This week: Kymriah receives European Commission approval and safety of MSC administration in ALS patients confirmed
The news highlights:
Too-expensive Yescarta is not recommended for NHS availability
Kymriah receives European Commission approval, prompts expansion of manufacturing facilities
Feasibility and safety of IV and spinal cord infused MSCs for ALS treatment confirmed
In draft guidance, the National Institute for Health and Care Excellence (NICE; UK), which publishes guidelines on health technologies in the NHS (UK), has not recommended axicabtagene ciloleucel to be available on the NHS. Axicabtagene ciloleucel, known as Yescarta (Kite, a Gilead company; CA, USA), recently received approval from the European Medicines Agency for use in the European Union.
In the guidelines, NICE commented that although “axicabtagene ciloleucel meets NICE’s criteria to be considered a life-extending treatment at the end of life”, lack of direct comparison data for Yescarta vs salvage chemotherapy combined with the high cost-effectiveness estimates meant that the treatment did not receive approval. However, these are only draft guidelines; NICE will make its final decision at the end of September.
Novartis (Switzerland) has received approval from the European Commission for Kymriah for the indications of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. This ruling makes Novartis the only company with an approved CAR-T cell therapy for pediatric r/r B-cell ALL as well as the first to receive approval in two distinct indications, both in the EU and the US.
“The Kymriah approval is a transformational milestone for patients in Europe in need of new treatment options,” said Liz Barrett, CEO, Novartis Oncology. “Novartis will continue to build a global infrastructure for delivering CAR-T cell therapies where none existed before remaining steadfast in our goal of reimagining cancer.”
Two small Phase I trials have demonstrated that stem cell transplants for the treatment of amyotrophic lateral sclerosis (ALS) are feasible whether administered intravenously or via the spinal cord. Iranian researchers carried out the autologous bone marrow-derived mesenchymal stromal cells (MSCs) transplants to assess the safety and feasibility of the two treatment modalities, and encountered no reports of adverse events. Both cohorts saw an improvement in ALS Functional Rating Scale score and forced vital capacity percentage, and the team will now assess allogeneic transplants.
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