Roche's Tecentriq 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 bladder cancer.
The decision means that patients with advanced cancer of the bladder and urinary system, which has spread and come back after one course of standard cancer chemotherapy, can now potentially access Tecentriq (atezolizumab) while the relevant regulatory bodies review its marketing application in Europe.
In the US, the drug is the first and only cancer immunotherapy cleared to treat locally advanced or metastatic urothelial carcinoma (mUC), on the back of data from the Phase II IMvigor 210 trial, which assessed Tecentriq in heavily pre-treated patients and showed median overall survival of 11.4 months in those with higher levels of PD-L1 expression, and 7.9 months overall.
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.
PIM for ProMetic's AS drug
ProMetic Life Sciences' lead drug candidate, PBI-4050, has just been issued a PIM designation for the treatment of Alström Syndrome (AS), a rare inherited autosomal recessive syndrome characterised by the onset of obesity in childhood or adolescence, type II diabetes with severe insulin resistance, dyslipidaemia, hypertension and severe multi-organ fibrosis, involving the liver, kidney and heart.
PBI-4050 is an orally active investigational drug which the firm says has excellent safety and efficacy profiles confirmed in several in vivo experiments targeting fibrosis, a very complex process by which continuing inflammation causes vital organs to lose their function as normal tissue is replaced by fibrotic scar tissue.
"We believe PBI-4050 to have first-in-class anti-fibrotic properties and look forward to continue working closely with the MHRA to advance PBI-4050 through its clinical programme and provide Alström Syndrome patients with a new treatment option as soon as possible," commented Pierre Laurin, president and chief executive of the firm.