Table 1.

Current globally marketed ERT products

Disease	Product	Company	US FDA* approval/EU EMA† approval	Treatment in India	Dose/frequency (IV infusion)	Approximate annual cost (INR) - 10 kg child
Gaucher	Imiglucerase	Sanofi-Genzyme	1994/1997	2002	60 units/kg/EOW‡	40 lakhs
	Velaglucerase α	Shire HGT	2010/2010	2014	60 units/kg/EOW	70 lakhs
	Taliglucerase α	Protalix	2012/not approved	No information	60 units/kg q EOW	No information
Fabry	Agalsidase β	Sanofi-Genzyme	2003/2001	2005	1 mg/kg EOW	20 lakhs
	Agalsidase α	Shire HGT	Not approved/2001	2014	0.2 mg/kg EOW	20 lakhs
Pompe	Alglucosidase α	Sanofi-Genzyme	2006/2006	2008	20 mg/kg EOW	49 lakhs
MPS§ I	Laronidase	Sanofi-Genzyme	2003/2003	2006	0.58 mg/kg weekly	46 lakhs
MPS II	Idursulfase	Shire HGT	2006/2007	2014	0.5 mg/kg weekly	1 crore
MPS IVA	Elosulfase α	BioMarin	2014/2014	No information	2 mg/kg weekly	1.3crores
MPS VI	Galsulfase	BioMarin	2005/2006	2017	1 mg/kg weekly	1.1 crore
MPS VII	Vestronidase α	Ultragenyx	November 2017	Not available	4 mg/kg EOW	No information
Lysosomal acid lipase deficiency	Sebelipase α	Alexion	2015/2015	Not available	1-3 mg/kg weekly	3.2 crores
Neuronal ceroidlipofuscinosis type 2	Cerliponase α	BioMarin	2017/2017	Not available	300 mg EOW Intraventricular	4.7 crores

*FDA: Food and drug administration, ^†EMA: European Medicines Agency, ^‡EOW: Every other week, ^§MPS: Mucopolysaccharidosis