Shots: The ongoing P-III GENEr8-1 study involves assessing of valoctocogene roxaparvovec (6e13 vg/kg dose) in 134 patients with with severe hemophilia A. The patients received a single dose of valoctocogene […]readmore
Tags : BioMarin
Eli Lilly and Incyte Receive FDA’s EUA for Baricitinib + Remdesivir to Treat Hospitalized Patients with COVID-19 Published: Nov 20,2020 | Tags: baricitinib, COVID-19, Eli Lilly, Emergency Use Authorization, FDA’s, […]readmore
AstraZeneca’s Brilinta (ticagrelor) Receives the US FDA’s Approval to Reduce the Risk of Stroke in Patients with an Acute Ischemic Stroke or High-Risk Transient Ischemic Attack Published: Nov 6, 2020 […]readmore
Shots: The acceptance marks the first marketing application accepted for achondroplasia in the US with an anticipated PDUFA date as of Aug 20, 2021. The US FDA is not planning […]readmore
Takeda Signs ~$1B Pact with Arrowhead for ARO-AAT Targeting Alpha-1 Antitrypsin-Associated Liver Disease Published: Oct 9, 2020 | Tags: Alpha-1 Antitrypsin-Associated Liver Disease, Arrowhead, Collaborates, Commercialize. ARO-AAT, Develop, Takeda, […]readmore
Shots: The US FDA has approved the sBLA to increase the maximum allowable dose of 60mg with Palynziq for PKU. Previously, the maximum dose was 40mg The label expansion is […]readmore
1. Janssen’s Tremfya (guselkumab) Receives the US FDA’s Approval as the First Selective IL-23 Inhibitor for Active Psoriatic Arthritis Published: Sept 10, 2020 | Tags: Janssen, Tremfya, Guselkumab, Biologic, US […]readmore
1. BioMarin Reports NDA Submission to the US FDA for Vosoritide to Treat Children with Achondroplasia Published: Aug 20, 2020 | Tags: BioMarin, Reports, NDA, Submission, US, FDA, Vosoritide, Children, […]readmore
Shots: BioMarin has submitted an NDA to the US FDA for vosoritide to treat children with achondroplasia The company also reported that EMA has begun validation of MAA for the […]readmore
Shots: The MAA is based on P-III study assessing the efficacy and safety of vosoritide, further supported by the long-term safety and efficacy from the ongoing P-II and P-III extension […]readmore