BioMarin gene therapy for haemophilia on course for EU approval
The EMA’s human medicines advisory committee has recommended that BioMarin Pharmaceutical’s haemophilia A gene therapy Roctavian be approved in the EU, removing one of the […]
The EMA’s human medicines advisory committee has recommended that BioMarin Pharmaceutical’s haemophilia A gene therapy Roctavian be approved in the EU, removing one of the […]
In 2015, NICE said that BioMarin’s Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected […]
Jazz’ Sunosi (solriamfetol) Receives NICE Recommendation for the Treatment of Excessive Daytime Sleepiness Caused by Narcolepsy Published: Nov 19, 2021 | Tags: Jazz, Sunosi, solriamfetol, […]
Shots: Entos will use its Fusogenix nucleic acid delivery system to develop BioMarin products for multiple genetic diseases. BioMarin will be responsible for the preclinical […]
A Phase 1/2 study of BioMarin Pharmaceutical’s gene therapy for phenylketonuria has been placed under a clinical hold after interim results from a preclinical study […]
BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of […]
Shots: The FDA’s clinical hold is based on interim safety findings from a pre-clinical study which showed that 6/7 animals treated with BMN 307 (2e14 […]
The second quarter of 2021 witnesses major acquisitions, approvals, and clinical data. There are major alliances in this quarter which include Merck signed ~$1.2B supply […]
Shots: BioMarin will receive an exclusive license to each program for research, development, and commercialization of the gene therapies The collaboration will utilize Allen institute […]
Pfizer and BioNTech Initiate COVID-19 Vaccine Trial in Children Under 12 Published: Mar 26, 2021 | Tags: Pfizer and BioNTech, Initiate, COVID-19 Vaccine Trial, Children, […]
Shots: The ongoing P-III OLE study involves assessing Vosoritide (15 mcg/kg) vs PBO in 119 children with achondroplasia. The CHMP opinion is expected in Europe […]
Novartis’ Ligelizumab (QGE031) Receives the US FDA’s Breakthrough Designation for Patients with Chronic Spontaneous Urticaria Published: Jan 15, 2020 | Tags: Novartis’, Ligelizumab (QGE031), Receives […]
The fourth quarter of 2020 contains multiple initiations of clinical trials, big approvals, and numerous deals. COVID-19 related news remains at the peak in this […]
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 […]
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 […]
2020 has been an exciting year for gene therapies targeting blood disorders. Despite FDA’s recent rejection of BioMarin’s hemophilia gene therapy program, the clinical landscape […]
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 […]
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 […]
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 […]
1. BioMarin Reports NDA Submission to the US FDA for Vosoritide to Treat Children with Achondroplasia Published: Aug 20, 2020 | Tags: BioMarin, Reports, NDA, […]
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 […]
US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” […]
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 […]
Copyright © 2024 | WordPress Theme by MH Themes