Givosiran sodium - Generic Drug Details

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

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

Givosiran sodium has two hundred and seventy-three patent family members in forty-five countries.

One supplier is listed for this compound.

Summary for givosiran sodium

International Patents:	273
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 givosiran sodium
What excipients (inactive ingredients) are in givosiran sodium?	givosiran sodium excipients list
DailyMed Link:	givosiran sodium at DailyMed

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

Dosage:

SOLUTION;SUBCUTANEOUS

^*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 givosiran sodium

Drug Class	Aminolevulinate Synthase 1-directed RNA Interaction Small Interfering RNA
Physiological Effect	Decreased RNA Integrity

Anatomical Therapeutic Chemical (ATC) Classes for givosiran sodium

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

US Patents and Regulatory Information for givosiran sodium

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y

Expired US Patents for givosiran sodium

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019

International Patents for givosiran sodium

Country	Patent Number	Title	Estimated Expiration
European Patent Office	4074344	LIPIDES DE CIBLAGE (TARGETING LIPIDS)
European Patent Office	2836595	COMPOSITIONS ET PROCÉDÉS PERMETTANT D'INHIBER L'EXPRESSION DU GÈNE ALAS1 (COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE)
Montenegro	02850	SASTAVI I METODE ZA INHIBIRANJE EKSPRESIJE ALAS1 GENA (COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE)
Japan	5519523
Poland	3052628
Australia	2003248708	OLIGOMERIC COMPOUNDS THAT INCLUDE CARBOCYCLIC NUCLEOSIDES AND THEIR USE IN GENE MODULATION

Supplementary Protection Certificates for givosiran sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3052628	132020000000106	Italy		PRODUCT NAME: GIVOSIRAN O UN SUO SALE FARMACEUTICAMENTE ACCETTABILE(GIVLAARI); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/20/1428, 20200304
3052628	37/2020	Austria		PRODUCT NAME: GIVOSIRAN ODER EIN PHARMAZEUTISCH ANNEHMBARES SALZ DAVON; REGISTRATION NO/DATE: EU/1/20/1428 (MITTEILUNG) 20200304
3052628	122020000045	Germany		PRODUCT NAME: GIVOSIRAN ODER EIN PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/20/1428 20200302
3052628	2090037-9	Sweden		PRODUCT NAME: GIVOSIRAN OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REG. NO/DATE: EU/1/20/1428 20200304
3052628	PA2020527	Lithuania		PRODUCT NAME: GIVOSIRANAS ARBA JO FARMACINIU POZIURIU PRIIMTINA DRUSKA; REGISTRATION NO/DATE: EU/1/20/1428 20200302
3052628	301061	Netherlands		PRODUCT NAME: GIVOSIRAN OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/20/1428 20200304

Market Dynamics and Financial Trajectory for Givosiran Sodium (Givlaari)

Introduction to Givosiran Sodium (Givlaari)

Givosiran sodium, marketed as Givlaari, is a revolutionary drug developed by Alnylam Pharmaceuticals for the treatment of acute hepatic porphyria (AHP) in adults. Approved by Health Canada in 2020 and the FDA in 2019, Givlaari has marked a significant milestone in the management of this rare and debilitating genetic disorder[1][4].

Mechanism of Action and Clinical Benefits

Givosiran works by inhibiting the production of aminolevulinic acid (ALA) and porphobilinogen (PBG), key precursors in the heme biosynthesis pathway that are elevated in AHP patients. This mechanism reduces the frequency and severity of porphyria attacks, significantly improving the quality of life for patients. Clinical trials have shown that monthly dosing of 2.5 mg/kg givosiran reduces the annualized attack rate (AAR) to 2.9 attacks per year, compared to 12.5 attacks per year with placebo[1].

Market Authorization and Regulatory Approval

Health Canada granted Givlaari a Notice of Compliance in October 2020, following a thorough review of its quality, non-clinical, and clinical data. The approval was based on the drug's favourable benefit-risk profile, with appropriate warnings and precautions in place to manage identified safety concerns[1].

Financial Performance and Market Impact

Revenue and Sales

Givlaari is part of Alnylam Pharmaceuticals' robust pipeline of RNA interference (RNAi) therapies. The drug's commercial success contributes to Alnylam's overall financial performance. As of the latest financial reports, Alnylam is executing its "Alnylam P 5x25" strategy, aiming to deliver transformative medicines and achieve sustainable innovation and financial performance. This strategy includes expanding the market presence of drugs like Givlaari, which are critical to the company's revenue growth[2].

