The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for velmanase alfa for the treatment of alpha-mannosidosis.
Alpha-mannosidosis is a lysosomal storage disorder characterized by a deficiency of the enzyme alpha-mannosidase, which results in an abnormal accumulation of sugars in the cells of the body. Symptoms of the disease can include facial and skeletal abnormalities, hearing loss, and intellectual disability.
Velmanase alfa is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase, thereby preventing the accumulation of mannose–rich oligosaccharides in various tissues in the body.
The application is supported by data from the SPARKLE cohort study (EUPAS29038), which was recently presented at the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2022 held in Freiburg, Germany. The SPARKLE registry was developed to gather insights into the natural history and disease progression of treated and untreated patients with alpha-mannosidosis. The study is expected to enroll up to 100 patients; preliminary baseline clinical status data were available for 49 patients, 24 of whom were currently receiving treatment with velmanase alfa.
Findings from a long-term efficacy analysis of patients with alpha-mannosidosis being treated with velmanase alfa were also presented at the meeting. Results showed mobility improvements in most patients, while biochemical measures and pulmonary function improved in all evaluable patients (N=16).
“The current management of alpha-mannosidosis is largely focused on treatment of the various symptoms,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. “We believe that our investigational enzyme replacement therapy has the potential to address the cause of the disease and look forward to working closely with the FDA throughout its review process.”
A Prescription Drug User Fee Act action date has been set for the first half of 2023.
- Chiesi Global Rare Diseases announces FDA acceptance of BLA filing for velmanase alfa for the proposed treatment of alpha-mannosidosis. News release. Chiesi Global Rare Diseases. Accessed September 12, 2022. https://www.prnewswire.com/news-releases/chiesi-global-rare-diseases-announces-fda-acceptance-of-bla-filing-for-velmanase-alfa-for-the-proposed-treatment-of-alpha-mannosidosis-301621738.html
- Hennermann J, Guffon N, Ballabeni A, et al. Baseline characteristics from the international retrospective and prospective SPARKLE registry of patients with alpha-mannosidosis. Abstract presented at: Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2022; August 30, 2022 to September 2, 2022. Abstract 2829.
- Guffon N, Gaches F, Daridon C, et al. Long-term efficacy of velmanase alfa in patients with alpha-mannosidosis: Retrospective analysis of a French registry for up to 9.5 years. Abstract presented at: Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2022; August 30, 2022 to September 2, 2022. Abstract 2659.
This article originally appeared on MPR