Feb. 16 Quick Takes: FDA approves Chiesi’s Lamzede for ultra-rare lysosomal storage disorder

FDA’s approval of Lamzede velmanase alfa-tycv from Chiesi Farmaceutici S.p.A. to treat non-CNS manifestations of alpha-mannosidosis comes almost five years after the recombinant form of human alpha-mannosidase was approved in Europe. Alpha-mannosidosis, an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme alpha-mannosidase, affects about one in every 500,000 to one in every 1,000,000 babies born worldwide, the company said. Chiesi gained the product through its 2013 acquisition of Zymenex A/S. 

Iveric bio Inc. (NASDAQ:ISEE) is looking ahead to an Aug. 19 PDUFA date for avacincaptad pegol after FDA accepted an NDA under priority review for the complement C5 inhibitor to treat geographic atrophy secondary to age-related macular degeneration (AMD). FDA is not currently planning to hold an advisory committee meeting to discuss the therapy, according to the company. ...