AbbVie venetoclax has been accepted onto the UK's Early Access to Medicines Scheme (EAMS), having picked up a positive scientific opinion from the Medicines and Healthcare products Regulatory Agency for the treatment of chronic lymphocytic leukaemia (CLL).
The decision means that adult patients with CLL and a 17p deletion or TP53 mutation, who are unsuitable for or have failed a B-cell receptor pathway inhibitor (BCRi), can now potentially gain access to the drug while the relevant regulatory bodies continue to review marketing application.
Venetoclax can also be used to treat CLL patients in the absence of 17p deletion or TP53 mutation, if they are unsuitable for or have failed both chemo-immunotherapy and a BCRi.
There are over 3,000 new cases of CLL in the UK each year, and treatment options remain limited for patients suffering relapses of the condition. Venetoclax works by targeting the B‐cell lymphoma‐2 protein, which regulates cell survival, restoring the body's ability to trigger cancer cell self-destruction (apoptosis).
The drug was approved in the US in April, on the back of efficacy data from a single-arm clinical trial of 106 pre-treated CLL patients with 17p deletion, which showed that 80 percent taking the drug experienced a complete or partial remission of their cancer.
"As part of AbbVie's fight against CLL we are delighted to receive the EAMS positive scientific opinion which will help ensure that eligible patients with limited current treatment options gain access to this therapy as soon as possible," noted Alice Butler, medical director, AbbVie UK.
The EAMS enables patients with life-threatening and seriously debilitating conditions to receive innovative and promising new drugs as soon as the MHRA has signalled that the benefits outweigh the risks, through a two-stage evaluation process.
In stage one, if the MHRA considers a product to be a possible candidate for EAMS, it will issue a Promising Innovative Medicines (PIM) designation, based on early clinical data. In stage two, the Agency will issue an EAMS Scientific Opinion if the quality, safety and efficacy data provided in support of the application is sufficient to support a positive benefit/risk balance and added clinical value.
Medicines in EAMS will typically be commissioned by NHS England through its specialised commissioning arrangements, and AbbVie will be providing its medicine free of charge to the NHS while it is prescribed under the scheme.