Common questions

What class of drug is Amifampridine?


What class of drug is Amifampridine?

Amifampridine is a voltage gated potassium channel blocker used to treat Lambert-Eaton myasthenic syndrome.

What is Lambert-Eaton disease?

Lambert-Eaton myasthenic syndrome (LEMS) is a very rare condition that affects the signals sent from the nerves to the muscles. It means the muscles are unable to tighten (contract) properly, resulting in muscle weakness and a range of other symptoms.

How is Lambert-Eaton syndrome treated?

Two potassium channel blockers are approved in the United States to treat LEMS. Firdapse (also known as 3,4-diaminopyridine, 3,4-DAP) was approved by the FDA in November 2018 for the treatment of adults with LEMS and is commercialized by Catalyst Pharmaceuticals.

What is the cost of firdapse?

The cost for Firdapse oral tablet 10 mg is around $24,724 for a supply of 120 tablets, depending on the pharmacy you visit. Prices are for cash paying customers only and are not valid with insurance plans. Firdapse is available as a brand name drug only, a generic version is not yet available.

What is the difference between firdapse and Ruzurgi?

Firdapse is a potassium channel blocker indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults (1). Ruzurgi is a potassium channel blocker indicated for the treatment of Labert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age (2).

What is pharmaceutical catalyst?

Catalyst Pharmaceuticals, Inc. is a commercial-stage, patient-centric biopharmaceutical company. The Company is focused on in-licensing, developing, and commercializing medicines for patients living with rare diseases. The Company is developing a pipeline of medicines for other rare diseases.

What diseases are associated with LEMS?

In 40% of patients with LEMS, cancer is present when the weakness begins or is found later. This is usually a small cell lung cancer (SCLC), although LEMS has also been associated with non-SCLC, lymphosarcoma, malignant thymoma, or carcinoma of the breast, stomach, colon, prostate, bladder, kidney, or gallbladder.

What causes Lambert-Eaton disease?

What causes Lambert-Eaton syndrome? This condition is often associated with a certain type of cancer called small cell lung cancer. This syndrome may result from your body’s efforts to fight the underlying cancer. In some of the remaining cases, Lambert-Eaton syndrome develops following another autoimmune disease.

Who makes firdapse?

Firdapse is approved to treat LEMS in adults, and Ruzurgi is approved to treat LEMS in pediatric patients….Official Answer.

Ruzurgi Firdapse
Date of FDA Approval May 6, 2019 November 28, 2018
Company Jacobus Pharmaceutical Company Inc. Catalyst Pharmaceuticals, Inc.

Where is Catalyst Pharmaceuticals located?

Coral Gables, Florida
Catalyst Pharmaceuticals is a biopharmaceutical company based in Coral Gables, Florida.

What is the difference between LEMS and MG?

The difference between LEMS and myasthenia gravis (MG) This is very similar to myasthenia gravis, however the target of the attack is different in MG as the acetylcholine receptor on the nerve is affected, whereas in LEMS it’s the voltage-gated calcium channel on the nerve.

What kind of muscle disease is amifampridine used for?

Amifampridine ( pyridine-3,4-diamine, 3,4-diaminopyridine, 3,4-DAP) is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and Lambert–Eaton myasthenic syndrome…

How is amifampridine used to treat LEMS in adults?

The only other treatment approved for LEMS (Firdapse) is only approved for use in adults. Amifampridine is used to treat many of the congenital myasthenic syndromes, particularly those with defects in choline acetyltransferase, downstream kinase 7, and those where any kind of defect causes “fast channel” behaviour of the acetylcholine receptor.

Why did amifampridine need to be licensed in France?

The company responded that it submitted the licensing request at the suggestion of the French government, and points out that the increased cost of a licensed drug also means that it is monitored by regulatory authorities (e.g. for uncommon side effects), a process that was previously not present in Europe.

Are there any side effects to taking amifampridine?

The dose-limiting side effects include tingling or numbness, difficulty sleeping, fatigue, and loss of muscle strength. Amifampridine can cause seizures, especially but not exclusively when given at high doses and/or in particularly vulnerable individuals who have a history of seizures.