Carglumic acid - Generic Drug Details

What are the generic drug sources for carglumic acid and what is the scope of patent protection?

Carglumic acid is the generic ingredient in two branded drugs marketed by Recordati Rare, Navinta Llc, and Novitium Pharma, and is included in three NDAs. Additional information is available in the individual branded drug profile pages.

There are two drug master file entries for carglumic acid. Four suppliers are listed for this compound.

Summary for carglumic acid

US Patents:	0
Tradenames:	2
Applicants:	3
NDAs:	3
Drug Master File Entries:	2
Finished Product Suppliers / Packagers:	4
Raw Ingredient (Bulk) Api Vendors:	85
Clinical Trials:	4
Patent Applications:	324
Drug Prices:	Drug price trends for carglumic acid
What excipients (inactive ingredients) are in carglumic acid?	carglumic acid excipients list
DailyMed Link:	carglumic acid at DailyMed

See drug prices for carglumic acid

Recent Clinical Trials for carglumic acid

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

Sponsor	Phase
National Taiwan University Hospital	N/A
King Abdullah International Medical Research Center	Phase 3
Children's National Research Institute	Phase 2

See all carglumic acid clinical trials

Pharmacology for carglumic acid

Drug Class	Carbamoyl Phosphate Synthetase 1 Activator
Mechanism of Action	Carbamoyl Phosphate Synthetase 1 Activators

Anatomical Therapeutic Chemical (ATC) Classes for carglumic acid

A16AA	Amino acids and derivatives
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 carglumic acid

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Recordati Rare	CARBAGLU	carglumic acid	TABLET, FOR SUSPENSION;ORAL	022562-001	Mar 18, 2010	AB	RX	Yes	Yes
Novitium Pharma	CARGLUMIC ACID	carglumic acid	TABLET, FOR SUSPENSION;ORAL	213729-001	Oct 13, 2021	AB	RX	No	No
Navinta Llc	CARGLUMIC ACID	carglumic acid	TABLET, FOR SUSPENSION;ORAL	213395-001	Jun 22, 2022	AB	RX	No	No

EU/EMA Drug Approvals for carglumic acid

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Eurocept International BV	Ucedane	carglumic acid	EMEA/H/C/004019 Ucedane is indicated in treatment of:hyperammonaemia due to N-acetylglutamate synthase primary deficiency;Hyperammonaemia due to isovaleric acidaemia;Hyperammonaemia due to methymalonic acidaemia;Hyperammonaemia due to propionic acidaemia.	Authorised	yes	no	no	2017-06-23
Recordati Rare Diseases	Carbaglu	carglumic acid	EMEA/H/C/000461 Carbaglu is indicated in treatment of:hyperammonaemia due to N-acetylglutamate-synthase primary deficiency;hyperammonaemia due to isovaleric acidaemia;hyperammonaemia due to methymalonic acidaemia;hyperammonaemia due to propionic acidaemia.	Authorised	no	no	no	2003-01-24

Carglumic acid Market Analysis and Financial Projection Experimental

Carglumic Acid Market Dynamics and Financial Trajectory

Introduction to Carglumic Acid

Carglumic acid, a medication derived from N-acetyl glutamate, plays a crucial role in the removal of ammonia and detoxification by activating the first enzyme in the urea cycle. It is particularly used for the treatment of rare diseases such as propionic acidemia and methylmalonic acidemia, which are autosomal recessive diseases often requiring lifelong medical treatment[5].

Market Size and Growth Projections

The global carglumic acid market has been valued at approximately USD 137.2 million in 2023. It is projected to grow at a compound annual growth rate (CAGR) of 7.38% to 7.43% from 2024 to 2030 or 2033, depending on the forecast. By 2033, the market is expected to reach USD 281.81 million to USD 314.4 million[1][4][5].

Key Growth Drivers

Rising Prevalence of Lifestyle-Related Diseases

The increasing prevalence of cancers and metabolic disorders linked to lifestyle choices such as excessive alcohol consumption, tobacco use, and sedentary behavior is a significant driver of the carglumic acid market. For instance, excessive alcohol consumption accounts for over 10% of the global non-communicable disease burden, including ailments like pancreatitis, cirrhosis, and cancer[4].

Growing Incidence of Metabolic Disorders

The rising incidence rates of metabolic disorders such as hepatic encephalopathy, Reye's syndrome, and cirrhosis globally are anticipated to boost overall market demand. Carglumic acid's role in reducing ammonia concentrations in the bloodstream makes it a critical medication for these conditions[1][4].

