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 one NDA. There are five patents protecting this compound. Additional information is available in the individual branded drug profile pages.

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

One supplier is listed for this compound.

Summary for risdiplam

International Patents:	130
US Patents:	5
Tradenames:	1
Applicants:	1
NDAs:	1
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

^*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	11,938,136		Y
Genentech Inc	EVRYSDI	risdiplam	FOR SOLUTION;ORAL	213535-001	Aug 7, 2020		RX	Yes	Yes	9,586,955		Y	Y
Genentech Inc	EVRYSDI	risdiplam	FOR SOLUTION;ORAL	213535-001	Aug 7, 2020		RX	Yes	Yes	11,827,646
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
Australia	2015261046	Compounds for treating spinal muscular atrophy
South Korea	102341596
Argentina	092794	COMPUESTOS PARA TRATAR LA ATROFIA MUSCULAR ESPINAL
Slovenia	3143025
Philippines	12016502081	COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY
Mexico	2022011699	COMPUESTOS PARA TRATAR LA ATROFIA MUSCULAR ESPINAL. (COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY.)

Supplementary Protection Certificates for risdiplam

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3143025	CA 2021 00037	Denmark		PRODUCT NAME: RISDIPLAM ELLER FARMACEUTISK ACCEPTABLE SALTE HERAF; REG. NO/DATE: EU/1/21/1531 20210329
3143025	SPC/GB21/050	United Kingdom		PRODUCT NAME: RISDIPLAM OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTERED: UK EU/1/21/1531(FOR NI) 20210329; UK FURTHER MA ON IPSUM 20210329
3143025	122021000051	Germany		PRODUCT NAME: RISDIPLAM ODER EIN PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1531 20210326
3143025	301128	Netherlands		PRODUCT NAME: RISDIPLAM OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/21/1531 20210329
3143025	CR 2021 00037	Denmark		PRODUCT NAME: RISDIPLAM ELLER FARMACEUTISK ACCEPTABLE SALTE HERAF; REG. NO/DATE: EU/1/21/1531 20210329
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

Risdiplam Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Risdiplam

Introduction

Risdiplam, a groundbreaking treatment for spinal muscular atrophy (SMA), has been making significant waves in the pharmaceutical industry since its approval. Developed by Genentech/Roche, this drug has transformed the treatment landscape for SMA patients. Here, we delve into the market dynamics and financial trajectory of risdiplam.

Approval and Regulatory Landscape

Risdiplam was approved by the U.S. Food and Drug Administration (FDA) on August 7, 2020, for the treatment of SMA in patients two months or older. This approval marked a crucial milestone, as it provided a new treatment option for a disease with limited therapeutic choices[3][5].

Market Size and Growth

The global SMA treatment market has been experiencing rapid growth, driven by increasing R&D initiatives and the introduction of new treatment options. In 2018, the global market size was USD 1.72 billion, and it is projected to reach USD 31.89 billion by 2032, exhibiting a Compound Annual Growth Rate (CAGR) of 28.9% during the forecast period (2019-2026)[4].

Competitive Landscape

Risdiplam enters a market dominated by other SMA treatments such as Biogen’s Spinraza (nusinersen) and Novartis’s Zolgensma (onasemnogene abeparvovec). However, risdiplam’s ease of administration and potential for cost-effectiveness make it a significant competitor. The market is characterized by strong R&D initiatives, with major players like Biogen and Novartis AG driving innovation[4].

Pricing and Cost-Effectiveness

The pricing of risdiplam is a critical factor in its market dynamics. Currently, the drug is expensive, with prices ranging from $117 to $232 per mg in high-income countries, translating to annual costs of $213,525 to $423,400 for a 5mg daily dose. However, the potential for generic versions could significantly reduce costs. If generic suppliers enter the market, prices are expected to fall dramatically, making the drug more affordable[1].

Access Programs and Affordability

Roche has implemented access programs in lower-income countries to make risdiplam more affordable. For example, in India, the program limits the number of bottles a patient pays for each year, although the price remains high relative to average incomes. These programs aim to increase access but highlight the ongoing challenge of affordability[1].

