Completion of NDA submission for velaglucerase alpha is expected before the fourth quarter.
Shire is reporting positive results from the first of three Phase III trials with velaglucerase alfa, an enzyme replacement therapy for the treatment of type 1 Gaucher’s disease. Additionally, the FDA accepted the firm’s treatment protocol related to this drug, so that it can be supplied to patients under certain guidelines ahead of approval. The firm saw its share price on the LSE nudge up a couple of percent.
Shire had been asked to help fill the treatment gap due to the shortfall in the current marketed therapy (Genzyme’s Cerezyme). The company also confirmed initiating a rolling NDA submission for velaglucerase alpha under FDA’s fast track approval process. It expects to complete the NDA submission by the end of this quarter.
The reported Phase III trial assessed two dose levels of velaglucerase alpha in 25 patients (aged two years and over) with type 1 Gaucher’s disease. Patients had not previously received any treatment for the disorder, and all demonstrated disease-related anaemia and at least one other clinical symptoms of type 1 Gaucher’s.
Trial results showed treatment with the lower 45 U/Kg IV dose resulted in statistically significant improvements in patients’ haemoglobin levels, platelet count, and spleen volume. There was also a trend to reduced liver volume. Results for the 60 U/Kg IV dose were either statistically as good as or better than the lower dose across all clinical endpoints. There were no serious drug-related events.
Velaglucerase alfa is produced in a human cell line. The company says that the enzyme has the exact human amino acid sequence and carries a human glycosylation pattern.