Heightened Awareness and Diagnosis of Rare Diseases

The growing awareness and diagnosis of rare diseases, including urea cycle disorders (UCD), are driving the market's growth. North America, in particular, has become the largest market for carglumic acid due to these factors[1].

Impact of COVID-19 Pandemic

The COVID-19 pandemic moderately impacted the carglumic acid industry, leading to an increased demand for the medication due to hyperammonemia-related concerns among COVID-19 patients. This heightened demand during the pandemic contributed to market growth[4].

Regional Market Dynamics

North America

North America is currently the largest market for carglumic acid, driven by the high prevalence of breast cancer, lung cancer, and hyperammonemia in the United States. The significant healthcare spending in the U.S., accounting for around 17% of the country’s GDP, also supports the market's expansion[5].

Asia-Pacific

The Asia-Pacific region is predicted to witness the fastest CAGR over the forecast period. Factors such as rapidly growing awareness regarding metabolic ailments, a rising geriatric population prone to chronic diseases, and increasing government initiatives are driving the market in this region[4].

Europe

In Europe, particularly in the U.K., the market is expected to grow at a CAGR of 7.2%, offering an absolute dollar opportunity worth US$ 16.4 million during the forecast period. This growth is driven by the need for efficient medical treatment for NAGS deficiency, a severe disorder affecting newborns in the region[5].

Distribution Channels

Hospital pharmacies account for around half of the total carglumic acid market. The established hospital infrastructure, quick access to medications, patient convenience, and preference for purchasing medications from hospital pharmacies are key factors contributing to this dominance. Hospital pharmacies are set to grow, creating an absolute $ opportunity of US$ 67.2 million between 2023-2033[5].

Financial Performance of Key Players

Eton Pharmaceuticals

Eton Pharmaceuticals, a company focused on developing and commercializing treatments for rare diseases, reported significant growth in its product sales and royalty revenue for carglumic acid. The company saw a 109% year-over-year increase in the fourth quarter of 2023, driven primarily by the ongoing momentum of ALKINDI SPRINKLE and Carglumic Acid. Eton generated $0.4 million in operating cash during the fourth quarter of 2023 and had total cash and cash equivalents of $21.4 million as of December 31, 2023[2].

Research and Development Activities

The upsurge in research and development activities by well-established industry leaders to develop novel orphan drugs is anticipated to propel the global carglumic acid market. Efforts to develop new treatments employing carglumic acid, particularly for various cancers such as pancreatic ductal adenocarcinoma and lung cancer, are expected to drive long-term growth opportunities[4][5].

Government Initiatives and Awareness Campaigns

Growing government initiatives and awareness campaigns regarding several metabolic disorders, including hyperammonemia disease management and treatment, are predicted to drive market demand. Regulatory agencies like the USFDA are initiating campaigns to raise awareness about the use and dose of carglumic acid, further supporting market expansion[4][5].

Key Takeaways

• The global carglumic acid market is projected to grow at a CAGR of 7.38% to 7.43% from 2024 to 2030 or 2033.
• The market is driven by the rising prevalence of lifestyle-related diseases, growing incidence of metabolic disorders, and heightened awareness of rare diseases.
• North America is the largest market, with significant contributions from the U.S. due to high healthcare spending.
• Asia-Pacific is expected to witness the fastest CAGR due to growing awareness and government initiatives.
• Hospital pharmacies dominate the distribution channels, offering substantial growth opportunities.

FAQs

Q: What is the current market size of the global carglumic acid market? A: The global carglumic acid market was valued at approximately USD 137.2 million in 2023[4].

Q: What is the projected CAGR for the global carglumic acid market from 2024 to 2033? A: The market is expected to grow at a CAGR of 7.38% to 7.43% from 2024 to 2033[1][4].

Q: Which region is expected to witness the fastest CAGR in the carglumic acid market? A: The Asia-Pacific region is predicted to witness the fastest CAGR over the forecast period[4].

Q: What are the primary distribution channels for carglumic acid? A: Hospital pharmacies account for around half of the total carglumic acid market[5].

Q: How has the COVID-19 pandemic impacted the carglumic acid market? A: The pandemic moderately impacted the market, leading to an increased demand for carglumic acid due to hyperammonemia-related concerns among COVID-19 patients[4].

Cited Sources:

1. The Brainy Insights: Carglumic Acid Market Size, Trend Analysis, Insights.
2. Biospace: Eton Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results.
3. GII Research: Carglumic Acid Market by Distribution Channel.
4. Grandview Research: Carglumic Acid Market Size, Share & Growth Report, 2030.
5. Fact.MR: Carglumic Acid Market Size, Demand & Growth Drivers 2023-2033.