Innovative Therapies
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News
FDA APPROVES AQVESME™ (mitapivat) | First Oral Therapy for Anaemia Across All Forms of Thalassaemia
This regulatory decision marks a landmark milestone in the management of thalassaemia, as AQVESME is now the first and only therapy approved in the United States indicated for treating anaemia…
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EU AGREES LANDMARK REFORM OF PHARMACEUTICAL LEGISLATION | What It Means for Patients and Public Health
This milestone agreement — part of what has been referred to as the “pharma package” — was negotiated between the European Commission, the European Parliament, and the Council of the…
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SICKLE CELL DISEASE | Limited Progress in Sickle Cell Disease Highlights Barriers to Treatment Access
According to the findings, there has been limited improvement in outcomes for SCD patients, in large part due to barriers in accessing first-line therapies such as hydroxyurea. Although several new…
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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…
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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…
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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…
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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.…
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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…
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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…
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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…
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