AMIFAMPRIDINE PHOSPHATE - Generic Drug Details

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

Amifampridine phosphate is the generic ingredient in one branded drug marketed by Catalyst Pharms and is included in one NDA. There are six patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Amifampridine phosphate has three patent family members in two countries.

One supplier is listed for this compound.

Summary for AMIFAMPRIDINE PHOSPHATE

International Patents:	3
US Patents:	6
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	18
Clinical Trials:	8
Patent Applications:	89
What excipients (inactive ingredients) are in AMIFAMPRIDINE PHOSPHATE?	AMIFAMPRIDINE PHOSPHATE excipients list
DailyMed Link:	AMIFAMPRIDINE PHOSPHATE at DailyMed

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

Constraining patent/regulatory exclusivity:

10,626,088

Dosage:

TABLET;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.

Recent Clinical Trials for AMIFAMPRIDINE PHOSPHATE

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

Sponsor	Phase
Catalyst Pharmaceuticals, Inc.	Phase 2
Catalyst Pharmaceuticals, Inc.	Phase 3
Catalyst Pharmaceuticals, Inc.

See all AMIFAMPRIDINE PHOSPHATE clinical trials

Pharmacology for AMIFAMPRIDINE PHOSPHATE

Drug Class	Potassium Channel Blocker
Mechanism of Action	Potassium Channel Antagonists

Anatomical Therapeutic Chemical (ATC) Classes for AMIFAMPRIDINE PHOSPHATE

N07XX	Other nervous system drugs
N07X	OTHER NERVOUS SYSTEM DRUGS
N07	OTHER NERVOUS SYSTEM DRUGS
N	Nervous system

N07XX	Other nervous system drugs
N07X	OTHER NERVOUS SYSTEM DRUGS
N07	OTHER NERVOUS SYSTEM DRUGS
N	Nervous system

Paragraph IV (Patent) Challenges for AMIFAMPRIDINE PHOSPHATE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
FIRDAPSE	Tablets	amifampridine phosphate	10 mg	208078	3	2022-11-28

US Patents and Regulatory Information for AMIFAMPRIDINE PHOSPHATE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Catalyst Pharms	FIRDAPSE	amifampridine phosphate	TABLET;ORAL	208078-001	Nov 28, 2018		RX	Yes	Yes	10,626,088		Y
Catalyst Pharms	FIRDAPSE	amifampridine phosphate	TABLET;ORAL	208078-001	Nov 28, 2018		RX	Yes	Yes	11,274,331
Catalyst Pharms	FIRDAPSE	amifampridine phosphate	TABLET;ORAL	208078-001	Nov 28, 2018		RX	Yes	Yes	10,793,893
Catalyst Pharms	FIRDAPSE	amifampridine phosphate	TABLET;ORAL	208078-001	Nov 28, 2018		RX	Yes	Yes	11,274,332
Catalyst Pharms	FIRDAPSE	amifampridine phosphate	TABLET;ORAL	208078-001	Nov 28, 2018		RX	Yes	Yes	11,060,128

International Patents for AMIFAMPRIDINE PHOSPHATE

Country	Patent Number	Title	Estimated Expiration
Canada	2840591	METHODES D'ADMINISTRATION DE 3,4-DIAMINOPYRIDINE (METHODS OF ADMINISTERING 3,4-DIAMINOPYRIDINE)
World Intellectual Property Organization (WIPO)	2013003708
World Intellectual Property Organization (WIPO)	2017214504

AMIFAMPRIDINE PHOSPHATE Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Amifampridine Phosphate (Firdapse)

Introduction to Amifampridine Phosphate

Amifampridine phosphate, marketed under the brand name Firdapse, is a potassium channel blocker used for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), a rare neuromuscular disorder. The drug has been a significant development in the treatment of LEMS, particularly for adults and, more recently, for pediatric patients.

Mechanism of Action and Clinical Use

Firdapse works by prolonging the signals released from nerves to muscles, thereby enhancing muscle stimulation. It is specifically designed to address the symptoms of LEMS, which include muscle weakness, fatigue, and autonomic dysfunction[1].

Market Outlook

The LEMS market, though niche, has shown significant growth potential. In 2017, the market size for LEMS in the seven major markets (7MM) was estimated to be $59.43 million, with the United States accounting for the majority at $32.85 million. Japan was the second-largest market, contributing $9.59 million[1].

Geographical Distribution

• United States: The primary market for Firdapse, with Catalyst Pharmaceuticals holding the marketing rights.
• EU5: Firdapse is also approved for use in the EU5 countries.
• Japan: The second-largest market for LEMS treatments[1].

