

Slovenia

South Africa

South Korea

Spain

Taiwan

Ukraine

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering EVRYSDI around the world.

Country	Patent Number	Title	Estimated Expiration
European Patent Office	3143025
China	106459092
Eurasian Patent Organization	029542	ПРОИЗВОДНЫЕ 4Н-ПИРИДО[1,2-а]ПИРИМИДИН-4-ОНА ДЛЯ ЛЕЧЕНИЯ СПИНАЛЬНОЙ МЫШЕЧНОЙ АТРОФИИ (4H-PYRIDO[1,2-a]PYRIMIDIN-4-ONE DERIVATIVES FOR TREATING SPINAL MUSCULAR ATROPHY)

Supplementary Protection Certificates for EVRYSDI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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	CA 2021 00037	Denmark		PRODUCT NAME: RISDIPLAM ELLER FARMACEUTISK ACCEPTABLE SALTE HERAF; REG. NO/DATE: EU/1/21/1531 20210329
3143025	122021000051	Germany		PRODUCT NAME: RISDIPLAM ODER EIN PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1531 20210326

Market Dynamics and Financial Trajectory for the Drug: Evrysdi

Introduction to Evrysdi

Evrysdi (risdiplam) is a groundbreaking oral small molecule approved by the U.S. FDA in 2020 for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Developed by PTC Therapeutics and marketed by Roche, Evrysdi has quickly become a dominant force in the SMA treatment market.

Mechanism and Clinical Impact

Evrysdi works as a survival motor neuron 2 (SMN2) splicing modifier, enhancing the production of the SMN protein, which is crucial for motor neuron function. Clinical trials have shown significant efficacy in maintaining and improving motor function in patients with SMA, making it a preferred treatment option for many[5].

Market Dominance

In a notable shift, Evrysdi has surpassed the sales of other SMA treatments, including the antisense oligonucleotide Spinraza and the gene therapy Zolgensma. This indicates that traditional small molecule therapies still hold significant market value despite the emergence of newer modalities. The collective sales of these SMA drugs reached a new high of $1.3 billion in the second quarter of 2024, exceeding the $1.15 billion total from the third quarter of 2023[1].

Sales Performance

Evrysdi's sales have been robust, with full-year 2023 sales reported at approximately CHF 1,419 million (around $1.4 billion), resulting in royalty revenue of $168.9 million for PTC Therapeutics. This represents a significant increase from the $113.5 million in royalty revenue for 2022. Additionally, PTC recorded a sales milestone of $100 million for achieving $1.5 billion in worldwide annual net sales from Evrysdi in the fourth quarter of 2023[3].

Royalty Monetization

In a strategic financial move, PTC Therapeutics sold up to $1.5 billion of its Evrysdi royalty stream to Royalty Pharma plc. This transaction included an upfront payment of $1.0 billion, with options for additional payments up to $500 million. This deal is expected to enhance PTC's financial stability, allowing the company to pay off debt and fuel its development pipeline[2][4].

Financial Projections

Royalty Pharma's acquisition of the Evrysdi royalty is projected to significantly contribute to its long-term growth. Evrysdi is expected to become one of the top four portfolio royalties for Royalty Pharma by 2025, with Adjusted Cash Receipts contributions exceeding $200 million based on consensus sales estimates. This transaction highlights the financial viability and market potential of Evrysdi[4].

Patient Impact and Clinical Efficacy

Clinical trials and real-world data have shown that Evrysdi effectively maintains and improves motor function in SMA patients. Patients like Angela, who lives with Type 2 SMA, have reported significant benefits in daily activities, such as brushing teeth and applying makeup, which are crucial for maintaining independence. Neurologists, like Dr. Shadé Moody, emphasize the importance of monitoring patient progress and managing potential side effects to ensure optimal treatment outcomes[5].

Market Competition

Despite the dominance of Evrysdi, the SMA market remains competitive with other treatments like Spinraza and Zolgensma. However, Evrysdi's oral administration and convenience have made it a preferred choice for many patients and healthcare providers. The market dynamics suggest that traditional small molecule therapies can still outperform newer modalities in terms of sales and patient preference[1].

Financial Growth and Diversification

The financial performance of Evrysdi has contributed significantly to the growth of both PTC Therapeutics and Royalty Pharma. For PTC, the royalty revenue from Evrysdi has been a key driver of its 34% year-over-year revenue growth in 2023. For Royalty Pharma, the acquisition of Evrysdi royalties is part of a broader strategy to enhance long-term growth and diversify its royalty portfolio[3][4].

Future Outlook

Given its strong sales performance and clinical efficacy, Evrysdi is poised to continue dominating the SMA market. The drug's ability to treat a wide range of SMA patients, from pediatric to adult, further solidifies its market position. As the SMA treatment landscape evolves, Evrysdi's convenience, efficacy, and financial performance make it a cornerstone in the therapeutic options available.

Key Takeaways

• Market Dominance: Evrysdi has surpassed the sales of other SMA treatments, including Spinraza and Zolgensma.
• Financial Performance: Full-year 2023 sales of Evrysdi reached approximately CHF 1,419 million, with significant royalty revenue for PTC Therapeutics.
• Royalty Monetization: PTC Therapeutics sold up to $1.5 billion of its Evrysdi royalty stream to Royalty Pharma plc.
• Clinical Efficacy: Evrysdi effectively maintains and improves motor function in SMA patients.
• Future Outlook: Evrysdi is expected to continue dominating the SMA market due to its convenience, efficacy, and strong financial performance.

FAQs

Q: What is Evrysdi and how does it work?

A: Evrysdi (risdiplam) is an oral small molecule that works as a survival motor neuron 2 (SMN2) splicing modifier to enhance the production of the SMN protein, crucial for motor neuron function.

Q: How has Evrysdi performed in the market?

A: Evrysdi has surpassed the sales of other SMA treatments like Spinraza and Zolgensma, with collective SMA drug sales reaching a new high of $1.3 billion in the second quarter of 2024.

Q: What was the financial impact of the royalty sale to Royalty Pharma?

A: The sale of up to $1.5 billion of Evrysdi's royalty stream to Royalty Pharma provided significant upfront capital to PTC Therapeutics, helping to pay off debt and fund its development pipeline.

Q: How does Evrysdi compare to other SMA treatments?

A: Evrysdi's oral administration and convenience have made it a preferred choice over other treatments like Spinraza and Zolgensma, despite the latter being newer modalities.

Q: What are the future prospects for Evrysdi in the SMA market?

A: Given its strong sales performance, clinical efficacy, and financial projections, Evrysdi is expected to continue dominating the SMA market, treating a wide range of patients from pediatric to adult.

Sources:

1. Biocentury: "Small molecule Evrysdi overtakes new modalities to dominate SMA market"
2. BioWorld: "PTC sells Evrysdi royalties in deal potentially worth $1.5B"
3. PTC Therapeutics: "PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results"
4. Royalty Pharma: "Royalty Pharma Announces Agreement to Purchase up to $1.5 Billion of PTC Therapeutics’ Royalty on Evrysdi"
5. Evrysdi-HCP: "Later-Onset SMA Efficacy Results in Clinical Trials & Studies"