An investigational therapy in development for the treatment of Niemann-Pick Type C1 disease (NPC) has been awarded Promising Innovative Medicine (PIM) status by the Medicines and Healthcare products Regulatory Agency.

The decision means that Vtesse's VTS-270 has completed the first of a two-stage process that could see it made available to patients via the UK's Early Access to Medicines Scheme before it is approved by European regulators.

NPC is a progressive, irreversible, chronically debilitating - and ultimately lethal - genetic disease, caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen.

Current treatment is focused on managing specific symptoms of the condition in each individual, as well as preventing secondary complications.

VTS-270, a mixture of 2-hydroxypropyl-B-cyclodextrin, targets cholesterol and sphingolipid storage. The firm says preliminary studies have shown that it can promote transport of the cholesterol that would normally accumulate in the lysosomes of cells carrying the mutation responsible for the disease, restoring normal cholesterol metabolism and regulation.

Results from the VTS-270 treated group in the intrapatient Phase I/II dose escalation study show that, after 12 months and 18 months of monthly dosing, disease progression as measured by the NPC Neurological Severity Score (NSS) was reduced as compared to a matched natural history study control group. Changes in hearing, which were anticipated as an adverse event, and transient ataxia and transient fatigue were observed in the study.

"This UK PIM designation, coupled with the US FDA Breakthrough Therapy Designation granted earlier this year, demonstrates the strength of the preliminary clinical data of NPC treatment with VTS-270," noted Kevin Johnson, vice president, Regulatory Affairs, at Vtesse. "Both designations afford us enhanced regulatory opportunities, which we will rely on as we complete our rigorous Phase IIb/III clinical trial and seek regulatory approvals to bring this drug to market as quickly as possible."

The firm's ongoing Phase IIb/III clinical trial of VTS-270 is enrolling patients in the UK at Birmingham Children's Hospital and Great Ormond Street Hospital, and is also ongoing at sites in the US, France, Germany, Spain, Turkey and Australia.