Innovative Therapies
-
News
BREAKING | Vertex and CRISPR’s Gene-editing Therapy Casgevy Receives Early FDA Approval for β-Thalassaemia Treatment
Just one month prior, Casgevy made history as the first CRISPR gene editing therapy to enter the US market when it was approved for sickle cell disease. Now, the FDA…
Read More » -
News
NEW TREATMENTS | Gene Editing, mRNA Hold Promise as Fanconi Anemia Therapies
That is the major finding of a newly published review that examined how a better understanding in the scientific and molecular underpinnings of the disease has translated into potential new…
Read More » -
News
TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
Read More » -
News
ADVANCED THERAPIES | Vertex, CRISPR To Submit Exa-Cel to FDA For β-Thalassaemia And Sickle Cell Disease
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission package by the end of Q1 2023.…
Read More » -
News
TIF Guide on COVID-19 Vaccinations & Therapeutic Drugs – The December Issue
During this last fortnight, experts have scrambled to gain an understanding of the new heavily mutated variant, what kind of symptoms and illness it can cause, and whether it undermines…
Read More » -
Clinical News
FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation…
Read More » -
TIF News
Juggling Between The Cost And Value of New Therapies: Does Science Still Serve Patient Needs?
In the aftermath of the withdrawal of Zynteglo, the first gene therapy for patients with β-thalassaemia, from Europe, TIF´s Executive Director, Dr Androulla Eleftheriou, reflects on the viability of the…
Read More » -
News
Coming Up This Thursday: Discussion On Access To Innovative Therapies – The Reblozyl Case
After receiving numerous questions from patients regarding the innovative treatment for β-thalassaemia Reblozyl (luspatercept), approved for use in the US, Europe, and Canada, TIF proceeds to organise an interactive, online…
Read More » -
News
Live Webinar on Thalassemia: Advances in 2021 with Dr M.D. Cappellini
The past decade has witnessed path-breaking advances with regard to novel therapeutic approaches of thalassaemia and, most significantly, phenomenal developments made in the direction of gene therapy of β- thalassaemia…
Read More » -
News
TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia
The Thalassaemia International Federation (TIF) expresses its disappointment about the outcome of the reimbursement negotiation process concerning gene therapy (Zynteglo™) for transfusion-dependent thalassaemia patients, announced last week. The Federation’s distress…
Read More »