Nusinersen sodium - Generic Drug Details

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

Nusinersen sodium is the generic ingredient in one branded drug marketed by Biogen Idec and is included in one NDA. There are nine patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Nusinersen sodium has one hundred and two patent family members in twenty-seven countries.

One supplier is listed for this compound.

Summary for nusinersen sodium

International Patents:	102
US Patents:	9
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for nusinersen sodium
DailyMed Link:	nusinersen sodium at DailyMed

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

Dosage:

SOLUTION;INTRATHECAL

^*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.

Pharmacology for nusinersen sodium

Drug Class	Antisense Oligonucleotide Survival Motor Neuron-2-directed RNA Interaction
Physiological Effect	Increased Protein Synthesis

Anatomical Therapeutic Chemical (ATC) Classes for nusinersen sodium

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

US Patents and Regulatory Information for nusinersen sodium

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes				Y
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes			Y	Y

Expired US Patents for nusinersen sodium

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016

International Patents for nusinersen sodium

Country	Patent Number	Title	Estimated Expiration
Japan	2022025131	被験体におけるＳＭＮ２スプライシングのモジュレーションのための組成物および方法
Cyprus	1120807
Chile	2017000594	Composiciones y métodos para detección de proteína smn en un sujeto y tratamiento de un sujeto
Denmark	2548560
Russian Federation	2566724	КОМПОЗИЦИИ И СПОСОБЫ МОДУЛЯЦИИ SMN2 СПЛАЙСИНГА У СУБЪЕКТА (COMPOSITIONS AND METHODS OF MODULATING SMN2 SPLICING IN SUBJECT)
Spain	2761614
Australia	2016200344	COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT

Supplementary Protection Certificates for nusinersen sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1910395	122017000099	Germany		PRODUCT NAME: NUSINERSEN ODER SALZE DAVON (INTERNATIONALER, NICHT FIRMENEIGENER NAME); REGISTRATION NO/DATE: EU/1/17/1188 20170530
2548560	C201730053	Spain		PRODUCT NAME: NUSINERSEN O SUS SALES; NATIONAL AUTHORISATION NUMBER: EU/1/17/1188; DATE OF AUTHORISATION: 20170530; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/17/1188; DATE OF FIRST AUTHORISATION IN EEA: 20170530
1910395	2017C/047	Belgium		PRODUCT NAME: SPINRAZA - NUSINERSEN; AUTHORISATION NUMBER AND DATE: EU/1/17/1188 20170601
2548560	132017000128699	Italy		PRODUCT NAME: NUSINERSEN O SUOI SALI(SPINRAZA); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/17/1188, 20170601
2548560	300902	Netherlands		PRODUCT NAME: NUSINERSEN, DESGEWENST IN DE VORM VAN EEN ZOUT; REGISTRATION NO/DATE: EU/1/17/1188 20170601
1910395	2017/051	Ireland		PRODUCT NAME: NUSINERSEN OR SALTS THEREOF; REGISTRATION NO/DATE: EU/1/17/1188 20170530
2548560	CA 2017 00055	Denmark		PRODUCT NAME: NUSINERSEN ELLER SALTE DERAF; REG. NO/DATE: EU/1/17/1188 20170601

Market Dynamics and Financial Trajectory for Nusinersen Sodium (Spinraza)

Introduction

Nusinersen sodium, marketed as Spinraza, is a groundbreaking medication for the treatment of spinal muscular atrophy (SMA), a rare and debilitating genetic disorder. Since its approval in December 2016, the drug has been at the center of significant discussions regarding its efficacy, safety, and particularly, its cost.

Approval and Regulatory Landscape

Nusinersen was developed through a collaboration between Adrian Krainer at Cold Spring Harbor Laboratory, Ionis Pharmaceuticals, and Biogen. It was approved by the FDA in December 2016 and subsequently by the European Medicines Agency (EMA) in May 2017. The drug has also been approved in several other countries, including Canada, Japan, Brazil, Switzerland, and China[4].

Clinical Efficacy and Patient Outcomes

Clinical trials have consistently shown that nusinersen improves motor function and survival rates in patients with SMA, especially when treatment is initiated early. Patients have reported improvements in respiratory function, endurance, upper limb and core strength, and voice. However, some patients have experienced temporary side effects such as headaches and discomfort from intrathecal injections[5].

