In the Rare Disease area, we plan to submit velmanase alfa (Lamazyn), a recombinant enzyme indicated for alpha-mannosidosis
, an inherited condition which leads to a progressive deterioration of motor and mental functions.
The European Medicines Agency (EMA) has given Chiesi Farmaceutici's Lamzede (velmanase alfa) marketing authorisation as the first long-term enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis
(AM), Pharma Times reported on Thursday.
The LSDs for which clinical trials are in various phases include Mucopolysaccharidosis Type-IIIA, Mucoploysaccharidosis Type-IVA, Mucoploysaccharidosis Type-VII, Metachromatic Leukodystrophy, Wolman Disease, Alpha-mannosidosis
and Niemann Pick C/D.
Due to the lesions with demyelinating characteristics, recurrent stress-triggered psychotic syndrome, pectus carinatum, and hypopneumatization of the sinus, the possibility of alpha-mannosidosis
and Krabbe's disease was suggested.