Exclusive license for Japanese rights to Ultragenyx's (Nasdaq: RARE) Mepsevii (vestronidase alfa), an enzyme replacement therapy (ERT) for the lysosomal disorder Mucopolysaccharidosis type VII (MPS VII
, Sly syndrome).
Mepsevii is an enzyme replacement therapy designed to replace the deficient lysosomal enzyme beta-glucuronidase in patients with MPS VII
, a progressive and debilitating rare genetic disease.
Food and Drug Administration (FDA) has approved MEPSEVII[TM] (vestronidase alfa), the first medicine approved for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome).
MPS VII is a rare genetic, metabolic lysosomal storage disorder (LSD) caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulfate, chondroitin sulfate and heparan sulfate.
The US Food and Drug Administration (FDA) has granted approval to Ultragenyx Pharmaceutical's enzyme replacement therapy, Mepsevii (vestronidase alfa), intended for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in both children and adults, it was reported yesterday.
MPS VII, Sly syndrome is a progressive and debilitating inherited, rare genetic disease, which has previously had no approved therapy.
Mean activity ([micro]mol/h/L blood) (a) Sample GALNS NAGLU ARSB Blank (n = 6) 0.0033 0.01 0.05 CDC QC low (b) (n = 2) 0.17 0.23 0.46 CDC QC high (b) (n =2) 3.0 5.8 5.1 Adults (n = 4) 1.1 4.1 1.2 Newborns (n = 62) 0.67 1.6 4.4 MPS IIIB patients (n =4) 0.83 0.01 1.7 MPS VII
patients (n =3) 1.0 3.2 1.8 LINCL patients (n = 7) 1.2 3.1 1.8 Mean activity ([micro]mol/h/L blood) (a) Sample I2S GUSB TPP1 Blank (n = 6) 0.06 0.06 0.03 CDC QC low (b) (n = 2) 0.98 13.0 5.0 CDC QC high (b) (n =2) 14.6 44.4 22.7 Adults (n = 4) 17.0 19.5 19.0 Newborns (n = 62) 16.1 28.5 35.9 MPS IIIB patients (n =4) 19.2 17.7 21.2 MPS VII
patients (n =3) 26.1 0.08 20.4 LINCL patients (n = 7) 17.1 27.0 1.3 (a) For SDs in these measurements see online Supplemental Table 2.
In humans the same disease, known as mucopolysaccharidosis type VII (MPS VII
), remains largely incurable.
Odiparcil has also the potential to address other MPS types, characterized by the accumulation of chondroitin or dermatan sulfate (MPS I or Hurler/Sheie syndrome, MPS II or Hunter syndrome, MPS IVa or Morquio syndrome and MPS VII
or Sly syndrome).
Biotechnology company Ultragenyx Pharmaceutical Inc reported on Wednesday the receipt of approval from the US Food and Drug Administration for Mepsevii (vestronidase alfa-vjbk) for the treatment of pediatric and adult patients with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII
), also known as Sly syndrome.
Enzyme Disease P (a)/C Reference [alpha]-L-Iduronidase MPS I 13/7 2 (EC 220.127.116.11) Iduronate sulfatase MPS II 10/8 3 (EC 18.104.22.168) MSD 1/2 Arylsulfatase B MPS VI 10/8 4 (EC 22.214.171.124) MSD 1/2 [beta]-D-Glucuronidase MPS VII
2/1 5 (EC 126.96.36.199) [beta]-D-Galactosidase GM1 gangliosidosis 10/10 5 (EC 188.8.131.52) Galactosialidosis 1/1 MPS IV B NA [alpha]-D-Mannosidase [alpha]-Mannosidosis 2/2 5 (EC 184.108.40.206) [alpha]-L-Fucosidase Fucosidosis NA 5 (EC 220.127.116.11) [beta]-Hexosaminidase Sandhoff 3/3 5 (EC 18.104.22.168) Mucolipidosis II/III 5/4 (a) P, patients; C, carriers; NA, not available.