Cost-Effectiveness and Economic Impact

While Givlaari offers significant clinical benefits, its cost-effectiveness is a subject of ongoing analysis. A study published on PubMed compared the total cost of care for AHP patients treated with either hemin or givosiran. The results indicated that hemin treatment is associated with lower annual total costs of care, ranging from $311,950 to $545,219 less than givosiran, depending on the treatment regimen. This suggests that while Givlaari is clinically effective, it may face economic challenges in terms of cost savings compared to traditional treatments like hemin[5].

Investment Trends and Industry Dynamics

Biotechnology and Precision Medicine

The pharmaceutical industry is witnessing a significant shift towards biotechnology and precision medicine, with investors favoring companies that develop targeted therapies. Givlaari, as an RNAi therapy, aligns with this trend, attracting investment and interest due to its innovative approach to treating rare genetic disorders[3].

Digital Health and Advanced Manufacturing

The industry is also seeing a surge in investments in digital health technologies and advanced manufacturing processes. While Givlaari itself is not directly related to these trends, Alnylam's overall strategy and pipeline are influenced by these broader industry shifts. For instance, the company's focus on sustainable innovation and exceptional financial performance is supported by advancements in digital health and manufacturing technologies[3].

Competitive Landscape

Givlaari operates in a niche market targeting a rare disease, which limits direct competition. However, it competes indirectly with other treatments for AHP, such as hemin, in terms of cost-effectiveness and patient preference. The drug's unique mechanism of action and clinical efficacy make it a preferred choice for many patients and healthcare providers, despite the higher cost[1][5].

Safety and Monitoring

Givosiran treatment is associated with several safety considerations, including the potential for renal impairment and interactions with CYP450 enzymes. Health Canada and other regulatory bodies have mandated careful monitoring of renal function and appropriate labelling to address these safety concerns. Long-term data collection and risk management plans (RMPs) are also in place to better characterize the short- and long-term effects of Givlaari[1][4].

Future Outlook and Growth Potential

Pipeline Expansion and Strategic Partnerships

Alnylam's "Alnylam P 5x25" strategy includes expanding its pipeline and forming strategic partnerships to enhance market presence and diversify revenue streams. This strategy is expected to drive growth for Givlaari and other Alnylam products, as the company continues to innovate and deliver transformative medicines[2].

Market Expansion and Patient Access

Efforts to expand market access and improve patient outcomes are crucial for the long-term success of Givlaari. This includes working with healthcare systems, payers, and patient advocacy groups to ensure that the drug is accessible to those who need it, despite its higher cost compared to traditional treatments[3].

Key Takeaways

• Clinical Efficacy: Givosiran sodium (Givlaari) significantly reduces the frequency and severity of porphyria attacks in AHP patients.
• Regulatory Approval: Approved by Health Canada and the FDA, with a favourable benefit-risk profile.
• Financial Performance: Contributes to Alnylam Pharmaceuticals' revenue growth as part of the company's "Alnylam P 5x25" strategy.
• Cost-Effectiveness: Higher costs compared to traditional treatments like hemin, but offers significant clinical benefits.
• Industry Trends: Aligns with trends in biotechnology, precision medicine, and digital health.
• Safety and Monitoring: Requires careful monitoring of renal function and management of potential interactions with CYP450 enzymes.

FAQs

What is Givosiran Sodium (Givlaari) used for?

Givosiran sodium, marketed as Givlaari, is used for the treatment of acute hepatic porphyria (AHP) in adults.

How does Givosiran work?

Givosiran inhibits the production of aminolevulinic acid (ALA) and porphobilinogen (PBG), reducing the frequency and severity of porphyria attacks.

Is Givlaari cost-effective compared to other treatments?

Givlaari is associated with higher costs compared to traditional treatments like hemin, although it offers significant clinical benefits.

What are the potential safety concerns with Givlaari?

Givosiran treatment can lead to renal impairment and interactions with CYP450 enzymes, requiring careful monitoring and appropriate labelling.

What is Alnylam's strategy for Givlaari and other products?

Alnylam is executing its "Alnylam P 5x25" strategy, focusing on sustainable innovation, financial performance, and expanding market presence through strategic partnerships.

Sources

1. Health Canada: Summary Basis of Decision for Givlaari[1].
2. Business Wire: Alnylam to Webcast Conference Call Discussing Third Quarter 2024 Financial Results[2].
3. PharmaSalmanac: What are the Current Investment Trends in the Pharmaceutical Industry[3].
4. European Medicines Agency: Givlaari, INN-givosiran[4].
5. PubMed: Cost savings with hemin versus givosiran for the treatment of acute intermittent porphyria[5].

Last updated: 2024-12-18