Chiesi gets FDA high five for enzyme replacement therapy Lamzede

Chiesi Farmaceutici’s regulatory success with Lamzede has come in five-year increments.

After purchasing the enzyme replacement therapy in its 2013 acquisition of Zymenex, Chiesi scored approval for it in Europe in 2018. Now, five more years later, the Italian pharma has pushed it over the finish line in the United States.

The FDA has endorsed Lamzede, which becomes the first enzyme replacement therapy to treat non-central nervous system manifestations of alpha-mannosidosis (AM).

The ultra-rare, progressive lysosomal storage disorder—which is caused by deficiency in the alpha-mannosidase enzyme—strikes between one in every 500,000 to 1 million people. The approval for the inherited condition covers adults and children.

The green light comes less than three weeks after Chiesi named Baxter veteran Giuseppe Accogli its new CEO to replace 12-year chief Ugo Di Francesco. Earlier last month, Chiesi revealed a $1.48 buyout of Dublin-based rare disease specialist Amryt.

The acquisition and Thursday’s approval are evidence of Chiesi’s push to diversify and grow in the U.S. In 2020, the company established Chiesi Global Rare Diseases, headquartered in Boston. Chiesi also has hubs in China, Sweden, Canada, France and England.

Those with alpha-mannosidosis lack the enzyme that breaks down sugars, causing an accumulation in various tissues. Symptoms present in a variety of ways including impaired hearing, speech and cognitive function. Chest and ear infections also are common.