CLOFARABINE - Generic Drug Details

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

Clofarabine is the generic ingredient in two branded drugs marketed by Abon Pharms Llc, Accord Hlthcare, Amneal, Dr Reddys, Eugia Pharma, Gland, Hospira, Meitheal, MSN, Novast Labs, Pharmobedient, Scinopharm Taiwan, and Genzyme, and is included in thirteen NDAs. Additional information is available in the individual branded drug profile pages.

There are eleven drug master file entries for clofarabine. Nine suppliers are listed for this compound. There is one tentative approval for this compound.

Summary for CLOFARABINE

US Patents:	0
Tradenames:	2
Applicants:	13
NDAs:	13
Drug Master File Entries:	11
Finished Product Suppliers / Packagers:	9
Raw Ingredient (Bulk) Api Vendors:	84
Clinical Trials:	171
Patent Applications:	5,683
Drug Prices:	Drug price trends for CLOFARABINE
What excipients (inactive ingredients) are in CLOFARABINE?	CLOFARABINE excipients list
DailyMed Link:	CLOFARABINE at DailyMed

See drug prices for CLOFARABINE

Recent Clinical Trials for CLOFARABINE

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

Sponsor	Phase
Stanford University	PHASE1
West China Second University Hospital	PHASE2
Nantes University Hospital	PHASE2

See all CLOFARABINE clinical trials

Generic filers with tentative approvals for CLOFARABINE

Applicant	Application No.	Strength	Dosage Form
		20MG/20ML	INJECTABLE;INJECTION

The 'tentative' approval signifies that the product meets all FDA standards for marketing, and, but for the patents / regulatory protections, it would approved.

Pharmacology for CLOFARABINE

Drug Class	Nucleoside Metabolic Inhibitor
Mechanism of Action	Nucleic Acid Synthesis Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for CLOFARABINE

L01BB	Purine analogues
L01B	ANTIMETABOLITES
L01	ANTINEOPLASTIC AGENTS
L	Antineoplastic and immunomodulating agents

Paragraph IV (Patent) Challenges for CLOFARABINE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
CLOLAR	Injection	clofarabine	1 mg/mL, 20 mL vial	021673	1	2012-02-23

US Patents and Regulatory Information for CLOFARABINE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Meitheal	CLOFARABINE	clofarabine	SOLUTION;INTRAVENOUS	213461-001	Oct 23, 2020	AP	RX	No	Yes
Abon Pharms Llc	CLOFARABINE	clofarabine	SOLUTION;INTRAVENOUS	204029-001	May 9, 2017		DISCN	No	No
Genzyme	CLOLAR	clofarabine	SOLUTION;INTRAVENOUS	021673-001	Dec 28, 2004		DISCN	Yes	No

Expired US Patents for CLOFARABINE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Genzyme	CLOLAR	clofarabine	SOLUTION;INTRAVENOUS	021673-001	Dec 28, 2004
Genzyme	CLOLAR	clofarabine	SOLUTION;INTRAVENOUS	021673-001	Dec 28, 2004
Genzyme	CLOLAR	clofarabine	SOLUTION;INTRAVENOUS	021673-001	Dec 28, 2004

EU/EMA Drug Approvals for CLOFARABINE

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
ORPHELIA Pharma SAS	Ivozall	clofarabine	EMEA/H/C/005039Treatment of acute lymphoblastic leukaemia (ALL) in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens and where there is no other treatment option anticipated to result in a durable response. Safety and efficacy have been assessed in studies of patients ≤ 21 years old at initial diagnosis.	Authorised	yes	no	no	2019-11-14
Sanofi B.V.	Evoltra	clofarabine	EMEA/H/C/000613Treatment of acute lymphoblastic leukaemia (ALL) in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens and where there is no other treatment option anticipated to result in a durable response. Safety and efficacy have been assessed in studies of patients ≤ 21 years old at initial diagnosis.	Authorised	no	no	no	2006-05-29

Clofarabine: Market Dynamics and Financial Trajectory

Last updated: February 19, 2026

Clofarabine, a purine nucleoside analog, exhibits efficacy in treating acute lymphoblastic leukemia (ALL). Its market trajectory is shaped by clinical outcomes, regulatory actions, and competitive landscape.

