UK Multiple Myeloma Patients Allowed Early Access To Sanofi’s Isatuximab
Executive Summary
The UK scheme allows patients with early access to promising new unlicensed medicines when there is a clear unmet medical need.
Sanofi has received permission to make its unlicensed monoclonal antibody, isatuximab, available to multiple myeloma patients in the UK under the government’s early access to medicines scheme (EAMS) while it is evaluated for EU-wide approval.
Isatuximab, which is designated as an orphan drug in the EU, was given a positive scientific opinion under EAMS by the UK’s Medicines and healthcare Products Regulatory Agency in December 2019.
As per the MHRA’s opinion, isatuximab may be used in combination with pomalidomide and dexamethasone for treating adult patients with relapsed and refractory multiple myeloma only if they have received three previous lines of therapies (that include lenalidomide and a proteasome inhibitor) and have demonstrated disease progression on the last therapy.
The EAMS aims to provide patients with life threatening or seriously debilitating conditions with access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. The EAMS scientific opinion lasts for a year and can be renewed.
According to the MHRA, treatment for multiple meyloma has “improved remarkably over the last two decades with the introduction of autologous stem cell transplantation and the introduction of numerous novel agents.” Nevertheless, once a patient becomes refractory to those agents, survival is limited, and the disease remains an incurable disease in the vast majority of patients, it said.
Isatuximab was awarded a positive opinion under the EAMS because it was shown to “significantly improve progression free survival and also to significantly improve overall response rate with an adverse event profile that can be considered to be clinically manageable,” the MHRA said.
The scientific opinion provides benefit and risk information to doctors who may wish to prescribe the unlicensed medicine. There are provisions to report side effects experienced by patients receiving isatuximab through the scheme.
Drugs under the scheme are usually provided free of charge to the National Health Service until either the EAMS designation lapses or the product receives EU marketing authorization. It is anticipated that medicines with a positive EAMS scientific opinion could be made available to patients 12 to 18 months before formal marketing authorization.
A benefit of the scheme for companies is that EAMS products that subsequently receive marketing approval and that are found to be cost-effective following a health technology appraisal by the National Institute of Health and Care Excellence are commissioned by NHS England within 30 days of the NICE guidance, rather than the standard three months.
Seeking Approval In EU, US
Sanofi has applied for the approval of isatuximab’s in both the EU and the US. In the EU, the a marketing authorization application for the drug was accepted for evaluation by the European Medicines Agency in the second quarter of 2019.
While Sanofi had initially asked the EMA to review its MAA under the EU’s accelerated assessment pathway, its request was rejected by the agency. This means isatuximab’s marketing application is being evaluated as per the standard review timelines. (Also see "EU Accelerated Assessment Tracker" - Pink Sheet, 6 Jun, 2019.)
In the US, isatuximab was accepted for review by the US Food and Drug Administration last July, and the target action date for the FDA’s decision is 30 April 2020. (Also see "Sanofi Poised To File Isatuximab In Multiple Myeloma, Going Up Against J&J's Darzalex" - Scrip, 5 Feb, 2019.) Isatuximab has also received orphan drug designation in the US.