Tags : BioMarin

PharmaShots Weekly Snapshot (Oct 5-9, 2020)

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, with Lilly and Incyte’s Olumiant (baricitinib) Reports Additional Data Demonstrating Reduction in COVID-19 Recovery Time  Published: Oct 9, 2020 | Tags: COVID-19, Demonstrate, Incyte, Lilly, […]Read More

BioMarin Receives the US FDA’s Approval Allowing Maximum Dose of

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 based on OLE study out to 3yrs. demonstrating that 66% had a blood Phe level ≤360 μmol/L consistent with the Phe target ACMG recommended guidelines […]Read More

PharmaShots Weekly Snapshot (Sept 07 -11, 2020)

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 FDA, Selective, IL-23 inhibitor, Active Psoriatic Arthritis, Severe Plaque Psoriasis, human mAb 2. Janssen Reports sBLA Submission to the US FDA for Darzalex Faspro (daratumumab […]Read More

PharmaShots Weekly Snapshot (Aug 17 -21 2020)

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, Achondroplasia 2. Junshi and Impact to Establish Joint Venture for Senaparib (IMP4297) in China Published: Aug 20, 2020 | Tags:  Junshi, Impact, Establish, Joint Venture, […]Read More

BioMarin Reports NDA Submission to the US FDA for Vosoritide

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 therapy on 13 Aug, 2020 Vosoritide is an investigational, once-daily injection analog of C-type Natriuretic Peptide (CNP) and is potentially the first treatment for achondroplasia […]Read More

BioMarin Reports the Submission of MAA to EMA for Vosoritide

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 studies and extensive natural history data Following the completion of EMA’s validation, BioMarin expects the initiation of the MAA review to commence in Aug’2020. The […]Read More

PharmaShots Weekly Snapshot (November 18-22, 2019)

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, Respiratory Diseases, India 2. BioMarin Reports Submission of MAA to EMA for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A Published: Nov 21, 2019 | Tags: BioMarin, […]Read More

BioMarin Reports Submission of MAA to EMA for Valoctocogene Roxaparvovec

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 gene therapy product targeting any type of hemophilia BioMarin is expected its MAA for Valoctocogene Roxaparvovec to be reviewed under expedited accelerated assessment designation in […]Read More