Catalyst Pharmaceuticals

Catalyst Pharmaceutical Partners, Inc.Type| Public company

|

Traded as

| NASDAQ: CPRX
Russell 2000 Component
S&P 600 Component
Industry| Biotechnology
Headquarters|

Coral Gables, Florida, U.S.

Key people

| Patrick J. McEnany, co-founder, chairman, president and chief executive officer
Products| FIRDAPSE® (amifampridine) Tablets 10 mg (commercialized 2019)

Operating income

|  US$ 52.38 million (2022)[1]
Total assets| US$ 267.31 million (2022)[1]
Total equity| US$ 237.79 million (2022)[1]

Number of employees

| 76 as of March 14, 2022[1]
Website| www.catalystpharma.com
Footnotes / references
[2] [3]

Catalyst Pharmaceuticals Inc. is a biopharmaceutical company based in Coral Gables, Florida. The company is developing medicines for rare diseases, including the phosphate salt of amifampridine for the treatment of Lambert–Eaton myasthenic syndrome (LEMS). The drug is referred to under the trade name Firdapse, which was approved by the FDA for approved use in children 6 years and older with LEMS in addition to the prior approval for use in adults with LEMS on November 28, 2018. Firdapse commercially launched in January 2019.[4]

## Contents

* 1 History
* 2 Lambert-Eaton myasthenic syndrome
* 3 Amifampridine
* 4 Litigation and Reception
* 5 References

## History

Catalyst was founded in 2002, and completed an IPO in 2006.[5] It focused primarily on developing therapies to prevent addiction until 2012.[6]

In 2009, Catalyst in-licensed worldwide rights to a family of GABA inhibitors including CPP-115 from Northwestern University.[7][8] In 2012, it in-licensed patents covering the use of amifampridine phosphate to treat LEMS for the North American market from BioMarin.[9]

In 2012, while BioMarin had a Phase III trial ongoing in the US, it licensed the US rights to 3,4-DAPP, including the orphan designation and the ongoing trial, to Catalyst Pharmaceuticals.[10]

In August 2013, analysts anticipated that FDA approval would be granted to Catalyst in LEMS by 2015.[11] The drug is used to treat Lambert–Eaton myasthenic syndrome (LEMS), which is a rare neuromuscular disorder characterized by muscle weakness of the limbs, affecting about 3.4 per million people.

In December 2015, Catalyst submitted its new drug application to the FDA,[12] and in February 2016 the FDA refused to accept it, on the basis that it wasn't complete. In April 2016 the FDA told Catalyst it would have to gather further data.[13][14][15] In March 2018 the company re-submitted its NDA.[16] The FDA approved amifampridine for the treatment of adults with Lambert-Eaton myasthenic syndrome on November 29, 2018.[17]

In 2018, Catalyst terminated its license for CPP-115 with Northwestern and stopped the development program for that compound.[18]

As of 2022, the company offered Catalyst Pathways, a program that provides financial aid, insurance navigation, bridge medicine, and Patient Access Liaisons.[19]

## Lambert-Eaton myasthenic syndrome

Tentative evidence supports 3,4-diaminopyridine treatment at least for a few weeks, with the goal to improve neuromuscular transmission.[20] The 3,4-diaminopyridine base or the water-soluble 3,4-diaminopyridine phosphate may be used.[21] Both 3,4-diaminopyridine formulations stall the repolarization of nerve terminals after a discharge, allowing more calcium to gather in the terminal.[22][23]

## Amifampridine

The development of amifampridine and its phosphate has brought attention to orphan drug policies that grant market exclusivity as an incentive for companies to develop therapies for conditions that affect small numbers of people. Amifampridine, also called 3,4-DAP, was discovered in Scotland in the 1970s, and doctors in Sweden first showed its use in LEMS in the 1980s.[24]

## Litigation and Reception

In December 2015 a group of neuromuscular doctors who had worked with both Jacobus and BioMarin/Catalyst published an editorial in the journal, Muscle & Nerve, with concerns about the potential for the price of the drug to be drastically increased should Catalyst obtain FDA approval, and stating that 3,4-DAPP represented no real innovation and didn't deserve exclusivity under the Orphan Drug Act, which was meant to spur innovation to meet unmet needs.[24][25] Catalyst responded to this editorial with a response in 2016 that explained they were conducting a full range of clinical and non-clinical studies necessary to obtain approval in order to specifically address the unmet need among the estimated 1500-3000 LEMs patients since about 200 were receiving the product through compassionate use – and that this is exactly what the Orphan Drug Act was intended to do.[26] Prior to the FDA approval, patients were able to get an investigational version of amifampridine for free through compassionate use programs in accordance with FDA Rules and Guidelines.[27][28][29]

