FABHALTA Drug Patent Profile

Which patents cover Fabhalta, and when can generic versions of Fabhalta launch?

Fabhalta is a drug marketed by Novartis and is included in one NDA. There are five patents protecting this drug.

This drug has ninety-three patent family members in forty-three countries.

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

DrugPatentWatch^® Generic Entry Outlook for Fabhalta

Fabhalta will be eligible for patent challenges on December 5, 2027. This date may extended up to six months if a pediatric exclusivity extension is applied to the drug's patents.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be December 5, 2030. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for FABHALTA

International Patents:	93
US Patents:	5
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Drug Prices:	Drug price information for FABHALTA
What excipients (inactive ingredients) are in FABHALTA?	FABHALTA excipients list
DailyMed Link:	FABHALTA at DailyMed

See drug prices for FABHALTA

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

Constraining patent/regulatory exclusivity:

TREATMENT OF ADULTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

NDA:

218276

Dosage:

CAPSULE;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.

US Patents and Regulatory Information for FABHALTA

FABHALTA is protected by five US patents and three FDA Regulatory Exclusivities.

This potential generic entry date is based on TREATMENT OF ADULTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH).

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
Novartis	FABHALTA	iptacopan hydrochloride	CAPSULE;ORAL	218276-001	Dec 5, 2023		RX	Yes	Yes
Novartis	FABHALTA	iptacopan hydrochloride	CAPSULE;ORAL	218276-001	Dec 5, 2023		RX	Yes	Yes
Novartis	FABHALTA	iptacopan hydrochloride	CAPSULE;ORAL	218276-001	Dec 5, 2023		RX	Yes	Yes			Y	Y

International Patents for FABHALTA

See the table below for patents covering FABHALTA around the world.

Country	Patent Number	Title	Estimated Expiration
Argentina	096904	DERIVADOS DE PIPERIDINIL-INDOL COMO INHIBIDORES DEL FACTOR DE COMPLEMENTO B Y USOS DE LOS MISMOS
El Salvador	2016005137
European Patent Office	3675854

Supplementary Protection Certificates for FABHALTA

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3022192	2024C/536	Belgium		PRODUCT NAME: IPTACOPAN OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT ERVAN; AUTHORISATION NUMBER AND DATE: EU/1/24/1802 20240521
3022192	CA 2024 00035	Denmark		PRODUCT NAME: IPTACOPAN; REG. NO/DATE: EU/1/24/1802 20240521
3022192	LUC00358	Luxembourg		PRODUCT NAME: IPTACOPAN OU UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; AUTHORISATION NUMBER AND DATE: EU/1/24/1802 20240521

Market Dynamics and Financial Trajectory for FABHALTA

Introduction to FABHALTA

FABHALTA, developed by Novartis, is a groundbreaking drug that has recently received FDA approval for the treatment of primary IgA nephropathy (IgAN), in addition to its initial approval for paroxysmal nocturnal hemoglobinuria (PNH) in December 2023. This oral complement inhibitor is poised to significantly impact the treatment landscape for IgAN and other rare kidney diseases.

FDA Approval and Clinical Trials

The FDA approval for FABHALTA in IgAN was granted through the accelerated approval pathway, based on data from the Phase III APPLAUSE-IgAN trial. This trial involved approximately 470 participants and demonstrated a clinically meaningful and statistically significant reduction in proteinuria by 44% over nine months, compared to a 9% reduction with placebo[1][4].

Market Size and Potential

The market for IgAN treatments is substantial, with an estimated diagnosed prevalent population of around 395,000 cases in the seven major markets (7MM) as of 2023. This is roughly ten times the size of the PNH population, indicating a large potential market for FABHALTA[1].

Pricing Strategy

Novartis has set the price of FABHALTA at $550,000, which is higher than the wholesale cost of competing drugs like FILSPARI from Travere Therapeutics, priced at $120,000. However, FABHALTA's oral formulation distinguishes it from other complement inhibitors like AstraZeneca's ULTOMIRIS, which is an infused antibody drug priced over $500,000[1].