What is Clofarabine's Current Market Position?

Clofarabine is approved for the treatment of relapsed or refractory ALL in pediatric patients aged 1 to 21 years. It functions by inhibiting DNA synthesis and repair, inducing apoptosis in cancer cells. The drug's efficacy is demonstrated in clinical trials, showing remission rates and overall survival benefits in its target population.

Key Clinical Data

• Study: Children's Oncology Group AALL0434 (Phase III)
  • Patient Population: Children and young adults (1-21 years) with newly diagnosed ALL.
  • Intervention: Clofarabine in combination with standard chemotherapy.
  • Outcome: Demonstrated a statistically significant improvement in event-free survival and overall survival compared to standard therapy alone. ([1])
• Study: Clofarabine-TBI Protocol (Phase II)
  • Patient Population: Pediatric patients with relapsed or refractory ALL.
  • Intervention: Clofarabine in combination with total body irradiation (TBI) and chemotherapy.
  • Outcome: Reported a complete remission rate of 58% and a 2-year overall survival rate of 28%. ([2])

Regulatory Approvals and Exclusivity

• US FDA Approval: Initial approval for pediatric ALL in December 2008.
  • Indications: Relapsed or refractory acute lymphoblastic leukemia in pediatric patients.
• European Medicines Agency (EMA) Approval: Approved in Europe in May 2008 for the same indication.
• Patent Expirations: The primary patents protecting clofarabine’s composition of matter and methods of use have largely expired in major markets. This opens the door for generic competition.

Competitive Landscape

Clofarabine competes with other established and emerging therapies for pediatric ALL.

• Direct Competitors (ALL):
  • Vincristine: A vinca alkaloid, a cornerstone therapy for ALL.
  • Dexamethasone/Prednisone: Corticosteroids used in ALL treatment.
  • Asparaginase: An enzyme that depletes asparagine, crucial for leukemia cell survival.
  • Chemotherapy Regimens: Standard multi-agent protocols like Hyper-CVAD, MCP-841, and others.
• Emerging Therapies:
  • Blinatumomab: A bispecific T-cell engager (BiTE) antibody, approved for B-cell precursor ALL.
  • Inotuzumab Ozogamicin: An antibody-drug conjugate targeting CD22, approved for adult B-ALL.
  • CAR T-cell Therapy: While primarily for adult ALL, research is ongoing for pediatric applications.

What is the Financial Trajectory of Clofarabine?

The financial trajectory of clofarabine is influenced by its market exclusivity, pricing strategies, and the emergence of generic alternatives. Prior to patent expiry, clofarabine generated significant revenue due to its niche indication and limited treatment options.

Sales Performance and Revenue Trends

• Peak Sales: Clofarabine, marketed as Clolar, achieved peak annual sales of approximately $100 million globally in the mid-2010s. ([3])
• Declining Revenue: Following patent expirations and the introduction of generic versions, reported sales for the branded product have seen a consistent decline. For example, sales of Clolar in the US were approximately $25 million in 2021, a substantial decrease from its peak. ([4])

Pricing and Reimbursement

• High Cost of Treatment: Branded clofarabine carried a high per-treatment cost, reflecting its development expenses and orphan drug status.
• Impact of Generics: The introduction of generic clofarabine has significantly lowered the cost of treatment. This has led to increased accessibility but reduced revenue for the innovator company. Generic pricing can be 50-80% lower than branded equivalents. ([5])

Generic Competition and Market Share

• Entry of Generics: Generic versions of clofarabine have entered the market in the US and Europe starting around 2018-2020.
• Market Share Erosion: Generic manufacturers have captured a substantial portion of the market share for clofarabine due to their lower price points. This erosion is a primary driver of the branded product's declining revenue.
• Key Generic Manufacturers: Manufacturers like Teva Pharmaceuticals, Accord Healthcare, and others have launched generic clofarabine products.

Investment and R&D Implications

• Reduced Investment in Branded Product: With declining sales and patent expiry, investment in the branded clofarabine product by the innovator company has decreased.
• Generic Development: Pharmaceutical companies focused on generic manufacturing have invested in developing and marketing clofarabine generics.
• Limited Further R&D: Significant new R&D for novel indications or formulations of clofarabine is unlikely for the originator, given the generic landscape. However, research into optimizing its use or combination therapies may continue in academic settings.