Cost-Effectiveness Analyses

National cost-effectiveness analyses have shown mixed results. While some analyses suggest that risdiplam could contribute to cost savings, others, such as those by the Dutch Zorginstituut, NICE, and NCPE, have concluded that the drug’s current price is too high to justify reimbursement due to uncertainties about long-term effects and cost estimates[1].

Safety and Adverse Events

Pharmacovigilance analyses are crucial for evaluating the safety of risdiplam. Studies using real-world data from the FDA Adverse Event Reporting System (FAERS) have identified various adverse events, including serious and non-serious AEs across 18 organ systems. Common AEs include fever, diarrhea, skin rash, and upper respiratory tract infections. New AE signals such as cardiac arrest, nephrolithiasis, and elevated hepatic enzyme activities have also been detected, emphasizing the need for long-term clinical studies to confirm these findings[3].

Patient Demographics and Reporting Trends

The FAERS database analysis shows that the majority of reported AEs involved individuals under 45 years old, with females accounting for 47.54% of the cases. The USA reported the highest number of AEs, followed by other countries like the UK, Netherlands, India, and Germany. The annual number of reports has shown an increasing trend from 2020 to 2023[3].

Financial Impact and Budget Considerations

The financial impact of risdiplam on healthcare systems is significant. For instance, the estimated annual per-patient drug cost in some regions is around €264,371. The gross budget impact for risdiplam over five years is estimated to be €107 million, which is lower than that for nusinersen but still substantial[1].

Generic Version Potential

The potential for a generic version of risdiplam is a game-changer in terms of affordability. Although the generic price per API is expected to be higher than the median price for drugs on the WHO Essential Medicines List, it would still be significantly lower than the current branded price. This could make the drug widely available without supplementary insurance or other third-party reimbursements[1].

Market Projections and Future Outlook

Given the strong R&D initiatives and increasing reimbursement access, the market for SMA treatments is poised for continued growth. Risdiplam’s unique position, with its ease of administration and potential for cost-effectiveness, places it as a key player in this expanding market. As generic versions become available, the drug is likely to become more accessible globally, further driving market growth[4].

Key Takeaways

• Approval and Market Entry: Risdiplam was approved by the FDA in 2020, marking a significant milestone in SMA treatment.
• Market Size and Growth: The global SMA treatment market is projected to reach USD 31.89 billion by 2032, with a CAGR of 28.9%.
• Pricing and Affordability: Current prices are high, but potential generic versions could significantly reduce costs.
• Safety and Adverse Events: Pharmacovigilance analyses have identified various AEs, emphasizing the need for long-term studies.
• Financial Impact: The drug has a substantial financial impact on healthcare systems, but generic versions could mitigate this.

FAQs

What is risdiplam used for?

Risdiplam is used for the treatment of spinal muscular atrophy (SMA) in patients two months or older.

How much does risdiplam cost?

The current cost of risdiplam ranges from $117 to $232 per mg, translating to annual costs of $213,525 to $423,400 for a 5mg daily dose.

What are the common adverse events associated with risdiplam?

Common adverse events include fever, diarrhea, skin rash, and upper respiratory tract infections. New signals include cardiac arrest, nephrolithiasis, and elevated hepatic enzyme activities.

Is risdiplam cost-effective?

Current cost-effectiveness analyses are mixed, with some suggesting potential cost savings but others concluding the drug is too expensive for reimbursement due to uncertainties about long-term effects.

What is the potential impact of generic versions of risdiplam?

Generic versions could significantly reduce the cost of risdiplam, making it more affordable and widely available without supplementary insurance or other third-party reimbursements.

Sources

1. PROPOSAL FOR THE INCLUSION OF RISDIPLAM IN THE WHO EML - WHO.
2. Q2 2023 Results - Investor presentation - Novartis.
3. Exploration of adverse events associated with risdiplam use - PLOS ONE.
4. Spinal Muscular Atrophy Treatment Market Size Report, 2032 - Fortune Business Insights.
5. FDA Approval of Risdiplam - FDA.