Firdapse Maker Secures 3 New Patents in US for LEMS Treatment

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Catalyst Pharmaceuticals has secured three new U.S. patents covering the use of Firdapse (amifampridine) for treating Lambert-Eaton myasthenic syndrome (LEMS).

The United States Patent and Trademark Office informed Catalyst that one new patent will be issued on March 8 and two more a week later on March 15.

“We are pleased to have been notified about these new U.S. patents that will further fortify our intellectual property estate to provide lasting durability for our flagship product,” Patrick McEnany, chairman and CEO of Catalyst, said in a press release.

“As part of our portfolio strategy, we will continue to execute on our key initiatives to strengthen and protect the long-term commercial potential of Firdapse, which currently has patent exclusivity protection in the U.S. until 2034,” McEnany said.

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LEMS is caused by an autoimmune attack that interferes with the ability of nerve cells to send signals to muscle cells, ultimately resulting in muscle weakness and other symptoms. Firdapse’s active ingredient, a molecule called amifampridine, helps to normalize neurological activity and nerve-muscle communications, reducing symptoms.

The three newly issued patents relate to the use of amifampridine by people who metabolize the medication differently due to genetic variations.

Amifampridine is processed in the body by a protein called N-acetyl transferase 2 (NAT2), which converts the medication into an inactive compound that is eventually excreted in the urine.

Every person has two alleles, or copies, of the gene that codes for NAT2, one from each biological parent. Based on subtle variations in the genetic code that provides instructions for making NAT2, some versions of it process, or metabolize, amifampridine much more quickly than others. As such, NAT2 alleles can broadly be classified as either “fast” or “slow,” based on their metabolic rate.

The three new patents cover all three possible combinations of these alleles — two fast, two slow, or one of each. According to Catalyst, these patents, in combination with two U.S. patents for Firdapse the company had previously secured, cover the treatment of all amifampridine metabolizer types within the LEMS patient population.

“The claims in these three new patents, combined with our two previously issued patents, provide comprehensive protection for the Firdapse franchise, regardless of the NAT2 metabolizer status of the patients,” said Steven Miller, PhD, chief operating officer and chief scientific officer of Catalyst.

Firdapse is currently the only approved treatment for adults with LEMS in the U.S. The company recently announced plans to ask the U.S. Food and Drug Administration to extend the therapy’s approval to children with LEMS.