Approved Therapies and Market Share

Firdapse is the only approved therapy for the treatment of LEMS in adults in the United States and EU5. For pediatric patients (ages 6 to less than 17), Ruzurgi, another formulation of amifampridine, was approved by the US FDA in May 2019. However, Catalyst Pharmaceuticals has contested this approval, seeking a reversal of the FDA's decision[1].

Drugs Uptake and Patient Enrollment

The uptake of Firdapse has been strong, driven by high patient persistency and new patient enrollments. In the third quarter of 2021, Catalyst Pharmaceuticals reported a 23.1% increase in Firdapse revenue compared to the same period in 2020, reaching $35.9 million[5].

Reimbursement Scenario

Reimbursement plays a crucial role in the market dynamics of Firdapse. The Canadian Drug Expert Committee (CDEC) recommends reimbursement for Firdapse under specific conditions, highlighting its cost-effectiveness compared to other treatments like amifampridine base. However, achieving reimbursement can be challenging, and the cost-effectiveness of Firdapse compared to best supportive care is still under evaluation[3][4].

Financial Performance of Catalyst Pharmaceuticals

Catalyst Pharmaceuticals, the company commercializing Firdapse, has shown strong financial performance. Here are some key financial highlights:

• Revenue Growth: In the third quarter of 2021, Firdapse revenue increased by 23.1% to $35.9 million compared to the third quarter of 2020[5].
• Cash Position: As of September 30, 2021, Catalyst Pharmaceuticals had approximately $174.8 million in cash and investments, supporting their long-term growth strategy[5].
• Operating Income: The company reported an operating income of $14.0 million in the third quarter of 2021, up from $11.7 million in the same period of 2020[5].

Research and Development

Catalyst Pharmaceuticals continues to invest in research and development, including work on a long-acting formulation of amifampridine phosphate and plans to file a supplemental New Drug Application (NDA) for the treatment of pediatric LEMS. These initiatives are aimed at expanding the therapeutic scope and improving patient outcomes[5].

Challenges and Opportunities

Competition and Regulatory Challenges

The approval of Ruzurgi for pediatric LEMS patients has introduced competition, although Catalyst Pharmaceuticals is contesting this approval. Regulatory challenges, including reimbursement hurdles, also impact the market dynamics of Firdapse[1].

Expansion and Growth Opportunities

• Pediatric Indication: Plans to expand the U.S. approval of Firdapse to include pediatric LEMS patients offer significant growth opportunities.
• Long-acting Formulation: Development of a long-acting formulation could enhance patient compliance and market share.
• Global Expansion: Potential for expansion into other markets beyond the 7MM[5].

Cost and Pricing

The cost of Firdapse is substantial, with an estimated annual cost per patient of approximately $51,993 at the public list price. This high cost necessitates careful consideration of reimbursement strategies and cost-effectiveness analyses[3].

"Based on public list prices, treatment with Firdapse is expected to cost approximately $51,993 per patient per year"[3].

Key Takeaways

• Market Growth: The LEMS market is expected to grow, driven by the success of Firdapse.
• Financial Performance: Catalyst Pharmaceuticals has shown strong revenue growth and a robust cash position.
• Regulatory and Reimbursement Challenges: Ongoing challenges include competition from Ruzurgi and reimbursement hurdles.
• Expansion Opportunities: Potential for expansion into pediatric indications and development of long-acting formulations.

FAQs

1. What is the primary mechanism of action of Firdapse?

Firdapse works by prolonging the signals released from nerves to muscles, thereby enhancing muscle stimulation.

2. Which company holds the marketing rights for Firdapse in North America?

Catalyst Pharmaceuticals holds the marketing rights for Firdapse in North America.

3. How much does Firdapse cost per patient per year?

The estimated annual cost per patient for Firdapse is approximately $51,993 at the public list price.

4. What are the key financial highlights for Catalyst Pharmaceuticals in 2021?

In the third quarter of 2021, Catalyst Pharmaceuticals reported $35.9 million in Firdapse revenue, a 23.1% increase from the same period in 2020, and had a cash position of approximately $174.8 million.

5. Are there any ongoing developments or expansions planned for Firdapse?

Yes, Catalyst Pharmaceuticals is working on a long-acting formulation of amifampridine phosphate and plans to file a supplemental NDA for the treatment of pediatric LEMS.

Cited Sources:

1. Lambert-Eaton Myasthenic Syndrome (LEMS) Markets, 2017-2030 - ResearchAndMarkets.com
2. 2018 ANNUAL REPORT - Catalyst Pharmaceuticals
3. Amifampridine Phosphate (Firdapse) - NCBI Bookshelf
4. Amifampridine phosphate (Firdapse) - Canada's Drug Agency
5. Catalyst Pharmaceuticals Reports Third Quarter 2021 Financial Results - Catalyst Pharmaceuticals