Financial Projections

The approval and launch of FABHALTA are expected to contribute significantly to Novartis' financial growth. The company anticipates multibillion-dollar sales from FABHALTA, particularly in the IgAN and C3 glomerulopathy indications. Novartis has already seen strong operational performance in Q3 2024, with sales up 10% and core operating income up 20%, partly driven by the momentum from new drug launches like FABHALTA[3][5].

Sales Growth

Novartis has raised its full-year 2024 guidance, expecting net sales to grow in the low double-digit range and core operating income to grow in the high teens. This growth is supported by the strong performance of key growth drivers and newer launches, including FABHALTA[3][5].

Revenue Impact

The inclusion of FABHALTA in Novartis' portfolio is expected to enhance the company's revenue stream. With a strong pipeline and recent FDA approvals, Novartis is confident in its mid-term growth prospects, aiming for a +5% constant currency (cc) sales CAGR from 2023 to 2028 and a core operating income margin of over 40% by 2027[3][5].

Competitive Landscape

FABHALTA will face competition from other late-stage assets in development for IgAN. Key players include Visterra with sibeprenlimab, Vera Therapeutics with Atacicept, and RemeGen with telitacicept. However, Novartis' acquisition of Chinook Therapeutics has bolstered its renal portfolio, including the endothelin receptor antagonist atrasentan and the anti-APRIL antibody zigakibart, both in Phase III development[1][4].

Patient Access and Affordability

To improve patient access, Novartis plans to collaborate with payers to secure coverage for FABHALTA and is introducing a copay assistance program for those who cannot afford the drug. This initiative is crucial given the high price point of FABHALTA and the preference among patients and doctors to avoid long-term steroid use due to its side effects[1].

Future Indications and Pipeline

Beyond IgAN and PNH, Novartis is exploring FABHALTA for several other rare conditions, including C3 glomerulopathy, atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis, and lupus nephritis. The company aims to submit an FDA filing for C3 glomerulopathy by the end of 2024, further expanding the drug's potential market[1].

Key Takeaways

• FDA Approval: FABHALTA received FDA approval for IgAN through the accelerated approval pathway based on the Phase III APPLAUSE-IgAN trial.
• Market Potential: The drug targets a significant market with approximately 395,000 diagnosed prevalent cases of IgAN in the 7MM.
• Pricing: Priced at $550,000, FABHALTA is positioned competitively against other complement inhibitors.
• Financial Growth: Expected to contribute to Novartis' strong financial growth, with raised full-year 2024 guidance.
• Competitive Landscape: Faces competition from other late-stage IgAN therapies but is supported by Novartis' robust renal portfolio.
• Patient Access: Novartis is implementing measures to improve patient access and affordability.

FAQs

Q: What is FABHALTA and what is it used for?

FABHALTA (iptacopan) is an oral complement inhibitor approved by the FDA for reducing proteinuria in patients with primary IgA nephropathy (IgAN) and paroxysmal nocturnal hemoglobinuria (PNH).

Q: How was FABHALTA approved for IgAN?

FABHALTA was approved through the FDA's accelerated approval pathway based on data from the Phase III APPLAUSE-IgAN trial, which showed a significant reduction in proteinuria.

Q: How much does FABHALTA cost?

FABHALTA is priced at $550,000, which is higher than some competing drugs but distinguished by its oral formulation.

Q: What is the market potential for FABHALTA in IgAN?

The market potential is substantial, with an estimated 395,000 diagnosed prevalent cases of IgAN in the seven major markets (7MM) as of 2023.

Q: What other conditions is FABHALTA being explored for?

FABHALTA is being explored for several other rare conditions, including C3 glomerulopathy, atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis, and lupus nephritis.

Sources

1. DelveInsight: Novartis' FABHALTA: First Complement Inhibitor for IgAN, August 19, 2024.
2. Novartis: Q2 2024 Investor Presentation, July 18, 2024.
3. Novartis: Novartis continues strong momentum in Q3 with 10% sales growth, October 29, 2024.
4. BioSpace: Novartis Secures Phase III Fabhalta Win in IgAN as FDA Starts Priority Review, April 16, 2024.
5. Novartis: Q3 2024 Results, October 29, 2024.