Shots: The FDA acceptance marks the first marketing application accepted for a gene therapy to treat hemophilia in the US. The application is based on ongoing P-III study with material […]readmore
Tags : BioMarin
1. Lupin Launches Smart Device for Metered-Dose Inhalers to Support Treatment of Respiratory Diseases in India Published: Nov 22, 2019 | Tags: Lupin, Launches, Smart Device, Metered-Dose Inhalers, Support, Treatment, […]readmore
Shots: The MAA submission is based on ongoing P-III study with material from the to-be-commercialized process and updated 3yrs. P-I/II data. The submission marks the first MAA submission for a […]readmore
Shots: Biomarin has received approval for its Vimizim, as a first approval by NMPA for patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome In May […]readmore
Shots: The P-III GENEr8-1 study results involves assessing of valoctocogene roxaparvovec (6e13 vg/kg) in 20 patients with hemophilia A The P-III GENEr8-1 study results: @23-26eks. 8 patients achieved Factor VIII […]readmore
Regulatory
BioMarin’s Palynziq (pegvaliase injection) Receives European Commission’s MAA Approval for
Shots: The approval is based on P-III PRISM-2 study results assessing Palynziq (pegvaliase injection, 20 & 40 mg) vs PBO in 215 PKU patients with inadequate blood Phe level >600 […]readmore
Shots: The study involves assessing Brineura (cerliponase alfa, 300mg) in 24 patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, aged 3-16 yrs. with motor-language (ML) score 3-6 […]readmore
Shots: The approval is based on P-III PRISM-2 study results assessing Palynziq (pegvaliase-pqpz, 20mg qd or 40mg qd) in patients in ratio (2:1) with high level of phenylalanine (Phe) The […]readmore