On January 28, 2022, in Catalyst Pharmaceuticals, Inc. v. Becerra, the Eleventh Circuit upheld orphan exclusivity for Catalyst Pharmaceuticals and its drug Firdapse. With this decision, the Eleventh Circuit rejected the FDA's interpretation of orphan exclusivity and concluded that the agency had improperly approved a competitor product from Jacobus Pharmaceutical Co.[30][31]

On February 4, 2019, Bernie Sanders, United States Senator from Vermont, publicly sent a letter to Catalyst asking why they raised the price of their drug Firdapse to an annual cost of $375,000, considering Firdapse was previously free of charge through an FDA compassionate use program. Sanders questioned the financial decision regarding the negative impact, specifically asking about how many patients would suffer or die, for patients who may no longer be able to afford the drug. The drug is used to treat Lambert–Eaton myasthenic syndrome (LEMS), which is a rare neuromuscular disorder.[29][32]

## References

1. ↑ 1.0 1.1 1.2 1.3 "Catalyst Pharmaceuticals Annual Report 2022". https://sec.report/Document/0001193125-22-077228/. [|permanent dead link|dead link}}]
2. ↑ "Catalyst Pharmaceuticals 2014 Annual Report Form (10-K)" (XBRL). United States Securities and Exchange Commission. https://www.sec.gov/Archives/edgar/data/1369568/000119312515091323/d838315d10k.htm.
3. ↑ "Catalyst Annual Report 2018". ir.catalystpharma.com. https://ir.catalystpharma.com/static-files/e2322ff0-2135-41d8-9990-70c82d08933e.htm. [|permanent dead link|dead link}}]
4. ↑ "Firdapse FDA Approval Letter". 2018-11-28. https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2018/208078Orig1s000ltr.pdf.
5. ↑ "Catalyst Pharmaceuticals Registration Statement Form S-1" (XBRL). United States Securities and Exchange Commission. https://www.sec.gov/Archives/edgar/data/1369568/000119312512153699/d329891ds1.htm.
6. ↑ Mann Jr., Joseph A. (July 18, 2015). "The big gamble: Catalyst Pharmaceuticals of Coral Gables bets on new drug for rare disease" (in en). Miami Herald. http://www.miamiherald.com/news/business/biz-monday/article27561916.html.
7. ↑ Hawker D. and R. Silverman. “Synthesis and evaluation of novel heteroaromatic substrates of GABA Aminotransferase”, Bioorganic & Medicinal Chemistry, October 1, 2012. Retrieved September 8, 2015.
8. ↑ Brian Bandell. “Catalyst Pharmaceutical signs licensing deal with Northwestern” “South Florida Business Journal”, August 31, 2009. Retrieved September 8, 2015.
9. ↑ “Catalyst Acquires Late-Stage Orphan Drug from BioMarin, “Genetic Engineering and Biotechnology News, November 1, 2012. Retrieved August 27, 2015.
10. ↑ Leuty, Ron (October 31, 2012). "BioMarin licenses North American rights to rare disease drug, invests $5M in Florida company". San Francisco Business Journal. http://www.bizjournals.com/sanfrancisco/blog/biotech/2012/10/biomarin-catalyst-firdapse-lems.html.
11. ↑ Baker, D. E. (2013). "Breakthrough Drug Approval Process and Postmarketing ADR Reporting". Hospital Pharmacy 48 (10): 796–798. doi:10.1310/hpj4810-796. PMID 24421428.
12. ↑ Tavernise, Sabrina (December 22, 2015). "Patients Fear Spike in Price of Old Drugs" (in en). New York Times. https://www.nytimes.com/2015/12/23/health/patients-fear-spike-in-price-of-old-drugs.html.
13. ↑ Adams, Ben (April 26, 2016). "Catalyst Pharmaceuticals hit by FDA extra studies request for Firdapse". FierceBiotech. http://www.fiercebiotech.com/biotech/catalyst-pharmaceuticals-hit-by-fda-extra-studies-request-for-firdapse.
14. ↑ Tavernise, Sabrina (February 17, 2016). "F.D.A. Deals Setback to Catalyst in Race for Drug Approval" (in en). New York Times. https://www.nytimes.com/2016/02/18/health/fda-deals-setback-to-catalyst-in-race-for-drug-approval.html.
