The decision to approve the two chimeric antigen receptor T-cell (CAR-T) therapies had been widely anticipated, after the European Medicines Agency recommended both treatments for approval earlier this year.
Both products will emerge on the European market with two indications: Kymriah for the treatment of paediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia (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 diffuse large B-cell lymphoma after two or more lines of systemic therapy.
Gilead receives approval for the latter indication and for the treatment of primary mediastinal large B-cell lymphoma, after two or more lines of systemic therapy.
The approvals were based on results achieved in clinical trials, with Novartis noting 60% of patients treated achieved complete remission for B-cell ALL, and Gilead achieving a 51% complete response rate in patients with refractory aggressive non-Hodgkin lymphoma.
Despite the good news, for Novartis, there still remain questions regarding its ability to manufacture the treatment on a large scale.
In a second quarter earnings call last month, Novartis CEO Vasant Narasimhan admitted that it had experienced issues with manufacturing the treatment for patients with B-cell ALL.
The company announced, in the press release, that it will not immediately begin treating patients in this group but will “launch initially in the paediatric ALL indication, as we continue to ramp up capacity”.
CEO of Lonza, a contract development and manufacturing organisation (CDMO), Richard Ridigner, has actually claimed that the problems that Novartis is facing should be shouldered by the CDMO industry.
Revolutionary treatments, with price tags to match
On the same day that Gilead received the EC approval, the UK’s National Institute for Health and Care Excellence (NICE) delivered guidance rejecting the Yescarta’s routine use on the NHS – after it was denied on cost-effectiveness ground.
The guidance stated that “there are no direct data comparing axicabtagene ciloleucel [Yescarta] with salvage chemotherapy (referred to as best supportive care by the company). This means that the exact size of the benefit of axicabtagene ciloleucel compared with salvage chemotherapy is unknown.”
This led NICE to conclude that “all cost-effectiveness estimated are above the range normally to be a cost-effective use of NHS resources.”
This will unlikely be the last debate that is had over the price of either treatment, with the question of cost arriving immediately after Novartis received the first FDA approval for its CAR-T treatment.
With the EC’s approval, it will now be down to individual payers to negotiate with both companies to decide a fair price for public access to the treatments.