According to Biotech and Pharma Industry News, British pharmaceutical manufacturer Mallinckrodt has reported inconclusive results in its Phase 2/3 trial of VTS-270, a drug candidate for the treatment of the rare genetic disease Niemann-Pick type C.

About Niemann-Pick Disease Type C

Niemann-Pick is a group of diseases caused by genetic mutations that affect the body’s metabolism.

In patients with Niemann-Pick Type C, cells in their bodies fail to transport cholesterol and other important lipids like they would in an otherwise healthy individual. This leads to an accumulation of fat deposits in and around major organs– including the liver, spleen, and brain.

It presents most commonly in early childhood, though in some cases, patients may be asymptomatic until later in life. There is currently no known cure, and the majority of patients do not live past 20.

Though Type C is one of the most commonly recognized types of Niemann-Pick disease, it is still quite rare, afflicting only a few hundred known individuals across the U.S. For more information, click here.

Inconclusive Trial Results

In a year-long Phase 2/3 trial (relatively early in the intensive clinical trial process), Mallinckrodt’s drug VTS-270 failed to outperform placebos in combating the progression of Niemann-Pick type C.

Across the study, regardless of whether they were being treated with VTS-270 or a placebo, patients’ Niemann-Pick did not observably progress– rendering the effectiveness of the drug uncertain.

Mallinckrodt has proposed that the frustrating result stemmed from inconsistencies in the population of the study group. Steve Romano, the chief science officer at Mallinckrodt, told Biotech and Pharma Industry News that the company intends to examine several variables potentially causing the confusion– focusing on the age of the participants, the severity of their cases, and the degree to which their Niemann-Pick has already progressed.

Though the future of the drug is not yet certain, this is an undeniably serious setback in research and development.

Hope for an Alternative

Pharmaceutical competitor Actelion’s drug Zavesca^® is intended for patients with Gaucher disease type 1, but has shown some promise in slowing the progression of Niemann-Pick type C. In Europe, Canada, and Japan, it has already been approved for this purpose.

In the United States, however, the FDA rejected this use of Zavesca^®, requesting additional clinical and preclinical information before agreeing to move forward. That was eight years ago.

Should the FDA approve Zavesca^® as a treatment for Niemann-Pick in lieu of VTS-270? Do drugs that benefit fewer individuals struggle to gain approval? Share your thoughts with the Patient Worthy community!