15. ↑ Adams, Ben (May 17, 2016). "Catalyst to ax 30% of workforce in wake of FDA trial demands" (in en). FierceBiotech. https://www.fiercebiotech.com/biotech/catalyst-to-ax-30-workforce-wake-fda-trial-demands.
16. ↑ Lima, Debora (March 29, 2018). "Catalyst Pharmaceuticals files new drug application with FDA". South Florida Business Journal. https://www.bizjournals.com/southflorida/news/2018/03/29/catalyst-files-new-drug-application-with-fda.html.
17. ↑ "Firdapse (amifampridine phosphate) FDA Approval History". https://www.drugs.com/history/firdapse.html.
18. ↑ "Catalyst Pharmaceuticals 2018 Annual Report. As of August 2020 Q2 filing, CPRX (ticker symbol) has had average quarterly revenue of 30M USD, and profits of 9M USD per quarter.". https://ir.catalystpharma.com/static-files/e2322ff0-2135-41d8-9990-70c82d08933e.
19. ↑ "Catalyst Pathways". https://www.needymeds.org/program_list.taf?_function=name&program_id=799de6d3dae4c924142cf245a1d7f703.
20. ↑ Keogh, M; Sedehizadeh, S; Maddison, P (16 February 2011). "Treatment for Lambert-Eaton myasthenic syndrome.". The Cochrane Database of Systematic Reviews 2011 (2): CD003279. doi:10.1002/14651858.CD003279.pub3. PMID 21328260.
21. ↑ Lindquist, S; Stangel, M; Ullah, I (2011). "Update on treatment options for Lambert-Eaton myasthenic syndrome: focus on use of amifampridine.". Neuropsychiatric Disease and Treatment 7: 341–9. doi:10.2147/NDT.S10464. PMID 21822385.
22. ↑ "Lambert–Eaton myasthenic syndrome". Semin. Neurol. 24 (2): 149–53. June 2004. doi:10.1055/s-2004-830900. PMID 15257511.
23. ↑ "Available treatment options for the management of Lambert–Eaton myasthenic syndrome". Expert Opin. Pharmacother. 7 (10): 1323–36. July 2006. doi:10.1517/14656566.7.10.1323. PMID 16805718.
24. ↑ 24.0 24.1 Deak, Dalia (February 22, 2016). "Jacobus and Catalyst Continue to Race for Approval of LEMS Drug". Bill of Health. http://blogs.harvard.edu/billofhealth/2016/02/22/jacobus-and-catalyst-approval/.
25. ↑ Burns, TM, et al.I (February 2016). "Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4-diaminopyridine.". Muscle & Nerve 53 (2): 165–8. doi:10.1002/mus.25009. PMID 26662952.
26. ↑ McEnany, Patrick J. (2017). "A response to a recent editorial by concerned physicians on 3,4-diaminopyridine" (in en). Muscle & Nerve 55 (1): 138. doi:10.1002/mus.25437. ISSN 1097-4598. PMID 27756108.
27. ↑ Commissioner, Office of the (2019-05-06). "Expanded Access" (in en). https://www.fda.gov/news-events/public-health-focus/expanded-access.
28. ↑ "NDA for Catalyst's LEMS Therapy Firdapse Accepted with Priority Review" (in en-US). 2018-06-04. https://lamberteatonnews.com/2018/06/04/fda-accepts-firdapse-nda-for-lems-priority-review-status/.
29. ↑ 29.0 29.1 Abutaleb, Yasmeen (4 February 2019). "Senator Sanders asks why drug, once free, now costs $375k". Reuters. https://www.reuters.com/article/us-usa-healthcare-catalyst/senator-sanders-to-ask-why-drug-once-free-now-costs-375k-idUSKCN1PT0ZJ.
30. ↑ Lovells, Hogan. "Eleventh Circuit decision could significantly expand scope of orphan exclusivity" (in English). https://www.jdsupra.com/legalnews/eleventh-circuit-decision-could-3664479/.
31. ↑ Koblitz, Sara (February 17, 2022). "Condition Critical: Court Interprets Orphan Drug Exclusivity Broadly" (in English). Hyman, Phelps & McNamara, P.C.. https://www.thefdalawblog.com/2022/02/condition-critical-court-interprets-orphan-drug-exclusivity-broadly/.
32. ↑ "Family outraged over life-changing treatment going from free to $375,000 a year". https://www.nbcnews.com/health/health-news/family-outraged-over-life-changing-treatment-going-free-375-000-n968906.

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