Cost and Economic Impact

The cost of nusinersen is one of its most contentious aspects. The list price in the USA is $125,000 per injection, resulting in a total cost of $750,000 in the first year and $375,000 annually thereafter[4].

Comparative Costs

When compared to other treatments like risdiplam, nusinersen is significantly more expensive. For instance, to be considered cost-effective at a $50,000 per QALY (Quality-Adjusted Life Year) threshold, nusinersen would need a price reduction of over 99%[1].

Healthcare System Impact

The high cost of nusinersen has led to varied funding decisions across different countries. In Denmark and Norway, the drug was initially rejected for funding due to its "unreasonably high price" but later approved for specific patient groups. In Ireland, the Health Service Executive decided not to fund the drug due to its high cost, although pricing arrangements were later negotiated through the BeneluxA initiative[4].

Price Adjustments

In some regions, efforts have been made to reduce the cost. For example, in China, the price was reduced from ¥697,000 per vial to around ¥33,000 (~US$5,100) per vial in December 2021, making the treatment more accessible[4].

Market Performance and Sales

Despite the high cost, nusinersen has seen strong market performance. The drug's sales have contributed significantly to the revenue of Biogen, the company responsible for its development and marketing. The financial reports from Biogen and other pharmaceutical companies indicate a steady growth trajectory for nusinersen, driven by its unique position as the first approved treatment for SMA[3].

Global Availability

As of May 2019, nusinersen was available in public healthcare systems in over 40 countries, reflecting its widespread adoption despite cost concerns[4].

Patient and Clinician Perspectives

Patients and clinicians have generally reported positive outcomes from nusinersen treatment, despite the challenges associated with its administration and cost. Many patients have switched to alternative treatments like risdiplam due to financial constraints and difficulties with intrathecal administration, but overall, the benefits of nusinersen are seen to outweigh its drawbacks[5].

Economic Evaluation and Cost-Effectiveness

Economic evaluations have highlighted the significant incremental costs associated with nusinersen compared to standard care. For instance, the incremental cost-effectiveness ratio (ICER) for nusinersen versus standard care was found to be $3,568,727 per QALY gained, indicating a high cost per unit of health benefit[1].

Future Outlook

The future of nusinersen is marked by ongoing clinical trials and efforts to improve its accessibility. Additional trials continue to validate its efficacy, particularly emphasizing the benefits of early intervention. Regulatory and pricing negotiations are also ongoing to make the drug more affordable in various markets[4].

Key Takeaways

• High Efficacy: Nusinersen has shown significant improvements in motor function and survival rates for SMA patients.
• High Cost: The drug is among the most expensive in the world, with a first-year cost of $750,000 and annual costs of $375,000 thereafter.
• Variable Funding: Different countries have made varied decisions on funding nusinersen due to its high cost.
• Strong Market Performance: Despite cost concerns, nusinersen has seen strong sales and revenue growth.
• Ongoing Evaluations: Continuous clinical trials and economic evaluations are being conducted to assess its long-term efficacy and cost-effectiveness.

FAQs

Q: What is the primary use of nusinersen sodium (Spinraza)?

A: Nusinersen sodium is used to treat spinal muscular atrophy (SMA), a rare genetic disorder that affects muscle strength and movement.

Q: How much does nusinersen sodium cost?

A: The list price of nusinersen in the USA is $125,000 per injection, resulting in a total cost of $750,000 in the first year and $375,000 annually thereafter.

Q: Why is nusinersen so expensive?

A: The high cost is due to several factors, including the complexity of its production, the rarity of the condition it treats, and the lack of comparative treatments.

Q: Is nusinersen available in public healthcare systems globally?

A: Yes, as of May 2019, nusinersen was available in public healthcare systems in over 40 countries, although funding decisions vary by country.

Q: What are the common side effects of nusinersen treatment?

A: Common side effects include temporary headaches, discomfort from intrathecal injections, and a wearing-off of treatment effects shortly before the next maintenance dose.

References

1. Pharmacoeconomic Review - Nusinersen (Spinraza) - NCBI Bookshelf
2. Concerns About the Approval of Nusinersen Sodium by the US Food and Drug Administration - JAMA Internal Medicine
3. Novartis Q2 2024 Investor Presentation
4. Nusinersen - Wikipedia
5. CADTH Reimbursement Review: Nusinersen (Spinraza)