FDA gives the OK to Chiesi for treatment of ultra-rare enzyme deficiency disease
The FDA handed down an approval Thursday for Chiesi’s velmanase alfa, an enzyme supplement designed to treat non-central nervous system manifestations of an ultra-rare progressive disease, the company announced.
Velmanase alfa is approved for adults and children and will be sold in the US as Lamzede, the same branded name it uses in Europe where it was approved in 2018. The disease it’s designed to treat is called alpha-mannosidosis (AM), which affects only one in every 500,000 to one in every one million babies born worldwide, Chiesi says.
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