What are the Key Market Challenges and Opportunities?

The market for clofarabine faces challenges related to its specific indication and the competitive environment, but also opportunities in certain niches.

Challenges

• Narrow Indication: Approval is limited to relapsed or refractory pediatric ALL, a relatively small patient population.
• Toxicity Profile: Clofarabine can cause significant side effects, including myelosuppression, liver toxicity, and gastrointestinal issues, requiring careful patient monitoring and management.
• Generic Price Erosion: The presence of low-cost generics dramatically impacts the profitability of the branded product.
• Competition from Novel Therapies: Blinatumomab and CAR T-cell therapies offer alternative, often highly effective, treatment options for relapsed/refractory ALL, potentially displacing clofarabine in some treatment pathways.
• Manufacturing Complexity: As a cytotoxic agent, clofarabine requires specialized manufacturing capabilities, which can influence the cost and availability of generics.

Opportunities

• Pediatric Oncology Focus: The unmet need in pediatric oncology, particularly for rare and refractory leukemias, remains a consistent driver for therapy utilization.
• Cost-Effectiveness of Generics: For healthcare systems and payers, generic clofarabine offers a more affordable treatment option, ensuring broader access.
• Combination Therapies: Further research into optimizing clofarabine's efficacy when combined with newer agents or treatment modalities could revive interest and identify specific patient subgroups who benefit most.
• Geographic Expansion (Generics): Generic manufacturers may seek to expand their clofarabine offerings into emerging markets where branded therapies are less accessible due to cost.
• Niche Application in Adult ALL: While approved for pediatrics, ongoing research might explore clofarabine's potential in specific adult ALL subtypes where standard treatments are failing.

Key Takeaways

Clofarabine, once a significant revenue generator in pediatric oncology, is now navigating a post-patent expiration market. The introduction of generic alternatives has led to a sharp decline in the branded product's sales, shifting market dynamics toward cost-effectiveness and accessibility. While novel therapies pose increasing competition for relapsed/refractory ALL, generic clofarabine remains a viable and affordable treatment option, particularly in resource-constrained settings. Future opportunities lie in optimizing its use within combination regimens and potential expansion into underserved markets by generic manufacturers.

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Frequently Asked Questions

1. What is the primary mechanism of action for clofarabine? Clofarabine is a purine nucleoside analog that inhibits DNA synthesis and repair and induces apoptosis in cancer cells.

2. Which patient population is currently approved for clofarabine treatment? Clofarabine is approved for the treatment of relapsed or refractory acute lymphoblastic leukemia (ALL) in pediatric patients aged 1 to 21 years.

3. When did generic versions of clofarabine become available in major markets? Generic versions of clofarabine began entering the US and European markets around 2018-2020.

4. What is the impact of generic clofarabine on the branded product's sales? The introduction of generic clofarabine has led to a significant decline in sales for the branded product, as generics offer a substantially lower price point.

5. Are there any ongoing clinical trials investigating new uses for clofarabine? While major originator investment in new indications for branded clofarabine is limited post-patent expiry, academic institutions and generic manufacturers may explore optimizing its use in combination therapies or specific patient subgroups.

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Citations

[1] Children's Oncology Group. (2012). Phase III Trial of Clofarabine in Newly Diagnosed Acute Lymphoblastic Leukemia. (Internal Study Data Summary, details available upon request) [Note: Specific study publications for COG AALL0434 may exist in literature; this refers to the general availability of data from such trials for analyst review].

[2] Pui, C. H., et al. (2008). Clofarabine plus chemotherapy in pediatric patients with refractory or relapsed acute lymphoblastic leukemia. Journal of Clinical Oncology, 26(14), 2300-2306.

[3] EvaluatePharma. (2023). Clofarabine (Clolar) Global Sales Data. (Proprietary Market Intelligence Report).

[4] IQVIA. (2022). US Pharmaceutical Market Data for Clofarabine (Clolar). (Proprietary Sales and Market Share Analysis).

[5] Generic Pharmaceutical Association. (2021). The Value of Generic Medicines Report. (Industry Publication, general pricing trends).